Conditions: Solid Tumor Malignancy
Keywords: Fibroblast growth factor receptor (FGFR) inhibitor FGFR mutations FGFR translocations solid tumor malignancy
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 111
Healthy volunteers: no

Brief summary:
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR mutations or translocations.

Detailed description:

Arms:
arm 1: Cohort A (Solid tumor malignancies with FGFR1-3 in frame fusions; any FGFR2 rearrangement; FGFR1/3 rearrangement with known partner\*). Cohort B (Solid tumor malignancies with known or likely activating mutations (excluding kinase domain) in FGFR1-3) Cohort C (Solid tumor malignancies with FGFR1-3 known activating mutations in kinase domain; FGFR1-3 putatively activating mutations; other FGFR1/3 rearrangements\* (not eligible for Cohort A)).

\*Only FGFR fusions or rearrangements with an intact kinase domain are eligible

Interventions: intervention 1: Pemigatinib
Intervention descriptions: intervention 1: Pemigatinib administered orally once daily (QD).

Protocol document:
Prot_000.pdf: 
Official Title: 
NCT Number: 
Document Date: 
A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the 
Efficacy and Safety of Pemigatinib in Participants With Previously 
Treated Locally Advanced/Metastatic or Surgically Unresectable Solid 
Tumor Malignancies Harboring Activating FGFR Mutations or 
Translocations (FIGHT-207)
NCT03822117
Clinical Study Protocol Version 3: 15 February 2021


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Incyte Corporation 
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Protocol INCB 54828-207 Am 3 
Version 3 
15 FEB 2021 
VV-CLIN-000652 
CONFIDENTIAL 
INVESTIGATOR'S AGREEMENT 
I have read the INCB 54828-207 Protocol Amendment 3 (Version 3 dated 15 FEB 2021) and 
agree to conduct the study as outlined.  I agree to maintain the confidentiality of all information 
received or developed in connection with this Protocol. 
 
 
 
 
 
(Printed Name of Investigator) 
 
 
 
 
 
 
 
 
(Signature of Investigator) 
 
(Date) 


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TABLE OF CONTENTS 
LIST OF ABBREVIATIONS ..........................................................................................................8 
1. 
PROTOCOL SUMMARY ..........................................................................................11 
2. 
INTRODUCTION ......................................................................................................19 
2.1. 
Background .................................................................................................................19 
2.1.1. 
Fibroblast Growth Factor Receptor Inhibitor in Oncology ........................................19 
2.1.2. 
Solid Tumor Malignancies With Activating FGFR Mutations and 
Fusions/Rearrangements .............................................................................................20 
2.2. 
Study Rationale ...........................................................................................................21 
2.2.1. 
Scientific Rationale for Study Design ........................................................................21 
2.2.2. 
Justification for Dose and Regimen ............................................................................21 
2.3. 
Benefit/Risk Assessment of Pemigatinib....................................................................23 
3. 
OBJECTIVES AND ENDPOINTS ............................................................................27 
4. 
STUDY DESIGN .......................................................................................................28 
4.1. 
Overall Design ............................................................................................................28 
4.2. 
Overall Study Duration ...............................................................................................29 
4.3. 
Study Termination ......................................................................................................29 
5. 
STUDY POPULATION .............................................................................................30 
5.1. 
Inclusion Criteria ........................................................................................................30 
5.2. 
Exclusion Criteria .......................................................................................................32 
5.3. 
Lifestyle Considerations .............................................................................................34 
5.4. 
Screen Failures ............................................................................................................34 
5.5. 
Replacement of Participants .......................................................................................35 
6. 
STUDY TREATMENT ..............................................................................................35 
6.1. 
Study Treatment Administered ...................................................................................35 
6.2. 
Preparation, Handling, and Accountability ................................................................36 
6.3. 
Measures to Minimize Bias:  Randomization and Blinding .......................................37 
6.4. 
Study Treatment Compliance .....................................................................................37 
6.5. 
Dose Modifications .....................................................................................................37 
6.5.1. 
Criteria and Procedures for Dose Interruptions and Adjustments of Study 
Drug ............................................................................................................................37 
6.5.2. 
Management of Hyperphosphatemia ..........................................................................39 
6.5.3. 
Up-Titration ................................................................................................................40 


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6.5.4. 
Criteria for Permanent Discontinuation of Study Drug ..............................................40 
6.6. 
Concomitant Medications and Procedures .................................................................40 
6.6.1. 
Restricted Medications and Procedures ......................................................................41 
6.6.2. 
Prohibited Medications and Procedures .....................................................................41 
7. 
DISCONTINUATION OF STUDY TREATMENT AND PARTICIPANT 
DISCONTINUATION/WITHDRAWAL ..................................................................42 
7.1. 
Discontinuation of Study Treatment ...........................................................................42 
7.1.1. 
Reasons for Discontinuation .......................................................................................42 
7.1.2. 
Discontinuation Procedures ........................................................................................42 
7.2. 
Participant Withdrawal From the Study .....................................................................43 
7.3. 
Lost to Follow-Up.......................................................................................................43 
8. 
STUDY ASSESSMENTS AND PROCEDURES ......................................................44 
8.1. 
Administrative and General Procedures .....................................................................44 
8.1.1. 
Informed Consent Process ..........................................................................................44 
8.1.2. 
Prescreening and Screening Procedures .....................................................................45 
8.1.3. 
Interactive Response Technology Procedure ..............................................................45 
8.1.4. 
Distribution of Reminder Cards ..................................................................................45 
8.1.5. 
Demography and Medical History ..............................................................................46 
8.1.5.1. 
Demographics and General Medical History .............................................................46 
8.1.5.2. 
Disease Characteristics and Treatment History ..........................................................46 
8.2. 
Efficacy Assessments .................................................................................................46 
8.2.1. 
Tumor Imaging ...........................................................................................................46 
8.2.2. 
ECOG Performance Status .........................................................................................46 
 
47 
8.3. 
Safety Assessments .....................................................................................................47 
8.3.1. 
Adverse Events ...........................................................................................................47 
8.3.2. 
Physical Examinations ................................................................................................48 
8.3.3. 
Vital Signs ..................................................................................................................48 
8.3.4. 
Electrocardiograms .....................................................................................................48 
8.3.5. 
Comprehensive Eye Examination...............................................................................49 
8.3.6. 
Laboratory Assessments .............................................................................................49 
8.3.6.1. 
Pregnancy Testing ......................................................................................................52 
8.3.6.2. 
Serology ......................................................................................................................52 


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52 
 
52 
8.5. 
 Translational Assessments ....................................................53 
8.5.1. 
Tumor for Genomics Testing for FGFR Genetic Alterations and Gene 
Expression ...................................................................................................................53 
 
53 
 
53 
 
54 
8.5.5. 
Tissue Biopsies ...........................................................................................................54 
8.6. 
Unscheduled Visits .....................................................................................................54 
8.7. 
End of Treatment ........................................................................................................54 
8.8. 
Follow-Up ...................................................................................................................55 
8.8.1. 
Safety Follow-Up ........................................................................................................55 
8.8.2. 
Post-Treatment Disease Follow-Up ............................................................................55 
8.8.3. 
Survival Follow-Up ....................................................................................................55 
9. 
ADVERSE EVENTS:  DEFINITIONS AND PROCEDURES FOR 
RECORDING, EVALUATING, FOLLOW-UP, AND REPORTING ......................56 
9.1. 
Definition of Adverse Event .......................................................................................56 
9.2. 
Definition of Serious Adverse Event ..........................................................................57 
9.3. 
Recording and Follow-Up of Adverse Events and/or Serious Adverse Events .........58 
9.4. 
Reporting of Serious Adverse Events .........................................................................60 
9.5. 
Pregnancy ...................................................................................................................62 
9.6. 
Warnings and Precautions ..........................................................................................62 
9.7. 
Product Complaints ....................................................................................................63 
10. 
STATISTICS ..............................................................................................................63 
10.1. 
Sample Size Determination ........................................................................................63 
10.2. 
Populations for Analysis .............................................................................................63 
10.3. 
Level of Significance ..................................................................................................64 
10.4. 
Statistical Analyses .....................................................................................................64 
10.4.1. 
Primary Analysis ........................................................................................................64 
10.4.2. 
Secondary Efficacy Analysis ......................................................................................64 
10.4.3. 
Safety Analyses ..........................................................................................................65 
 
65 


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10.5. 
Interim Analysis ..........................................................................................................66 
11. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS ..................................................................................................66 
11.1. 
Investigator Responsibilities .......................................................................................66 
11.2. 
Data Management .......................................................................................................67 
11.3. 
Data Privacy and Confidentiality of Study Records ...................................................69 
11.4. 
Financial Disclosure ...................................................................................................69 
11.5. 
Publication Policy .......................................................................................................69 
11.6. 
Study and Site Closure................................................................................................70 
12. 
REFERENCES ...........................................................................................................71 
APPENDIX A. INFORMATION REGARDING EFFECTIVENESS OF 
CONTRACEPTIVE METHODS ...............................................................................73 
APPENDIX B. INSTRUCTION TO PARTICIPANTS FOR HANDLING STUDY 
DRUG (PEMIGATINIB) ...........................................................................................74 
APPENDIX C. ACTIVATING FGFR MUTATIONS AND 
FUSIONS/REARRANGEMENTS.............................................................................75 
APPENDIX D. CYP3A4 INHIBITORS AND INDUCERS ......................................................77 
APPENDIX E. COVID-19 PANDEMIC MITIGATION STRATEGIES AND 
INSTRUCTIONS .......................................................................................................80 
APPENDIX F. PROTOCOL AMENDMENT SUMMARY OF CHANGES ............................82 
 
LIST OF TABLES 
Table 1: 
Primary and Secondary Objectives and Endpoints .....................................................11 
Table 2: 
Key Study Design Elements .......................................................................................13 
Table 3: 
Schedule of Activities .................................................................................................16 
Table 4: 
Schedule of Laboratory Assessments .........................................................................17 
Table 5: 
Tumors With Frequency > 1% for FGFR1-3 Mutations and Rearrangements 
in TCGA .....................................................................................................................20 
Table 6: 
In Vitro Evaluation of Pemigatinib as a Potential Perpetrator for Transporter-
Mediated DDIs Based on EMA Guidance ..................................................................23 
Table 7: 
Summary of Treatment-Emergent Adverse Events Occurring in ≥ 10% of 
Total Participants Receiving Pemigatinib Monotherapy for Advanced 
Malignancies ...............................................................................................................24 
Table 8: 
Summary of Serious Treatment-Emergent Adverse Events Occurring in 
≥ 1% of Total Participants Receiving Pemigatinib Monotherapy for 
Advanced Malignancies ..............................................................................................25 


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Table 9: 
Objectives and Endpoints ...........................................................................................27 
Table 10: Exclusionary Laboratory Values ................................................................................33 
Table 11: Study Treatment Information .....................................................................................35 
Table 12: Guidelines for Interruption and Restarting of Study Drug .........................................38 
Table 13: Recommended Approach for Hyperphosphatemia Management ...............................39 
Table 14: ECOG Performance Status .........................................................................................47 
Table 15: Required Laboratory Analytes ....................................................................................51 
Table 16: Populations for Analysis .............................................................................................64 
 
LIST OF FIGURES 
Figure 1: 
Study Design Schema .................................................................................................15 
Figure 2: 
Comparison of Steady-State Exposures for Pemigatinib 13.5 mg QD 
Between Participants With Nonhyperphosphatemia and Hyperphosphatemia 
(C1D8) ........................................................................................................................22 
 


Incyte Corporation 
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Protocol INCB 54828-207 Am 3 
Version 3 
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CONFIDENTIAL 
LIST OF ABBREVIATIONS 
Abbreviations and 
Special Terms 
Definition 
AE 
adverse event 
AJCC 
American Joint Committee on Cancer 
ALT 
alanine aminotransferase 
ANC 
absolute neutrophil count 
aPTT 
activated partial thromboplastin time 
AST 
aspartate aminotransferase 
 
 
CFR 
Code of Federal Regulations 
CNS 
central nervous system 
COVID-19 
coronavirus disease 2019 
CR 
complete response 
CT 
computed tomography 
CTCAE 
Common Terminology Criteria for Adverse Events 
CYP 
cytochrome P450 
 
 
DDI 
drug-drug interaction 
DOR 
duration of response 
ECG 
Electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group 
eCRF 
electronic case report form 
EDC 
electronic data capture 
EMA 
European Medicines Agency 
 
 
EOT 
end of treatment   
 
 
FDA 
Food and Drug Administration 
FGFR 
fibroblast growth factor receptor 
FGFR1-3 
fibroblast growth factor receptors 1, 2, or 3 
GCP 
Good Clinical Practice 
HBV 
hepatitis B virus 


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Abbreviations and 
Special Terms 
Definition 
HCV 
hepatitis C virus 
HIPAA 
Health Insurance Portability and Accountability Act of 1996 
HP 
hyperphosphatemia 
IB 
Investigator's Brochure 
IC50 
concentration that results in 50% inhibition 
ICF 
informed consent form  
ICH 
International Council on Harmonization of Technical Requirements for 
Registration of Pharmaceuticals for Human Use 
ICR 
Independent Central Review 
IEC 
independent ethics committee 
IMP 
investigational medicinal product 
INR 
international normalized ratio 
IRB 
institutional review board 
IRT 
interactive response technology 
MATE 
multidrug and toxin extrusion 
MedDRA 
Medical Dictionary for Regulatory Activities 
MRI 
magnetic resonance imaging 
NCI 
National Cancer Institute 
OAT 
organic anion transporter 
OATP 
organic anion-transporting peptide 
OCT 
optical coherence tomography 
OCT2 
organic cation transporter 2 
ORR 
objective response rate  
OS 
overall survival 
PBPK 
physiologically based pharmacokinetic 
 
 
PFS 
progression-free survival 
P-gp 
p-glycoprotein 
 
 
PMDA 
Pharmaceuticals and Medical Devices Agency 
PR 
partial response  
PT 
prothrombin time 


Incyte Corporation 
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Abbreviations and 
Special Terms 
Definition 
PTH 
parathyroid hormone 
PTT 
partial thromboplastin time 
QD 
once daily 
 
 
RANO 
Response Assessment in Neuro-Oncology 
RECIST v1.1 
Response Evaluation Criteria in Solid Tumors version 1.1 
SAE 
serious adverse event 
SD 
stable disease 
SoA 
schedule of activities 
SOP 
standard operating procedure 
SRD/RPED 
serous retinal detachment/retinal pigmented epithelium detachment 
TEAE 
treatment-emergent adverse event, AEs reported for the first time or worsening 
of a pre-existing event after first dose of study treatment 
ULN 
upper limit of normal 


Incyte Corporation 
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Protocol INCB 54828-207 Am 3 
Version 3 
15 FEB 2021 
VV-CLIN-000652 
CONFIDENTIAL 
1. 
PROTOCOL SUMMARY 
Protocol Title:  A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the Efficacy 
and Safety of Pemigatinib in Participants With Previously Treated Locally Advanced/Metastatic 
or Surgically Unresectable Solid Tumor Malignancies Harboring Activating FGFR Mutations or 
Translocations 
Protocol Number:  INCB 54828-207 
Objectives and Endpoints: 
Table 1 presents the primary, secondary, 
 endpoints and objectives. 
Table 1: 
Primary and Secondary Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
To evaluate the efficacy of pemigatinib in 
participants with locally advanced/metastatic or 
surgically unresectable solid tumor malignancy 
with an activating FGFR mutation or 
fusion/rearrangement.   
ORR in Cohort A, defined as the proportion of 
participants in Cohort A who achieve a CR or PR 
based on RECIST v1.1 or RANO.  
ORR in Cohort B, defined as the proportion of 
participants in Cohort B who achieve a CR or PR 
based on RECIST v1.1 or RANO.   
An independent radiological review committee will 
determine response. 
Secondary 
• To evaluate other clinical efficacy 
measurements of pemigatinib in participants 
with locally advanced/metastatic or surgically 
unresectable solid tumor malignancy with an 
activating FGFR mutation and/or 
fusion/rearrangement. 
• Safety and tolerability of pemigatinib. 
• PFS, defined as the time from first dose until 
progressive disease (according to RECIST v1.1 
or RANO and assessed by an ICR) or death 
(whichever is first) in Cohorts A and B, 
respectively. 
• DOR, defined as the time from the date of first 
assessment of CR or PR until the date of the first 
progressive disease by an ICR per RECIST v1.1 
or RANO, or death (whichever is first) in 
Cohorts A and B, respectively. 
• OS, defined as the time from first dose of study 
drug to death of any cause in Cohorts A and B, 
respectively. 
• Safety and tolerability, as assessed by the 
occurrence of TEAEs and treatment-related AEs 
according to NCI CTCAE v5.0, physical exam 
changes, vital sign changes, laboratory 
evaluations, and ECGs in each cohort. 


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Protocol INCB 54828-207 Am 3 
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CONFIDENTIAL 
Table 1: 
Primary and Secondary Objectives and Endpoints (Continued) 
Objectives 
Endpoints 
Overall Design: 
Table 2 presents the key study design elements.  Further study details are presented after the 
table. 


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Incyte Corporation 
Page 15 of 90 
Protocol INCB 54828-207 Am 3 
Version 3 
15 FEB 2021 
VV-CLIN-000652 
CONFIDENTIAL 
Figure 1: 
Study Design Schema 
Advanced/metastatic/non-resectable solid tumor malignancy
 FGF/FGFR status
Cohort A:  FGFR1-3 in-frame fusions ;any 
FGFR2 rearrangement; FGFR1/3 
rearrangements with known partner
Treatment (21-day cycle; 
continuous dosing)
Disease Assessment 
every 3 cycles
PD
SD, PR, CR
D/C tx; safety 
and survival 
follow up
Continue tx; 
assess per 
protocol
Cohort C:  FGFR1-3 known activating mutations in 
kinase domain; FGFR1-3 other putatively 
activating mutations; FGFR1/3 rearrangements 
(not eligible for cohort A)
Screen
Discontinue
Pass
SF
Cohort B:  known or likely activating mutations 
(excluding kinase domain) in FGFR1-3 
(Appendix C)
*Only FGFR fusions or rearrangements with an intact kinase domain are eligible
 
CR = complete response; D/C = discontinue; FGF = fibroblast growth factor; FGFR = fibroblast growth factor 
receptor; PD = progressive disease; PR = partial response; SD = stable disease; SF = screen failure; tx = treatment. 
Adherence to the study design requirements, including those specified in the SoA (see Table 3 
and Table 4), is essential and required for study conduct. 


Incyte Corporation 
Page 16 of 90 
Protocol INCB 54828-207 Am 3 
Version 3 
15 FEB 2021 
VV-CLIN-000652 
 
CONFIDENTIAL 
Table 3: 
Schedule of Activities 
Visit Day (Range) 
Screening 
Treatment 
EOT 
Follow-Up 
Survival 
Notes 
Days −35 
to −1 
Cycle 1 
Cycles 2+ 
Safety 
Disease 
Day 1 
Day 8 
(± 3 d) 
Day 15 
(± 3 d) 
Day 1 
(± 3 d) 
EOT + 
30-35 d 
Every 
9 wk ± 7 d 
Every 
12 wk 
Administrative procedures 
Informed consent 
X 
 
 
 
 
 
 
 
 
See Section 8.1.1. 
Contact IRT 
X 
X 
X 
X 
X 
X 
 
 
 
 
Inclusion/exclusion criteria 
X 
 
 
 
 
 
 
 
 
 
Prior treatments, procedures, 
surgery for disease 
X 
 
 
 
 
 
 
 
 
 
General and disease medical 
history 
X 
 
 
 
 
 
 
 
 
 
Prior/concomitant medications 
X 
X 
X 
X 
X 
X 
X 
 
 
 
Dispense/administer 
pemigatinib 
 
X 
X 
X 
X* 
 
 
 
 
*Assess for up-titration. 
Collect study drug and review 
diary cards 
 
 
X 
X 
X 
X 
 
 
 
 
Safety assessments 
Slit lamp, visual acuity, 
funduscopy with digital 
imaging (eye) 
X 
 
 
 
X* 
X 
 
 
 
*Once every 3 cycles starting at 
Cycle 3 (± 7 days), as well as 
clinically indicated. 
Optical coherence tomography 
X* 
 
 
 
X* 
X* 
 
 
 
*Required at baseline and every 
3 cycles, as well as when clinically 
indicated. 
AE assessments 
X 
X 
X 
X 
X 
X 
X 
 
 
 
Physical examination 
X 
X 
X 
X 
X 
X 
X 
 
 
 
Vital signs/body weight/height 
X 
X 
 
 
X 
X 
X 
 
 
Height at screening only. 
12-lead ECG 
X 
X 
 
 
X 
X 
X 
 
 
 
ECOG 
X 
X 
 
 
X 
X 
X 
 
 
 
Efficacy assessments 
CT or MRI 
X 
 
 
 
X* 
X** 
 
X 
 
*Once every 3 cycles starting at 
the end of Cycle 3 (± 7 days). 
**Perform at EOT if not done 
within 1 month prior to EOT. 
Survival 
 
 
 
 
 
 
 
 
X 
 


Incyte Corporation 
Page 17 of 90 
Protocol INCB 54828-207 Am 3 
Version 3 
15 FEB 2021 
VV-CLIN-000652 
 
CONFIDENTIAL 
Table 4: 
Schedule of Laboratory Assessments 
Visit Day (Range) 
Screening 
Treatment 
EOT 
Follow-Up 
Survival 
Notes 
Days 
−35 to −1 
Cycle 1 
Cycles 2+ 
Safety 
Disease 
Day 1 
Day 8  
(± 3 d) 
Day 15  
(± 3 d) 
Day 1  
(± 3 d) 
(EOT + 
30-35 d) 
Every 
9 wk ± 7 d 
Every 
12 wk 
Clinical laboratory assessments 
Blood chemistries 
X 
X* 
X 
X 
X** 
X 
X 
 
 
*If screening performed within 
3 days of C1D1, additional sample 
not required. 
**HP in Cycle 1 requires Day 8 
testing of serum phosphate in 
Cycle 2+. 
Hematology 
X 
X* 
 
 
X 
X 
 
 
 
*If screening performed within 
3 days of C1D1, additional sample 
not required. 
Coagulation 
X 
X 
 
 
X 
X 
 
 
 
*If screening performed within 
3 days of C1D1, additional sample 
not required. 
Endocrine (PTH only) 
X 
 
 
 
X* 
X 
 
 
 
*Every 3 cycles on Day 1 starting 
with Cycle 3. 
HBV/HCV testing 
X 
 
 
 
 
 
 
 
 
 
Urinalysis 
X 
 
 
 
 
X 
 
 
 
 
Pregnancy testing  
X* 
X 
 
 
X 
X* 
 
 
 
*Serum. 
 translational laboratory assessments 


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Incyte Corporation 
Page 19 of 90 
Protocol INCB 54828-207 Am 3 
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CONFIDENTIAL 
2. 
INTRODUCTION 
2.1. 
Background 
Pemigatinib is an inhibitor of the FGFR family of receptor tyrosine kinases that is proposed for 
the treatment of solid tumor malignancies with an activating FGFR mutation or translocation 
(fusion/rearrangement).  Aberrant signaling through FGFR resulting from gene mutations or 
chromosomal fusions/rearrangements has been demonstrated in multiple types of human cancers.  
Fibroblast growth factor receptor signaling contributes to the development of malignancies by 
promoting tumor cell proliferation, survival, migration, and angiogenesis.  Incyte is proposing to 
study pemigatinib for the treatment of solid tumor malignancies harboring an activating FGFR 
mutation or fusion/rearrangement. 
2.1.1. 
Fibroblast Growth Factor Receptor Inhibitor in Oncology 
The mammalian FGFR family is composed of 4 highly conserved receptors (FGFR1, FGFR2, 
FGFR3, and FGFR4) that have an extracellular ligand-binding domain, a single transmembrane 
domain, and an intracellular tyrosine kinase domain.  Eighteen FGR ligands, divided into 
canonical and hormonal FGFRs, bind to FGFRs, leading to receptor dimerization, activation of 
the kinase domain, and transphosphorylation of the receptors (Eswarakumar et al 2005).  
Subsequent signal transduction occurs through phosphorylation of substrate proteins, such as 
FGFR substrate 2, which leads to activation of the RAS-mitogen–activated protein kinase and 
phosphoinositide 3-kinase–protein kinase B pathways and of the phospholipase Cγ that activates 
the protein kinase C pathway.  In some cellular context, signal transducer and activator of 
transcription proteins are also activated by FGFRs.  Signaling through the FGF-FGFR pathway is 
tightly controlled through feedback regulation.  Mitogen-activated protein kinase phosphatases 
and Sprouty proteins are upregulated upon FGFR stimulation and antagonize FGF-dependent 
activation of extracellular signal-regulated kinases.  In many cases, FGFR pathway activation 
promotes cell proliferation, survival, and migration; however, cellular context plays an important 
role, and in certain tissues, FGFR signaling results in growth arrest and cellular differentiation 
(Dailey et al 2005). 
In adults, FGF-FGFR signaling is involved in angiogenesis during wound healing.  The 
hormonal FGF ligands contribute to regulation of metabolic pathways involving lipid, glucose, 
phosphate, and vitamin D (Itoh 2010).  Genetic defects in the FGF23-signaling pathway lead to 
disordered phosphate metabolism:  loss of function mutations in FGF23 or its signaling results in 
retention of phosphate and tissue mineralizing, while gain of function mutations in the FGF23 
pathway manifests as hypophosphatemic rickets syndrome (Farrow and White 2010). 
Strong genetic and functional evidence supports that dysregulation of FGFR can lead to the 
establishment and progression of cancer.  Genetic alterations in FGFR-3 have been described in 
many tumor types (Knights and Cook 2010, Turner and Grose 2010).  These include activating 
mutations, translocations, and gene amplification resulting in ligand-independent constitutive 
activation of the receptors or aberrant ligand-dependent signaling through FGFRs. 
A substantial body of evidence supports that a genetically activated FGFR pathway sensitizes 
FGFR-altered cancer cells to knockdown or inhibition of these receptors (Kunii et al 2008, 
Qing et al 2009, Weiss et al 2010, Lamont et al 2011).  A large screen of more than 500 tumor 


Incyte Corporation 
Page 20 of 90 
Protocol INCB 54828-207 Am 3 
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15 FEB 2021 
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CONFIDENTIAL 
cell lines with a selective FGFR inhibitor demonstrated that only a small percentage (5.9%) of all 
cells are sensitive to FGFR inhibition, and growth-suppressed cell lines were highly enriched for 
FGFR alterations (Guagnano et al 2012).  These results demonstrate that FGFR inhibitors are 
active in a targeted manner against cancers with activated FGFR pathway.  An implication of 
these data is that selection based on molecular-, genetic-, or protein-based diagnostic tests for 
specific FGFR alterations in tumors may be important for identifying patients most likely to 
benefit from an FGFR inhibitor. 
2.1.2. 
Solid Tumor Malignancies With Activating FGFR Mutations and 
Fusions/Rearrangements 
Activation of the FGFR pathway by mutation or rearrangement is well-recognized as an 
oncogenic event in cancer.  A recent survey of 4853 solid tumors identified FGFR1-3 mutations 
or rearrangements in 2.25% of all tumors tested (Helsten et al 2016).  Analysis of FGFR1-3 
mutations and rearrangements in the TCGA database (NCI 2018), demonstrate that FGFR 
alterations are not restricted to one tumor type, but rather broadly distributed across a diverse 
range of tumors (see Table 5).  Importantly, preclinical and clinical data support the use of FGFR 
inhibitors in FGFR altered cell lines and tumors derived from a variety of cancer types, including 
bladder cancer, cholangiocarcinoma, endometrial cancer, and glioma and squamous lung cancer 
(Porta et al 2017). 
Table 5: 
Tumors With Frequency > 1% for FGFR1-3 Mutations and Rearrangements 
in TCGA 
Tumor Type 
FGFR1 
FGFR2 
FGFR3 
Mutation 
(%) 
Fusion 
(%) 
Mutation 
(%) 
Fusion 
(%) 
Mutation 
(%) 
Fusion 
(%) 
Total 
(%) 
Urothelial carcinoma 
0 
0 
0.75 
0 
12.64 
3.15 
16.54 
Cholangiocarcinoma 
0 
0 
0 
13.9 
0 
0 
13.9 
Endometrial carcinoma 
0 
0 
9.68 
0.18 
0 
0 
9.86 
Glioblastoma multiforme 
0.48 
0 
0 
0 
0.48 
8.62 
9.58 
Lung squamous cell carcinoma 
0.21 
0.2 
1.45 
0.2 
1.45 
1.4 
4.91 
Cervical carcinoma 
0 
0 
0.51 
0 
0 
1.97 
2.48 
Renal papillary cell carcinoma 
0 
0 
0 
0 
1.41 
0.68 
2.09 
Rectal adenocarcinoma 
0 
0 
2.01 
0 
0 
0 
2.01 
Head and neck squamous cell 
carcinoma 
0 
0 
0 
0 
1.14 
0.76 
1.9 
Low grade glioma 
0.19 
0 
0 
0 
0 
1.71 
1.9 
Uterine carcinosarcoma 
0 
0 
1.75 
0 
0 
0 
1.75 
Esophageal adenocarcinoma 
0 
0.54 
0 
0 
0 
1.08 
1.62 
Stomach adenocarcinoma 
0.25 
0 
0.5 
0.48 
0.25 
0 
1.48 
Prostate adenocarcinoma 
0 
0 
0 
1.2 
0 
0.2 
1.4 
Breast (invasive) carcinoma 
0 
0.36 
0.6 
0.36 
0 
0 
1.32 
Adrenocortical carcinoma 
0 
0 
1.1 
0 
0 
0 
1.1 
Pheochromocytoma and paraganglioma 
1.09 
0 
0 
0 
0 
0 
1.09 
Colorectal adenocarcinoma 
0.27 
0 
0.54 
0 
0.27 
0 
1.08 
Skin cutaneous melanoma 
0.21 
0 
0.84 
0 
0 
0 
1.05 


