Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients
Phase: PHASE2
Conditions: Basal Cell Nevus Syndrome
Sponsor: PellePharm, Inc.

PellePharm, Inc. 
Proof of Concept Study in Gorlin Syndrome Patients 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 1 of 81 
 
 
 
 
Clinical Study Protocol 
Protocol Pelle-926-201 
Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of Patidegib Gel 
2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in 
Gorlin Syndrome Patients 
Development Phase of Study: 
2A 
Study design: 
 
 
EUDRACT #: 
Date of Original Protocol: 
Amendment 1 – Protocol Version 2.0: 
Amendment 2 – Protocol Version 3.0 
 
Amendment 3 – Protocol Version 4.0 
(not approved and not implemented) 
 
Amendment 4-Protocol Version 5.0 
Multicenter, Double-Blind, 
Randomized, Parallel-Group, 
Controlled Clinical Study 
2015-004274-15 
14 December 2015 
 
27 January 2016 
 
02 March 2016 
 
24 May 2016 
 
 
18 August 2016 
 
 
Sponsor Representative: 
 
 
 
Sponsor: 
PellePharm, Inc. 
275 Middlefield Rd., Suite 100 
Menlo Park, CA 94025 
 
CONFIDENTIAL 
PPD 
PPD 
Phone: PPD 
Email: PPD 

PellePharm, Inc. 
Proof of Concept Study in Gorlin Syndrome Patients 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 2 of 81 
 
 
Nothing herein is to be disclosed without prior approval of the sponsor. 
This study will be performed in compliance with Good Clinical Practice (GCP) including the archiving of essential 
study documents. This protocol follows guidelines outlined by the International Conference on Harmonization 
(ICH). All data furnished to the Investigator and his/her staff, and all data obtained through this study, will be 
regarded as confidential and proprietary in nature and will not be disclosed to any third party, except for the 
Medicines and Healthcare Products Regulatory Agency (MHRA) or other local authority, without written consent 
from the sponsor. 

 
PPD 
PPD 
._ 
...., 
_ 
 
 
PellePh ann , Inc. 
Proof of Concept Study in Gorlin Syndrome Patients 
 
Protocol Review and Approvals 
 
Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical Trial of 
Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal 
Cell Carcinomas in Gorlin Syndrome Patients 
 
 
Reviewed and approved: 
 
 
 
 
PellePharm, Inc. 
 
30 August 2016 
Date 
 
 
 
 
 
 
 
CMS International, Inc. 
 
 
 
 
 
PellePharm, Inc. 
 
1gnature 
 
 
 
 
 
- 
:signature 
  
30 August 2016 
Date 
 
 
 
 
2. 
 /4 u9 ?.o/c, 
Date 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL  
Page 3 of &l 
PPD 
PPD 
PPD 

 
tered in Engla 
PellePhann 
, Inc. 
Proof of Concept Study in Godin Syndrome Patients 
 
Personnel Responsible for Conducting the Study 
Double-Blind, Randomized, Vehicle-ControlledProof of Concept Clinical Trial of 
Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal 
Cell Carcinomas in Gorlin Syndrome Patients 
 
 
Contract Research Organization 
PPD 
 
 
Premier Research 
1st Floor Rubra 2 
Mulbeny Business Park 
Fishponds Road 
Wokingham 
RG41 2GY 
Te l 
P D  - 
- 
- 
- 
Direct PPD 
F a x: PD 
Mob1 
PD 
Regis -. .-.- -=--.. -nd No.4671020 
PPD 
 
 
PPD 
 
 
QST Consultations, Ltd. 
11275 Edgewater Dr. 
Allendale, MI 49401 
Telephone: PD 
Fax number: PPD 
PD 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Pelle-926-201 , VS, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 4 of81 

PellePharm, Inc. 
Proof of Concept Study in Gorlin Syndrome Patients 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 5 of 81 
 
 
 