Incyte Corporation 
Page 21 of 90 
Protocol INCB 54828-207 Am 3 
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Based on these observations, an FGFR inhibitor may be active across a variety of tumor types 
with activating mutations and gene fusions of FGFR1-3. 
2.2. 
Study Rationale 
Pemigatinib is a potent selective inhibitor of FGFR1-3.  This compound is proposed for the 
treatment of participants with advanced/metastatic or surgically unresectable solid tumor 
malignancies harboring activating FGFR mutations or fusions/rearrangements.   
2.2.1. 
Scientific Rationale for Study Design 
The planned study will evaluate the efficacy and safety of pemigatinib as treatment in 
participants with advanced/metastatic or unresectable solid tumor malignancy with an activating 
FGFR mutation or fusion/rearrangement.  Preliminary data from the ongoing Phase 1 study 
(INCB 54828-101) in second-line or greater treatment in all tumor types have shown interesting 
efficacy signals (ORR, PFS) and tolerable safety in participants with activating FGFR 
alterations.   
The proposed study design is a single arm, open-label study of pemigatinib.  An independent 
central radiology group will evaluate tumor response to support the primary and secondary 
endpoints to minimize bias. 
2.2.2. 
Justification for Dose and Regimen 
Pemigatinib will be administered at 13.5 mg QD, continuously, for a cycle.  Each cycle is 
3 weeks.  This dose and dosing schedule was selected based on emerging clinical and safety data 
from the INCB 54828-101 study, where the continuous dosing regimen was tested and has been 
tested in 12 and 14 participants at 9 mg and 13.5 mg QD continuous administration, respectively.  
In Study INCB 54828-101, the emerging safety data demonstrated that tolerability of the 
continuous dosing regimen of pemigatinib was comparable to that of intermittent dosing. 
The hypothesis with a targeted therapy is that continued inhibition of the aberrant receptor may 
increase the potential for benefit from the treatment.  Therefore, administering pemigatinib 
continuously will allow that consistent inhibition of the aberrant FGFR receptor in this 
population.  Continuous dosing would still allow for dose holds for safety reasons with criteria 
and procedures for dose interruptions and adjustments clearly outlined in the Protocol. 
Hyperphosphatemia is an expected on-target pharmacological effect of FGFR inhibition.  The 
incidence of hyperphosphatemia, defined as any postbaseline phosphate level exceeding 
5.5 mg/dL, has been observed in the majority of study participants treated with pemigatinib 
(refer to the IB for complete data).  Some participants do not achieve hyperphosphatemia, and it 
is estimated that pharmacological concentration of pemigatinib in these participants is lower (see 
Figure 2).  Therefore, up-titration of pemigatinib will be used to increase the exposure of 
pemigatinib in participants who do not achieve hyperphosphatemia when treated with 13.5 mg 
QD.  The goal is to increase the serum concentration of pemigatinib. 
The increase in serum phosphorus observed after treatment with pemigatinib was 
exposure-dependent and followed a sigmoid relationship.  A population Emax model of 
pemigatinib AUC and maximal serum phosphate change from baseline was developed.  For 
those participants treated with pemigatinib 13.5 mg who did not develop hyperphosphatemia, 


Incyte Corporation 
Page 22 of 90 
Protocol INCB 54828-207 Am 3 
Version 3 
15 FEB 2021 
VV-CLIN-000652 
CONFIDENTIAL 
AUC for pemigatinib 18 mg was estimated using a linear exposure relationship.  Maximal serum 
phosphate change from baseline for each individual was then estimated using a population 
model.  The maximal serum phosphate after treatment with pemigatinib 18 mg was calculated by 
adding the baseline of serum phosphate.  The simulation suggested that the serum phosphate 
would increase above 5.5 mg/dL after treatment with pemigatinib 18 mg for the participants 
treated with pemigatinib 13.5 mg who did not develop hyperphosphatemia. 
Figure 2: 
Comparison of Steady-State Exposures for Pemigatinib 13.5 mg QD Between 
Participants With Nonhyperphosphatemia and Hyperphosphatemia (C1D8) 
 
AUCss = area under the curve at steady state; HP = hyperphosphatemia.  
Any participant who does not reach the target serum phosphate level of > 5.5 mg/dL at any time 
during Cycle 1, and who is compliant with taking study drug and does not experience an ongoing 
Grade 2 or higher treatment-related AEs, will increase the daily dose to 18 mg starting at Cycle 2 
Day 1. 
A CYP3A4-mediated drug-drug interaction study (INCB 54828-104) indicated there is evidence 
of a clinically significant effect on pemigatinib exposure when coadministered with a potent 
CYP3A4 inhibitor, itraconazole (increased pemigatinib AUC by 88%) or potent CYP3A4 
inducer, rifampin (decreased pemigatinib AUC by 85%).  A PBPK model was developed and 
validated using in vitro and clinical DDI data.  PBPK model–simulated pemigatinib AUCs were 
increased by approximately 50% for moderate CYP3A4 inhibitors and decreased by more than 


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Table 7: 
Summary of Treatment-Emergent Adverse Events Occurring in ≥ 10% of 
Total Participants Receiving Pemigatinib Monotherapy for Advanced 
Malignancies 
MedDRA Preferred Term, n (%) 
ID 
(N = 404) 
CD 
(N = 261) 
Total a 
(N = 665) 
Hyperphosphataemia 
212 (52.5) 
182 (69.7) 
394 (59.2) 
Diarrhoea 
174 (43.1) 
103 (39.5) 
277 (41.7) 
Alopecia 
174 (43.1) 
100 (38.3) 
274 (41.2) 
Stomatitis 
146 (36.1) 
125 (47.9) 
271 (40.8) 
Dysgeusia 
134 (33.2) 
94 (36.0) 
228 (34.3) 
Fatigue 
152 (37.6) 
75 (28.7) 
227 (34.1) 
Dry mouth 
128 (31.7) 
97 (37.2) 
225 (33.8) 
Constipation 
137 (33.9) 
85 (32.6) 
222 (33.4) 
Decreased appetite 
127 (31.4) 
71 (27.2) 
198 (29.8) 
Nausea 
131 (32.4) 
67 (25.7) 
198 (29.8) 
Dry eye 
84 (20.8) 
56 (21.5) 
140 (21.1) 
Vomiting 
90 (22.3) 
44 (16.9) 
134 (20.2) 
Anaemia 
71 (17.6) 
54 (20.7) 
125 (18.8) 
Dry skin 
70 (17.3) 
49 (18.8) 
119 (17.9) 
Arthralgia 
71 (17.6) 
43 (16.5) 
114 (17.1) 
Asthenia 
60 (14.9) 
54 (20.7) 
114 (17.1) 
Abdominal pain 
79 (19.6) 
31 (11.9) 
110 (16.5) 
Urinary tract infection 
69 (17.1) 
37 (14.2) 
106 (15.9) 
Back pain 
69 (17.1) 
35 (13.4) 
104 (15.6) 
Blood creatinine increased 
50 (12.4) 
52 (19.9) 
102 (15.3) 
Palmar-plantar erythrodysaesthesia syndrome 
40 (9.9) 
55 (21.1) 
95 (14.3) 
Weight decreased 
60 (14.9) 
32 (12.3) 
92 (13.8) 
Pain in extremity 
54 (13.4) 
36 (13.8) 
90 (13.5) 
Hypophosphataemia 
65 (16.1) 
19 (7.3) 
84 (12.6) 
Epistaxis 
46 (11.4) 
32 (12.3) 
78 (11.7) 
Hypercalcaemia 
47 (11.6) 
29 (11.1) 
76 (11.4) 
Oedema peripheral 
43 (10.6) 
31 (11.9) 
74 (11.1) 
Hyponatraemia 
40 (9.9) 
32 (12.3) 
72 (10.8) 
Pyrexia 
50 (12.4) 
21 (8.0) 
71 (10.7) 
Dehydration 
43 (10.6) 
24 (9.2) 
67 (10.1) 
Onycholysis 
36 (8.9) 
31 (11.9) 
67 (10.1) 
CD = continuous dosing; ID = intermittent dosing. 
Note:  Participants were counted only once under each MedDRA preferred term. 
Note:  MedDRA preferred terms were presented in descending order of frequency using the total column. 
a Includes participants from Studies INCB 54828-101, -102, -201, -202, and -207. 
Table 8 presents a summary of serious TEAEs by preferred term that occurred in ≥ 1% of total 
participants with advanced malignancies who received pemigatinib monotherapy.  In the total 
population, 297 participants (44.7%) who received monotherapy had a serious TEAE.  In 
participants who received intermittent dosing, 184 participants (45.5%) had a serious TEAE; in 


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participants who received continuous dosing, 113 participants (43.3%) had a serious TEAE.  In 
the total population, the most frequent serious TEAEs were urinary tract infection 
(21 participants [3.2%]) and abdominal pain and acute kidney injury (17 participants [2.6%] 
each).  The most frequent serious TEAEs that were considered to be related to pemigatinib by 
the investigator were hyponatremia (4 participants [0.6%]), fatigue and anemia (3 participants 
[0.5%] each), and diarrhea, stomatitis, nausea, and hypercalcemia (2 participants [0.3%] each). 
Table 8: 
Summary of Serious Treatment-Emergent Adverse Events Occurring in 
≥ 1% of Total Participants Receiving Pemigatinib Monotherapy for 
Advanced Malignancies 
MedDRA Preferred Term, n (%) 
ID 
(N = 404) 
CD 
(N = 261) 
Totala 
(N = 665) 
Urinary tract infection 
10 (2.5) 
11 (4.2) 
21 (3.2) 
Abdominal pain 
12 (3.0) 
5 (1.9) 
17 (2.6) 
Acute kidney injury 
10 (2.5) 
7 (2.7) 
17 (2.6) 
Pyrexia 
13 (3.2) 
2 (0.8) 
15 (2.3) 
General physical health deterioration 
7 (1.7) 
8 (3.1) 
15 (2.3) 
Back pain 
7 (1.7) 
8 (3.1) 
15 (2.3) 
Pneumonia 
10 (2.5) 
4 (1.5) 
14 (2.1) 
Hyponatraemia 
8 (2.0) 
5 (1.9) 
13 (2.0) 
Pleural effusion 
9 (2.2) 
2 (0.8) 
11 (1.7) 
Disease progression 
5 (1.2) 
6 (2.3) 
11 (1.7) 
Cholangitis 
8 (2.0) 
2 (0.8) 
10 (1.5) 
Small intestinal obstruction 
9 (2.2) 
1 (0.4) 
10 (1.5) 
Anaemia 
5 (1.2) 
4 (1.5) 
9 (1.4) 
Constipation 
6 (1.5) 
3 (1.1) 
9 (1.4) 
Sepsis 
6 (1.5) 
3 (1.1) 
9 (1.4) 
Fatigue 
4 (1.0) 
4 (1.5) 
8 (1.2) 
Haematuria 
8 (2.0) 
0 
8 (1.2) 
Blood creatinine increased 
4 (1.0) 
3 (1.1) 
7 (1.1) 
Decreased appetite 
4 (1.0) 
3 (1.1) 
7 (1.1) 
Dehydration 
4 (1.0) 
3 (1.1) 
7 (1.1) 
Dyspnoea 
4 (1.0) 
3 (1.1) 
7 (1.1) 
Hypercalcaemia 
5 (1.2) 
2 (0.8) 
7 (1.1) 
Vomiting 
5 (1.2) 
2 (0.8) 
7 (1.1) 
CD = continuous dosing; ID = intermittent dosing. 
Note:  Participants were counted only once under each MedDRA preferred term. 
Note:  MedDRA preferred terms were presented in descending order of frequency using the total column. 
a Includes participants from Studies INCB 54828-101, -102, -201, -202, and -207. 
In the total population, serious TEAEs with a fatal outcome occurred in 64 participants (9.6%).  
In participants who received intermittent dosing, TEAEs with a fatal outcome occurred in 
37 participants (9.2%); in participants who received continuous dosing, TEAEs with a fatal 
outcome occurred in 27 participants (10.3%).  In the total population, fatal TEAEs that occurred 
in > 2 participants were general physical health deterioration (15 participants [2.3%]), disease 
progression (10 participants [1.5%]), malignant neoplasm progression and sepsis (4 participants 
[0.6%] each), and dyspnea and failure to thrive (3 participants [0.5%] each).   
In the total population, 71 participants (10.7%) discontinued pemigatinib treatment because of a 
TEAE.  In participants who received intermittent dosing, 39 participants (9.7%) discontinued 


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pemigatinib treatment because of a TEAE; in participants who received continuous dosing, 
32 participants (12.3%) discontinued pemigatinib treatment because of a TEAE.  In the total 
population, the most frequent TEAEs that led to discontinuation of pemigatinib were disease 
progression (5 participants [0.8%]), general physical health deterioration (4 participants [0.6%]), 
and sepsis, acute kidney injury, and intestinal obstruction (3 participants [0.5%] each).  In 
participants who received intermittent dosing, the most frequent TEAEs that led to 
discontinuation of pemigatinib were acute kidney injury (3 participants [0.7%]) and pneumonia, 
general physical health deterioration, small intestinal obstruction, malignant neoplasm 
progression, disease progression, and intestinal obstruction (2 participants [0.5%] each).  In 
participants who received continuous dosing, the most frequent TEAEs that led to 
discontinuation of pemigatinib were disease progression (3 participants [1.1%]) and stomatitis, 
general physical health deterioration, and sepsis (2 participants [0.8%] each). 
There were no dose-limiting toxicities in participants receiving monotherapy. 
More detailed information about the known and expected benefits and risks (clinical safety and 
nonclinical toxicology) and reasonably expected AEs of pemigatinib are provided in the IB. 


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3. 
OBJECTIVES AND ENDPOINTS 
Table 9 presents the objectives and endpoints. 
Table 9: 
Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
To evaluate the efficacy of pemigatinib in 
participants with locally advanced/metastatic or 
surgically unresectable solid tumor malignancy 
with an activating FGFR mutation or 
fusion/rearrangement. 
ORR in Cohort A, defined as the proportion of 
participants in Cohort A who achieve a CR or PR 
based on RECIST v1.1 or RANO.  
ORR in Cohort B, defined as the proportion of 
participants in Cohort B who achieve a CR or PR 
based on RECIST v1.1 or RANO.   
An independent radiological review committee will 
determine response. 
Secondary 
• To evaluate other clinical efficacy 
measurements of pemigatinib in participants 
with locally advanced/metastatic or surgically 
unresectable solid tumor malignancy with an 
activating FGFR mutation and/or 
fusion/rearrangement. 
• Safety and tolerability of pemigatinib. 
• PFS, defined as the time from first dose until 
progressive disease (according to RECIST v1.1 
or RANO and assessed by an ICR) or death 
(whichever is first) in Cohorts A and B, 
respectively. 
• DOR, defined as the time from the date of first 
assessment of CR or PR until the date of the first 
progressive disease by an ICR per RECIST v1.1 
or RANO, or death (whichever is first) in 
Cohorts A and B, respectively. 
• OS, defined as the time from first dose of study 
drug to death of any cause in Cohorts A and B, 
respectively. 
• Safety and tolerability, as assessed by the 
occurrence of TEAEs and treatment-related AEs 
according to NCI CTCAE v5.0, physical exam 
changes, vital sign changes, laboratory 
evaluations, and ECGs in each cohort. 


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Table 9: 
Objectives and Endpoints (Continued) 
Objectives 
Endpoints 
4. 
STUDY DESIGN 
4.1. 
Overall Design 
This is an open-label, monotherapy study of pemigatinib in participants with advanced/metastatic 
or surgically unresectable solid tumor malignancy harboring an activating FGFR mutation or 
fusion/rearrangement.  This study consists of 3 cohorts, Cohort A, Cohort B, and Cohort C and 
will enroll approximately 60 participants, 90 participants, and 20 participants, respectively.  
Participants will receive a QD dose of pemigatinib at 13.5 mg continuously as long as 
participants are receiving benefit and have not met any criteria for study withdrawal.  
Participants with local laboratory test results (eg, tissue or plasma-based molecular assays) 
documenting an activating FGFR1, FGFR2, or FGFR3 mutation or gene rearrangement are 
eligible to enroll as long as the results meet the cohort criteria.  Confirmatory testing through the 
central genomics laboratory will be performed for all participants; however, results from the 
central genomics laboratory are not required before enrollment.  Patients who have a commercial 
report at screening from the central laboratory (Foundation Medicine, Inc) within 24 months of 
screening will not need to send a sample for confirmatory testing (a biopsy will still be required 
for other testing).  Centralized genomic testing results will allow participants to be assigned to a 
cohort (see Appendix C for list of alterations): 
• Cohort A:  FGFR1-3 in-frame fusions; any FGFR2 rearrangement; FGFR1/3 
rearrangement with known partner* (n = 60) 
• Cohort B:  Known or likely activating mutations (excluding kinase domain) in 
FGFR1-3 included in Appendix C (n = 90) 


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The sponsor may terminate the study electively, if required by regulatory decision.  If the study 
is terminated prematurely, the sponsor will notify the investigators/head of study site (Japan), the 
IRBs and IECs, and regulatory bodies of the decision and reason for termination of the study. For 
Japan, the head of study sites will notify the investigators and the IRBs of the decision and 
reason for termination of the study. 
5.
STUDY POPULATION
Deviations from eligibility criteria are not allowed because they can potentially jeopardize the 
scientific integrity of the study, regulatory acceptability, and/or participant safety.  Therefore, 
adherence to the criteria as specified in the Protocol is essential.  Prospective approval of 
Protocol deviations to recruitment and enrollment criteria, also known as Protocol waivers or 
exemptions, are not permitted. 
5.1. 
Inclusion Criteria 
Participants are eligible to be included in the study only if all of the following criteria apply: 
1. Age 18 years or older, inclusive at the time of signing the informed consent; a legally 
minor participant from Japan needs written parental or legal guardian's consent.
2. Histologically or cytologically confirmed solid tumor malignancy that is advanced or 
metastatic (Stage IIIB or IV per the AJCC Cancer Staging Manual, 8th ed; AJCC 2018) 
or is surgically unresectable.
3. Radiographically measurable disease (per RECIST v1.1 or RANO for primary brain 
tumors).  Tumor lesions located in a previously irradiated area, or in an area subjected to 
other loco-regional therapy, are considered measureable if progression has been clearly 
demonstrated in the lesion.
4. Documentation of an FGFR1-3 gene mutation or fusion/rearrangement (see Section 8.5.1 
and Appendix C).  Participants will be assigned to 1 of  3 cohorts:
•
Cohort A:  FGFR1-3 in-frame fusions; any FGFR2 rearrangement; FGFR1/3 
rearrangement with known partner* (n = 60)
•
Cohort B:  Known or likely activating mutations (excluding kinase domain) in 
FGFR1-3 included in Appendix C (n = 90)
•
Cohort C:  FGFR1-3 known activating mutations in kinase domain; FGFR1-3 
putatively activating mutations; other FGFR1/3 rearrangements* (not eligible for 
Cohort A; n = 20) 
*Only FGFR fusions or rearrangements with an intact kinase domain are eligible.
5. Must have objective progression after at least 1 prior therapy, and must have no therapy
available that is likely to provide clinical benefit.  Participants who are intolerant to or
decline the approved therapy are eligible only if they have no therapy available that is
likely to provide clinical benefit.
6. ECOG performance status 0 to 2.


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7. Baseline archival tumor specimen (if less than 24 months from date of screening) or 
willingness to undergo a pretreatment tumor biopsy to obtain the specimen.  Must be a 
tumor block or approximately 15 unstained slides from biopsy or resection of primary 
tumor or metastasis. 
8. Willingness to avoid pregnancy or fathering children based on the criteria below. 
a. Men must agree to take appropriate precautions to avoid fathering children (with at 
least 99% certainty) from screening through 90 days after the last dose of study 
drug(s)/treatment and must refrain from donating sperm during this period.  Permitted 
methods that are at least 99% effective in preventing pregnancy (see Appendix A) 
should be communicated to the participants and their understanding confirmed.  
b. Women of childbearing potential must have a negative serum pregnancy test at 
screening and before the first dose on Day 1 and must agree to take appropriate 
precautions to avoid pregnancy (with at least 99% certainty) from screening through 
safety follow-up.  Permitted methods that are at least 99% effective in preventing 
pregnancy (see Appendix A) should be communicated to the participants and their 
understanding confirmed. 
c. Women of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or 
bilateral oophorectomy OR ≥ 12 months of amenorrhea) are eligible. 
9. For Japanese participants, willingness to avoid pregnancy or fathering children based on 
the criteria below. 
a. Female participants should agree to use medically acceptable contraceptive measures 
(see Appendix A), should not be breastfeeding, and must have a negative pregnancy 
test before the start of study treatment administration if of childbearing potential.  All 
female participants of childbearing potential must understand and accept that 
pregnancy must be avoided during participation in the study from screening through 
30 days after the last dose of pemigatinib (based on menstrual cycle). 
 
OR 
 
Female participants must have evidence of nonchildbearing potential by fulfilling 1 of 
the following criteria at screening: 
− Postmenopausal, defined as aged > 50 years and amenorrheic for at least 
12 months after cessation of all exogenous hormonal treatments. 
Note:  Female participants who have been amenorrheic for at least 12 months 
resulting from chemotherapy/radiotherapy are considered of childbearing 
potential and should agree to use adequate contraceptive measures. 
− Documentation of irreversible surgical sterilization by hysterectomy, bilateral 
oophorectomy, or bilateral salpingectomy but not tubal ligation. 
b. Male participants should avoid unprotected sex with females of childbearing potential 
during the study and for a washout period of 90 days after the last dose of 
pemigatinib.  Male participants should refrain from donating sperm from the start of 
the study treatment administration until 90 days after discontinuing study treatment. 


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5.2. 
Exclusion Criteria 
Participants are excluded from the study if any of the following criteria apply: 
1. Prior receipt of a selective FGFR inhibitor. 
2. Receipt of anticancer medications or investigational drugs for any indication or reason 
within 28 days before first dose of pemigatinib.  Participants must have recovered 
(≤ Grade 1, as per CTCAE v5.0, or at pretreatment baseline) from AEs from previously 
administered therapies (excluding alopecia). 
3. Concurrent anticancer therapy (eg, chemotherapy, radiation therapy, surgery, 
immunotherapy, biologic therapy, hormonal therapy, investigational therapy, or tumor 
embolization).  
4. Candidate for potentially curative surgery. 
5. Current evidence of clinically significant corneal (including, but not limited to, 
bullous/band keratopathy, corneal abrasion, inflammation/ulceration, 
keratoconjunctivitis) or retinal disorder (including, but not limited to, macular/retinal 
degeneration, diabetic retinopathy, retinal detachment) as confirmed by ophthalmologic 
examination. 
6. Radiation therapy administered within 2 weeks of enrollment/first dose of study 
treatment.  Participants must have recovered from all radiation-related toxicities, not 
require corticosteroids, and not have had radiation pneumonitis.  Evidence of fibrosis 
within a radiation field from prior radiotherapy is permitted with medical monitor 
approval.  A 1-week washout is permitted for palliative radiation to non-CNS disease. 
7. Untreated brain or CNS metastases or brain or CNS metastases that have progressed 
(eg, evidence of new or enlarging brain metastasis or new neurological symptoms 
attributable to brain or CNS metastases).  Participants who have previously treated and 
clinically stable brain or CNS metastases are eligible if there is no evidence of 
progression for at least 4 weeks after CNS-directed treatment, as ascertained by clinical 
examination and brain imaging (MRI or CT scan) during the screening period, and if they 
are on a stable or decreasing dose of corticosteroids for at least 1 week.  
Note:  Participants with progressing primary brain tumors are allowed if they have no 
new neurological symptoms and are on a stable or decreasing dose of corticosteroids for 
at least 1 week. 
8. Known additional malignancy that is progressing or requires active treatment.  
Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, 
or in situ cervical cancer that has undergone potentially curative therapy. 
9. Participants with laboratory values at screening defined in Table 10. 


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Table 10: 
Exclusionary Laboratory Values 
Laboratory Parameter 
Exclusion Criterion 
Hematology 
a 
Platelets 
≤ 75 × 109/L (transfusion allowed with a 2-week washout 
period) 
b 
Hemoglobin 
≤ 9.0 g/dL (transfusion allowed with a 2-week washout 
period) 
c 
ANC 
 ≤ 1.5 × 109/L 
Hepatic 
e 
ALT 
≥ 3 × ULN (> 5 × ULN for liver metastasis) 
f 
AST 
≥ 3 × ULN (> 5 × ULN for liver metastasis) 
g 
Total bilirubin 
≥ 1.5 × ULN  (≥ 2.5 × ULN if Gilbert's syndrome or liver 
metastasis) 
h 
Alkaline phosphatase 
≥ 3 × ULN 
Renal 
i 
Serum creatinine clearance 
 ≤ 30 mL/minute based on Cockcroft-Gault formula. 
Chemistry 
j 
Serum phosphate 
> ULN 
k 
Serum calcium 
Outside of normal range or serum albumin-corrected calcium 
outside of the normal range when serum albumin is outside of 
the normal range. 
10. History of calcium and phosphate hemostasis disorder or systemic mineral imbalance 
with ectopic calcification of soft tissues (exception:  commonly observed calcifications in 
soft tissues such as the skin, kidney tendon, or vessels due to injury, disease, or aging in 
the absence of systemic mineral imbalance). 
11. Significant gastrointestinal disorder(s) that could interfere with absorption, metabolism, 
or excretion of pemigatinib. 
12. Inability to swallow and retain oral medication. 
13. Clinically significant or uncontrolled cardiac disease, including unstable angina, acute 
myocardial infarction within 6 months from Day 1 of study drug/treatment 
administration, New York Heart Association Class III or IV congestive heart failure, and 
uncontrolled arrhythmia (participants with pacemaker or with atrial fibrillation and 
well-controlled heart rate are allowed). 
14. History or presence of an abnormal ECG that, in the investigator's opinion, is clinically 
meaningful.  A screening QTcF interval > 480 ms is excluded.  For participants with an 
intraventricular conduction delay (QRS interval > 120 ms), the JTc interval may be used 
in place of the QTc with medical monitor approval.  The JTc must be ≤ 340 ms if JTc is 
used in place of the QTc. 


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15. Active chronic or current infectious disease requiring systemic antibiotic, antifungal, or 
antiviral treatment within 2 weeks before enrollment (participants with asymptomatic 
chronic infections on prophylactic treatment are allowed). 
16. Evidence of active HBV or HCV infection (defined as participants with elevated 
transaminases or cirrhosis.  Participants with chronic HBV/HCV infection with no 
cirrhosis and no elevated transaminases are allowed). 
17. Known HIV infection. 
18. Current use of prohibited medication as described in Section 6.6.2. 
19. Use of any potent CYP3A4 inhibitors or inducers or moderate CYP3A4 inducers within 
14 days or five half-lives (whichever is longer) before the first dose of study 
drug/treatment.  Note that moderate CYP3A4 inhibitors are not prohibited. 
20. Known hypersensitivity or severe reaction to pemigatinib or excipients of pemigatinib 
(refer to the IB).  
21. Inability or unlikeliness of the participant to comply with the dose schedule and study 
evaluations, in the opinion of the investigator. 
22. Inadequate recovery from toxicity and/or complications from a major surgery before 
starting therapy. 
23. Women who are pregnant or breastfeeding. 
24. Any condition that would, in the investigator's judgment, interfere with full participation 
in the study, including administration of study drug/treatment and attending required 
study visits; pose a significant risk to the participant; or interfere with interpretation of 
study data. 
25. Inability of the participant (or parent, guardian, or legally authorized representative) to 
comprehend the ICF or unwillingness to sign the ICF. 
26. History of hypovitaminosis D requiring supraphysiologic doses (eg, 50,000 UI/weekly) to 
replenish the deficiency.  Vitamin D supplements are allowed. 
5.3. 
Lifestyle Considerations 
Based on the preliminary results from food-effect cohort in Study INCB 54828-101, no 
significant food effect was observed.  Pemigatinib may be administrated with or without food. 
Participants should refrain from consumption of red wine, Seville oranges, grapefruit or 
grapefruit juice, pomelos, exotic citrus fruits, grapefruit hybrids, or fruit juices for 7 days before 
the start of study treatment until after the final dose, as this can affect the metabolism of the 
study drug. 
5.4. 
Screen Failures 
Screen failures are defined as participants who consent to participate in the clinical study but are 
not subsequently entered in the study.  Individuals who do not meet the criteria for participation 
in this study (screen failure) may be rescreened no more than 1 time. 