Principal Investigator Protocol Agreement Page 
 
I have carefully read the protocol entitled: “Double-Blind, Randomized, Vehicle-Controlled 
Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number 
of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients” and, 
I declare that this clinical protocol was subject to critical review and is approved by the Sponsor. 
I agree to conduct this study in compliance with procedures outlined in this document according 
to International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, 
the Declaration of Helsinki, and applicable regulatory requirements. This study will not be 
initiated without the approval of the Institutional Review Board (IRB) or Independent Ethics 
Committee (IEC) and the competent authority, if applicable. 
I understand that any substantial changes to the protocol must be approved in writing by the 
IRB/IEC and the competent authority, if applicable, before it can be implemented except where 
necessary to eliminate immediate harm to the patient. I will provide copies of the protocol and 
access to all information furnished by PellePharm to study personnel under my supervision and 
will discuss this material with them to ensure they are fully informed about the study. I 
understand that the study may be terminated or enrollment suspended at any time by PellePharm 
with or without cause, or by me if it becomes necessary to protect the best interests of the study 
patients. 
 
 
 
Investigator Signature 
 
 
 
 
 
Printed Name 
Date 
 
 
Institution Name 
 
 
 
Address 
 
 
 
City, State Zip Code 
Phone Number 

PellePharm, Inc. 
Proof of Concept Study in Gorlin Syndrome Patients 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
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1. SYNOPSIS 
 
Name of Sponsor: PellePharm, Inc. 
Name of Investigational Product: patidegib gel 
Name of Active Ingredients: patidegib HCl 
Title of Study: Double-Blind, Randomized, Vehicle-Controlled Proof of Concept Clinical 
Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible 
Basal Cell Carcinomas in Gorlin Syndrome Patients 
Number of Clinical Centers: Approximately 3 investigational centers in Europe will 
participate in this study. 
Objective: 
The primary objectives of the study are to evaluate the following: 
1. The clinical efficacy of patidegib as defined by the percent decrease in greatest 
diameter of baseline treatment targeted Surgically Eligible basal cell carcinomas 
(SEBs) after 26 weeks of treatment (SEBs are defined as clinically diagnosed basal 
cell carcinoma (BCC) 5 mm or greater in diameter on the face excluding the nose and 
periorbital skin, and 9 mm or greater at sites other than the face). 
2. The molecular efficacy of treatment as defined by reduction in the hedgehog (HH) 
signaling pathway target gene GLI1 after treatment with patidegib gel 2% or 4% or 
vehicle applied twice daily for 6 weeks to treatment-targeted SEBs. 
3. The safety and tolerability of treatment with patidegib gel 2% or 4% or vehicle 
applied twice daily for 26 weeks. 
The secondary objectives of the study are to evaluate the following: 
1. The clinical efficacy of treatment as defined by percent decrease in greatest diameter 
of baseline central facial SEBs (BCCs 3 mm or greater in diameter at Baseline 
located on the nose or periorbital area). 
2. The frequency of new SEBs on the face. (A new SEB is defined as a SEB first noted 
at a visit after Week 2 and that developed at a site where there was no visible BCC of 
any size at the Baseline or the Week 2 visit.) 
3. The proportion of non-central facial BCCs that at Baseline and/or Week 2 visit were 
less than 5 mm in greatest diameter but by the Week 6, 10, 14, 18, 22, or 26 visit 
have become greater than 5 mm in greatest diameter. 
4. The proportion of baseline treatment targeted SEBs that at the end of 26 weeks of 
treatment are no longer large enough to be classified as SEBs (i.e., that is the 
proportion of baseline treatment targeted SEBs on the face that become less than 5 
mm in greatest diameter and non-facial baseline treatment targeted SEBs that become 
less than 9 mm in greatest diameter). 

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Exploratory objective: 
• To evaluate the utility of an investigator static global tumor assessment (ISGTA) in 
assessing the proportion of baseline treatment targeted SEBs that are evaluated as 
being clear or almost clear. 
Methodology: 
This is a multicenter, double-blind, randomized, vehicle-controlled study designed to 
compare the efficacy and safety of patidegib gel, 2% and 4% in comparison with that of 
vehicle. To be eligible for the study, subjects must be at least 18 years of age and must meet 
the diagnostic criteria for basal cell nevus syndrome (BCNS). 
Approximately 18 subjects who meet the study entry criteria will be randomized in a 1:1:1 
ratio to receive patidegib gel 2%, patidegib gel 4%, vehicle gel. One or two tubes of the 
assigned study drug will be dispensed to the subject at the Baseline visit. Additional tubes 
will be dispensed at subsequent visits through Week 22. The study drug will be applied 
topically to the entire face as well as to treatment-targeted SEBs at other anatomical sites 
twice daily for 26 weeks of treatment. 
Information on reported and observed adverse events (AEs) will be obtained at each visit. 
An abbreviated physical examination (PE) will be performed at Baseline, Week 14, and 
Week 26. 
At Baseline and Weeks 2, 6, 10, 14, 18, 22, and 26, all visible BCCs (excluding areas below 
the knees) will be identified by the Investigator, circled in ink, photographed, measured, and 
recorded on a body diagram. Treatment targeted SEBs are defined as the five SEBs on the 
face and/or other anatomical areas identified at Baseline as SEBs will be treated during the 
26 week treatment phase. If a subject has 5 eligible previously untreated facial SEBs 
(excluding tumors on nose and eyelids) these tumors will be the subject’s 5 baseline 
treatment targeted SEBs and non-facial baseline SEBs will not be treated with study 
medication. 
Biomarkers: A single treatment targeted baseline SEB, preferably with the clinical features 
of a nodular BCC, will be biopsied with a 2 mm punch biopsy both at Baseline and at the 
Week 6 visit for determination of GLI1 mRNA levels. In addition, whenever possible, 
available non-treatment targeted tumors that are biopsied or any tumor treated by surgical 
removal during the trial will be evaluated for GLI1 mRNA levels. 
Blood samples for complete blood count and serum chemistry and urine for urinalysis will 
be collected from subjects at Screening, Week 6, Week 14, and Week 26. Additionally, 
blood samples will be collected at Screening and Weeks 6, 14, and 26 to determine plasma 
concentrations of patidegib. 
Subjects who terminate study participation early will be asked to complete all Week 26 
assessments, as appropriate, prior to commencement of any alternative therapy for BCC (if 
possible). Subjects who discontinue from the study during the treatment period will not be 
replaced. 
If signs or symptoms develop in the treatment areas during the treatment period that restrict 
daily activities or make continued application of the study drug difficult due to discomfort, 

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the Investigator may instruct the subject to interrupt use of the study drug temporarily and to 
resume application of the study drug once the signs and symptoms have resolved adequately 
to the point that the Investigator believes that treatment can safely be resumed. The 
Investigator should try to minimize study drug interruptions; and if needed, make best efforts 
to limit a “drug holiday” to 7 days. If the study drug interruption exceeds 4 consecutive days, 
the Investigator should consult with the Medical Monitor to determine a course of action. If 
the study drug is interrupted, discontinued, or a concomitant medication is used to treat a 
sign or symptom, an AE shall be recorded. 
Subjects who discontinue from the study due to clinically significant laboratory 
abnormalities, AEs or any other reason will be asked to complete all Week 26 evaluations. 
Any subject who has an AE during the treatment period will be monitored by the 
Investigator until resolution (return to normal or to the baseline state) or stabilization, as 
determined by the Investigator. 
In addition, application of study drug may be delayed or halted at any time if ongoing safety 
data evaluations raise concern for subject safety. If the subject participation is suspended, all 
of the subject’s safety data will be reviewed by the Medical Monitor in conjunction with the 
Investigator to determine the course of action. 
Number of Subjects Planned: 
Approximately 18 adult subjects (approximately 6 subjects per investigational center) will be 
enrolled and randomized in the study. With a 1:1:1 randomization ratio, it is anticipated that: 
• 6 subjects will be randomized to receive patidegib gel 4% 
• 6 subjects will be randomized to receive patidegib gel 2% 
• 6 subjects will be randomized to receive vehicle gel 
Inclusion Criteria: 
1. The subject is from 18 to 85 years of age, inclusive. 
2. The subject must provide written informed consent prior to any study procedures. 
3. The subject must meet diagnostic criteria for basal cell nevus syndrome (BCNS) 
including major criterion #3a plus one additional major criterion or major criterion #3a 
plus two of the minor criteria outlined below: 
Major Criteria: 
a. More than 2 histologically confirmed BCCs or one under the age of 20 years 
b. Odontogenic keratocysts of the jaw proven by histology 
c. Three or more palmar and/or plantar pits 
d. Bilamellar calcification of the falx cerebri (if less than 20 years old) 
e. Fused, bifid, or markedly splayed ribs. 
f. First degree relative with basal cell nevus syndrome (BCNS) 
g. PTCH1 gene mutation in normal tissue 
Minor Criteria 
a. Macrocephaly 
b. Congenital malformations: cleft lip or palate, frontal bossing, “coarse face”, 