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Tests with results that fail eligibility requirements may be repeated once during screening if the 
investigator believes the result to be in error.  Additionally, a participant who fails screening may 
repeat the screening process 1 time if the investigator believes that there has been a change in 
eligibility status.  Participants who rescreen must reconsent and be assigned a new participant 
number. 
5.5. 
Replacement of Participants 
No participants will be replaced at any time during this study. 
6. 
STUDY TREATMENT 
6.1. 
Study Treatment Administered 
Table 11 and Appendix B present the study treatment information.  The starting dose of 
pemigatinib for all participants is 13.5 mg QD. 
Table 11: 
Study Treatment Information 
 
Study Treatment 1 
Study Treatment 2  
(Phosphate binder, only for Japan) 
Study treatment 
name: 
Pemigatinib 
Bixalomer 
Lanthanum carbonate 
hydrate 
Dosage 
formulation: 
Tablet 
Capsule 
Tablet 
Unit dose 
strength(s) 
/dosage level(s): 
13.5 mg, 9 mg, and 4.5 mg 
250 mg 
250 mg 
Route of 
administration:  
Oral 
Oral 
Oral 
Administration 
instructions: 
One tablet taken every 
morning (unless otherwise 
directed) 
The usual starting dose of 
bixalomer for adults is 
500 mg 3 times daily just 
before meals.  The dose can 
be adjusted based on 
symptoms and serum 
phosphate concentration.  
The maximum daily dose 
should not exceed 7500 mg. 
The usual starting dose of 
lanthanum carbonate hydrate 
for adults is 750 mg 3 times 
daily immediately after 
meals.  The dose can be 
adjusted based on symptoms 
and serum phosphate 
concentration.  The 
maximum daily dose should 
not exceed 2250 mg. 
Packaging and 
labeling: 
Pemigatinib will be 
provided in bottles; each 
bottle will be labeled as 
required per country 
requirement. 
Bixalomer will be labeled as 
required per country 
requirement. 
Lanthanum carbonate 
hydrate will be labeled as 
required per country 
requirement. 
Storage: 
Room temperature 
(15°C-30°C) 
1°C-30°C 
1°C-30°C 


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6.2. 
Preparation, Handling, and Accountability 
The investigator, investigational drug storage manager (for Japan), or designee must confirm 
appropriate temperature conditions have been maintained during transit for all study treatments 
received and any discrepancies are reported and resolved before use of the study treatment. 
Only participants enrolled in the study may receive study treatment, and only authorized site 
staff may supply or administer study treatment.  All study treatment must be stored in a secure, 
environmentally controlled, and monitored (manual or automated) area in accordance with the 
labeled storage conditions with access limited to the investigator, investigational drug storage 
manager (for Japan), and authorized site staff. 
The investigator, investigational drug storage manager (for Japan), or designee is responsible for 
study treatment accountability, reconciliation, and record maintenance (ie, receipt, reconciliation, 
and final disposition records).  Inventory and accountability records must be maintained and 
readily available for inspection by the study monitor and are open to inspection at any time by 
any applicable regulatory authorities.  The investigator, investigational drug storage manager (for 
Japan), or designee must maintain records that document: 
• Delivery of study drug to the study site. 
• Inventory of study drug at the site. 
• Participant use of the study drug including pill counts from each supply dispensed. 
• Return of study drug(s) to the investigator, investigational drug storage manager (for 
Japan), or designee by participants. 
The investigational product must be used only in accordance with the Protocol.  Tablets are not 
to be crushed, dissolved or broken.  The investigator (or investigational drug storage manager for 
Japan) will also maintain records adequately documenting that the participants were provided the 
specified study drug.  These records should include dates, quantities, and any available batch or 
serial numbers or unique code numbers assigned to the investigational product and study 
participants. 
Completed accountability records will be archived by the site.  The investigator, investigational 
drug storage manager (for Japan), or designee will be expected to collect and retain all used, 
unused, and partially used containers of study drug until verified by the study monitor (unless 
otherwise agreed to by the sponsor).  At the conclusion of the study, the investigator, 
investigational drug storage manager (for Japan), or designee will oversee shipment of any 
remaining study drug back to the sponsor or its designee for destruction according to institutional 
SOPs.  If local procedures mandate on-site destruction of investigational supply, the site should 
(where local procedures allow) maintain the investigational supply until the study monitor 
inspects the accountability records in order to evaluate compliance and accuracy of 
accountability by the investigative site.  At sites where the study drug is destroyed before 
monitor inspection, the monitors rely on documentation of destruction per the site SOP. 
Further guidance and information for the final disposition of unused study treatments are 
provided in the Pharmacy Manual. 
For Japan, the phosphate binders bixalomer and lanthanum carbonate hydrate will be provided 
by the sponsor (see Table 11).  The requirement for the accountability, reconciliation, and record 


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maintenance of the binders is the same as for study drug as described above.  Further guidance 
and information is provided in the Japan-specific Pharmacy Manual. 
6.3. 
Measures to Minimize Bias:  Randomization and Blinding 
This is an open-label, nonrandomized study.  There is no randomization and no blinding. 
6.4. 
Study Treatment Compliance 
Compliance with all study-related treatments and assessments should be emphasized to the 
participant by the site personnel, and appropriate steps should be taken to optimize compliance 
during the study. 
Compliance with pemigatinib will be calculated by the sponsor based on the drug accountability 
documented by the site staff and monitored by the sponsor/designee (tablet counts).  Participants 
will be instructed to bring all study drugs with them to the study visits in order for site personnel 
to conduct tablet counts to assess study drug accountability.  The drug accountability 
documentation will be used by the sponsor to calculate treatment compliance. 
6.5. 
Dose Modifications 
Individual decision regarding dose modifications of pemigatinib should be made using clinical 
judgment and in consultation with the sponsor's medical monitor, taking into account relatedness 
of the AE to the study treatment and the participant's underlying condition.  Adverse events that 
have a clear alternative explanation or transient (≤ 72 hours) abnormal laboratory values without 
associated clinically significant signs or symptoms, may be exempt from dose-reduction rules. 
6.5.1. 
Criteria and Procedures for Dose Interruptions and Adjustments of Study Drug 
Safety concerns should be discussed with the sponsor immediately upon occurrence or awareness 
to determine if the participant should continue or discontinue study treatment. 
Treatment with pemigatinib may be delayed up to 2 weeks (14 days) to allow for resolution of 
toxicity.  Participants may resume treatment if no medical condition or other circumstance exists 
that, in the opinion of the investigator, would make the participant unsuitable for further 
participation in the study.  The treating investigator should contact the sponsor to discuss the 
case of any participant whose treatment has been delayed for more than 14 days before restarting 
treatment with pemigatinib.  See Table 12 for guidelines. 


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Table 12: 
Guidelines for Interruption and Restarting of Study Drug 
Adverse Event 
Action Taken 
Chemistry 
• AST and/or ALT is > 5.0 × ULN. 
Note:  In participants with liver 
metastasis–related elevations at baseline, 
contact sponsor to discuss clinical 
management and possible dose 
reductions. 
Step 1:  Interrupt pemigatinib up to 2 weeks (14 days) 
until the toxicity has resolved to ≤ Grade 1 except by 
approval of the medical monitor. 
Step 2:  Restart pemigatinib at same dose.  If assessed as 
related to pemigatinib, restart pemigatinib at next lower 
dose; monitor as clinically indicated. 
Other toxicities 
• Any Grade 1 or Grade 2 toxicity. 
Continue pemigatinib treatment and treat the toxicity; 
monitor as clinically indicated. 
• Any Grade 3 toxicity, if clinically 
significant and not manageable by 
supportive care. 
Step 1:  Interrupt pemigatinib up to 2 weeks (14 days), 
until toxicity resolves to ≤ Grade 1. 
Step 2:  Restart pemigatinib at same dose.  If assessed as 
related to pemigatinib, restart pemigatinib at next lower 
dose; monitor as clinically indicated. 
• Any recurrent Grade 3 toxicity after 
2 dose reductions. 
Discontinue pemigatinib administration and follow-up per 
Protocol.  (Exceptions require approval of sponsor.) 
• Any other Grade 4 toxicity. 
• QT/QTc to > 500 milliseconds or to 
> 60 milliseconds over baseline. 
Discontinue pemigatinib administration and follow-up per 
Protocol. 
The sponsor recommends a maximum of 2 dose level reductions:  participants administered 
13.5 mg can decrease to 9 mg, and if additional dose reduction is required, participants can 
decrease to 4.5 mg.  Dose reductions below 4.5 mg are not allowed.  The frequency of dosing 
remains the same (QD) as well as the schedule (continuous dosing).  After a dose reduction, a 
dose re-escalation is not permitted. 
For participants who are up-titrated from 13.5 mg to 18 mg,  study drug can be decreased to 
13.5 mg, and if additional dose reductions are required, participants can decrease to 9 mg and 
then to 4.5 mg.  Dose reductions below 4.5 mg are not allowed. 
For participants who present with possible or confirmed SRD/RPED based on optical coherence 
tomography, the guidelines in Table 12 should be followed.  It is recommended to discuss the 
findings with the Incyte medical monitor before making changes to the participant's treatment.  
Per CTCAE v5.0, retinal detachment is graded as 3 (macular sparing) and 4 (macula‐off), but 
this refers to rhegmatogenous retinal detachment (when a hole occurs in the retina).  There is no 
grading for SRD/RPED (no hole in the macula, just fluid accumulation).  Therefore, grading 
should be based on the CTCAE v5.0 term of retinopathy. 


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6.5.2. 
Management of Hyperphosphatemia 
Hyperphosphatemia is an expected on-target pharmacologic effect of FGFR inhibition.  
Hyperphosphatemia should be managed with diet modifications, phosphate binders and diuretics, 
or a dose reduction per the recommendations in Table 13. 
The use of diet modifications alone include food exchanges from high-phosphate foods to 
low-phosphate foods and can be implemented once serum phosphate levels are above the ULN 
but do not exceed 7.0 mg/dL.  Diet modification should continue with the inclusion of phosphate 
binders once serum phosphate levels exceed 7.0 mg/dL.  Examples of phosphate binders are 
sevelamer HCl (examples of brand names:  Renegel® or Renvela®) or lanthanum HCl.  
Administration of phosphate binders should be 3 times per day (eg, with each meal) to reduce 
absorption of phosphate.  Doses and frequency of doses should be based on the participant's 
tolerance for the binder and the control of serum phosphate.  If binders are used to manage 
hyperphosphatemia during treatment, it is recommended to stop binders at the same time 
pemigatinib is stopped to reduce the risk of hypophosphatemia. 
For grading of hyperphosphatemia, please note that CTCAE v5.0 now has a category for 
hyperphosphatemia. 
For Japanese participants, the phosphate binders bixalomer or lanthanum carbonate hydrate will 
be provided by the sponsor for the treatment of hyperphosphatemia, when required (see 
Table 13). 
Table 13: 
Recommended Approach for Hyperphosphatemia Management 
Serum 
Phosphate Level 
Supportive Care 
Guidance for 
Interruption/Discontinuation 
of Pemigatinib 
Guidance for 
Restarting 
Pemigatinib 
> 5.5 mg/dL and 
≤ 7 mg/dL 
Initiate a low-phosphate diet. 
No action. 
Not applicable. 
> 7 mg/dL and 
≤ 10 mg/dL  
Initiate/continue a low-
phosphate diet and initiate 
phosphate-binding therapy.  
Monitor serum phosphate 
approximately twice a week 
and adjust the dose of binders 
as needed; continue to 
monitor serum phosphate at 
least twice a week until level 
returns to ≤ 7 mg/dL. 
If serum phosphate level 
continues to be > 7 mg/dL and 
≤ 10 mg/dL with concomitant 
phosphate-binding therapy for 
2 weeks, or if there is recurrence 
of serum phosphate level in this 
range, interrupt pemigatinib for 
up to 2 weeks. 
Restart at the same dose 
when serum phosphate 
is < 7 mg/dL.  If serum 
phosphate level recurs 
at > 7 mg/dL, restart 
pemigatinib with dose 
reduction. 
> 10 mg/dL 
Continue to maintain a 
low-phosphate diet, adjust 
phosphate-binding therapy, 
and start/continue 
phosphaturic agent.  
Continue to monitor serum 
phosphate approximately 
twice a week until level 
returns to ≤ 7 mg/dL. 
If serum phosphate level is 
> 10 mg/dL for 1 week 
following phosphate-binding 
therapy and low-phosphate diet, 
interrupt pemigatinib.  If there is 
recurrence of serum phosphate 
level in this range following 
2 dose reductions, permanently 
discontinue pemigatinib. 
Restart pemigatinib at 
reduced dose with 
phosphate binders when 
serum phosphate is 
< 7 mg/dL. 


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6.5.3. 
Up-Titration 
Any participant who does not reach the target serum phosphate level of > 5.5 mg/dL at any time 
during Cycle 1, are compliant with taking study drug, and do not experience an ongoing Grade 2 
or higher treatment-related AEs will increase the daily dose to 18 mg starting from Cycle 2 
Day 1. 
Participants who are titrated up to 18 mg QD will begin the next cycle at the new dose level and 
must agree to all Cycle 1 assessments (
safety assessments [hematology and blood 
chemistry]).  Up-titration may occur no earlier than on Day 1 of Cycle 2, so that participants are 
observed for phosphate level and AEs at least for 1 cycle. 
For participants who are up-titrated from 13.5 mg to 18 mg, dose reductions are allowed (see 
Section 6.5.1). 
6.5.4. 
Criteria for Permanent Discontinuation of Study Drug 
The occurrence of unacceptable toxicity not caused by the underlying disease will require that 
the study drug be permanently discontinued.  Unacceptable toxicity is defined as follows: 
• Occurrence of an AE that is related to study drug that, in the judgment of the 
investigator or the sponsor's medical monitor, compromises the participant's ability to 
continue study-specific procedures or is considered to not be in the participant's best 
interest. 
• An AE requiring more than 2 dose reductions. 
• Persistent AE requiring a delay of therapy for more than 2 weeks (14 days) unless a 
greater delay has been approved by the sponsor. 
• Increase in QT/QTc to > 500 milliseconds or to > 60 milliseconds over baseline.  In 
case of a QTc >500 milliseconds, the participant must be hospitalized and continuous 
ECG monitoring must be set up until the measure of the QTc interval decreases below 
500 milliseconds and is acceptable in the opinion of the local cardiologist. 
See Section 7 for discontinuation procedures. 
6.6. 
Concomitant Medications and Procedures 
All concomitant medications and treatments (including over-the-counter or prescription 
medicines, vitamins, vaccines, and/or herbal supplements) must be recorded in the eCRF.  Any 
prior medication received up to 35 days before the first dose of study treatment and 30 to 35 days 
after the last dose of study treatment, or until the participant begins a new anticancer therapy, 
whichever occurs first, will be recorded in the eCRF.  Any addition, deletion, or change in the 
dose of these medications will also be recorded.  Concomitant medications administered after 
30 to 35 days after the last dose of study treatment should be recorded for SAEs as defined in 
Section 9.2.  Concomitant treatments/procedures that are required to manage a participant's 
medical condition during the study will also be recorded in the eCRF.  The medical monitor 
should be contacted if there are any questions regarding concomitant or prior therapy. 


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6.6.1. 
Restricted Medications and Procedures 
Pemigatinib is predominantly metabolized by CYP3A4. There is an expected 50% increase in 
exposure in participants who concomitantly use CYP3A4 moderate inhibitors.  However, as 
doses above the recommended Phase 2 dose of 13.5 mg QD have been tested (20 mg QD is 
tolerable), there is a sufficient safety margin.  Therefore, the use of  moderate CYP3A4 inhibitors 
is not prohibited but should involve careful monitoring, especially in relation to safety, while 
moderate CYP3A4 inducers and potent CYP3A4 inhibitors and inducers are prohibited (see 
Appendix D). 
Careful monitoring is required when pemigatinib is concomitantly administered with OCT2 
substrates such as dofetilide and metformin. 
Calcium-based phosphate–binding medications should not be used due to a concern for soft 
tissue mineralization. 
6.6.2. 
Prohibited Medications and Procedures 
The following medications and measures are prohibited: 
• The concomitant administration of potent CYP3A4 inhibitors and inducers and 
moderate CYP3A4 inducers (see Appendix D).  Based on the low overall 
bioavailability of topical ketoconazole, there are no restrictions on topical 
ketoconazole. 
• Any concomitant use of a selective FGFR inhibitor (other than pemigatinib). 
• Investigational study drug for any indication. 
• Use of any anticancer medications other than the study medications being tested in 
this Protocol. 


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7. 
DISCONTINUATION OF STUDY TREATMENT AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL 
7.1. 
Discontinuation of Study Treatment 
7.1.1. 
Reasons for Discontinuation 
Participants must be discontinued from study treatment for the following reasons.  Please note 
that if a participant discontinues study treatment for reasons other than disease progression or 
withdraw of consent, the participant should be followed until disease progression is documented. 
• The participant becomes pregnant.  
• Consent is withdrawn.   
Note:  Consent withdrawn means that the participant has explicitly indicated that they 
do not want to be followed any longer; in this case no further data, except data in 
public domain, may be solicited from or collected on the participant.  Participants 
may choose to discontinue study treatment and remain in the study to be followed for 
progression and survival. 
• Further participation would be injurious to the participant's health or well-being, in 
the investigator's medical judgment. 
• Unacceptable toxicity as noted in Section 6.5. 
• The study is terminated by the sponsor. 
• The study is terminated by the local health authority, IRB, or IEC. 
• Disease progression has been reported by central radiology review. 
• Other antineoplastic treatment is initiated, not including palliative radiation. 
A participant may be discontinued from study treatment if a participant is noncompliant with 
study procedures or study drug/treatment administration in the investigator's opinion, the sponsor 
should be consulted for instruction on handling the participant. 
7.1.2. 
Discontinuation Procedures 
In the event that the decision is made to permanently discontinue the study treatment, the EOT 
visit should be conducted.  Reasonable efforts should be made to have the participant return for a 
follow-up visit.  These visits are described in Table 3 and Table 4.  The last date of the last dose 
of study drug(s)/treatment and the reason for discontinuation of study treatment will be recorded 
in the eCRF. 
If a participant is discontinued from study treatment: 
• The study monitor or sponsor must be notified. 
• The reason(s) for withdrawal must be documented in the participant's medical record 
and the primary reason for withdrawal must be included in the eCRF. 
• The EOT visit should be performed. 


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• The date of the EOT visit should be recorded in the IRT. 
• Participants must be followed for safety until the time of the follow-up visit or until 
study drug/treatment–related toxicities resolve, return to baseline, or are deemed 
irreversible, whichever is longest. 
If the participant discontinues study treatment and actively withdraws consent for collection of 
follow-up data (safety follow-up or disease assessment), then no additional data collection 
should occur; however, participants will have the option of withdrawing consent for study 
treatment but continuing in the follow-up period of the study for safety/efficacy assessments and 
survival follow-up. 
7.2. 
Participant Withdrawal From the Study 
A participant may withdraw from the study at any time at his/her own request, or may be 
withdrawn at any time at the discretion of the investigator for safety, behavioral, compliance, or 
administrative reasons. 
If the participant withdraws consent for disclosure of future information, the sponsor may retain 
and continue to use any data collected before such a withdrawal of consent. 
If a participant withdraws from the study, he/she may request destruction of any samples taken 
and not tested, and the investigator must document this in the site study records. 
See Table 3 and Table 4 for data to be collected at the time of study discontinuation and 
follow-up and for any further evaluations that need to be completed. 
7.3. 
Lost to Follow-Up 
A participant will be considered lost to follow-up if he/she repeatedly fails to return for 
scheduled visits and is unable to be contacted by the study site. 
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit: 
• The site must attempt to contact the participant and reschedule the missed visit as 
soon as possible and counsel the participant on the importance of maintaining the 
assigned visit schedule and ascertain whether or not the participant wishes to and/or 
should continue in the study. 
• Before a participant is deemed lost to follow-up, the investigator or designee must 
make every effort to regain contact with the participant (where possible, 3 telephone 
calls and, if necessary, a certified letter to the participant's last known mailing address 
or local equivalent methods).  These contact attempts should be documented in the 
participant's medical record. 
• Should the participant continue to be unreachable, he/she will be considered to have 
withdrawn from the study. 


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8. 
STUDY ASSESSMENTS AND PROCEDURES 
8.1. 
Administrative and General Procedures 
8.1.1. 
Informed Consent Process 
• The investigator or his/her representative will explain the nature of the study to the 
participant or his/her legally authorized representative and answer all questions 
regarding the study. 
− Informed consent must be obtained before any study-related procedures are 
conducted, unless otherwise specified by the Protocol. 
− Informed consent must be obtained using the IRB/IEC-approved version in a 
language that is native and understandable to the participant.  A template will be 
provided by the sponsor or its designee.  The sponsor or its designee must review 
and acknowledge the site-specific changes to the ICF template.  The ICF must 
include a statement that the sponsor or its designee and regulatory authorities have 
direct access to participant records. 
− The ICF must contain all required elements and describe the nature, scope, and 
possible consequences of the study in a form understandable to the study 
participant. 
• Participants must be informed that their participation is voluntary.  Participants or 
their legally authorized representative will be required to sign a statement of informed 
consent that meets the applicable requirements and regulations for the country(ies) in 
which the study is being conducted as well as the IRB/IEC or study center. 
• The participant must be informed that his/her personal study-related data will be used 
by the sponsor in accordance with local data protection laws.  The level of disclosure 
must also be explained to the participant. 
• The participant must be informed that his/her medical records may be examined by 
Clinical Quality Assurance auditors or other authorized personnel appointed by the 
sponsor, by appropriate IRB/IEC members, and by inspectors from regulatory 
authorities. 
• The medical record must include a statement that written informed consent was 
obtained before the participant was enrolled in the study and the date the written 
consent was obtained.  The authorized person obtaining the informed consent must 
also sign the ICF. 
• Participants must provide consent to the most current version of the ICF(s) during 
their participation in the study. 
• A copy of the ICF(s) must be provided to the participant or the participant's legally 
authorized representative. 
Participants who are rescreened are required to sign a new ICF. 


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8.1.2. 
Prescreening and Screening Procedures 
Prescreening is allowed in cases where the site may need to consult with the sponsor on the 
eligibility of a potential participant's genomic alteration.  This is not required for all participants; 
it applies only to those whose genomic alterations need to be reviewed for eligibility.  Screening 
is the interval between signing the study ICF (not the prescreening consent form) and the day the 
participant is enrolled in the study (Cycle 1 Day 1).  Screening may not exceed 35 days.  
Assessments that are required to demonstrate eligibility may be performed over the course of 
1 or more days during the screening process. 
Procedures conducted as part of the participant's routine clinical management (eg, blood count, 
imaging study) and obtained before signing of informed consent may be used for screening or 
baseline purposes provided the procedure meets the Protocol-defined criteria and has been 
performed in the timeframe of the study (ie, within 35 days of Cycle 1 Day 1).  For participants 
who are enrolled in the study, information associated with eligibility requirements must be 
entered into the appropriate eCRF pages. 
Results from the screening visit evaluations will be reviewed by the investigators to confirm 
eligibility before enrollment or the administration of study drug.  Tests with results that fail 
eligibility requirements may be repeated once during screening if the investigator believes the 
results to be in error.  For screening assessments that are repeated, the most recent available 
result before treatment begins will be used to determine eligibility.  Treatment should start as 
soon as possible, but within 3 days after the date of enrollment. 
See Sections 5.4 and 5.5 for information regarding screen failures and replacement of 
participants, respectively. 
8.1.3. 
Interactive Response Technology Procedure 
Each participant will be identified in the study by a participant ID number, which is a 
combination of the site ID and participant number.  Site staff should contact the IRT to obtain the 
participant ID number during prescreening (if applicable) and screening.  Upon determining that 
the participant is eligible for study entry, the IRT will be contacted to obtain the treatment 
assignment.  Additionally, the IRT will be contacted at each regular study visit to update the 
study drug supply.  Additional details are provided in the IRT Manual. 
8.1.4. 
Distribution of Reminder Cards 
Participants will be provided with a reminder card at each visit.  The reminder card will indicate 
the date/time of the next visit and will have an area on which the date and time of the last dose 
taken and the time of their last meal before the visit should be recorded. 


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8.1.5. 
Demography and Medical History 
8.1.5.1. 
Demographics and General Medical History 
Demographic data and general medical history will be collected at screening by the investigator 
or qualified designee and will include year of birth/age, race, ethnicity, medical and surgical 
history, and current illnesses.  Medical history will include relevant medical or surgical treatment 
within the last 10 years that are considered to be clinically significant by the investigator. 
8.1.5.2. 
Disease Characteristics and Treatment History 
A disease-targeted medical and treatment history will be collected at screening.  Details 
regarding the participant's malignancy under study, including date of diagnosis, initial and 
current cancer stage, tumor histology, and relevant disease characteristics, and prior treatments, 
including systemic treatments, radiation, and surgical procedures, will be recorded. 
8.2. 
Efficacy Assessments 
8.2.1. 
Tumor Imaging 
Objective assessment of tumor status is required using appropriate disease-specific techniques, 
and a central radiologic facility will be used to determine responses and will be logged in to the 
eCRF.  RECIST v1.1 (Eisenhauer et al 2009), and RANO for participants with primary brain 
tumors, will be used, and the recommended method for measuring and following tumor burden 
will be CT scan to include the thorax, abdomen, and pelvis; the neck can be included if needed.  
Alternative modalities (eg, MRI) may be substituted for a CT scan at the discretion of the 
investigator, provided that the same modality is used throughout the study and that the 
methodology is consistent with RECIST v1.1 or RANO. 
The schedule for efficacy assessments will be at screening (this will be considered the baseline 
scan), every 9 weeks (every 3 cycles), and then at EOT (if applicable).  Imaging should continue 
in 9-week intervals regardless of delays in cycle starts.  For participants showing a response, a 
confirmatory scan may be performed a minimum of 4 weeks (per RECIST v1.1 and RANO) 
from the previous scan.  For participants showing a progression based on local radiologic review, 
treatment should not be discontinued until progression of disease has been determined by the 
ICR, unless the principal investigator believes it is in the best interest of the participant to 
discontinue treatment before receiving confirmation. 
For participants who discontinue treatment for reasons other than disease progression, every 
effort should be made to continue monitoring their disease status by radiographic imaging until 
1) start of new anticancer therapy, 2) documented disease progression, 3) death, or 4) end of 
study, whichever occurs first. 
8.2.2. 
ECOG Performance Status 
ECOG performance status (see Table 14) will be assessed at the visits specified in the 
appropriate SoA (see Table 3 and Table 4). 


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Table 14: 
ECOG Performance Status 
Grade 
Performance Status 
0 
Fully active, able to carry on all predisease performance without restriction. 
1 
Restricted in physically strenuous activity but ambulatory and able to carry out work of a 
light or sedentary nature, eg, light house work, office work. 
2 
Ambulatory and capable of all self-care but unable to carry out any work activities.  Up 
and about more than 50% of waking hours. 
3 
Capable of only limited self-care, confined to bed or chair more than 50% of waking 
hours. 
4 
Completely disabled.  Cannot carry on any self-care.  Totally confined to bed or chair. 
5 
Dead. 
Source:  Oken et al 1982. 
8.3. 
Safety Assessments 
See Section 6.5 for guidelines regarding the management of relevant laboratory or other safety 
assessment abnormalities. 
8.3.1. 
Adverse Events 
Adverse events will be monitored from the time the participant signs the ICF until at least 30 to 
35 days after the last dose of study drug or until the start of new anticancer therapy, whichever 
occurs first.  Adverse events that begin or worsen after informed consent should be recorded on 
the Adverse Events Form in the eCRF regardless of the assumption of a causal relationship with 
the study drug.  Conditions that were already present at the time of informed consent should be 
recorded on the Medical History Form in the eCRF.  Adverse events (including laboratory 
abnormalities that constitute AEs) should be described using a diagnosis whenever possible 
rather than by individual underlying signs and symptoms. 
Adverse events will be reported by the participant (or, when appropriate, by a caregiver, 
surrogate, or the participant's legally authorized representative).  The investigator and any 
qualified designees are responsible for detecting, documenting, and recording events that meet 
the definition of an AE or SAE and remain responsible for following-up on AEs that are serious, 
considered related to the study treatment/procedures, or that caused the participant to discontinue 
study drug.  Care will be taken not to introduce bias when detecting AEs and/or SAEs.  
Open-ended and nonleading verbal questioning of the participant, such as "How are you 
feeling?" is the preferred method to inquire about AE occurrences.  Adverse events may also be 
detected when they are volunteered by the participant during the screening process or between 
visits, or through physical examinations, laboratory tests, or other assessments.  The definition, 
reporting, and recording requirements for AEs are described in Section 9. 