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moderate or severe hypertelorism 
c. Skeletal abnormalities: sprengel deformity, marked pectus deformity, or 
marked syndactyly of the digits 
d. Radiological abnormalities: bridging of the sella turcica, vertebral anomalies 
such as hemivertebrae, fusion or elongation of the vertebral bodies, modeling 
defects of the hands and feet, or flame shaped lucencies of the hands or feet 
e. Ovarian fibroma 
f. 
Medulloblastoma 
(modification of criteria outlined by Kimonis et al, Am J Med Genetics 69:299- 308, 
1997) 
4. The subject must have a history of at least 10 BCC in toto present at Baseline and/or 
treated within 24 months prior to screening. 
5. The subject has at Baseline a total of at least 5 previously untreated SEBs (greatest 
diameter 5 mm or greater on the face excluding the nose and periorbital skin, 9 mm or 
greater on non-facial areas excluding the skin below the knees), as documented clinically 
by the Investigator at Baseline. Untreated is defined as no previous surgical or topical or 
intralesional drug treatment. Previous treatment with systemically administered drugs 
more than 6 months prior to baseline is not considered previous treatment as long as 
there was no clinical evidence of resistance to oral HH (e.g., vismodegib, patidegib, and 
sonidegib inhibitors. Baseline treatment targeted SEBs must not exceed a diameter of 
>2cm. At least one of these tumors must be appropriate for a 2 mm punch biopsy for 
biomarker analysis and Baseline and Week 6 visits. If a subject has 5 or more facial, 
excluding periorbital and nasal skin, SEBs at Baseline, non-facial SEBs will not be 
treatment targeted SEBs. 
6. The subject is willing to have SEBs biopsied for biomarkers and plasma to be collected 
to measure drug levels as required in the protocol. 
7. The subject is willing to abstain from application of non-study topical prescription and 
over the counter medications to facial skin and within 5 cm of treatment targeted SEBs at 
other anatomical areas for the duration of the study, except as prescribed by the 
Investigator. Moisturizers and emollients are allowable. Subjects will be encouraged to 
use sunscreen with a sunscreen protection factor (SPF 15 or higher) at least once daily on 
all exposed skin sites. 
8. Female subjects must have a negative serum pregnancy test at Screening. 
9. If the subject is a male with a female sexual partner who is of childbearing potential the 
couple is willing to use two effective methods of birth control during the duration of the 
trial and for one month after the last application of the gel. A female of childbearing 
potential is a sexually mature woman who: 1) has not undergone a hysterectomy or 
bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 
consecutive months (i.e., has had menses at any time in the preceding 24 consecutive 
months, must agree to use two effective methods of contraception for the duration of the 
study and at least 1 month after the last study drug application. The two forms of birth 
control authorized are defined as the use of a barrier method of contraception (condom 
with spermicide) in association with one of the following methods of birth control: 

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bilateral tubal ligation; combined oral contraceptives (estrogens and progesterone) or 
implanted or injectable contraceptives with a stable dose for at least 1 month prior to 
Baseline; hormonal intra-uterine device (IUD) inserted at least 1 month prior to Baseline. 
10. The subject is willing to contact the study center after each primary skin care physician 
(PSCP) visit to provide the study center details of the visit and any treatment of skin 
tumors. 
11. The subject is willing to forego treatment of the treatment targeted baseline SEBs except 
when the Investigator and/or PSCP believes that delay in treatment potentially might 
compromise the health of the subject. 
Exclusion Criteria: 
1. The subject is a woman of childbearing potential. This proscription is based on the key 
role of the HH pathway in embryogenesis, the known preclinical teratogenic effects of 
systemic cyclopamine, a naturally occurring inhibitor of SMO, and the unknown level of 
systemic exposure following topical application of patidegib in humans. A female of 
childbearing potential is a sexually mature woman who: 1) has not undergone a 
hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for 
at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 
consecutive months. 
2. The subject has used topical products to the face or within 5 cm of a treatment targeted 
SEB or systemic therapies that might interfere with the evaluation of the study 
medication during the study. Specifically these include the use of: 
a. Topical glucocorticoids 30 days prior to screening 
b. Retinoids (e.g., etretinate, isotretinoin, tazarotene, tretinoin, adapalene) 
systemically or topically or > 5% of an alphahydroxy acid (e.g., glycolic acid, 
lactic acid) or 5-fluorouracil or imiquimod (except as topical treatment to discrete 
BCCs) systemically or topically to the skin during the six months prior to entry. 
c. Systemic chemotherapy within one year prior to screening. (Note: field therapy 
with topically applied treatments can be done as long as they are not applied 
within 5 cm of a treatment targeted tumor). 
d. Known inhibitors of the HH signaling pathway (e.g., vismodegib patidegib, and 
sonidegib) topically or systemically within 6 months of entry into the study. 
3. The subject has a history of hypersensitivity to any of the ingredients in the study 
medication formulation. 
4. The subject is unable or unwilling to make a good faith effort to return for all follow-up 
visits and tests. 
5. The subject has uncontrolled systemic disease. 
6. The subject has clinically important history of liver disease, including viral hepatitis, 
current alcohol abuse, or cirrhosis. 
7. The subject has any condition or situation which in the Investigator’s opinion may put 
the subject at significant risk, could confound the study results, or could interfere 
significantly with the subject’s participation in the study. This includes history of other 

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skin conditions or diseases, metabolic dysfunction, physical examination findings, or 
clinical laboratory findings giving reasonable suspicion of a disease or condition that 
contraindicates use of an investigational drug or that might affect interpretation of the 
results of the study or render the patient at high risk from treatment complications. 
8. The subject has a history of invasive cancer within the past five years excluding non- 
melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of breast, or 
chronic lymphocytic lymphoma (CLL) (Stage 0). 
9. The subject has current, recent (within 4 weeks of Baseline visit), or planned 
participation in an experimental drug study while enrolled in this study. 
10. Female sexual partner(s) of male subjects are unwilling or unable to comply with 
pregnancy prevention measures. 
Investigational Product, Reference Therapy, Dosage and Mode of Administration: 
• patidegib gel 2%, applied topically, twice daily for 26 weeks. 
• patidegib gel 4%, applied topically, twice daily for 26 weeks. 
• vehicle gel, applied topically, twice daily for 26 weeks 
Application Instructions: 
The Study Coordinator or designee will instruct the subject on how to apply the study drug 
to the affected treatment areas identified at the Baseline visit by the Investigator. The staff 
member will instruct the subject on the proper application procedure during the Baseline 
visit. Subjects will be instructed to treat their entire face and if instructed to do so, to treat 
non-facial treatment targeted SEBs as well twice daily for 26 weeks. The quantity of gel to 
be applied will be defined using laminated dosing cards. It is approximately 330 mg of gel 
for the entire face and 20 mg of gel for each non-facial treatment targeted SEBs. In addition 
to the verbal instructions given during the visit, the subjects will be provided with written 
instructions for proper application technique. 
Because sunlight can increase the development of skin cancers subjects will be advised to 
avoid or minimize exposure to direct sunlight while in the study. Subjects will also be 
advised to wash their hands before and after application of the study drug. 
The amount of study drug used by the subjects will be monitored by instructing them in the 
use of dosing cards and weighing each newly dispensed study drug tube and re-weighing 
each returned study drug tube at all applicable study visits. 
Duration of Treatment: 
All subjects will be treated for 26 weeks 
Criteria for Evaluation: 
The study will be conducted as outlined in the Schedule of Assessments (Table 2). For all 
efficacy measurements, the Investigators will be provided with training to ensure consistent 
evaluations across investigational centers. The assessments for a particular subject should be 
performed by the same investigator at all study visits whenever possible. 