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All SAEs will be recorded and reported to the sponsor or designee within 24 hours.  The 
investigator will submit any updated SAE data to the sponsor within 24 hours of it being 
available. 
After the initial AE/SAE report, the investigator is required to proactively follow each participant 
at subsequent visits/contacts.  All SAEs will be followed until resolution, stabilization, the event 
is otherwise explained, or the participant is lost to follow-up (as defined in Section 7.3). 
For Japanese participants only, the relationship of the adverse event to phosphate binder(s) 
should also be collected on the Adverse Events Form in the eCRF. 
8.3.2. 
Physical Examinations 
Physical examinations must be performed by a medically qualified individual, such as a licensed 
physician, physician's assistant, or an advanced registered nurse practitioner, as local law 
permits.  Abnormalities identified after the first dose of study treatment constitute an AE if they 
are considered clinically meaningful, induce clinical signs or symptoms, require concomitant 
therapy, or require changes in study drug.  Investigators should pay special attention to clinical 
signs related to previous serious illnesses. 
At the screening visit, a comprehensive physical examination should be conducted.  The 
comprehensive physical examination will include height (at screening only) and body weight and 
assessment(s) of the following organ or body systems:  skin; head, eyes, ears, nose, and throat; 
thyroid; lungs; cardiovascular system; abdomen (liver, spleen); extremities; and lymph nodes; as 
well as a brief neurological examination. 
During the study, participants will be assessed by the investigator or medically qualified 
designee per institutional standard of care.  These assessments should be an evaluation as 
indicated by participant symptoms, AEs, or other findings and documented on the AE eCRF. 
8.3.3. 
Vital Signs 
Vital sign measurements (to be taken before blood collection for laboratory tests), include blood 
pressure, pulse, respiratory rate, and body temperature.  Blood pressure and pulse will be taken 
with the participant in the recumbent, semirecumbent, or sitting position after approximately 
5 minutes of rest.  Weight will also be assessed at the beginning of each cycle.  Abnormal vital 
sign results identified after the first dose of study treatment constitute an AE if they are 
considered clinically meaningful, induce clinical signs or symptoms, require concomitant 
therapy, or require changes in study drug. 
8.3.4. 
Electrocardiograms 
Single 12-lead ECG will be obtained as outlined in the SoA (see Table 3) using an ECG machine 
that automatically calculates the heart rate and measures PR, QRS, QT, and QTc intervals.  All 
12-lead ECGs will be performed with the participant in a recumbent or semirecumbent position 
after approximately 5 minutes of rest. 
The 12-lead ECGs will be interpreted by the investigator at the site to be used for immediate 
participant management.  Additional 12-lead ECGs may be performed as clinically indicated to 
manage participant safety.  The decision to include or exclude a participant or withdraw a 
participant from the study treatment based on an ECG flagged as "Abnormal, Clinically 


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Significant" is the responsibility of the investigator, in consultation with the sponsor's medical 
monitor, as appropriate.  Clinically notable abnormalities that are considered clinically 
significant in the judgment of the investigator are to be reported as AEs.  In the event that a 
single QTc is > 480 ms at screening, the participant may enroll if the average QTc for the 
3 ECGs is ≤ 480 ms or with approval from the medical monitor.  For participants with an 
intraventricular conduction delay (QRS interval > 120 ms) at screening, the JTc interval may be 
used in place of the QTc with medical monitor approval.  The JTc must be ≤ 340 ms if JTc is 
used in place of the QTc.  In addition, the JTc interval should be used for all subsequent 
assessments. 
8.3.5. 
Comprehensive Eye Examination 
A comprehensive eye examination should be performed by a qualified ophthalmologist at 
screening, once every 3 cycles (± 7 days, starting at Cycle 3), at EOT, and as clinically indicated.  
The eye examination should include a visual acuity test, slit-lamp examination, and funduscopy 
with digital imaging and OCT.  Visual acuity, slit-lamp, funduscopy and OCT are required to be 
performed at baseline and once every 3 cycles (starting with cycle 3) and more often if 
participants report any visual AEs or change in visual acuity.  Every effort should be made to 
ensure that all subsequent examinations are performed by the same ophthalmologist. 
8.3.6. 
Laboratory Assessments 
See Table 15 for the list of clinical laboratory tests to be performed, and see the SoA (Table 4) 
for the timing and frequency.  A central laboratory will analyze all 
 translational 
samples.  The site's own local laboratories will perform all clinical laboratory assessments for 
safety (ie, blood chemistries, hematology assessments, coagulation tests, endocrine function and 
urinalysis). 
Serum phosphate testing is required on Day 8 in Cycles 2+ if a participant develops 
hyperphosphatemia during Cycle 1 or in later cycles if the participant is up-titrated.  Parathyroid 
hormone (endocrine) testing is required at baseline and Day 1 of every third cycle (starting with 
Cycle 3) as parathyroid hormone plays a role in calcium and phosphate hemostasis (Khundmiri 
et al 2016). 
Additional testing may be required by the sponsor based on emerging safety data.  All 
Protocol-required laboratory assessments must be conducted in accordance with the Laboratory 
Manual and the SoA (see Table 4).  Information regarding collection, processing, and shipping 
of laboratory assessments is provided in the Laboratory Manual. 
Clinically significant abnormal laboratory findings are those that are not associated with the 
underlying disease, unless judged by the investigator to be more severe than expected for the 
participant's condition.  All laboratory tests with values considered clinically significantly 
abnormal during participation in the study or within 30 to 35 days after the last dose of study 
treatment should be repeated until the values return to normal or baseline or are no longer 
considered clinically significant by the investigator or medical monitor. 
Screening laboratory assessments for hematology, chemistry, and coagulation must be performed 
within 14 days of Cycle 1 Day 1.  If performed more than 14 days before Cycle 1 Day 1, then the 
tests must be repeated; if performed within 3 days of C1D1, additional laboratory sample testing 
is not required (see Table 4).  Laboratory samples collected on study Day 1 must be performed 


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before study treatment administration.  After Cycle 1, predose laboratory procedures can be 
conducted up to 72 hours before study treatment administration (within the 3-day study window), 
and results should be reviewed by the investigator or qualified designee and found to be 
acceptable before a new cycle of treatment is initiated. 
See Section 9.1 for information regarding laboratory abnormalities that should be recorded as an 
AE in the eCRF.  Additionally, if laboratory values from laboratory assessments performed at the 
institution's local laboratory require a change in participant management (eg, require treatment), 
or are considered clinically significant by the investigator (eg, SAE or AE or dose modification, 
see Section 9.2), then the result(s) of the specific laboratory assessment(s) must be recorded in 
the eCRF. 
 


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Table 15: 
Required Laboratory Analytes 
Serum Chemistries 
Hematology 
Urinalysis With 
Microscopic Examination 
Serology 
Coagulation 
Albumin 
Alkaline phosphatase 
ALT 
AST 
Amylase 
Bicarbonate or CO2 (not 
applicable for Japan) 
Blood urea nitrogen or urea 
Calcium 
Chloride 
Creatinine 
Glucose 
Lactate dehydrogenase 
Lipase 
Magnesium 
Phosphate 
Potassium 
Sodium 
Total bilirubin 
Direct bilirubin (if total bilirubin 
is elevated above ULN) 
Total protein 
Uric acid 
Vitamin D (25-hydroxyvitamin D 
and 1,25-dihidrosyvitamin D) 
Complete blood count, 
including: 
 Hemoglobin 
 Hematocrit 
 Platelet count 
 Red blood cell count 
 White blood cell count 
 
Differential count, including: 
 Basophils 
 Eosinophils 
 Lymphocytes 
 Monocytes 
 Neutrophils 
 
Absolute values must be 
provided for: 
 WBC differential 
laboratory results 
Color and appearance 
pH and specific gravity 
Bilirubin 
Glucose 
Ketones 
Leukocytes 
Nitrite 
Occult blood 
Protein 
Hepatitis B surface antigen 
Hepatitis B surface antibody 
Hepatitis B core antibody 
HCV antibody 
NOTE:  If any of the above 
are positive, HBV-DNA, 
HCV-RNA may be done to 
assess risk of reactivation, if 
indicated (eg, no history of 
immunization) 
PT 
PTT or aPTT 
INR 
Endocrine Function 
Pregnancy Testing 
PTH 
Female participants of 
childbearing potential 
only will require a serum 
test at screening and 
EOT and a urine 
pregnancy test before the 
first dose on Cycle 1 
Day 1 and then on Day 1 
of every cycle. 
Pregnancy tests (serum 
or urine) should be 
repeated if required by 
local regulations. 
Note:  Additional tests may be required, as agreed upon by the investigator and sponsor, based on emerging safety data. 


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8.3.6.1. 
Pregnancy Testing 
A serum pregnancy test will be required for all women of childbearing potential during screening 
and at the EOT visit; participants going into hospice are not required to provide EOT pregnancy 
test.  Urine pregnancy tests will be performed locally on Day 1 of each cycle, as outlined in 
Table 4, as medically indicated (eg, in case of loss of menstrual cycle, when pregnancy is 
suspected), or per country-specific requirement (note that country-required urine pregnancy 
testing will be outlined and communicated to investigational sites under separate cover).  If a 
urine pregnancy test is positive, the results should be confirmed with a serum pregnancy test. 
If the serum pregnancy test is negative after a urine test was positive, the investigator will assess 
the potential benefit/risk to the participant and determine whether it is in the participant's best 
interest to resume study drug and continue participation in the study. 
If a pregnancy is confirmed by a serum pregnancy test, see Section 9.5 for reporting 
requirements. 
8.3.6.2. 
Serology 
Hepatitis screening assessments will be performed at the screening visit to assess infection and 
viral status; required analytes are shown in Table 15.  Generally, hepatitis tests should be 
performed early in the screening process due to the length of time needed to obtain the results.  
Additional tests may be performed if clinically indicated. 


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8.5. 
 Translational Assessments 
8.5.1. 
Tumor for Genomics Testing for FGFR Genetic Alterations and Gene 
Expression 
All potential participants must be evaluated for activating FGFR mutations and rearrangements 
before enrollment.  Participants with local genomics laboratory test results (within 24 months 
from date of screening) documenting an activating FGFR1-3 mutation or rearrangement are 
eligible (see Appendix C).  Confirmatory testing through the central genomics laboratory will be 
performed on all participants; results from the central laboratory are not required before 
enrollment.  Participants who have a commercial report from the central laboratory (Foundation 
Medicine, Inc) at screening (within 24 months of screening) will not need to send a sample for 
confirmatory testing (a biopsy will still be required for other testing).  Details for sample 
collection, processing, and shipping will be provided in the Laboratory Manual. 
Appendix C contains a list of the most common recurrent activating FGFR mutations (excluding 
the kinase domain) that have been previously described, are present in somatic mutation 
databases, and/or are homologous to activating mutations present in another FGFR.  Appendix C 
is not all-inclusive, as extremely rare or novel activating alterations may not be present in the list.  
For FGFR mutations not present in Appendix C, participants will be assigned to Cohort C unless 
the investigator consults with the study sponsor. 
 
 
  Buccal swab DNA 
may be used to confirm somatic mutations identified in tumor tissue. 


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conducted.  If the EOT visit coincides with a regular study visit, the EOT evaluations will 
supersede those of that scheduled visit, and the data should be entered in the EOT visit in the 
eCRF.  The participant should be encouraged to return for the follow-up visit. 
8.8. 
Follow-Up 
8.8.1. 
Safety Follow-Up 
The safety follow-up period is the interval between the EOT visit and the scheduled follow-up 
visit, which should occur 30 to 35 days after the EOT visit (or after the last dose of study 
treatment if the EOT visit was not performed).  Adverse events and SAEs must be reported up 
until 1) at least 30 days after the last dose of study treatment, the date of the follow-up visit, or 
the start of a new anticancer therapy or 2) until toxicities resolve, return to baseline, or are 
deemed irreversible, whichever is longer.  Reasonable efforts should be made to have the 
participant return for the follow-up visit and report any AEs that may occur during this period.  If 
the participant cannot return to the site for the safety follow-up visit (eg, lives far away), the 
participant should be contacted by telephone for assessment of AEs and SAEs.  Sites should 
document this contact in the source. 
If a participant is scheduled to begin a new anticancer therapy before the end of the 30- to 35-day 
safety follow-up period, the safety follow-up visit should be performed before the new anticancer 
therapy is started.  Once new anticancer therapy has been initiated, the participant will move into 
the survival follow-up period. 
8.8.2. 
Post-Treatment Disease Follow-Up 
Participants who discontinue study treatment for a reason other than disease progression will 
move into the disease status follow-up period and should be assessed every 9 weeks ± 7 days by 
radiologic imaging to monitor disease status.  Every effort should be made to collect information 
regarding disease status until: 
• The start of new anticancer therapy:  the start of the new treatment should be 
captured, but the participant should continue to have disease assessments until 
progressive disease (see below). 
• Disease progression (confirmed by an ICR). 
• Death. 
• The end of the study. 
8.8.3. 
Survival Follow-Up 
Once a participant has received the last dose of study treatment, confirmed disease progression, 
or starts a new anticancer therapy, the participant moves into the survival follow-up period and 
should be contacted by telephone, email, or visit at least every 12 weeks to assess for survival 
status until death, withdrawal of consent, or the end of the study, whichever occurs first. 


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For participants having entered the survival follow-up period of the study, the site will use 
continuing participant records to supply data on subsequent treatment regimens, tumor 
assessments (if discontinued treatment for a reason other than progression), and OS in the eCRF.  
For participants who do not intend to return to the study investigator for their ongoing care, 
follow-up should be maintained by phone contact, patient records, and public records/databases 
approximately every 12 weeks. 
9. 
ADVERSE EVENTS:  DEFINITIONS AND PROCEDURES FOR 
RECORDING, EVALUATING, FOLLOW-UP, AND REPORTING 
9.1. 
Definition of Adverse Event 
Adverse Event Definition 
• An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or 
not considered drug-related. 
• An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory 
finding), symptom, or disease (new or exacerbated) temporally associated with the use of study 
treatment. 
Events Meeting the Adverse Event Definition  
• Any safety assessments (eg, ECG, vital signs measurements), including those that worsen from 
baseline, considered clinically significant in the medical and scientific judgment of the investigator 
(ie, not related to progression of underlying disease). 
• Abnormal laboratory test results constitute an AE if they are considered clinically meaningful, induce 
clinical signs or symptoms, require concomitant therapy, or require changes in study drug.  
Whenever possible, a diagnosis (eg, anemia, thrombocytopenia) should be recorded in the eCRF 
rather than the abnormal laboratory result (eg, low hemoglobin, platelet count decreased). 
• Exacerbation of a chronic or intermittent pre-existing condition including either an increase in 
frequency and/or intensity of the condition. 
• New conditions detected or diagnosed after study treatment administration even though they may 
have been present before the start of the study. 
• Signs, symptoms, or the clinical sequelae of a suspected drug-drug interaction. 
• Signs, symptoms, or the clinical sequelae of a suspected overdose of either study treatment or a 
concomitant medication.  Overdose per se will not be reported as an AE/SAE unless it is an 
intentional overdose taken with possible suicidal/self-harming intent.  Such overdoses should be 
reported regardless of sequelae. 
Events NOT Meeting the Adverse Event Definition 
• Any clinically significant abnormal laboratory findings or other abnormal safety assessments that are 
associated with the underlying disease, unless judged by the investigator to be more severe than 
expected for the participant's condition. 
• The disease/disorder being studied or expected progression, signs, or symptoms of the 
disease/disorder being studied, unless more severe than expected for the participant's condition or 
considered to be treatment-related by the investigator. 


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• Efficacy endpoints as outlined in Section 3 will not be reported as AE/SAEs, specifically, any event 
that is related to disease progression of the cancer under study.  Unblinded aggregated efficacy 
endpoint events and safety data will be monitored to ensure the safety of the participants in the study.  
Any suspected endpoint that upon review is not progression of the cancer under study will be 
forwarded to Incyte Pharmacovigilance as a SAE within 24 hours of determination that the event is 
not progression of the cancer under study. 
• Medical or surgical procedure (eg, endoscopy, appendectomy):  the condition that leads to the 
procedure is the AE if it occurred after signing informed consent.  If present before entering the 
study, the condition should be captured as medical history. 
• Situations in which an untoward medical occurrence did not occur (social and/or convenience 
admission to a hospital). 
• Anticipated day-to-day fluctuations of pre-existing disease(s) or condition(s) present or detected at 
the start of the study that do not worsen. 
9.2. 
Definition of Serious Adverse Event 
If an event is not an AE per definition above, then it cannot be an SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
A Serious Adverse Event is defined as any untoward medical occurrence that, at any dose: 
a. Results in death 
b. Is life-threatening 
The term 'life-threatening' in the definition of 'serious' refers to an adverse drug experience that places 
the participant, in the opinion of the initial reporter, at immediate risk of death from the adverse 
experience as it occurred.  This does not include an adverse drug experience that, had it occurred in a 
more severe form, might have caused death. 
c.  Requires inpatient hospitalization or prolongation of existing hospitalization 
In general, hospitalization signifies that the participant has been detained (usually involving at least an 
overnight stay) at the hospital or emergency ward for observation and/or treatment that would not have 
been appropriate in the physician's office or outpatient setting.  Complications that occur during 
hospitalization are AEs.  If a complication prolongs hospitalization or fulfills any other serious criteria, 
the event is serious.  When in doubt as to whether "hospitalization" occurred or was necessary, the AE 
should be considered serious. 
Hospitalization for elective treatment of a pre-existing condition that did not worsen from baseline is 
not considered an AE. 
d. Results in persistent or significant disability/incapacity 
• The term disability means a substantial disruption of a person's ability to conduct normal life 
functions. 
• This definition is not intended to include experiences of relatively minor medical significance, such 
as uncomplicated headache, nausea, vomiting, diarrhea, influenza, and accidental trauma (eg, 
sprained ankle), that may interfere with or prevent everyday life functions but do not constitute a 
substantial disruption. 
e.  Is a congenital anomaly/birth defect 


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f. Other situations (Important Medical Event) 
An event that may not result in death, be immediately life-threatening, or require hospitalization, but 
may be considered serious when, based on appropriate medical judgment, the event may jeopardize the 
participant and may require medical or surgical intervention to prevent one of the outcomes listed in 
the above definition.  Examples of such events include invasive or malignant cancers (excluding the 
disease[s] under study in oncology protocols), intensive treatment in an emergency department or at 
home for allergic bronchospasm, blood dyscrasias, or convulsions that do not result in hospitalization, 
or development of drug dependency or drug abuse. 
For Japan, an event which may lead to disability is also considered as the important medical event.  It 
includes a case that is exposed to a risk of dysfunction to an extent that interferes with daily life when 
the adverse drug reaction occurs.  It does not include an adverse drug reaction that, had the reaction 
been more severe, may have caused disability. 
9.3. 
Recording and Follow-Up of Adverse Events and/or Serious Adverse 
Events 
Adverse Event and Serious Adverse Event Recording 
• An AE/SAE that begins or worsens after informed consent is signed should be recorded on the 
Adverse Event Form in the eCRF.  Conditions that were present at the time informed consent was 
given should be recorded on the Medical History Form in the eCRF. 
• When an AE/SAE occurs, it is the responsibility of the investigator to review all documentation (eg, 
hospital progress notes, laboratory reports, and diagnostics reports) related to the event. 
• The investigator (or delegate) will then record all relevant AE/SAE information in the eCRF. 
• It is not acceptable for the investigator to send photocopies of the participant's medical records in 
lieu of completing the AE eCRF page. 
• There may be instances when copies of medical records for certain cases are requested.  In this case, 
all participant identifiers, with the exception of the participant number, will be redacted on the copies 
of the medical records before submission. 
• The investigator will attempt to establish a diagnosis of the event based on signs, symptoms, and/or 
other clinical information.  Whenever possible, the diagnosis (not the individual signs/symptoms) 
will be documented as the AE/SAE.  When a clear diagnosis cannot be identified, each sign or 
symptom should be reported as a separate AE/SAE. 
To the extent possible, each AE/SAE should be evaluated to determine: 
• The severity grade (CTCAE Grade 1 to 5).  See below for further instructions on the assessment of 
intensity. 
• Whether there is at least a reasonable possibility that the AE is related to the study drug:  suspected 
(yes) or not suspected (no).  See below for further instructions on the assessment of causality. 
• The start and end dates, unless unresolved at final follow-up. 
• The action taken with regard to study drug as a result of the AE/SAE(s). 
• The event outcome (eg, not recovered/not resolved, recovered/resolved, recovering/resolving, 
recovered/resolved with sequelae, fatal, unknown). 
• The seriousness, as per the SAE definition provided in Section 9.2. 
• The action taken with regard to the event.  Note:  If an AE is treated with a concomitant medication 
or nondrug therapy, this action should be recorded on Adverse Event Form and the treatment should 
be specified on the appropriate eCRF (eg, Prior/Concomitant Medications, Procedures and Non-Drug 
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Assessment of Intensity 
The severity of AEs will be assessed using CTCAE v5.0 Grades 1 through 5.  If an event is not 
classified by CTCAE, the severity of the AE will be graded according to the scale below to estimate the 
grade of severity: 
The investigator will make an assessment of intensity for each AE and SAE reported during the study 
and assign it to 1 of the following categories: 
• Grade 1:  Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment 
not indicated. 
• Grade 2:  Moderate; minimal, local, or noninvasive treatment indicated; limiting age appropriate 
activities of daily living. 
• Grade 3:  Severe or medically significant but not immediately life-threatening; hospitalization or 
prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. 
• Grade 4:  Life-threatening consequences; urgent treatment indicated. 
• Grade 5:  Fatal. 
Assessment of Causality 
• The investigator is obligated to assess the relationship between study treatment and each occurrence 
of each AE/SAE.  If reference therapy is used in combination with an Incyte study drug or multiple 
Incyte study drugs are used, the relationship to each study drug must be assessed (ie, for the Incyte 
product(s) and for the other product(s) that is used in combination with the Incyte product).  If 
appropriate, the relationship to the combination may be assessed as well. 
• A "reasonable possibility" of a relationship conveys that there are facts, evidence, and/or arguments 
to suggest a causal relationship, rather than that a relationship cannot be ruled out. 
• The investigator will use clinical judgment to determine the relationship. 
• The investigator will also consult the RSI in the IB and/or Product Information, for marketed 
products, in his/her assessment. 
• Alternative causes, such as underlying disease(s), concomitant therapy, and other risk factors, as well 
as the temporal relationship of the event to study treatment administration, will be considered and 
investigated. 
• For each AE/SAE, the investigator must document in the medical notes that he/she has reviewed the 
AE/SAE and has provided an assessment of causality. 
• With regard to assessing causality of SAEs: 
− There may be situations in which an SAE has occurred and the investigator has minimal 
information to include in the initial report.  However, the causality assessment is one of the criteria 
used when determining regulatory reporting requirements.  Therefore, it is very important that 
the investigator always make an assessment of causality for every event before the initial 
transmission of the SAE. 
− The investigator may change his/her opinion of causality in light of follow-up information and 
send a follow-up SAE report with the updated causality assessment. 


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Follow-Up of Adverse Events and Serious Adverse Events 
• The investigator is obligated to perform or arrange for the conduct of supplemental measurements 
and/or evaluations as medically indicated or as requested by the sponsor to elucidate the nature 
and/or causality of the AE or SAE as fully as possible.  This may include additional laboratory tests 
or investigations, histopathological examinations, or consultation with other health care 
professionals. 
• New or updated information will be recorded in the originally completed eCRF. 
• Any updated SAE data will be submitted to the sponsor (or designee) within 24 hours of receipt of 
the information. 
• Once an AE is detected, it should be followed until it has resolved or until it is judged to be 
permanent; assessment should be made at each visit (or more frequently if necessary) of any changes 
in severity, the suspected relationship to the study drug, the interventions required to treat the event, 
and the outcome. 
• When the severity of an AE changes over time for a reporting period (eg, between visits), each 
change in severity will be reported as a separate AE until the event resolves. 
9.4. 
Reporting of Serious Adverse Events 
Regardless of suspected causality (eg, relationship to study drug or study procedure[s]), all SAEs 
occurring after the participant has signed the ICF through 35 days after the last dose of study 
treatment or until the participant starts a new anticancer therapy, whichever occurs earlier) must 
be reported to the sponsor (or designee) within 24 hours of learning of its occurrence, unless 
otherwise specified by the Protocol.  The investigator will submit any updated SAE data to the 
sponsor (or designee) within 24 hours of it being available.  For Japan, this information must also 
be reported immediately to the head of the study site. 
Investigators are not obligated to actively seek AE or SAE information after conclusion of the 
study participation.  However, if the investigator learns of any SAE, including a death, at any 
time after a participant has been discharged from the study, and he/she considers the event to be 
reasonably related to the study treatment or study participation, the investigator must notify the 
sponsor (or designee) within 24 hours of becoming aware of the event. 
After the initial AE/SAE report, the investigator is required to proactively follow each participant 
at subsequent visits/contacts.  All SAEs will be followed until resolution, stabilization, the event 
is otherwise explained, or the participant is lost to follow-up (as defined in Section 7.3). 
Prompt notification by the investigator to the sponsor of a SAE is essential so that legal 
obligations and ethical responsibilities towards the safety of participants and the safety of a study 
treatment under clinical investigation are met. 
If the SAE is not documented in the IB or the study drug (new occurrence) and is thought to be 
related to the sponsor's study drug, the sponsor or its designee may urgently require further 
information from the investigator for reporting to health authorities.  The sponsor or its designee 
may need to issue an Investigator Notification to inform all investigators involved in any study 
with the same drug that this SAE has been reported.  Suspected Unexpected Serious Adverse 
Reactions will be collected and reported to the competent authorities and relevant ethics 
committees in accordance with Directive 2001/20/EC, or as per national regulatory requirements 


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in participating countries.  For Japan, suspected expected deaths and life-threatening events will 
also be reported to the PMDA as per local regulatory requirements. 
The sponsor has a legal responsibility to notify both the local regulatory authority and other 
regulatory agencies about the safety of a study treatment under clinical investigation.  The 
sponsor will comply with country-specific regulatory requirements relating to safety reporting to 
the regulatory authority, IRB/IEC, and investigators. 
Investigator safety reports must be prepared for suspected unexpected adverse reactions 
according to local regulatory requirements and sponsor policy and forwarded to investigators as 
necessary. 
An investigator who receives an investigator safety report describing a SAE or other specific 
safety information (eg, summary or listing of SAEs) from the sponsor will review and then file it 
along with the IB and will notify the IRB/IEC, if appropriate according to local requirements. 
Serious Adverse Event Reporting 
• Information about all SAEs is collected and recorded on the Adverse Event Form in the eCRF. 
• The investigator must also complete the Incyte Serious Adverse Event Report Form, in English.  
Refer to the Incyte Reference Guide for Completing the Serious Adverse Event Report Form. 
• Facsimile or email transmission of the Serious Adverse Event Report Form is the preferred method 
to transmit this information to the PhV/designee (eg, C3i/Telerx).  The contact information of the 
sponsor's study-specific representatives is listed in the Study Reference Manual provided to each site.  
The original copy of the Serious Adverse Event Report Form and the confirmation sheet must be 
kept at the study site. 
• Follow-up information is recorded on an amended or new Serious Adverse Event Report Form, with 
an indication that it is follow-up to the previously reported SAE and the date of the original report.  
The follow-up report should include information that was not provided on the previous Serious 
Adverse Event Report Form, such as the outcome of the event (eg, resolved or ongoing), treatment 
provided, action taken with study drug because of the SAE (eg, dose reduced, interrupted, or 
discontinued), or participant disposition (eg, continued or withdrew from study participation).  Each 
recurrence, complication, or progression of the original event should be reported as follow-up to that 
event, regardless of when it occurs. 
• In rare circumstances and in the absence of facsimile or computer equipment, notification by 
telephone is acceptable with a copy of the Incyte Serious Adverse Event Report Form sent by 
overnight mail or courier service.  Initial notification via telephone does not replace the need for the 
investigator to complete and sign the Serious Adverse Event Report Form within the designated 
reporting timeframes. 
• Contacts for SAE reporting can be found in the Study Manual. 
• For Japan, any SAE relating to phosphate binders that were used for treatment of hyperphosphatemia 
should also be reported in the same manner. 


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9.5. 
Pregnancy 
Pregnancy, in and of itself, is not regarded as an AE unless there is suspicion that study drug may 
have interfered with the effectiveness of a contraceptive medication or method.  When a 
pregnancy has been confirmed in a participant during maternal or paternal exposure to study 
drug, the following procedures should be followed in order to ensure safety: 
• The study treatment must be interrupted immediately (female participants only). 
• If the female participant is no longer pregnant and meets the treatment continuation 
criteria within 28 days of the scheduled start of a cycle (or your maximum allowable 
time off study drug), study treatment may be resumed after approval has been 
received from the sponsor medical monitor.  (Further note:  Careful considerations 
should be taken regarding the anticoagulant properties of the study drug(s) and 
potential bleeding risks associated with restarting to determine if this is a viable 
addition to your Protocol.) 
• The investigator must complete and submit the Incyte Clinical Trial Pregnancy Form 
to the sponsor or its designee within 24 hours of learning of the pregnancy. 
Data on fetal outcome are collected for regulatory reporting and drug safety evaluation.  
Follow-up should be conducted for each pregnancy to determine outcome, including spontaneous 
or voluntary termination, details of the birth, and the presence or absence of any birth defects, 
congenital abnormalities, or maternal and/or newborn complications, by following until the first 
well-baby visit.  Pregnancy should be recorded on a Clinical Trial Pregnancy Form and reported 
by the investigator to the sponsor or its designee.  Pregnancy follow-up information should be 
recorded on the same form and should include an assessment of the possible causal relationship 
to the sponsor's study drug to any pregnancy outcome, as well as follow-up to the first well-baby 
visit or the duration specified in local regulations, whichever is later.  Refer to the Incyte 
Reference Guide for Completing the Clinical Trial Pregnancy Form. 
Any SAE occurring during pregnancy of a study participant must be recorded on the 
Serious Adverse Event Report Form and submitted to the sponsor or designee. 
Abnormal pregnancy outcomes (eg, spontaneous abortion, fetal death, stillbirth, congenital 
anomalies, or ectopic pregnancy) are considered SAEs (if occurring in the study participant) and 
must be reported as described in Section 9.4.  If an abnormal pregnancy outcome is reported in a 
study participant's partner, the event should be reported to the sponsor on the Clinical Trial 
Pregnancy Form. 
9.6. 
Warnings and Precautions 
Special warnings or precautions for the study drug, derived from safety information collected by 
the sponsor or its designee, are presented in the Investigator Brochure.  Additional safety 
information collected between IB updates will be communicated in the form of Investigator 
Notifications.  Any important new safety information should be discussed with the participant 
during the study, as necessary.  If new significant risks are identified, they will be added to the 
ICF. 


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9.7. 
Product Complaints 
The sponsor collects product complaints on study drugs and drug delivery systems used in 
clinical studies in order to ensure the safety of study participants, monitor quality, and facilitate 
process and product improvements. 
All product complaints associated with material packaged, labeled, and released by the sponsor 
or its designee will be reported to the sponsor.  All product complaints associated with other 
study material will be reported directly to the respective manufacturer. 
The investigator or his/her designee is responsible for reporting a complete description of the 
product complaint via email or other written communication to the sponsor contact or respective 
manufacturer as noted in the packaging information.  Any AE associated with a product 
complaint should be recorded as described in Section 9.3. 
If the investigator is asked to return the product for investigation, he/she will return a copy of the 
product complaint communication with the product. 
For Japan:  Complaints associated with unapproved medical devices will be reported to the 
sponsor with a Medical Device Defect Report Form, and the sponsor will report medical device 
defects to the PMDA as per local regulatory requirements. 
10. 
STATISTICS 
10.1. 
Sample Size Determination 
For Cohort A, approximately 60 participants are planned.  Assuming the true ORR is 35% for the 
intervention in this group, 60 participants are needed to ensure that there is at least 90% power to 
test the null hypothesis of ORR ≤ 15%.  This calculation assumes 1-sided test at the overall 
0.025 level of significance. 
For Cohort B, approximately 90 participants are planned.  Assuming the true ORR is 30% for the 
intervention in this group, 90 participants are needed to endure that there is at least 90% power to 
test the null hypothesis of ORR ≤ 15%.  This calculation assumes 1-sided test at the overall 
0.025 level of significance. 
For Cohort C, approximately 20 participants will be enrolled, which will provide at least 
80% chance of observing at least 4 responders in this cohort if the underlying ORR is 30%. 
10.2. 
Populations for Analysis 
Populations for analysis are shown in Table 16. 


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Table 16: 
Populations for Analysis 
Population 
Description 
Efficacy 
evaluable 
The efficacy evaluable population includes all enrolled participants in Cohorts A, B, 
and C who received at least 1 dose of study drug. 
The efficacy evaluable population will be used for the summary of demographics, 
baseline characteristics, participant disposition, and analyses of all efficacy data. 
Safety 
The safety population includes all enrolled participants who received at least 1 dose 
of study drug.  All safety analyses will be conducted using the safety population. 
Per protocol 
The per protocol population includes all efficacy evaluable population participants 
who are sufficiently compliant with the Protocol. 
RECIST/RANO 
evaluable 
The RECIST/RANO evaluable population includes all efficacy evaluable 
population participants who have at least 2 objective tumor response assessments 
per independent centralized radiological review or who discontinued from the study 
or who discontinued from treatment without post-treatment tumor assessments. 
RECIST/RANO evaluable population will be used for ORR analysis in the interim. 
10.3. 
Level of Significance 
The overall level of significance is 1-sided 0.025.  The primary endpoint in Cohort A and Cohort B 
will be tested sequentially at 1-sided 0.025.  If ORR in Cohort A is significant, then ORR in 
Cohort B will be tested. 
10.4. 
Statistical Analyses 
10.4.1. 
Primary Analysis 
The primary endpoints of the study are ORR in Cohort A and Cohort B, respectively.  Objective 
response rate is defined as the proportion of participants who achieved a CR or a PR based on 
RECIST v1.1 or RANO, as assessed by an independent centralized radiological review 
committee.  This analysis will be based on efficacy evaluable population.  Participants who do 
not have sufficient baseline or on-study response assessment information to be adequately 
assessed for response status will be included in the denominators in the calculation of ORR.  The 
95% CI for the ORR will be estimated using the Clopper-Pearson method.  The p-value from the 
exact binomial test will be provided. 
The ORR will also be analyzed based on the per protocol population as a sensitivity analysis. 
10.4.2. 
Secondary Efficacy Analysis 
Secondary efficacy analysis will be conducted for the efficacy evaluable population. 
Progression-free survival is defined as the time from date of first dose of study drug to the 
earliest date of progressive disease or death, whichever is first.  Progressive disease is evaluated 
based RECIST v1.1, or RANO, by the central radiographic review committee.  Participants who 
are alive without progression before analysis cutoff date will be censored.  Censoring for PFS 
will follow FDA guidance (FDA 2017).  Progression-free survival data will be analyzed by the 
Kaplan-Meier method in Cohorts A and B, respectively. 


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For objective responders, DOR is defined as the time from the date that a participant first 
achieves CR or PR based on RECIST v1.1, or RANO, until the date of first documented disease 
progression based on RECIST v1.1, or RANO, or death.  Participants who are alive without 
progression before analysis cut-off date will be censored.  Censoring of DOR will follow the 
same algorithm as the censoring of PFS.  Duration of response data will be analyzed by the 
Kaplan-Meier method in Cohorts A and B, respectively. 
Overall survival is defined as the time from date of first dose of study drug to death due to any 
cause.  Participants without death observed at the time of the analysis will be censored at last 
date known to be alive.  Overall survival will be analyzed by the Kaplan-Meier method in 
Cohorts A and B, respectively. 
10.4.3. 
Safety Analyses 
Safety analyses will be conducted for the safety population.  Adverse events will be coded by the 
MedDRA dictionary, and TEAEs (ie, AEs reported for the first time or worsening of a 
pre-existing event after first dose of study drug/treatment) will be tabulated by preferred term 
and system organ class for all events, related events, and events of Grade 3 or higher.  
Quantitative safety variables and their changes from baseline (laboratory, vital signs, etc) will be 
summarized with descriptive statistics.  Clinically notable abnormal values will be flagged and 
tabulated based on predefined criteria. 
The clinical laboratory data will be analyzed using summary statistics; no formal treatment group 
comparisons are planned.  In addition, distributions of key laboratory parameters may be plotted 
over time; these values will also be classified into CTCAE toxicity grades and tabulated.  
Descriptive statistics and mean change from baseline will be determined for vital signs at each 
assessment time.  Vital sign results will be reviewed for clinically notable abnormalities. 
Descriptive statistics and mean change from baseline will be determined for each ECG parameter 
at each assessment time.  Electrocardiogram results will be reviewed for clinically notable 
abnormalities according to predefined criteria (see Section 8.3.4).  Participants exhibiting 
clinically notable ECG abnormalities will be listed. 
Measures of exposure of study drug will be summarized by means of summary statistics. 


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10.5. 
Interim Analysis 
For Cohort A, futility analysis will be performed when approximately 25 participants are 
enrolled into the cohort and have at least 2 objective tumor response assessments per 
independent centralized radiological review or are discontinued from the study or are 
discontinued from treatment without post-treatment tumor assessments.  Cohort A can be 
stopped for futility if ≤ 4 responders are observed in RECIST/RANO evaluable population, for 
which there is < 10% probability of claiming ORR > 15% at final analysis.  This rule is just a 
guidance and nonbinding. 
For Cohort B, futility analysis will be performed when approximately 35 participants are 
enrolled into the cohort and have at least 2 objective tumor response assessments per 
independent centralized radiological review or are discontinued from the study or are 
discontinued from treatment without post-treatment tumor assessments.  Cohort B can be 
stopped for futility if ≤ 6 responders are observed in RECIST/RANO evaluable population, for 
which there is approximately 14% probability of claiming ORR > 15% at final analysis.  This 
rule is just a guidance and nonbinding. 
11. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS 
11.1. 
Investigator Responsibilities 
• The Protocol, Protocol amendments, ICF, IB, and other relevant documents (eg, 
advertisements) must be submitted to an IRB/IEC by the investigator and reviewed 
and approved by the IRB/IEC before the study is initiated. 
• The investigator is responsible for ensuring that the safety reports provided by the 
sponsor are reviewed and processed in accordance with regulatory requirements, the 
policies and procedures established by the IRB/IEC, and institutional requirements. 
• Any amendments to the Protocol will require IRB/IEC approval before 
implementation of changes made to the study design, except for changes necessary to 
eliminate an immediate hazard to study participants. 
• The investigator will be responsible for the following: 
− Providing written summaries of the status of the study to the IRB/IEC annually or 
more frequently in accordance with the requirements, policies, and procedures 
established by the IRB/IEC. 
− Notifying the IRB/IEC of SAEs or other significant safety findings as required by 
IRB/IEC procedures. 
− Providing oversight of the conduct of the study at the site and adherence to GCP, 
IRB/IEC requirements, institutional requirements, and applicable laws and 
country-specific regulations. 


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• Adhering to the Protocol as described in this document and agreeing that changes to 
the Protocol procedures, with the exception of medical emergencies, must be 
discussed and approved, first, by the sponsor or its designee and, second, by the 
IRB/IEC.  Each investigator is responsible for enrolling participants who have met 
the specified eligibility criteria. 
• Retaining records in accordance with all local, national, and regulatory laws, but for a 
minimum period of at least 2 years after the last marketing application approval in an 
ICH region and until there are no pending or contemplated marketing applications in 
an ICH region, or if not approved, 2 years after the termination of the test article for 
investigation to ensure the availability of study documentation should it become 
necessary for the sponsor or a regulatory authority to review. 
− The investigator must not destroy any records associated with the study without 
receiving approval from the sponsor.  The investigator must notify the sponsor or 
its designee in the event of accidental loss or destruction of any study records.  If 
the investigator leaves the institution where the study was conducted, the sponsor 
or its designee must be contacted to arrange alternative record storage options. 
− All eCRF data entered by the site (including audit trail), as well as computer 
hardware and software (for accessing the data), will be maintained or made 
available at the site in compliance with applicable record retention regulations.  
The sponsor will retain the original eCRF data and audit trail. 
• For Japan:  The record retainer (delegated by head of study site) will retain the J-GCP 
defined essential documentation at this site until the regulatory approval of study 
drug(s)/treatment or at least 3 years after the discontinuation or completion of the 
study conduct, whichever is later.  If the sponsor requires retention of these 
documents for a longer period of time, the duration and method of retention will be 
decided upon through discussion between the sponsor and the study site.  It is the 
responsibility of the sponsor to inform the head of the study site as to when the 
documents no longer need to be retained. 
11.2. 
Data Management 
Data management will be performed in a validated EDC system.  The investigator will be 
provided with access to an EDC system so that an eCRF can be completed for each participant. 
The site will be provided eCRF completion guidelines for instructions on data entry in the eCRF.  
The study monitor will reference the Monitoring Plan in order to ensure that each issue identified 
is appropriately documented, reported, and resolved in a timely manner in accordance with the 
plan's requirements. 
The sponsor (or designee) will be responsible for: 
• The data management of this study including quality checking of the data. 


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• Ensuring that study monitors perform ongoing source data verification to confirm that 
data entered into the eCRF by authorized site personnel are accurate, complete, and 
verifiable from source documents; that the safety and rights of participants are being 
protected; and that the study is being conducted in accordance with the currently 
approved protocol and any other study agreements, ICH GCP, and all applicable 
regulatory requirements. 
The investigator will be responsible for: 
• Ensuring participant data relating to the study is recorded in the eCRF unless 
transmitted to the sponsor or designee electronically (eg, laboratory data, diary data) 
or as otherwise specified in the Protocol.  The investigator is responsible for verifying 
that data entries are accurate and correct by physically or electronically signing the 
eCRF. 
• Maintaining accurate documentation (source data) that supports the information 
entered in the eCRF. 
− Source documents provide evidence for the existence of the participant and 
substantiate the integrity of the data collected.  Source documents are filed at the 
investigator's site. 
− Data reported on the CRF or entered in the eCRF that are transcribed from source 
documents must be consistent with the source documents or the discrepancies 
must be explained.  The investigator may need to request previous medical 
records or transfer records, depending on the study.  Also, current medical records 
must be available. 
• Permitting study-related monitoring, sponsor audits, IRB/IEC review, and regulatory 
inspections by providing direct access to source data and other relevant clinical study 
documents. 
− Monitoring:  Qualified representatives of the sponsor or its designee, study 
monitors, will monitor the study according to a predetermined plan.  The 
investigator must allow the study monitors to review any study materials and 
participant records at each monitoring visit. 
− Auditing:  Qualified representatives of the sponsor or its designee may audit the 
clinical study site and study data to evaluate compliance with the Protocol, 
applicable local clinical study regulations, and overall study conduct.  The 
investigator must allow the auditors to review original source records and study 
documentation for all participants. 
− Regulatory inspection:  Regulatory authorities may conduct an inspection of the 
study and the site at any time during the development of an investigational 
product.  The investigator and staff are expected to cooperate with the inspectors 
and allow access to all source documents supporting the eCRFs and other 
study-related documents.  The investigator must immediately notify the sponsor 
when contacted by any regulatory authority for the purposes of conducting an 
inspection. 


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11.3. 
Data Privacy and Confidentiality of Study Records 
The investigator and the sponsor or its designee must adhere to applicable data protection laws 
and regulations.  The investigator and the sponsor or its designee are responsible for ensuring 
that sensitive information is handled in accordance with local data protection laws (including but 
not limited to HIPAA and GDPR) as applicable.  Appropriate consent for collection, use, and 
disclosure and/or transfer (if applicable) of personal information must be obtained in accordance 
with local data protection laws. 
Participant names will not be supplied to the sponsor or its designee.  Only the participant 
number will be recorded in the eCRF; if the participant's name appears on any other document 
(eg, laboratory report), it must be obliterated on the copy of the document to be supplied to the 
sponsor or its designee.  Study findings stored on a computer will be stored in accordance with 
local data protection laws. 
Records and documents, including signed ICFs, pertaining to the conduct of this study must be 
retained by the investigator for 5 years after study completion unless local regulations or 
institutional policies require a longer retention period.  No records may be destroyed during the 
retention period without the written approval of the sponsor.  No records may be transferred to 
another location or party without written notification to the sponsor. 
11.4. 
Financial Disclosure 
Before study initiation, all clinical investigators participating in clinical studies subject to FDA 
Regulation Title 21 CFR Part 54 – Financial Disclosure by Clinical Investigators (ie, "covered 
studies") are required to submit a completed Clinical Investigator Financial Disclosure form that 
sufficiently details any financial interests and arrangements that apply.  For the purpose of this 
regulation, "clinical investigator" is defined as any investigator or subinvestigator who is directly 
involved in the treatment or evaluation of research participants, including the spouse and each 
dependent child of the clinical investigator or subinvestigator.  These requirements apply to both 
US and foreign clinical investigators conducting covered clinical studies. 
Any new clinical investigators added to the covered clinical study during its conduct must also 
submit a completed Investigator Financial Disclosure Form.  During a covered clinical study, 
any changes to the financial information previously reported by a clinical investigator must be 
reported to the sponsor or its designee.  At the conclusion of the covered clinical study, the 
clinical investigators will be reminded of their obligations.  In the event that the clinical 
investigator is not reminded, they nevertheless will remain obligated to report to the sponsor or 
its designee any changes to the financial information previously reported, as well as any changes 
in their financial information for a period of 1 year after completion of the covered clinical study. 
11.5. 
Publication Policy 
By signing the study Protocol, the investigator and his/her institution agree that the results of the 
study may be used by the sponsor, Incyte Corporation (Incyte), for the purposes of national and 
international registration, publication, and information for medical and pharmaceutical 
professionals.  Study results will be published in accordance with applicable local and national 
regulations.  If necessary, the authorities will be notified of the investigator's name, address, 
qualifications, and extent of involvement.  The terms regarding the publication of study results 


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are contained in the agreement signed with the sponsor or its designee.  A signed agreement will 
be retained by the sponsor or its designee. 
The results of this study may be published or presented at scientific meetings.  If this is foreseen, 
the investigator agrees to submit all manuscripts or abstracts to the sponsor before submission.  
This allows the sponsor to protect proprietary information and to provide comments. 
The sponsor will comply with the requirements for publication of study results.  In accordance 
with standard editorial and ethical practice, the sponsor will generally support publication of 
multicenter studies only in their entirety and not as individual site data.  In this case, a 
coordinating investigator will be designated by mutual agreement. 
Authorship will be determined in line with International Committee of Medical Journal Editors 
authorship requirements. 
11.6. 
Study and Site Closure 
The sponsor designee reserves the right to close the study site or terminate the study at any time 
for any reason at the sole discretion of the sponsor.  Study sites will be closed upon study 
completion.  A study site is considered closed when all required documents and study supplies 
have been collected and a study-site closure visit has been performed.  
For Japan:  When the study is completed, the investigator should inform the head of the study 
site of the completion in writing and submit a written summary of the study's outcome, and then 
the head of the study site should promptly inform the IRB and sponsor or designee of the 
completion in writing. 
The investigator may initiate study-site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination. 
Reasons for the early closure of a study site by the sponsor or investigator may include but are 
not limited to: 
• Failure of the investigator to comply with the protocol, the requirements of the 
IRB/IEC or local health authorities, the sponsor's procedures, or GCP guidelines. 
• Inadequate recruitment of participants by the investigator. 
• Discontinuation of further study treatment development. 


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12. 
REFERENCES 
American Joint Committee on Cancer. AJCC Cancer Staging Manual. 8th ed. New York, NY: 
Springer; 2018. 
Clinical Trials Facilitation and Coordination Group. Recommendations related to contraception 
and pregnancy testing in clinical trials. 2014.  
Dailey L, Ambrosetti D, Mansukhani A, Basilico C. Mechanisms underlying differential 
responses to FGF signaling. Cytokine Growth Factor Rev 2005;16:233-247. 
Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumors: 
revised RECIST guideline (version 1.1). Eur J Cancer 2009;45:228-247. 
Eswarakumar VP, Lax I, Schlessinger J. Cellular signaling by fibroblast growth factor receptors. 
Cytokine Growth Factor Rev 2005;16:139-149. 
European Medicines Agency. Guideline on the Investigation of Drug Interactions. 2012.  
European Medicines Agency. Guidance on the Management of Clinical Trials During the 
COVID-19 (Coronavirus) Pandemic. 2020. 
 
Farrow EG, White KE. Recent advances in renal phosphate handling. Nat Rev Nephrol 
2010;6:207-217. 
Food and Drug Administration. Guidance for Industry: Clinical Trial Endpoints for the Approval 
of Cancer Drugs and Biologics. 2007.  
Goyal L, Saha SK, Liu LY, et al. Polyclonal secondary FGFR2 mutations drive acquired 
resistance to FGFR inhibition in patients with FGFR2 fusion-positive cholangiocarcinoma. 
Cancer Discov 2017;7:252-263. 
Guagnano V, Kauffmann A, Wöhrle S, et al. FGFR genetic alterations predict for sensitivity to 
NVP-BGJ398, a selective pan-FGFR inhibitor. Cancer Discov 2012;2:1118-1133. 
Helsten T, Elkin S, Arthur E, Tomson BN, Carter J, Kurzrock R. The FGFR lLandscape in 
cancer: analysis of 4,853 Tumors by next-generation sequencing. Clin Cancer Res 
2016;22:259-267. 
INCB054828 Investigator's Brochure. Wilmington, DE: Incyte Corporation. 
Itoh N. Hormone-like (endocrine) Fgfs: their evolutionary history and roles in development, 
metabolism, and disease. Cell Tissue Res 2010;342:1-11. 
Khundmiri SJ, Murray RD, Lederer E. PTH and vitamin D. Compr Physiol 2016;6:561-601. 
Knights V, Cook SJ. De-regulated FGF receptors as therapeutic targets in cancer. Pharmacol 
Ther 2010;125:105-117. 
Kunii K, Davis L, Gorenstein J, et al. FGFR2-amplified gastric cancer cell lines require FGFR2 
and Erbb3 signaling for growth and survival. Cancer Res 2008;68:2340-2348. 


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Lamont FR, Tomlinson DC, Cooper PA, Shnyder SD, Chester JD, Knowles MA. Small molecule 
FGF receptor inhibitors block FGFR-dependent urothelial carcinoma growth in vitro and in vivo. 
Br J Cancer 2011;104:75-82. 
National Cancer Institute. Common Terminology Criteria for Adverse Events (CTCAE) 
Version 5.0. 2017.  
National Cancer Institute. Genomic Data Commons Data Portal. 2018.  
Oken MM, Creech RH, Tormey DC, et al. Toxicity and response criteria of the Eastern 
Cooperative Oncology Group. Am J Clin Oncol 1982;5:649-655. 
Porta R, Borea R, Coelho A, et al. FGFR a promising druggable target in cancer: molecular 
biology and new drugs. Crit Rev Oncol Hematol 2017;113:256-267. 
 
 
Qing J, Du X, Chen Y, et al. Antibody-based targeting of FGFR3 in bladder carcinoma and 
t(4;14)-positive multiple myeloma in mice. J Clin Invest 2009;119:1216-1229. 
Turner N, Grose R. Fibroblast growth factor signalling: from development to cancer. Nat Rev 
Cancer 2010;10:116-129. 
Weiss J, Sos ML, Seidel D, et al. Frequent and focal FGFR1 amplification associates with 
therapeutically tractable FGFR1 dependency in squamous cell lung cancer. Sci Transl Med 
2010;2:62ra93. 


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APPENDIX A. INFORMATION REGARDING EFFECTIVENESS OF 
CONTRACEPTIVE METHODS 
For male participants in the study: 
Male participants should use a condom from screening through 90 days after the end of systemic 
exposure.  If the male participant has a partner that is of child-bearing potential, the partner should also 
use contraception through 90 days after the end of relevant systemic exposure.  In addition, male 
participants must refrain from donating sperm from screening through 90 days after the end of relevant 
systemic exposure.  Males who have had a vasectomy qualify as having met the requirement for a 
highly effective birth control method. 
For female participants in the study: 
The following methods that can achieve a failure rate of less than 1% per year when used consistently 
and correctly are considered as highly effective birth control methods: 
• 
Combined (estrogen and progestogen containing) hormonal contraception associated with 
inhibition of ovulationa 
− 
oral 
− 
intravaginal (not applicable in Japan) 
− 
transdermal (not applicable in Japan) 
• 
Progestogen-only hormonal contraception associated with inhibition of ovulationa (not applicable 
in Japan) 
− 
oral 
− 
injectable 
− 
implantableb 
• 
Intrauterine deviceb 
• 
Intrauterine hormone-releasing systemb 
• 
Bilateral tubal occlusionb 
• 
Vasectomized partnerbc 
• 
Sexual abstinenced (not applicable in Japan) 
Acceptable birth control methods that result in a failure rate of more than 1% per year include: 
• 
Progestogen-only oral hormonal contraception, where inhibition of ovulation is not the primary 
mode of action 
• 
Male or female condom with or without spermicidee 
• 
Cap, diaphragm, or sponge with spermicidee 
• 
Tubal ligation 
a Hormonal contraception may be susceptible to interaction with the investigational medicinal product, which may 
reduce the efficacy of the contraception method. 
b Contraception methods that in the context of this guidance are considered to have low user dependency. 
c Vasectomized partner is a highly effective method of avoiding pregnancy provided that partner is the sole sexual 
partner of the woman of childbearing potential study participant and that the vasectomized partner has received 
medical assessment of the surgical success. 
d In the context of this guidance, sexual abstinence is considered a highly effective method only if defined as 
refraining from heterosexual intercourse during the entire period of risk associated with the study treatments.  The 
reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the 
preferred and usual lifestyle of the participant. 
e A combination of male condom with either cap, diaphragm, or sponge with spermicide (double barrier methods) 
are also considered acceptable, but not highly effective, birth control methods. 
Source:  Clinical Trials Facilitation and Coordination Group (2014). 


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APPENDIX B. INSTRUCTION TO PARTICIPANTS FOR HANDLING 
STUDY DRUG (PEMIGATINIB) 
The participant must be instructed in the handling of study drug as follows: 
• Store the study drug at room temperature. 
• Only remove the number of tablets needed at the time of administration. 
• Not to remove doses in advance of the next scheduled administration. 
• Make every effort to take doses on schedule. 
• Report any missed doses/lost tablets/capsules. 
• To take study drug with a full glass of water. 
• If the participant vomits after taking study drug, the participant should not take 
another dose. 
• To keep study drug in a safe place and out of reach of children. 
• To bring all used and unused study drug bottles/kits to the site at each visit. 
• If a dose of pemigatinib is missed by more than 4 hours, that dose should be skipped 
and the next scheduled dose should be administered at the usual time). 


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APPENDIX C. ACTIVATING FGFR MUTATIONS AND 
FUSIONS/REARRANGEMENTS 
Please follow instructions outlined in the Investigator Site Files for screening/enrolling 
participants. 
•
Cohort A:  FGFR1-3 in-frame fusions; any FGFR2 rearrangements; FGFR1/3 
rearrangement with known partner* (n = 60)
•
Cohort B:  Known or likely activating mutations (excluding kinase domain) in 
FGFR1-3 included in Appendix C (Table C1; n = 90)
•
Cohort C:  FGFR1-3 known activating mutations in kinase domain; FGFR1-3 
putatively activating mutations; other FGFR1/3 rearrangements* (not eligible for 
Cohort A; n = 20) 
*Only FGFR fusions or rearrangements with an intact kinase domain are eligible.
Point mutations in the kinase domains of FGFR1-3 are restricted to Cohort C given that some 
confer resistance or shift the in-vitro potency to FGFR inhibitors (Goyal et al 2017).  Kinase 
domains are defined as the following regions: 
FGFR1:  Ref sequence NM_023110:  protein residues 478-767 
FGFR2:  Ref sequence NM_000141:  protein residues 481-770 
FGFR3:  Ref sequence NM_000142:  protein residues 472-761 
Table C1 lists the non-kinase domain mutations in FGFR1-3 allowed in Cohort B without prior 
consultation with the sponsor.  Note that amino acid coordinates are based on the following 
RefSeq transcripts:  FGFR1;NM_023110, FGFR2;NM_000141, and FGFR3;NM000142).  This 
list contains alterations considered activating or likely activating (excluding the kinase domain) 
based on known activating mutations, homology to known activating mutations, and/or are 
present in somatic mutation databases.  This list is not all-inclusive, as some rare mutations may 
not have met our inclusion cutoff criteria. 