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Safety Measurements: 
De1mal Safety and Tolerability: Safety and tolerability will be evaluated through assessment 
of selected local signs and symptoms (pain / burning, prnritus, e1ythema, edema, and 
scabbing/crnsting). Each of the 5 baseline treatment-targeted tumor sites, as well as the face 
in general, will be evaluated separately for these signs and symptoms of application site 
reactions. Any local skin reaction that requires use of a concomitant therapy or causing study 
drng inte1n1ption or discontinuation should be reported as an AE. The scales to be used for 
assessing local skin reactions follow: 
Score Grade 
Description 
Pain/Burning:as reported by the subject as being the greatest intensity they have 
experienced at the application site within the last 24 hours at Baselineor since the last 
visit at subsequent visits. 
0 
None 
No pain/burning 
1 
Mild 
Slight burning/stinging sensation; not really bothersome 
2 
Moderate Definite waim , burning/stinging that is somewhat bothersome 
3 
Severe 
Hot burning/stinging sensation that causes definite discomfort and 
may inte1n1pt daily activities and/or sleep 
Pruritus: as reported by the subject as being the greatest intensity they have 
experienced at the application site within the last 24 hours at Baselineor since the last 
visit at subsequent visits. 
0 
None 
No prnritus 
1 
Mild 
Slight prnritus, not really bothersome 
2 
Moderate Definite prnritus that is somewhat bothersome 
3 
Severe 
Intense prnritus that may inte1n1pt daily activities and/or sleep 
E1ythema: as assessed by the Investigatorat each site 
0 
None 
No e1ythema present 
1 
Mild 
Slight pink coloration 
2 
Moderate Definite redness 
3 
Severe 
Mai·ked e1ythema, bright red to dusky dai·k red in color 
Score Grade 
Description 
Edema: as assessed by the Investigator at each site 
0 
None 
No edema 