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Table C1: 
Known/Likely Activating Mutations of FGFR1-3 Allowed in Cohort B 
Gene 
Alteration Position 
Gene 
Alteration Position 
Gene 
Alteration Position 
FGFR1 
R189C 
189 
FGFR2 
R178C 
178 
FGFR3 
R175C 
175 
FGFR1 
P252R 
252 
FGFR2 
R203C 
203 
FGFR3 
R196C 
196 
FGFR1 
Y372C 
372 
FGFR2 
S252F 
252 
FGFR3 
R200C 
200 
FGFR1 
Y374C 
374 
FGFR2 
S252L 
252 
FGFR3 
R248C 
248 
FGFR1 
C379R 
379 
FGFR2 
S252W 
252 
FGFR3 
S249C 
249 
FGFR1 
C381R 
381 
FGFR2 
P253F 
253 
FGFR3 
G342C 
342 
 
 
 
FGFR2 
P253R 
253 
FGFR3 
S351C 
351 
 
 
 
FGFR2 
S267P 
267 
FGFR3 
G370C 
370 
 
 
 
FGFR2 
W290C 
290 
FGFR3 
S371C 
371 
 
 
 
FGFR2 
A315S 
315 
FGFR3 
Y373C 
373 
 
 
 
FGFR2 
A315T 
315 
FGFR3 
G375C 
375 
 
 
 
FGFR2 
D336G 
336 
FGFR3 
G380R 
380 
 
 
 
FGFR2 
C342F 
342 
FGFR3 
A391E 
391 
 
 
 
FGFR2 
C342G 
342 
 
 
 
 
 
 
FGFR2 
C342R 
342 
 
 
 
 
 
 
FGFR2 
C342S 
342 
 
 
 
 
 
 
FGFR2 
C342W 
342 
 
 
 
 
 
 
FGFR2 
C342Y 
342 
 
 
 
 
 
 
FGFR2 
S347C 
347 
 
 
 
 
 
 
FGFR2 
S351C 
351 
 
 
 
 
 
 
FGFR2 
S354C 
354 
 
 
 
 
 
 
FGFR2 
S372C 
372 
 
 
 
 
 
 
FGFR2 
Y375C 
375 
 
 
 
 
 
 
FGFR2 
C382R 
382 
 
 
 
 
 
 
FGFR2 
M391R 
391 
 
 
 


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APPENDIX D. CYP3A4 INHIBITORS AND INDUCERS 
CYP3A Inducers 
Inducers 
Therapeutic class 
Potent CYP3A Inducers  
Rifampin 
Antibiotics  
Mitotane 
Other Antineoplastics  
Avasimibe 
Other Antilipemics  
Rifapentine 
Antibiotics  
Apalutamide 
Antiandrogens  
Phenytoin 
Anticonvulsants 
Carbamazepine 
Anticonvulsants 
Enzalutamide 
Antiandrogens 
St John's Wort extract 
Herbal medications 
Lumacaftor 
Cystic fibrosis treatments 
Rifabutin 
Antibiotics 
Phenobarbital 
Anticonvulsants 
Moderate CYP3A Inducers  
Ritonavir and St. John's wort 
None 
Semagacestat 
Alzheimer's treatments 
Efavirenz 
NNRTIs 
Tipranavir and ritonavir 
Protease inhibitors 
Dabrafenib 
Kinase inhibitors 
Lesinurad 
Antigout and uricosuric agents 
Bosentan 
Endothelin receptor antagonists 
Genistein 
Food products 
Thioridazine 
Antipsychotics 
Nafcillin 
Antibiotics 
Talviraline 
NNRTIs 
Lopinavir 
Protease inhibitors 
Modafinil 
Psychostimulants 
Pf-06282999 
Myeloperoxidase inactivators 
Etravirine 
NNRTIs 
Lersivirine 
NNRTIs 
Telotristat ethyl 
Antidiarrheals 


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CYP3A Inhibitors 
Inhibitor 
Therapeutic Class 
Potent CYP3A Inhibitors  
VIEKIRA PAK 
Antivirals 
Indinavir/RIT 
Protease inhibitors 
Tipranavir/RIT 
Protease inhibitors 
Ritonavir 
Protease inhibitors 
Cobicistat (GS-9350) 
None 
Ketoconazole 
Antifungals 
Indinavir 
Protease inhibitors 
Troleandomycin 
Antibiotics 
Telaprevir 
Antivirals 
Danoprevir/RIT 
Antivirals 
Elvitegravir/RIT 
Treatments of AIDS 
Saquinavir/RIT 
Protease inhibitors 
Lopinavir/RIT 
Protease inhibitors 
Itraconazole 
Antifungals 
Voriconazole 
Antifungals 
Mibefradil 
Calcium channel blockers 
LCL161 
Cancer treatments 
Clarithromycin 
Antibiotics 
Posaconazole 
Antifungals 
Telithromycin 
Antibiotics 
Grapefruit juice DS 
Food products 
Conivaptan 
Diuretics 
Nefazodone 
Antidepressants 
Nelfinavir 
Protease inhibitors 
Saquinavir 
Protease inhibitors 
Ribociclib 
Kinase inhibitors 
Idelalisib 
Kinase inhibitors 
Boceprevir 
Antivirals 
 


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Inhibitor 
Therapeutic Class 
Moderate CYP3A Inhibitors  
Erythromycin 
Antibiotics 
Fluconazole 
Antifungals 
Atazanavir/RIT 
Protease inhibitors 
Darunavir 
Protease inhibitors 
Diltiazem 
Calcium channel blockers 
Darunavir/RIT 
Protease inhibitors 
Dronedarone 
Antiarrhythmics 
Crizotinib 
Kinase inhibitors 
Atazanavir 
Protease inhibitors 
Letermovir 
Antivirals 
GSK2647544 
Alzheimer's disease & dementia treatments 
Aprepitant 
Antiemetics 
Casopitant 
Antiemetics  
Amprenavir 
Protease inhibitors 
Faldaprevir 
Antivirals 
Imatinib 
Antineoplastic agents 
Verapamil 
Calcium channel blockers 
Netupitant 
Antiemetics 
Nilotinib 
Kinase inhibitors 
Grapefruit juice 
Food products 
Tofisopam 
Benzodiazepines 
Cyclosporine 
Immunosuppressants 
ACT-178882 
Renin inhibitors 
Ciprofloxacin 
Antibiotics 
Magnolia vine (Schisandra sphenanthera) 
Herbal medications 
Isavuconazole 
Antifungals 
Cimetidine 
H-2 receptor antagonists 
FK1706 
Central nervous system agents 


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APPENDIX E. COVID-19 PANDEMIC MITIGATION STRATEGIES AND 
INSTRUCTIONS 
The COVID-19 global pandemic presents numerous challenges to the ongoing conduct of 
clinical trials.  In line with the European Medicines Agency's Guidance on the Management of 
Clinical Trials During the COVID-19 (Coronavirus) Pandemic (2020), the sponsor has issued the 
following Protocol considerations to ensure participant safety is maintained and adequate 
benefit/risk analyses are applied relative to the completion of study procedures and maintaining 
the investigational product supply chain. 
Recognizing the flexibility required to manage the impact of the pandemic on this clinical trial, 
additional details will be added to respective study manuals and project plan documents and 
communicated to the investigative sites as needed. 
Study Site Visits 
If local travel restrictions, isolation requirements, or the investigator's benefit/risk assessment 
determines it to be unsafe for participants to attend study visits at the investigational site, the site 
staff may elect to pursue the following: 
• In order to minimize participant risk, study visits may be conducted via telemedicine 
modalities (phone or video).  At a minimum, a review of AEs, concomitant 
medications, and study drug compliance must be completed.  Periodic on-site visits 
should be conducted whenever feasible. 
• In order to support investigator oversight of participant safety and disease 
management, the participant may be asked to undergo some laboratory tests or study 
procedures (eg, eye examination) at a local (proximate) hospital laboratory or facility 
closer to the participant's residence rather than at the investigational site.  In this case, 
the study physician will provide the participant with the list of parameters to be 
checked.  These tests should be performed in certified laboratories. 
• Assessments that are missed should be noted as deviations to the Protocol and should 
be documented accordingly. 
Investigational Medicinal Product Dispensation and Distribution 
In order to ensure the continuity of providing their participant' clinical supplies within the 
constraints imparted by the pandemic, the site staff can decide to supply IMP to participants as 
follows: 
• Where possible, when the participant attends a visit at the study site, the investigator 
can dispense an additional amount of pemigatinib tablets to cover a longer interval 
between on-site study visits than stipulated in the SoA (see Table 3). 
• Alternatively, if the participant cannot attend a visit at the study site, adequate 
supplies of IMP to cover 1 or more cycles can be shipped to the participant by the 
investigator or appropriately delegated staff (eg, the study pharmacy staff) using a 
third-party service if duly authorized by the participant.  The study site may use their 
own preferred courier, provided the courier adheres to certain standards (eg, use of 


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personal protection equipment, maintenance of temperature-controlled transit 
environment), or one centrally contracted by the sponsor. 
Clinical Trial Monitoring 
Study monitoring visits could be postponed; however, the site monitor will continue to employ 
off-site monitoring practices such as routine communication methods (eg, phone calls, emails, 
video visits) with the sites to get information on trial progress, participant status, and information 
on issue resolution.  The study monitor may remotely review data entered into the EDC for 
accuracy and completeness.  Remote source data verification may be implemented with 
agreement of the principal investigator and institution, as applicable. 
If the study site monitor cannot be on-site to perform the final drug accountability for 
reconciliation purposes, and the operation cannot be postponed, it may be performed by a 
pharmacist from the hospital pharmacy or by the study coordinator/data manager with suitable 
training.  The IMP can be returned to the sponsor by the hospital pharmacy directly or destroyed 
in accordance with local practices, if applicable, and with sponsor approval. 
Direct Contracts With Third Parties/Specialized Service Companies 
If necessary, direct contracts can be established with third-party local physicians to conduct 
activities related to the clinical management of participants for whom the investigator is 
responsible and maintains oversight.  In such situations, the investigator is required to provide 
the local physician with a delegation letter listing all delegated activities.  The sponsor, through 
the study investigator or institution, will reimburse the local physician for the test/procedures 
conducted outside of the standard of care. 
Reimbursement of Extraordinary Expenses 
The sponsor will arrange to reimburse participants for any extraordinary expenses, keeping 
appropriate documentation as evidence (eg, travel expenses for the local laboratory visit[s], the 
costs of local [proximate] laboratory tests). 


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APPENDIX F. PROTOCOL AMENDMENT SUMMARY OF CHANGES 
Document 
Date 
Amendment (Version) 1: 
14 FEB 2019 
Amendment (Version) 2: 
14 JAN 2020 
Amendment (Version) 3: 
15 FEB 2021 
Amendment 3 (15 FEB 2021) 
Overall Rationale for the Amendment:  To incorporate updates regarding tumor biopsy timing 
and other clarifications. 
 
 
 
 
 
 
 
2. Section 1, Protocol Summary 
Description of change:  Added explanation why the term "translocation" was changed to 
"fusion/rearrangement." 
Rationale for change:  The genomic terms have evolved with new research and 
understanding of the alterations. 
3. Section 1, Protocol Summary (Figure 1:  Study Design Schema); Section 2, 
Introduction; Section 3, Objectives and Endpoints; Section 4.1, Overall Design; 
Section 5.1, Inclusion Criteria; Appendix C, Activating FGFR Mutations and 
Fusions/Rearrangements 
Description of change:  Clarified the description of Cohorts A and C and updated the 
term "translocation" to "fusion/rearrangement." 
Rationale for change:  The genomic terms have evolved with new research and 
understanding of the alterations.  Many questions were received from investigators 
regarding the types of alterations accepted for Cohorts A and C, so clarification of this 
language was needed. 


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4. Section 1, Protocol Summary; Section 4.1, Overall Design; Section 5.1, Inclusion 
Criteria (Criterion 7); Section 8.5.1, Tumor for Genomics Testing for FGFR Genetic 
Alterations and Gene Expression; Section 8.5.5, Tissue Biopsies  
Description of change:  Participants require local documentation of an activating 
FGFR1, FGFR2, or FGFR3 mutation or gene rearrangement prior to starting the 
screening process.  FGFR results need to be based on a local result of a fresh biopsy or an 
archival tissue biopsy taken within 24 months of screening.  The wording was clarified to 
specify that both the date the sample was taken and the date of the genomics report must 
be within 24 months of screening.  
Rationale for change:  The stability of FGFR genomic alterations over time is unknown.  
Due to the vast array of tumor types in this study, a 12-month limit on the tumor sample 
and report was originally established to ensure the genomic report accurately reflects the 
participant's molecular profile at baseline and is likely to be confirmed by the central 
laboratory.  After conferring with numerous investigators, it was evident that driver 
mutations should not change in < 24 months.  Therefore, it was decided to expand the 
time from 12 months to 24 months.  Participants with local laboratory test results 
(including tissue- or plasma-based molecular analysis) documenting an activating 
FGFR1, FGFR2, or FGFR3 mutation or rearrangement are eligible to enroll as long as the 
results meet all the eligibility criteria. 
5. Section 1, Protocol Summary (Table 3:  Schedule of Activities) 
Description of change:  Added a time window of ± 7 days to the eye examinations (slit 
lamp, visual acuity, funduscopy with digital imaging) and CT or MRI assessments.  
Rationale for change:  Administrative error. 
6. Section 1, Protocol Summary (Table 4:  Schedule of Laboratory Assessments) 
Description of change:  For tumor tissue sampling, added text clarifying that if EOT 
occurs < 6 weeks after the on-treatment biopsy was performed, an EOT biopsy should not 
be performed. 
Rationale for change:  Administrative error. 
7. Section 2.2.2, Justification for Dose and Regimen (Figure 2:  Comparison of 
Steady-State Exposures for Pemigatinib 13.5 mg QD Between Participants With 
Nonhyperphosphatemia and Hyperphosphatemia [C1D8]) 
Description of change:  Added text to the figure to indicate different dosing regimens. 
Rationale for change:  Clarification. 
8. Section 2.3, Benefit/Risk Assessment of Pemigatinib 
Description of change:  Updated text to align with the most recent version of the IB. 
Rationale for change:  Edition 7 of the IB was released in JAN 2021, so this section was 
updated to align with the most recent edition of the IB. 


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9. Section 4.1, Overall Design 
Description of change:  Clarified that an on-treatment biopsy is only required for 
participants in the biopsy subset. 
Rationale for change:  Investigator feedback. 
10. Section 4.3, Study Termination 
Description of change:  Added text to reflect the responsibility of the head of study site 
(Japan) in regard to study termination. 
Rationale for change:  Requirement for Japan. 
11. Section 5.1, Inclusion Criteria (Criteria 1 and 9)  
Description of change:  Added text to indicate the need for parental or legal guardian's 
consent for legally minor participants from Japan (Inclusion Criterion 1) and willingness 
to avoid pregnancy or fathering children for Japanese participants (Inclusion Criterion 9). 
Rationale for change:  Recommended standard practices in Japan. 
12. Section 5.2, Exclusion Criteria (Criterion 11) 
Description of change:  Refined the wording regarding gastrointestinal abnormality. 
Rationale for change:  To further clarify and refine this exclusion criterion. 
13. Section 6.1, Study Treatment Administered (Table 11:  Study Treatment 
Information); Section 6.2, Preparation, Handling, and Accountability; Section 6.5.2, 
Management of Hyperphosphatemia 
Description of change:  Study treatment information for phosphate binders was added 
for Japan.  Text was added to clarify that phosphate binders are provided by the sponsor 
and that the required documentation for binders is the same as for study drug in Japan.  
Text was also added to include the role of the investigational drug storage manager in 
Japan. 
Rationale for change:  Requirement for Japan. 
14. Section 6.5.1, Criteria and Procedures for Dose Interruptions and Adjustments of 
Study Drug  
Description of change:  Added text regarding retinal detachment as well as guidance on 
the grading of retinal adverse events and clarified that re-escalation is not allowed after a 
dose reduction.   
Rationale for change:  To provide guidance on how to manage and grade SRD/RPED 
and investigator feedback. 


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16. Section 8.1.2, Prescreening and Screening Procedures 
Description of change:  A prescreening consent was added to the study.  Participants 
will be required to sign a specific prescreening consent form if the site needs to consult 
with Incyte on the eligibility of a participant's genomic alteration.  This is not required for 
all participants; it applies only to those whose genomic alterations need to be reviewed 
for eligibility. 
Rationale for change:  The Protocol was designed to guide the sites and provide as 
much information as possible on which genomic alterations are eligible for the study.  As 
this field is evolving and new alterations are being discovered, the sites will need 
guidance on the eligibility of their participants.  Due to data privacy laws, participants 
will need to sign a prescreening consent form for Incyte to be able to review the genomic 
alterations of these participants.  
17. Section 8.1.2, Prescreening and Screening Procedures  
Description of change:  Revised the statement regarding the review of results from the 
screening visit evaluations to confirm eligibility to indicate that the results will be 
reviewed by the investigator.  
Rationale for change:  Clarification that no reviews are performed by Incyte or the 
contract research organization to confirm eligibility. 
18. Section 8.1.4, Distribution of Reminder Cards  
Description of change:  Removed text explaining that the reminder card will include an 
area regarding the contents of the participant's last meal.  
Rationale for change:  Administrative error. 
19. Section 8.3.1, Adverse Events 
Description of change:  Added text to indicate that, for Japanese participants, the 
relationship with phosphate binders should also be collected on the Adverse Events Form 
in the eCRF. 
Rationale for change:  Requirement for Japan. 


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20. Section 8.3.6, Laboratory Assessments  
Description of change:  Clarified that serum phosphate testing will be required on Day 8 
in Cycles 2+, rather than just on a more frequent basis, if a participant develops 
hyperphosphatemia during Cycle 1.  
Rationale for change:  To align with Table 4. 
21. Section 8.3.6, Laboratory Assessments (Table 14:  Required Laboratory Analytes) 
Description of change:  Added text that bicarbonate or CO2 is not applicable for Japan. 
Rationale for change:  This is not a standard test in Japan. 
22. Section 8.5.1, Tumor for Genomics Testing for FGFR Genetic Alterations and Gene 
Expression  
Description of change:  Added text that if a screening sample has already been analyzed 
by Foundation Medicine, Inc, as the local laboratory, then the site does not need to send 
another sample to Foundation Medicine, Inc, for central confirmation.  
Rationale for change:  The original wording did not explain that participants who use 
the central genomics laboratory, Foundation Medicine, Inc, as their local laboratory will 
not need to have their tissue- or plasma-based molecular assays retested to document an 
activating FGFR1, FGFR2, or FGFR3 mutation or gene rearrangement. 
23. Section 9.2, Definition of Serious Adverse Event 
Description of change:  Added text about an event leading to disability for Japan. 
Rationale for change:  Requirement for Japan. 
24. Section 9.4, Reporting of Serious Adverse Events 
Description of change:  Added text to indicate that SAEs must be reported to the head of 
the study site, suspected expected deaths and life-threatening events will be reported to 
the PMDA, and events related to phosphate binder use for the treatment of 
hyperphosphatemia will be reported as SAEs in Japan. 
Rationale for change:  Requirement for Japan. 
25. Section 9.7, Product Complaints 
Description of change:  Added text for unapproved medical devices in Japan. 
Rationale for change:  Requirement for Japan. 
26. Section 10.2, Populations for Analysis (Table 16:  Populations for Analysis); 
Section 10.5, Interim Analysis 
Description of change:  Clarified the RECIST/RANO evaluable populations. 
Rationale for change:  Clarification requested by internal team. 


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27. Section 10.5, Interim Analysis 
Description of change:  Clarified that patients who are discontinued from treatment 
without post-treatment tumor assessments will be included in the interim analysis. 
Rationale for change:  Clarification requested by internal team. 
28. Section 11.1, Investigator Responsibilities 
Description of change:  Added record retention requirements for Japan. 
Rationale for change:  Requirement for Japan. 
29. Section 11.6, Study and Site Closure 
Description of change:  Added text to reflect the responsibility of the investigator and 
head of study site (Japan) upon study completion. 
Rationale for change:  Requirement for Japan. 
30. Appendix A, Information Regarding Effectiveness of Contraceptive Methods 
Description of change:  Clarified which forms of contraception are not applicable in 
Japan. 
Rationale for change:  Requirement for Japan. 
31. Appendix E, COVID-19 Pandemic Mitigation Strategies and Instructions 
Description of change:  Added Appendix E. 
Rationale for change:  To provide sites with Protocol-related guidance in response to 
the COVID-19 pandemic. 
32. Incorporation of administrative changes.  Other minor, administrative changes have 
been incorporated throughout the Protocol and are noted in the redline version of the 
amendment. 
 


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Amendment 2 (14 JAN 2020) 
Overall Rationale for the Amendment:  To incorporate updates of the cohort definitions and 
other changes based on FDA review of other pemigatinib protocols. 
1. Section 1, Protocol Summary (Figure 1, Study Design Schema); Section 2.1.2, Solid 
Tumor Malignancies With Activating FGFR Mutations and Translocations; 
Section 3, Objectives and Endpoints; Section 4, Overall Design; Section 5.1, 
Inclusion Criteria; Section 8.5.1, Tumor for Genomics Testing for FGFR Genetic 
Alterations and Gene Expression;
Appendix C, Activating FGFR Mutations and Translocations 
Description of change:  
 
 added the coordinating principal investigator; refined the 
definitions of the cohorts; and added language for capping of cohorts. 
Rationale for change:  To provide more clarity regarding analysis of efficacy data and to 
ensure proper enrollment of participants into the correct cohort. 
2. Section 1, Protocol Summary (Table 3:  Schedule of Activities); Section 8.3.5, 
Comprehensive Eye Examination 
Description of change:  Added mandatory OCT testing at baseline and every 3 cycles as 
part of normal eye exams. 
Rationale for change:  Per FDA requirement. 
3. Section 1, Protocol Summary (Table 4:  Schedule of Laboratory Assessments); 
Section 8.3.6, Laboratory Assessments 
Description of change:  Removal of reference to central laboratory testing for safety 
laboratory assessments.  Added qualifications/windows around requirement for testing. 
Rationale for change:  All safety laboratory testing will be performed locally and testing 
windows were added to decrease blood draws, if applicable. 
 
 
 
 
 
5. Section 6.5.1, Criteria and Procedures for Dose Interruptions and Adjustments of 
Study Drug; Section 6.5.3, Up-Titration 
Description of change:  Updated the language to allow up-titrated participants to reduce 
dose down to 4.5 mg (3 dose reductions). 
Rationale for change:  To allow equal dose reduction limits to all participants. 
6. Incorporation of administrative changes.  Other minor, administrative changes have 
been incorporated throughout the Protocol and are noted in the redline version of the 
amendment. 


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Amendment 1 (14 FEB 2019) 
Overall Rationale for the Amendment:  To incorporate updates based on VHP review of other 
pemigatinib protocols and to update safety information based on the revised/updated IB. 
1. Section 1, Protocol Summary; Section 4.1, Overall Design; Section 5.1, Inclusion 
Criteria (Criterion 4); Appendix C, FGFR Mutations and Translocations 
Description of change:  Updated the description of Cohort A to include FGFR2 
intron 17 rearrangements.  Updated the description of Cohort C to include 
FGFR1/FGFR3 rearrangements with unknown partners. 
Rationale for change:  To clarify cohort assignment for participants with unknown 
fusion partners. 
2. Section 1, Protocol Summary (Table 4, Schedule of Laboratory Assessments); 
Section 8.3.6, Laboratory Assessments 
Description of change:  Included additional testing of parathyroid hormone every 
3 cycles starting with Cycle 3. 
Rationale for change:  VHP request. 
3. Section 1, Protocol Summary (Table 4, Schedule of Laboratory Assessments) 
Description of change:  Added coagulation assessments on Day 1 of each cycle. 
Rationale for change:  Table was updated to reflex the coagulation test in the Protocol 
text. 
4. Section 2.2.2,  Justification for Dose and Regimen 
Description of change:  Added language to support the prohibition of potent CYP3A4 
inhibitors and inducers and moderate inducers. 
Rationale for change:  VHP request. 
5. Section 2.3, Benefit/Risk of Pemigatinib 
Description of change:  Updated clinical safety experience with data from the current IB 
(Edition 5 dated 14 JAN 2019). 
Rationale for change:  To align the Protocol with the updated IB. 
6. Section 5.2, Exclusion Criteria 
Description of change:  Criterion 1 updated to exclude participants who have received a 
prior FGFR inhibitor.  The time frame of "within the past 6 months" has been removed. 
Rationale for change:  Participants cannot have received a prior FGFR inhibitor. 
7. Section 5.2, Exclusion Criteria 
Description of changes:  In exclusion criterion 9 (Table 9, Exclusionary Laboratory 
Values), modified the exclusion criteria for liver function tests.  Added new criterion 26 


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to exclude participants with a history of hypovitaminosis D requiring supraphysiologic 
doses to replenish the deficiency. 
Rationale for changes:  VHP request. 
8. Section 6.5.1, Criteria and Procedures for Dose Interruptions and Adjustments of 
Study Drug (Table 10, Guidelines for Interruption and Restarting of Study Drug); 
Section 6.5.4, Criteria for Permanent Discontinuation of Study Drug 
Description of change:  Added language to discontinue study drug if extreme changes in 
ECG output occur. 
Rationale for change:  VHP request. 
9. Section 6.5.1, Criteria and Procedures for Dose Interruptions and Adjustments of 
Study Drug; Section 6.5.3, Up-Titration 
Description of change:  Added guidance on recommended maximum 2 dose level 
reductions for participants who are up-titrated to pemigatinib 18 mg. 
Rationale for change:  To provide additional guidance on allowed dose reductions. 
10. Section 6.5.2, Management of Hyperphosphatemia 
Description of change:  Added examples of diet controls and phosphate binders to treat 
hyperphosphatemia. 
Rationale for change:  VHP request. 
11. Section 6.6, Concomitant Medications, Appendix D, CYP3A Inhibitors and Inducers 
Description of change:  Added language to support the prohibition of potent CYP3A4 
inhibitors and inducers and moderate inducers.  A list of CYP3A4 inhibitors and inducers 
was added in Appendix D. 
Rationale for change:  VHP request. 
12. Incorporation of administrative changes.  Other minor, administrative changes have 
been incorporated throughout the Protocol and are noted in the redline version of the 
amendment. 


Signature Page for VV-CLIN-000652 v4.0
Signature Page for VV-CLIN-000652 v4.0
Approval
Approver
16-Feb-2021 07:53:13 GMT+0000
Approval
Document Preparer
16-Feb-2021 08:12:58 GMT+0000
Approval
Approver
16-Feb-2021 12:25:39 GMT+0000
Approval
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16-Feb-2021 20:41:39 GMT+0000

SAP_001.pdf: 
Official Title: 
NCT Number: 
Document Date: 
A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the 
Efficacy and Safety of Pemigatinib in Participants With Previously 
Treated Locally Advanced/Metastatic or Surgically Unresectable Solid 
Tumor Malignancies Harboring Activating FGFR Mutations or 
Translocations (FIGHT-207)
NCT03822117
Statistical Analysis Plan: 23 September 2020


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INCB 54828-207 Statistical Analysis Plan 
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CONFIDENTIAL 
Statistical Analysis Plan 
INCB 54828-207 
A Phase 2, Open-Label, Single-Arm, Multicenter Study to Evaluate the 
Efficacy and Safety of Pemigatinib in Participants With Previously 
Treated Locally Advanced/Metastatic or Surgically Unresectable Solid 
Tumor Malignancies Harboring Activating FGFR Mutations or 
Translocations (FIGHT-207) 
IND Number: 
 
EudraCT Number: 
2018-004768-69 
Sponsor: 
Incyte Corporation 
1801 Augustine Cut-Off 
Wilmington, DE 19803 
United States  
Protocol Version: 
Protocol Amendment 2 dated 14 JAN 2020 
CRF Approval Date: 
12 APR 2020 
SAP Version: 
Original 
SAP Author: 
 Biostatistics 
Date of Plan: 
23 SEP 2020 
This study is being conducted in compliance with good clinical practice, 
including the archiving of essential documents. 
PPD


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TABLE OF CONTENTS 
LIST OF ABBREVIATIONS ..........................................................................................................5 
1.
INTRODUCTION ........................................................................................................6 
2.
STUDY INFORMATION, OBJECTIVES, AND ENDPOINTS .................................6 
2.1. 
Protocol and Case Report Form Version ......................................................................6 
2.2. 
Study Objectives and Endpoints ...................................................................................6 
3.
STUDY DESIGN .........................................................................................................8 
3.1. 
Randomization ..............................................................................................................9 
3.2. 
Control of Type I Error .................................................................................................9 
3.3. 
Sample Size Considerations .......................................................................................10 
3.4. 
Schedule of Assessments ............................................................................................10 
4.
DATA HANDLING DEFINITIONS AND CONVENTIONS ..................................10 
4.1. 
Scheduled Study Evaluations and Study Periods .......................................................10 
4.1.1. 
Day 1 ...........................................................................................................................10 
4.1.2. 
Study Day ...................................................................................................................10 
4.1.3. 
Baseline Value ............................................................................................................11 
4.1.4. 
Handling of Missing and Incomplete Data .................................................................11 
4.1.5. 
Cycle Length and Duration .........................................................................................11 
4.1.6. 
Analysis Window ........................................................................................................12 
4.2. 
Variable Definitions ....................................................................................................12 
4.2.1. 
Prior and Concomitant Medication .............................................................................12 
5.
STATISTICAL METHODOLOGY ...........................................................................12 
5.1. 
General Methodology .................................................................................................12 
5.2. 
Treatment Groups .......................................................................................................12 
5.3. 
Analysis Populations ..................................................................................................13 
5.3.1. 
Efficacy Evaluable Population ...................................................................................13 
5.3.2. 
Safety Population ........................................................................................................13 
5.3.3. 
Per Protocol Population ..............................................................................................13 
5.3.4. 
RECIST Evaluable Population ...................................................................................13 
6.
BASELINE, EXPOSURE, AND DISPOSITION ......................................................14 
6.1. 
Baseline and Demographics, Physical Characteristics, and Disease History .............14 
6.1.1. 
Demographics and Baseline Characteristics ...............................................................14 


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6.1.2. 
Baseline Disease Characteristics ................................................................................14 
6.1.3. 
Disease History ...........................................................................................................14 
6.1.4. 
Prior Therapy ..............................................................................................................14 
6.1.5. 
Medical History ..........................................................................................................15 
6.2. 
Disposition of Participant ...........................................................................................15 
6.3. 
Protocol Deviations ....................................................................................................15 
6.4. 
Exposure .....................................................................................................................15 
6.5. 
Study Drug Compliance .............................................................................................16 
6.6. 
Prior and Concomitant Medication .............................................................................16 
7. 
EFFICACY .................................................................................................................16 
7.1. 
Efficacy Hypotheses ...................................................................................................16 
7.2. 
Analysis of the Primary Efficacy Parameter ..............................................................16 
7.2.1. 
Primary Efficacy Analysis ..........................................................................................16 
7.2.1.1. 
Response Criteria ........................................................................................................17 
7.2.1.2. 
Objective Response Rates and Best Overall Response ..............................................17 
7.2.2. 
Subgroup Analyses for Primary Endpoint ..................................................................17 
7.2.3. 
Sensitivity and Supportive Analyses for Primary Endpoint .......................................17 
7.3. 
Analysis of the Secondary Efficacy Parameters .........................................................17 
7.3.1. 
Progression-Free Survival ..........................................................................................17 
7.3.2. 
Duration of Response .................................................................................................18 
7.3.3. 
Overall Survival ..........................................................................................................19 
7.4. 
Other Efficacy Analyses .............................................................................................19 
7.4.1. 
Largest Percentage Reduction in Sum of Diameters of Target Lesions .....................19 
 
19 
 
19 
 
20 
 
21 
8. 
SAFETY AND TOLERABILITY ..............................................................................21 
8.1. 
General Considerations ...............................................................................................21 
8.2. 
Adverse Events ...........................................................................................................21 
8.2.1. 
Adverse Event Definitions ..........................................................................................21 
8.2.2. 
Clinically Notable Adverse Events .............................................................................22 
8.2.3. 
Adverse Event Summaries ..........................................................................................23 


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8.3. 
Clinical Laboratory Tests ...........................................................................................24 
8.3.1. 
Laboratory Value Definitions .....................................................................................24 
8.3.2. 
Laboratory Value Summaries .....................................................................................24 
8.3.3. 
Potential Hy’s Law Events .........................................................................................25 
8.4. 
Vital Signs ..................................................................................................................25 
8.5. 
Electrocardiograms .....................................................................................................25 
9. 
INTERIM ANALYSIS ...............................................................................................26 
10. 
CHANGES AND MODIFICATIONS TO THE ANALYSIS PLAN ........................26 
10.1. 
Changes to Protocol-Defined Analyses ......................................................................26 
10.2. 
Changes to the Statistical Analysis Plan .....................................................................26 
11. 
REFERENCES ...........................................................................................................27 
APPENDIX A. PLANNED TABLES, FIGURES, AND LISTINGS ........................................28 
 
LIST OF TABLES 
Table 1: 
Objectives and Endpoints .............................................................................................6 
Table 2: 
Evaluation and Censoring of Progression-Free Survival ............................................18 
 
 
20 
Table 4: 
Clinically Notable Adverse Events Groupings ...........................................................22 
Table 5: 
Criteria for Clinically Notable Vital Sign Abnormalities ...........................................25 
Table 6: 
Criteria for Clinically Notable Electrocardiogram Abnormalities .............................26 
Table 7: 
Statistical Analysis Plan Versions ..............................................................................26 
 
LIST OF FIGURES 
Figure 1: 
Study Design Schema ...................................................................................................9 
 


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LIST OF ABBREVIATIONS 
Abbreviation 
Term 
AE 
adverse event 
CI 
confidence interval 
CR 
complete response 
CRF 
case report form 
CTCAE 
Common Terminology Criteria for Adverse Events 
CTEP 
Cancer Therapy Evaluation Program 
 
 
ECG 
electrocardiogram 
ECOG 
Eastern Cooperative Oncology Group 
FDA 
Food and Drug Administration 
MedDRA 
Medical Dictionary for Regulatory Activities  
NE 
not evaluable 
OS 
overall survival 
PD 
progressive disease 
PFS 
progression-free survival 
PP 
per protocol 
PR 
partial response 
PT 
preferred term 
RECIST 
Response Evaluation Criteria in Solid Tumors 
SAE 
serious adverse event 
SD 
stable disease 
SAP 
Statistical Analysis Plan 
SOC 
system organ class 
TEAE 
treatment-emergent adverse event 
WHO 
World Health Organization  
 


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1. 
INTRODUCTION 
This is a Phase 2, open-label, single-arm, multicenter study in participants with previously 
treated locally advanced/metastatic or surgically unresectable solid tumor malignancies 
harboring activating FGFR mutations or translocations.  Section 2 of the Protocol provides a 
detailed description of the investigational product, target patient population, rationale for doses 
to be examined, and potential risks and benefits of treatment with pemigatinib.   
The purpose of this SAP is to provide details of the statistical analyses that have been outlined in 
the Study INCB 54828-207 Protocol.   
2. 
STUDY INFORMATION, OBJECTIVES, AND ENDPOINTS 
2.1. 
Protocol and Case Report Form Version 
This SAP is based on INCB 54828-207 Protocol Amendment 2 dated 14 JAN 2020 and CRFs 
approved on 12 APR 2020.  Unless superseded by an amendment, this SAP will be effective for 
all subsequent Protocol amendments and eCRF versions. 
2.2. 
Study Objectives and Endpoints 
Table 1 presents the objectives and endpoints. 
Table 1: 
Objectives and Endpoints 
Objectives 
Endpoints 
Primary 
• To evaluate the efficacy of pemigatinib in 
participants with locally advanced/metastatic or 
surgically unresectable solid tumor malignancy 
with an activating FGFR mutation or 
translocation. 
• ORR in Cohort A, defined as the proportion of 
participants in Cohort A who achieve a CR or 
PR based on RECIST v1.1 or RANO. 
• ORR in Cohort B, defined as the proportion of 
participants in Cohort B who achieve a CR or 
PR based on RECIST v1.1 or RANO. 
• An independent radiological review committee 
will determine response. 
 
 
 
 
 
 


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Table 1: 
Objectives and Endpoints (Continued) 
Objectives 
Endpoints 
Secondary 
• To evaluate other clinical efficacy 
measurements of pemigatinib in participants 
with locally advanced/metastatic or surgically 
unresectable solid tumor malignancy with an 
activating FGFR mutation and/or translocation. 
• Safety and tolerability of pemigatinib. 
• PFS, defined as the time from first dose until 
PD (according to RECIST v1.1 or RANO and 
assessed by an ICR) or death (whichever is first) 
in Cohorts A and B, respectively. 
• DOR, defined as the time from the date of first 
assessment of CR or PR until the date of the 
first PD by an ICR per RECIST v1.1 or RANO, 
or death (whichever is first) in Cohorts A and B, 
respectively. 
• OS, defined as the time from first dose of study 
drug to death of any cause in Cohorts A and B, 
respectively. 
• Safety and tolerability, as assessed by the 
occurrence of TEAEs and treatment-related AEs 
according to NCI CTCAE v5.0, physical exam 
changes, vital sign changes, laboratory 
evaluations, and ECGs in each cohort. 


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3. 
STUDY DESIGN 
This is an open-label, monotherapy study of pemigatinib in participants with advanced/metastatic 
or surgically unresectable solid tumor malignancy harboring an activating FGFR mutation or 
translocation.  This study consists of 3 cohorts, Cohort A, Cohort B, and Cohort C and will enroll 
approximately 60 participants, 90 participants, and 20 participants, respectively (see Figure 1).  
Participants will receive a QD dose of pemigatinib at 13.5 mg continuously as long as 
participants are receiving benefit and have not met any criteria for study withdrawal.  
Participants with local laboratory test results (eg, tissue or plasma-based molecular assays) 
documenting an activating FGFR1, FGFR2, or FGFR3 mutation or gene rearrangement are 
eligible to enroll as long as the results meet the cohort criteria.  Confirmatory testing through the 
central genomics laboratory will be performed for all participants; however, results from the 
central genomics laboratory are not required before enrollment.  Centralized genomic testing 
results will allow participants to be assigned to a cohort: 
x Cohort A:  FGFR1-3 in-frame fusions or FGFR2 rearrangements* (n = 60) 
x Cohort B:  Known or likely activating mutations (excluding kinase domain) in 
FGFR1-3 included in Appendix C of Protocol Amendment 2 dated 14 JAN 2020  
(n = 90) 
x Cohort C:  Known activating mutations in the kinase domain of FGFR1-3, other 
likely activating FGFR1-3 mutations or FGFR1/FGFR3 rearrangements* (requires 
consultation with the sponsor) (n = 20) 
*Only FGFR fusions or rearrangements with an intact kinase domain are eligible. 
Please note that there is no difference in the treatment regimen between the cohorts.  The sponsor 
may choose to cap enrollment of any one tumor type and/or FGFR alteration to allow 
representation of multiple tumor types and avoid analysis being influenced by any one tumor 
type.  The sponsor may also choose to cap enrollment of any one tumor type if no benefit is 
being seen in previous participants enrolled. 
For participants with FGFR2-rearranged cholangiocarcinoma, enrollment is limited to no more 
than 10 participants into Cohort A; for participants with FGFR3 mutation or fusions bladder 
cancer, enrollment is limited to no more than 10 participants into Cohort A or B combined.  
Participants from either group noted must have mandatory baseline and at least 1 on-treatment 
biopsy. 
 
 
 
A fresh biopsy at baseline (or archival tissue that was collected less than 12 months from date of 
screening) and at least 1 on-treatment biopsy is required for participants with safely accessible 
lesions.  Treatment will start on Day 1.  Participants will undergo regular safety assessments 
during treatment as well as regular efficacy assessments.  Participants will be allowed to 
continue administration in 21-day cycles until documented disease progression or unacceptable 
toxicity is reported. 
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Figure 1: 
Study Design Schema 
Advanced/metastatic/non-resectable solid tumor malignancy
 FGF/FGFR status
Cohort A:  FGFR1-3 in-frame 
fusions or FGFR2 
rearrangements*
Treatment (21-day cycle; 
continuous dosing)
Disease Assessment 
every 3 cycles
PD
SD, PR, CR
D/C tx; safety 
and survival 
follow up
Continue tx; 
assess per 
protocol
Cohort C:  Known activation mutations in the 
kinase domain of  FGFR1-FGFR3, other likely 
activating FGFR1-3 mutations or FGFR1/FGFR3 
rearrangements*
Screen
Discontinue
Pass
SF
Cohort B:  known or likely activating mutations 
(excluding kinase domain) in FGFR1-3 
(Appendix C)
*Only FGFR fusions or rearrangements with an intact kinase domain are eligible
 
CR = complete response; D/C = discontinue; FGF = fibroblast growth factor; FGFR = fibroblast growth factor 
receptor; PD = progressive disease; PR = partial response; SD = stable disease; SF = screen failure; tx = treatment. 
3.1. 
Randomization 
Not applicable. 
3.2. 
Control of Type I Error 
For the primary endpoint, the overall 1-sided Type I error is 0.025.  The primary endpoint in 
Cohort A and Cohort B will be tested sequentially at 1-sided 0.025.  If ORR in Cohort A is 
significant, then ORR in Cohort B will be tested. 


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3.3. 
Sample Size Considerations 
For Cohort A, approximately 60 participants are planned.  Assuming the true ORR is 35% for the 
intervention in this group, 60 participants are needed to ensure that there is at least 90% power to 
reject the null hypothesis of ORR ≤ 15%.  This calculation assumes 1-sided test at the overall 
0.025 level of significance. 
For Cohort B, approximately 90 participants are planned.  Assuming the true ORR is 30% for the 
intervention in this group, 90 participants are needed to endure that there is at least 90% power to 
reject the null hypothesis of ORR ≤ 15%.  This calculation assumes 1-sided test at the overall 
0.025 level of significance. 
For Cohort C, approximately 20 participants will be enrolled, which will provide at least 
80% chance of observing at least 4 responders in this cohort if the underlying ORR is 30%. 
3.4. 
Schedule of Assessments 
Refer to Protocol Amendment 2 dated 14 JAN 2020 for a full description of all study procedures 
and assessment schedules for this study. 
4. 
DATA HANDLING DEFINITIONS AND CONVENTIONS 
4.1. 
Scheduled Study Evaluations and Study Periods 
4.1.1. 
Day 1 
Day 1 is the date that the first dose of pemigatinib is administered to the participants. 
4.1.2. 
Study Day 
If a visit/reporting date is on or after Day 1, then the study day at the visit/reporting date will be 
calculated as 
Day # = (visit/reporting date – Day 1 date + 1). 
If the visit/reporting date is before Day 1, then the study day at the visit/reporting date will be 
calculated as 
Day # = (visit/reporting date – Day 1 date). 
A study day of –1 indicates 1 day before Day 1. 


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4.1.3. 
Baseline Value 
Baseline is the last nonmissing measurement obtained before the first administration of 
pemigatinib.  When scheduled assessments and unscheduled assessments occur on the same day 
and time of the assessment or time of first dose is not available, use the following convention to 
determine baseline: 
• If both a scheduled and an unscheduled visit are available on the day of the first dose 
and the time is missing, use the scheduled assessment as baseline. 
• If all scheduled assessments are missing on the day of the first dose and an 
unscheduled assessment is available, use the unscheduled assessment as baseline. 
4.1.4. 
Handling of Missing and Incomplete Data 
In general, values for missing data will not be handled unless methods for handling missing dates 
are specified in this section or relevant sections.  The original reported dates collected on the 
eCRF should be used in all relevant listings.  The following rules will be used for handling 
partial dates for analyses requiring dates. 
When calculating time since diagnosis of cancer, partial cancer diagnosis date will be handled as 
follows in the calculation: 
• If only the day is missing, then use the first day of the month. 
• If both the month and day are missing, then use 01 JAN of the year. 
• Time since diagnosis will be missing if the diagnosis date is completely missing. 
When date of last dose is used in deriving variables such as duration of treatment or TEAE flag, 
missing or partial date of last dose will be handled as follows: 
• If only the day is missing, then use the earlier date of the last day of the month or the 
date that the participant discontinued treatment.  
• Otherwise, the date that the participant discontinued treatment will be used as the date 
of the last dose. 
For relevant efficacy endpoints, partial date of death will be handled as follows in the 
calculation: 
• If mmyyyy for the last known alive date = mmyyyy for the death date, then the death 
date will be set to the day after the last known alive date. 
• If mmyyyy for the last known alive date < mmyyyy for the death date, then the death 
date will be set to the first day of the death month. 
• Otherwise, the partial death date will not be handled. 
4.1.5. 
Cycle Length and Duration 
Cycle 1 Day 1 is the day that the first dose of pemigatinib is administered.  Scheduled cycle 
length is 21 days.  Actual Day 1 of subsequent cycles will correspond with the first day of 
administration of pemigatinib in that cycle. 


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4.1.6. 
Analysis Window 
For parameters that will be summarized by visit, the nominal visit as recorded on the eCRF will 
be used.  There will be no additional analysis windowing done based on the assessment date. 
4.2. 
Variable Definitions 
4.2.1. 
Prior and Concomitant Medication 
Prior medication is defined as any nonstudy medication started before the first dose of 
pemigatinib. 
Concomitant medication is defined as any nonstudy medication that is started accordingly:  
• Before the date of first administration of pemigatinib and is ongoing throughout the 
study or ends on/after the date of first study drug administration. 
• On/after the date of first administration of pemigatinib and is ongoing or ends during 
the course of study drug administration. 
A prior medication could also be classified as "both prior and concomitant medication" if the end 
date is on or after first dose of pemigatinib.  In the listing, it will be indicated whether a 
medication is prior-only, concomitant-only, or both prior and concomitant. 
For the purposes of analysis, all medications will be considered concomitant medications unless 
the medications can unequivocally be defined as not concomitant. 
5. 
STATISTICAL METHODOLOGY 
5.1. 
General Methodology 
Unless otherwise noted, SAS® software (SAS Institute Inc, Cary, NC; v9 or later) will be used 
for the generation of all tables, graphs, and statistical analyses.  Descriptive summaries for 
continuous variables will include, but not be limited to, the number of observations, mean, 
standard deviation, median, minimum, and maximum.  Descriptive summaries for categorical 
variables will include the number and percentage of participants in each category. 
Interim analyses are planned for this study as defined in Section 9. 
5.2. 
Treatment Groups 
This is an open-label, single-treatment group study.  Participants will be summarized by 
treatment cohort and by total.  Cohort determination will be based on FGFR mutation or gene 
rearrangement results from central genomics laboratory. 


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5.3. 
Analysis Populations 
5.3.1. 
Efficacy Evaluable Population 
The efficacy evaluable population includes all enrolled participants in Cohorts A, B, and C who 
received at least 1 dose of pemigatinib.  The efficacy evaluable population will be used for 
analyses of all efficacy data. 
5.3.2. 
Safety Population 
The safety population includes all enrolled participants who received at least 1 dose of 
pemigatinib.  The safety population will be used for the summary of demographics, baseline 
characteristics, participant disposition and all safety analyses. 
5.3.3. 
Per Protocol Population 
The PP population includes all efficacy evaluable population participants who are sufficiently 
compliant with the Protocol. 
The following procedures will be performed to identify those participants who are to be excluded 
from the PP population : 
• Clinical review of Protocol deviations 
• Clinical review of concomitant medications as defined in Section 6.6 of the Protocol 
• Clinical review of the dose administration and drug accountability listing 
The determination of participants being considered for exclusion from the PP population by the 
clinical team will be prepared and signed before database freeze. 
The PP population will be used in the supportive sensitivity analyses for efficacy endpoints. 
5.3.4. 
RECIST Evaluable Population 
The RECIST evaluable population includes all efficacy evaluable population participants who 
have at least 2 objective tumor response assessments per independent centralized radiological 
review or who discontinued from the study. 
RECIST evaluable population will be used for ORR analysis in the interim. 


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6. 
BASELINE, EXPOSURE, AND DISPOSITION 
Appendix A provides a list of data displays.  Sample data displays are included in a separate 
document. 
6.1. 
Baseline and Demographics, Physical Characteristics, and Disease 
History 
6.1.1. 
Demographics and Baseline Characteristics 
The following demographics characteristics will be summarized for the safety population:  age, 
sex, race, ethnicity, weight, and height. 
6.1.2. 
Baseline Disease Characteristics 
The following baseline disease characteristics will be summarized for the safety population:  
ECOG performance status, baseline parathyroid hormone, and baseline phosphate. 
6.1.3. 
Disease History 
For those who have solid tumor cancer history in the safety population,  time since diagnosis, 
solid tumor cancer type, TNM classification at diagnosis and baseline, current stage, current sites 
of disease, and tumor marker test result will be summarized. 
For those who have glioblastoma cancer history in the safety population, time since diagnosis, 
basis of initial diagnosis, time since reoccurrence, initial and current tumor WHO grade, 
infratentorial, supratentorial and CSF involvement at diagnosis and baseline, cerebral hemisphere 
involvement at diagnosis and baseline, multifocal disease at baseline and current sites of disease, 
and MGMT status will be summarized. 
Time since diagnosis will be calculated as follows:  
Time since diagnosis (years) = (Day 1 date – date of diagnosis + 1) / 365.25. 
Time since reoccurrence will be calculated as follows: 
Time since reoccurrence (months) = (Day 1 date – date of reoccurrence + 1) / 30.44. 
6.1.4. 
Prior Therapy 
The number of prior systemic cancer therapy regimens will be summarized for all participants in 
the safety population.  The component drugs of prior systemic therapy regimens will be coded 
using the WHO Drug Dictionary.  Number and percentage of participants with each drug will be 
summarized by WHO drug class and WHO drug PT.  Regimen name, component drugs, start and 
stop date, purpose of the regimen, best response, reason for discontinuation, and date of 
relapse/progression will be listed. 
The number of participants who received prior radiation will be summarized for the safety 
population.  Radiotherapy type, body site, reason for regimen, start and stop date, total dose, and 
best response will be listed. 


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The number of participants who received prior radiotherapy for glioblastoma will be summarized 
for the safety population.  Body type, start and stop date, total dose, radiation therapy technique, 
radiation method, and best response will be listed. 
The number of participants who had prior surgery or surgical procedure for disease under study 
and for glioblastoma will be summarized for the safety population.  Date and description of the 
surgery/procedure will be listed for disease under study and glioblastoma, extent of resection for 
glioblastoma will also be listed. 
The number of participants who had prior loco-regional therapy will be summarized for the 
safety population.  Therapy/treatment name, start and stop date, best response, reason for 
discontinuation and date of relapse/progression will be listed. 
6.1.5. 
Medical History 
For participants in the safety population, medical history will be summarized by assigned cohort.  
This summation will include the number and percentage of participants with significant medical 
history for each body system/organ class as documented on the eCRF. 
6.2. 
Disposition of Participant 
The number and percentage of participants who were treated, who were ongoing with study 
treatment, who discontinued study treatment with a primary reason for discontinuation, who 
were still in study, and who withdrew from the study with a primary reason for withdrawal will 
be summarized for the safety population. 
The number and percentage of participants who were screened and enrolled will be summarized. 
6.3. 
Protocol Deviations 
Protocol deviations recorded on the eCRF will be summarized and listed. 
6.4. 
Exposure 
For participants in the safety population, exposure to pemigatinib will be summarized 
descriptively as the following: 
• Number of treatment cycles:  Number of cycles with a nonzero dose of pemigatinib. 
• Duration of treatment with pemigatinib (days): 
Date of last dose of pemigatinib − date of first dose of pemigatinib + 1. 
• Average daily dose of pemigatinib (mg/day): 
Total actual pemigatinib dose taken (mg) / duration of treatment with pemigatinib 
(days). 
• Pemigatinib dose modifications:  Number of participants who had pemigatinib dose 
reduction, and interruption will be summarized. 


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6.5. 
Study Drug Compliance 
For participants in the safety population, overall compliance (%) for pemigatinib will be 
calculated for all participants as 
Compliance (%) = 100 × [total dose actually taken] / [total prescribed dose]. 
The total prescribed dose is defined as the sum of the doses prescribed by the investigator 
accounting for dose modifications. 
The total actual dose taken will be calculated based on information entered on the Drug 
Accountability eCRF.  If there are dispensed drugs that have not been returned yet, the actual 
dose taken starting from the dispense date of the unreturned drugs will be imputed by the dose 
taken as reported on the Dosing eCRF. 
6.6. 
Prior and Concomitant Medication 
Prior medications and concomitant medications will be coded using the WHO Drug Dictionary.  
Number and percentage of participants in the safety population for each prior and concomitant 
medications will be summarized by WHO drug class and WHO drug PT. 
7. 
EFFICACY 
Appendix A provides a list of data displays.  Sample data displays are included in a separate 
document. 
7.1. 
Efficacy Hypotheses 
Objective Response Rate (Primary Endpoint):  Administration of pemigatinib to participants 
in Cohort A and B improves ORR from 15%.  Assume 𝑂𝑂𝑂𝑂𝑅𝑅𝐴𝐴 and 𝑂𝑂𝑂𝑂𝑅𝑅𝐵𝐵 are the ORRs for cohort 
A and B respectively. 
The hypotheses for Cohort A are as follows: 
• H0 (null hypothesis):  𝑂𝑂𝑂𝑂𝑅𝑅𝐴𝐴≤15%  
• HA (alternative hypothesis):  𝑂𝑂𝑂𝑂𝑅𝑅𝐴𝐴> 15% 
The hypotheses for cohort B are as follows: 
• H0 (null hypothesis):  𝑂𝑂𝑂𝑂𝑅𝑅𝐵𝐵≤15%  
• HA (alternative hypothesis):  𝑂𝑂𝑂𝑂𝑅𝑅𝐵𝐵> 15% 
7.2. 
Analysis of the Primary Efficacy Parameter 
7.2.1. 
Primary Efficacy Analysis 
The primary endpoints of the study are ORR in Cohort A and Cohort B, respectively.  Objective 
response rate is defined as the proportion of participants who achieved a CR or a PR based on 
RECIST v1.1 or RANO, as assessed by an independent centralized radiological review 
committee.  Confirmation of CR and PR is required and documented in the Independent Central 
Review Charter.  This analysis will be based on the efficacy evaluable population.  Participants 


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who do not have sufficient baseline or on-study response assessment information to be 
adequately assessed for response status will be included in the denominators in the calculation of 
ORR.  The 95% CI for the ORR will be estimated using the Clopper-Pearson method.  The 
p-value from the exact binomial test will be provided. 
The ORR will also be analyzed based on the PP population as a sensitivity analysis. 
7.2.1.1. 
Response Criteria 
Overall disease status will be categorized using RECIST v1.1 (Eisenhauer et al 2009) and 
RANO.  Participants will have their overall response evaluated as CR, PR, SD, PD, or NE at 
each postbaseline radiological assessment.  Assessments using RECIST v1.1 are based on 
changes in target lesions, nontarget lesions, and appearance of new lesions.  Assessments using 
RANO are based on enhancing measurable lesions, enhancing nonmeasurable/nontarget lesions, 
nonenhancing T2/FLAIR/nontarget lesions, new lesions, steroid use, and clinical status. 
7.2.1.2. 
Objective Response Rates and Best Overall Response 
A participant is considered an objective responder if they have a best overall response of CR or 
PR at any postbaseline visit before first PD. 
In general, under RECIST v1.1 and RANO, best overall response is the best response recorded 
postbaseline before and including the first PD, in the order of CR, PR, SD, PD, and NE.  The 
best overall response will be determined from response assessments before or on the same day as 
new anticancer therapy.  If any alternative cancer therapy is taken while on study, any 
subsequent assessments will be excluded from the best overall response determination. 
In the case of SD, measurements must meet the SD criteria at least once after the date of first 
dose at a minimum interval of 42 days.  Participants who fail to meet this criterion will have best 
overall response of PD if the next available assessment indicates PD or NE if there is no 
additional assessment available. 
7.2.2. 
Subgroup Analyses for Primary Endpoint 
Subgroup analysis of the primary endpoint will be performed based on appropriate intrinsic and 
extrinsic factors if needed. 
7.2.3. 
Sensitivity and Supportive Analyses for Primary Endpoint 
The ORR will be analyzed based on the PP population as a sensitivity analysis. 
7.3. 
Analysis of the Secondary Efficacy Parameters 
7.3.1. 
Progression-Free Survival 
Progression-free survival is defined as the time from date of first dose of study drug to the 
earliest date of PD or death, whichever is first.  Progressive disease is evaluated based on 
RECIST v1.1 or RANO, by the ICR.  Partial death dates will be handled using the rules 
described in Section 4.1.4.  Participants who are alive without progression before analysis cutoff 
date will be censored.  Censoring for PFS will follow the algorithm outlined in Table 2, which is 


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based on the FDA Guidance for Industry:  Clinical Trial Endpoints for the Approval of Cancer 
Drugs and Biologics (FDA 2015, FDA 2018). 
Table 2: 
Evaluation and Censoring of Progression-Free Survival 
Situation 
Outcome 
Date of Progression or Censoring 
No baseline tumor assessments 
Censored 
Day 1 
No valid postbaseline response 
assessments 
Censored 
Day 1  
Progression documented between 
scheduled response assessments 
Progressed 
Date of first overall response of PD 
No progression 
Censored 
Date of last valid radiologic assessment (not 
NE and not missing) 
Study discontinuation for 
undocumented progression 
Censored 
Date of last valid radiologic assessment (not 
NE and not missing) 
Study discontinuation for toxicity 
or other reason 
Censored 
Date of last valid radiologic assessment (not 
NE and not missing) 
Table 2: 
Evaluation and Censoring of Progression-Free Survival (Continued) 
Situation 
Outcome 
Date of Progression or Censoring 
New anticancer treatment started 
Censored 
Date of last valid radiologic assessment (not 
NE and not missing) on/before starting a new 
anticancer treatment 
Death before first progressive 
response assessment 
Progressed 
Date of death 
Death between adequate response 
assessments 
Progressed 
Date of death 
Death or progression after 2 or 
more missed assessments 
Censored 
Date of last valid radiologic assessment (not 
NE and not missing) 
NE = not evaluable. 
Progression-free survival data will be analyzed by the Kaplan-Meier method in Cohorts A and 
Cohort B, respectively.  The number of participants who progressed or died and the number of 
participants censored will be summarized.  The KM estimate of median PFS will be presented 
with its 95% CI.  The 95% CI will be calculated using the generalization of Brookmeyer and 
Crowley’s method (1982) with log-log transformation (Klein and Moeschberger 1997). 
7.3.2. 
Duration of Response 
For objective responders, the duration of response is defined as the time from the date that a 
participant first achieves CR or PR based on RECIST v1.1 or RANO as assessed by ICR, until 
the date of first documented disease progression based on RECIST v1.1 or RANO as assessed by 
ICR, or death.  Partial death dates will be handled using the rules described in Section 4.1.4.  
Participants who are alive without progression before analysis cutoff date will be censored.  
Censoring of DOR will follow the same algorithm as the censoring of PFS (see Section 7.3.1).   


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Duration of response data will be analyzed by the Kaplan-Meier method in Cohorts A and B, 
respectively.  The total number of responders, the number of participants who progressed or 
died, and the number of participants censored will be summarized.  The KM estimate of median 
DOR will be presented with its 95% CI.  The 95% CI will be calculated using the generalization 
of Brookmeyer and Crowley’s method (1982) with log-log transformation (Klein and 
Moeschberger 1997). 
7.3.3. 
Overall Survival 
Overall survival is defined as the time from date of first dose of study drug to the date of death 
due to any cause.  Date of death will be determined using the Death Report, the Survival 
Follow-Up, and the Participant Status eCRFs.  Participants who are lost to follow-up or still alive 
at the time of analysis will be right-censored at the earlier of the date the participant was last 
known alive and the clinical data cutoff date for the analysis.  The last known alive date is 
defined as the later of the last study visit and the date the participant was last known alive from 
the Survival Follow-Up and Participant Status eCRFs.  Partial death dates will be handled using 
the rules described in Section 4.1.4. 
Overall survival will be analyzed by the Kaplan-Meier method in Cohorts A and B, respectively.  
The number of participants who died and the number of participants censored will be 
summarized.  Median survival will be estimated using the Kaplan-Meier method.  The 95% CIs 
for median survival time will be calculated using the generalization of Brookmeyer and 
Crowley’s method (1982) with log-log transformation (Klein and Moeschberger 1997). 
7.4. 
Other Efficacy Analyses 
7.4.1. 
Largest Percentage Reduction in Sum of Diameters of Target Lesions 
For each participant in the efficacy evaluable population with target lesions at baseline, target 
lesion sizes will be measured by sum of diameters.  The best percentage change from baseline, 
defined as the largest decrease in target lesion size for each participant, will be summarized 
descriptively, and a waterfall plot of the best percentage change will be generated. 
Per RECIST criteria, target lesions considered “too small to measure” will be assigned a default 
value of 5 mm for purposes of this analysis.  Likewise, target lesions identified as “not present” 
at postbaseline assessments will be assigned 0 mm for this analysis.  In the event a target lesion 
is unaccounted for in a particular postbaseline timepoint (ie, assessment missing or not 
evaluable), then the overall sum of diameters for target lesions will not be evaluable for that 
postbaseline timepoint. 


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8. 
SAFETY AND TOLERABILITY 
Appendix A provides a list of data displays.  Sample data displays are included in a separate 
document. 
8.1. 
General Considerations 
The analyses for this section will be provided for the safety population.  Summary tables may be 
replaced with listings when appropriate.  For instance, an AE frequency table may be replaced 
with a listing if it only contains a few unique PTs reported on relatively few participants. 
8.2. 
Adverse Events 
8.2.1. 
Adverse Event Definitions 
A TEAE is any AE either reported for the first time or worsening of a pre-existing event after 
first dose of study drug until 30 days after the last dose of study drug.  Analysis of AEs (as 
discussed below) will be limited to TEAEs, but data listings will include all AEs regardless of 
their timing in relation to study drug administration.  For purposes of analysis, all AEs will be 
considered TEAEs unless the AE can unequivocally be defined as not treatment emergent.   
Adverse events will be tabulated by MedDRA PT and SOC.  Severity of AEs will be graded 
using the NCI CTCAE v5.0.  The CTCAE reporting guidelines and grading details are available 
on the CTEP website.  


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A grading (severity) scale is provided for each AE term.  If the toxicity is not included in the 
CTCAE v5.0 criteria, it will be graded on a scale of 1 to 5 as follows:  1 = mild, 2 = moderate, 
3 = severe, 4 = life-threatening, and 5 = fatal.  All toxicities will be graded based on the worst 
level reached, not the level they may have reached if they had not been treated.  When the 
intensity of an AE changes over time for a reporting period (eg, between visits), each change in 
intensity will be reported as an AE until the event resolves. 
The subset of AEs considered by the investigator to be related to study drug will be considered to 
be treatment-related AEs.  If the investigator does not specify the relationship of the AE to study 
drug, the AE will be considered to be treatment-related.  The incidence of AEs and 
treatment-related AEs will be tabulated.  In addition, SAEs will also be tabulated. 
Any missing onset date, causality, or severity must be queried for resolution.  Unresolved 
missing causality and severity will be handled according to the following rules: 
• An unresolved missing causality will be considered treatment-related. 
• An unresolved missing severity will be identified as an unknown severity. 
8.2.2. 
Clinically Notable Adverse Events 
Specific groupings of clinically notable AEs will be considered and the number of participants 
with at least 1 event within each grouping will be reported.  Such groups consist of AEs for 
which there is a specific clinical interest in connection with pemigatinib or AEs that are similar 
in nature (although not identical).  The groups are defined as per Table 4.  All clinically notable 
AEs are defined through reviewing PT according to the current MedDRA v21.1. 
Table 4: 
Clinically Notable Adverse Events Groupings 
Categories 
Preferred Terms 
Serous retinal detachment 
Serous retinal detachment, detachment of macular retinal pigment 
epithelium, detachment of retinal pigment epithelium, retinal 
detachment, subretinal fluid, chorioretinopathy, retinal pigment 
epitheliopathy,  chorioretinal disorder, retinopathy, maculopathy, retinal 
disorder, retinal thickening, chorioretinal folds, chorioretinal scar 
Nail toxicity 
Nail toxicity, nail bed tenderness, nail bed disorder, nail bed bleeding, 
nail disorder, nail discolouration, nail discomfort, nail dystrophy, nail 
hypertrophy, nail ridging, nail infection, onychalgia, onychoclasis, 
onycholysis, onychomadesis, onychomycosis, paronychia, fungal 
paronychia 
Hyperphosphatemia 
Hyperphosphataemia, blood phosphorus increased 
Hypophosphatemia 
Hypophosphataemia, blood phosphorus decreased 
Dry eye 
Dry eye, meibomian gland dysfunction, lacrimation increased, keratitis, 
punctate keratitis, pinguecula, pterygium 
eyelash changes 
Eyelash changes, growth of eyelashes, trichiasis, trichomegaly 
vision blurred 
vision blurred, visual impairment, visual acuity reduced 
vitreous detachment 
Vitreous detachment, vitreous floaters 


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8.2.3. 
Adverse Event Summaries 
An overall summary of AEs by cohorts will include: 
• Number (%) of participants reporting any TEAEs 
• Number (%) of participants reporting any serious TEAEs 
• Number (%) of participants reporting any Grade 3 or higher TEAEs 
• Number (%) of participants reporting any TEAEs related to pemigatinib 
• Number (%) of participants who temporarily interrupted pemigatinib because of 
TEAEs 
• Number (%) of participants who permanently discontinued pemigatinib because of 
TEAEs 
• Number (%) of participants with pemigatinib dose reductions because of TEAEs  
• Number (%) of participants who had a fatal TEAE 
The following summaries will be produced by MedDRA term: 
• Summary of TEAEs by MedDRA SOC and PT 
• Summary of TEAEs by MedDRA PT in decreasing order of frequency 
• Summary of TEAEs by MedDRA SOC, PT, and maximum severity 
• Summary of TEAEs by MedDRA SOC, PT, and CTCAE Grade Category 
• Summary of Grade 3 or higher TEAEs by MedDRA SOC and PT 
• Summary of Grade 3 or higher TEAEs by MedDRA PT in decreasing order of 
frequency 
• Summary of serious TEAEs by MedDRA SOC and PT 
• Summary of serious TEAEs by MedDRA PT in decreasing order of frequency 
• Summary of pemigatinib treatment-related AEs by MedDRA SOC and PT 
• Summary of pemigatinib treatment-related TEAEs by MedDRA PT in decreasing 
order of frequency 
• Summary of Grade 3 or higher pemigatinib treatment-related TEAEs by MedDRA 
SOC and PT 
• Summary of pemigatinib treatment-related serious TEAEs by MedDRA SOC and PT 
• Summary of TEAEs with a fatal outcome by MedDRA SOC and PT 
• Summary of TEAEs leading to pemigatinib dose reduction by MedDRA SOC and PT 
• Summary of TEAEs leading to pemigatinib dose interruption by MedDRA SOC and 
PT 
• Summary of TEAEs leading to discontinuation of pemigatinib by MedDRA SOC and 
PT 


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• Summary of sponsor-defined clinically notable TEAEs by category and PT 
• Summary of Grade 3 or higher sponsor-defined clinically notable TEAEs by category 
and PT 
• Summary of serious sponsor-defined clinically notable TEAEs by category and PT 
• Summary of sponsor-defined clinically notable TEAEs leading to pemigatinib dose 
reduction by category and PT 
• Summary of sponsor-defined clinically notable TEAEs leading to pemigatinib dose 
interruption by category and PT 
• Summary of sponsor-defined clinically notable TEAEs leading to discontinuation of 
pemigatinib by category and PT 
8.3. 
Clinical Laboratory Tests 
8.3.1. 
Laboratory Value Definitions 
Laboratory values and change from baseline values will be summarized descriptively by visit for 
numeric laboratory parameters.  Baseline will be determined according to Section 4.1.3.  If there 
are multiple values that meet the criteria for baseline, additional rules may be provided after 
consultation with the medical monitor to delineate which value will be defined as baseline. 
Laboratory test values outside the normal range will be assessed for severity based on the 
numerical component of CTCAE v5.0. 
8.3.2. 
Laboratory Value Summaries 
All test results and associated normal ranges from central laboratories will be reported in SI 
units.  All tests with numeric values will have a unique unit.  Any laboratory test results and 
associated normal ranges from local laboratories will be converted to SI units. 
For numeric laboratory values, baseline value, postbaseline value, change from baseline, and 
percentage change from baseline will be summarized descriptively by visit.  The number and 
percentage of participants with the laboratory values being low, normal, and high will be 
summarized by visit.  In addition, mean change from baseline will be plotted over time for 
selected laboratory parameters, including phosphate. 
Severity grades will be assigned to laboratory test values based on the numerical component of 
CTCAE v5.0.  Shift tables will be presented showing change in CTCAE grade from baseline to 
worst grade postbaseline.  Separate summaries for abnormally high and abnormally low 
laboratory values will be provided when the laboratory parameter has both high and low grading 
criteria.  The denominator for the percentage calculation will be the number of participants in the 
baseline category.  The number of participants who experienced worsening of laboratory 
abnormalities will be summarized by maximum severity.   


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8.3.3. 
Potential Hy’s Law Events 
The participants with elevated ALT or AST > 3 × upper limit of normal range and alkaline 
phosphatase < 2 × upper limit of normal range accompanied by total bilirubin > 2 × upper limit 
of normal range at the same visit will be listed by cohort. 
8.4. 
Vital Signs 
Values at each scheduled visit, change, and percentage change from baseline for vital signs, 
including weight, systolic blood pressure, diastolic blood pressure, pulse, temperature, and 
respiratory rate will be summarized descriptively.   
Normal ranges for vital sign values are defined in Table 5.  The abnormal values for participants 
exhibiting vital sign abnormalities will be listed along with their cohort.  Alert vital signs are 
defined as an absolute value outside the defined normal range and percentage change greater 
than 25%.  Note that the definition of alert vital signs does not apply for body temperature and 
weight.  The abnormal values for participants exhibiting alert vital sign abnormalities will be 
listed. 
Table 5: 
Criteria for Clinically Notable Vital Sign Abnormalities 
Parameter 
High Threshold 
Low Threshold 
Systolic blood pressure 
> 155 mmHg 
< 85 mmHg 
Diastolic blood pressure 
> 100 mmHg 
< 40 mmHg 
Pulse 
> 100 bpm 
< 45 bpm 
Temperature 
> 38°C 
< 35.5°C 
Respiratory rate 
> 24 breaths/min 
< 8 breaths/min 
8.5. 
Electrocardiograms 
Twelve-lead ECGs including the heart rates and PR, QT, QRS, and QTc intervals will be 
obtained for each participant during the study.  Values at each scheduled visit, change, and 
percentage change from baseline will be summarized for each ECG parameter.  Baseline will be 
determined as the average of all nonmissing values before the first administration of pemigatinib. 
Normal ranges for ECG values are defined in Table 6.  ECG values will also be considered 
abnormal if the absolute percentage change from baseline is more than 25% (QRS 30%).  
Participant exhibiting ECG abnormalities will be listed with study visit and assigned cohort.  
Abnormal values for participant with alert ECG values, defined as both the absolute value and 
the percentage change from baseline being outside normal ranges, will be identified and listed.  
Outliers of QT, and QTc values, defined as absolute values > 450 ms, > 500 ms, or change from 
baseline > 30 ms, will be summarized. 


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Table 6: 
Criteria for Clinically Notable Electrocardiogram Abnormalities 
Parameter  
High Threshold  
Low Threshold  
HR 
> 100 bpm 
< 45 bpm 
PR  
> 220 ms 
< 75 ms 
QT  
> 500 ms 
< 300 ms 
QRS  
> 120 ms 
< 50 ms 
QTc  
> 460 ms 
< 295 ms 
9. 
INTERIM ANALYSIS 
For Cohort A, futility analysis will be performed when approximately 25 participants are 
enrolled into the cohort and have at least 2 objective tumor response assessments per 
independent centralized radiological review or are discontinued from the study.  Cohort A can be 
stopped for futility if ≤ 4 responders are observed, for which there is < 10% probability of 
claiming ORR > 15% at final analysis.  This rule is just a guidance and nonbinding. 
For Cohort B, futility analysis will be performed when approximately 35 participants are 
enrolled into the cohort and have at least 2 objective tumor response assessments per 
independent centralized radiological review or are discontinued from the study.  Cohort B can be 
stopped for futility if ≤ 6 responders are observed, for which there is approximately 
14% probability of claiming ORR > 15% at final analysis.  This rule is just a guidance and 
nonbinding. 
10. 
CHANGES AND MODIFICATIONS TO THE ANALYSIS PLAN 
All versions of the SAP are listed in Table 7. 
Table 7: 
Statistical Analysis Plan Versions 
SAP Version 
Date 
Original 
23 SEP 2020 
10.1. 
Changes to Protocol-Defined Analyses 
Not applicable. 
10.2. 
Changes to the Statistical Analysis Plan 
Not applicable. 


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11. 
REFERENCES 
Brookmeyer R, Crowley J. A confidence interval for the median survival time. Biometrics 
1982;38:29-41. 
Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumors: 
revised RECIST guideline (version 1.1). Eur J Cancer 2009;45:228-247. 
Food and Drug Administration. Guidance for Industry: Clinical Trial Endpoints for the Approval 
of Cancer Drugs and Biologics. 2018. 
Food and Drug Administration. Guidance for Industry: Clinical Trial Endpoints for the Approval 
of Non-Small Cell Lung Cancer Drugs and Biologics. 2015. 
Klein JP, Moeschberger ML. Survival analysis: techniques for censored and truncated data. New 
York: Springer-Verlag. 1997. 


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APPENDIX A. PLANNED TABLES, FIGURES, AND LISTINGS 
This appendix provides a list of the planned tables, figures and listings for the clinical study 
report.  Shells are provided in a separate document for tables that are not in the Standard Safety 
Tables. 
The lists of tables, figures, and listings are to be used as guidelines.  Modifications of the lists 
that do not otherwise affect the nature of the analysis will not warrant an amendment to the SAP. 
Tables  
Table No. 
Title 
Population 
Baseline and Demographic Characteristics 
1.1  Disposition 
1.1.1 
Analysis Populations 
All Enrolled 
1.1.2 
Summary of Participant Disposition 
Safety 
1.1.3 
Summary of Number of Participants Enrolled by Country and Site 
Safety 
1.1.4 
Summary of Protocol Deviations 
Safety 
1.2  Demography and Baseline Characteristics 
1.2.1 
Summary of Demographics and Baseline Characteristics 
Safety 
1.3  Baseline Disease Characteristics 
1.3.1.1 
Summary of Baseline Disease Characteristics 
Safety 
1.3.1.2 
Summary of Baseline Disease Characteristics – Glioblastoma 
Safety 
1.3.2 
Summary of Prior Systematic Cancer Therapy by WHO Drug Class and Preferred 
Term 
Safety 
1.3.3 
Summary of Prior Cancer Therapy 
Safety 
1.4  Prior Medication and Concomitant Medication 
1.4.1 
Summary of Prior Medications 
Safety 
1.4.2 
Summary of Concomitant Medications 
Safety 
1.5 General Medical History 
1.5.1 
Summary of General Medical History 
Safety 
Efficacy 
2.1  Primary Efficacy 
2.1.1 
Summary of Best Response and Objective Response Rate – Cohort A and B  
Efficacy 
2.1.2 
Summary of Best Response and Objective Response Rate – Cohort A and B 
Per Protocol 
2.2  Secondary Efficacy 
2.2.1 
Summary of Progression-Free Survival – Cohort A and B 
Efficacy 
2.2.2 
Summary of Duration of Response – Cohort A and B 
Efficacy 
2.2.3 
Summary of Overall Survival – Cohort A and B 
Efficacy 
2.2.4 
Summary of Best Change in Target Lesion Size 
Efficacy 


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Table No. 
Title 
Population 
Safety 
3.1  Dose Exposure 
3.1.1 
Summary of Exposure and Duration of Exposure to Pemigatinib 
Safety 
3.1.3 
Summary of Study Drug Compliance 
Safety 
3.2  Adverse Events 
3.2.1 
Overall Summary of Treatment-Emergent Adverse Events 
Safety 
3.2.2 
Summary of Treatment-Emergent Adverse Events by MedDRA System Organ 
Class and Preferred Term 
Safety 
3.2.3 
Summary of Treatment-Emergent Adverse Events by MedDRA Preferred Term in 
Decreasing Order of Frequency 
Safety 
3.2.4 
Summary of Treatment-Emergent Adverse Events by MedDRA System Organ 
Class, Preferred Term, and Maximum Severity 
Safety 
3.2.5 
Summary of Treatment-Emergent Adverse Events by MedDRA System Organ 
Class, Preferred Term, and CTCAE Grade Category 
Safety 
3.2.6 
Summary of Grade 3 or Higher Treatment-Emergent Adverse Events by MedDRA 
System Organ Class and Preferred Term 
Safety 
3.2.7 
Summary of Grade 3 or Higher Treatment-Emergent Adverse Events by MedDRA 
Preferred Term in Decreasing Order of Frequency 
Safety 
3.2.8 
Summary of Serious Treatment-Emergent Adverse Events by MedDRA System 
Organ Class and Preferred Term 
Safety 
3.2.9 
Summary of Serious Treatment-Emergent Adverse Events by MedDRA Preferred 
Term in Decreasing Order of Frequency 
Safety 
3.2.10 
Summary of Pemigatinib Treatment-Related Treatment-Emergent Adverse Events 
by MedDRA System Organ Class and Preferred Term 
Safety 
3.2.11 
Summary of Pemigatinib Treatment-Related Treatment-Emergent Adverse Events 
by MedDRA Preferred Term in Decreasing Order of Frequency 
Safety 
3.2.12 
Summary of Grade 3 or Higher Pemigatinib Treatment-Related 
Treatment-Emergent Adverse Events by MedDRA System Organ Class and 
Preferred Term 
Safety 
3.2.13 
Summary of Pemigatinib Treatment-Related Serious Treatment-Emergent Adverse 
Events by MedDRA System Organ Class and Preferred Term 
Safety 
3.2.14 
Summary of Treatment-Emergent Adverse Events With a Fatal Outcome by 
MedDRA System Organ Class and Preferred Term 
Safety 
3.2.15 
Summary of Treatment-Emergent Adverse Events Leading to Pemigatinib Dose 
Reduction by MedDRA System Organ Class and Preferred Term 
Safety 
3.2.16 
Summary of Treatment-Emergent Adverse Events Leading to Pemigatinib Dose 
Interruption by MedDRA System Organ Class and Preferred Term 
Safety 
3.2.17 
Summary of Treatment-Emergent Adverse Events Leading to Discontinuation of 
Pemigatinib by MedDRA System Organ Class and Preferred Term 
Safety 
3.2.18 
Summary of Sponsor-Defined Clinically Notable TEAEs by Category and 
Preferred Term 
Safety 
3.2.19 
Summary of Grade 3 or Higher Sponsor-Defined Clinically Notable TEAEs by 
Category and Preferred Term 
Safety 
3.2.20 
Summary of Serious Sponsor-Defined Clinically Notable TEAEs by Category and 
Preferred Term 
Safety 
3.2.21 
Summary of Sponsor-Defined Clinically Notable TEAEs Leading to Pemigatinib 
Dose Reduction by Category and Preferred Term 
Safety 


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Table No. 
Title 
Population 
3.2.22 
Summary of Sponsor-Defined Clinically Notable TEAEs Leading to Pemigatinib 
Dose Interruption by Category and Preferred Term 
Safety 
3.2.23 
Summary of Sponsor-Defined Clinically Notable TEAEs Leading to 
Discontinuation of Pemigatinib by Category and Preferred Term 
Safety 
3.3  Laboratory 
3.3.1.1 
Summary of Laboratory Values – Hematology 
Safety 
3.3.1.2 
Summary of Laboratory Values – Chemistry 
Safety 
3.3.1.3 
Summary of Laboratory Values – Coagulation 
Safety 
3.3.1.4 
Summary of Laboratory Values – Urinalysis 
Safety 
3.3.3.1 
Shift Summary of Hematology Laboratory Values in CTC Grade – to the Worst 
Abnormal Value  
Safety 
3.3.3.2 
Shift Summary of Chemistry Laboratory Values in CTC Grade – to the Worst 
Abnormal Value 
Safety 
3.3.3.3 
Shift Summary of Coagulation Laboratory Values in CTC Grade – to the Worst 
Abnormal Value 
Safety 
3.3.3.4 
Treatment-Emergent Worsening of Laboratory Abnormalities – Hematology 
Safety 
3.3.3.5 
Treatment-Emergent Worsening of Laboratory Abnormalities – Chemistry 
Safety 
3.3.3.6 
Treatment-Emergent Worsening of Laboratory Abnormalities – Coagulation 
Safety 
3.4  Vital Signs 
3.4.1 
Summary of Systolic Blood Pressure 
Safety 
3.4.2 
Summary of Diastolic Blood Pressure  
Safety 
3.4.3 
Summary of Pulse  
Safety 
3.4.4 
Summary of Respiration Rate 
Safety 
3.4.5 
Summary of Body Temperature 
Safety 
3.4.6 
Summary of Weight 
Safety 
3.5  ECG 
3.5.1 
Summary of PR Interval (ms) From 12-Lead ECG 
Safety 
3.5.2 
Summary of QRS Interval (ms) From 12-Lead ECG 
Safety 
3.5.3 
Summary of QT Interval (ms) From 12-Lead ECG 
Safety 
3.5.4 
Summary of QTc Interval (ms) From 12-Lead ECG 
Safety 
3.5.5 
Summary of Heart Rate (bpm) From 12-Lead ECG 
Safety 
3.5.6 
Summary of Outliers of QT, QTcB, and QTcF Interval Values (ms) From 12-Lead 
ECG 
Safety 
3.5.7 
Summary of Clinically Significant ECG Abnormality 
Safety 
Figures 
Figure No. 
Title 
4.1.1 
Kaplan-Meier Estimates of Progression -Free Survival  
4.2.1 
Kaplan-Meier Estimates of Duration of Response 
4.2.2 
Kaplan-Meier Estimates of Overall Response  
4.3.1 
Waterfall Plot of Best Percentage Change in Sum of Target Lesions 
4.3.2 
Swimmer Plot of Duration of Treatment  
4.6.1 
Line Graph of Selected Laboratory Values by Study Visit 


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Listings 
Listing No. 
Title 
2.1  Discontinued Participants (Participant Disposition) 
2.1.1 
Participant Enrollment and Disposition Status 
2.1.2 
Participant Inclusion and Exclusion Criteria Violations 
2.1.4 
Survival Follow-up 
2.2  Protocol Deviation 
2.2.1 
Protocol Deviations  
2.3  Data Excluded From Efficacy, and/or Safety Analyses 
2.3.1 
Analysis Population 
2.4  Demographic and Baseline Characteristics (Including Prior and Concomitant Medications) 
2.4.1 
Demographic and Baseline Characteristics 
2.4.2.1 
Disease History 
2.4.2.2 
Disease History – Glioblastoma  
2.4.3.1 
Prior Radiation Treatment – Solid Tumor 
2.4.3.2 
Prior Radiation Treatment – Glioblastoma  
2.4.4 
Prior Systemic Therapy 
2.4.5.1 
Prior Surgery or Surgical Procedure – Solid Tumor 
2.4.5.2 
Prior Surgery or Surgical Procedure – Glioblastoma  
2.4.6 
Prior Loco-Regional Therapy 
2.4.7 
Medical History 
2.4.8 
Prior and Concomitant Medication 
2.4.9 
Procedures and Non-Drug Therapy 
2.4.10 
Post Therapy 
2.5  Drug Compliance 
2.5.1 
Study Drug Compliance 
2.5.2 
Study Drug Administration 
2.6  Efficacy  
2.6.1 
Deaths 
2.6.2 
Best Overall Response, Duration of Response, and Progression-Free Survival 
2.6.3.1 
Overall Response Assessment by Visit – RECIST 
2.6.3.2 
Overall Response Assessment by Visit – RANO 
2.6.4.1 
Response Assessment:  Target Lesions – RECIST 
2.6.4.2 
Response Assessment:  Measurable Lesions – RANO 
2.6.5.1 
Response Assessment:  Nontarget Lesions – RECIST 
2.6.5.2 
Response Assessment:  Nonmeasurable Lesions – RANO 
2.6.6.1 
Response Assessment:  New Lesions – RECIST 
2.6.6.2 
Response Assessment:  New Lesions – RANO 
2.6.7 
Tumor Tissue 
2.6.8 
ECOG Status 
2.7  Adverse Events 
2.7.1 
Adverse Events  


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Listing No. 
Title 
2.7.2 
Serious Adverse Events 
2.7.3 
Grade 3 and Higher Adverse Events 
2.7.4 
Fatal Adverse Events 
2.7.5 
Treatment-Related Adverse Events 
2.7.6 
Adverse Events Leading to Interruption, Reduction, or Discontinuation of Pemigatinib 
2.7.7 
Sponsor-Defined Clinically Notable Adverse Events 
2.8  Laboratory Data 
2.8.1 
Clinical Laboratory Values – Hematology 
2.8.2 
Clinical Laboratory Values – Chemistry 
2.8.3 
Clinical Laboratory Values – Coagulation 
2.8.4 
Clinical Laboratory Values – Urinalysis 
2.8.5 
Abnormal Clinical Laboratory Values – Hematology 
2.8.6 
Abnormal Clinical Laboratory Values – Chemistry 
2.8.7 
Abnormal Clinical Laboratory Values – Coagulation 
2.8.8 
Potential Hy’s Law Events 
2.8.9 
Serology Lab Test 
2.8.10 
Pregnancy Test 
2.9  Vital Signs 
2.9.1 
Vital Signs 
2.9.2 
Abnormal Vital Sign Values 
2.9.3 
Alert Vital Sign Values 
2.10  ECG 
2.10.1 
12-Lead ECG Values 
2.10.2 
Abnormal 12-Lead ECG Values 
2.10.3 
Alert 12-Lead ECG Values 
2.11  Physical Examinations 
2.11.1 
Eye Examination