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1 
Mild 
Slight, but definite edema 
2 
Moderate Definite edema 
3 
Severe 
Marked edema 
Scabbing/Crusting: as assessed by the Investigator at each site 
0 
None 
No scabbing/cmsting 
1 
Mild 
Slight, but definite scabbing/cmsting 
2 
Moderate Definite scabbing/cmsting 
3 
Severe 
Marked scabbing/cmsting 
Symptoms of Hedgehog Inhibitor Toxicity 
At each visit subjects will be asked if they have experienced any symptoms that have been 
associated with this class of diugs. 
Adverse Events: Dming the study, subjects will be assessed for the occunence of new and 
ongoing AEs. Descriptions of AEs will include the dates of onset and resolution (if 
resolved), maximum severity, and seriousness, action taken regarding the study diug, 
con ective ti·eatment, outcome, and the Investigator's assessment of causality. AEs present at 
any visit will be followed to resolution (return to nonnal or to the baseline state) or until 
clinically stable as detennined by the Investigator. 
Safety Laborato1y Tests: Routine safety laborato1y tests (complete blood count/differential 
minalysis, and sennn chemistry) will be perfonned at Screening, Week 6, Week 14, and 
Week 26. Any out-of-range laborato1y result that is considered clinically significant by the 
Investigator will be recorded as an AE and should be confmned by repeat testing at the 
discretion of the Investigator. Clinically significant laborato1y abnonnalities at any visit will 
be followed to resolution (retmn to n01mal or to the baseline state) or until clinically stable 
as detennined by the Investigator. 
Physical Examinations: An abbreviated physical examination (including measmements of 
height, weight, and vital signs (blood pressme, hea1t rate, respiration rate, and oral 
temperatme) will be perfo1m ed at Baseline, Week 14, and Week 26. 
Pregnancy Tests: All female subjects will have a pregnancytest perfo1m ed as specified visits 
dming the study. A semm pregnancy test will be perfonned at Screening and Weeks 6, 14, 
and 26. 
Data Safety Management Board: Sho1tly after the last subject completes 14 weeks of 
h'eatment an independent data safety management board (DSMB) will evaluate all safety 
data as detailed in the DSMB chatter. 
Efficacy Measurements: 
The clinical efficacy of patidegib as defined by the percent decrease in greatest diaineter 
from Baseline of h'eatment targeted SEBs after 26 weeks of ti·eatment. 
At Baseline and Weeks 2, 6, 10, 14, 18, 22, and 26, all visible BCCs will be identified by the 

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Investigator, circled in ink, photographed, measured, and recorded on a body diagram. 
Biomarkers: A single baseline SEB designated as a treatment targeted tumor at Baseline 
will be biopsied with a 2 mm punch biopsy and at the end of 6 weeks of treatment for 
determination of GLI1 mRNA levels. In addition and whenever possible non-treatment 
targeted tumors or any tumor treated by surgical removal during the treatment phase of the 
trial will also be evaluated for GLI1 mRNA levels. 
In addition, if non-treatment targeted tumors have been biopsied or any tumor treated by 
surgical removal during the trial are available they will be evaluated for GLI1 mRNA levels. 
Subjects will complete the Dermatology Life Quality Index (DLQI) and the aBCCdex 
questionnaires at Baseline and Weeks 14 and 26. 
Statistical Methods: 
All subjects who are randomized and dispensed study drug will be included in the intent-to- 
treat (ITT) analysis set. All subjects who are randomized, receive at least 1 confirmed dose 
of study drug, and have at least 1 post-baseline safety assessment will be included in the 
safety population. 
Last observation carried forward (LOCF) will be used to impute efficacy data that are 
missing post-baseline through Week 26. No imputations will be made for missing safety 
data. 
No adjustments for multiplicity will be made. 
If determined appropriate by the Sponsor, limited efficacy analysis on tumor shrinkage and 
biomarkers may be performed after the last subject completes 14 weeks of treatment. 
Efficacy: 
Efficacy Endpoints: 
The efficacy endpoints are intended to compare twice daily application of 2% patidegib gel, 
4% patidegib gel, and vehicle gel. Efficacy assessments will be summarized descriptively by 
treatment group and visit. 
Primary Efficacy Endpoints: 
• The primary efficacy endpoint is the decrease in tumor size defined as percent 
decrease in the sum of the greatest diameter of baseline treatment targeted SEBs at 
the Week 26. 
• Change in GLI1 mRNA levels in drug-treated vs. vehicle-treated tumors after 6 
weeks of treatment. 
Secondary Efficacy Endpoints: 
The secondary efficacy endpoints include: 
• Change in tumor size defined as percent decrease in the sum of the greatest diameter 
of baseline treatment targeted SEBs at Weeks 6, 10, 14, 18, and 22. 
• Change in tumor size defined as percent change in greatest diameter from Baseline of 

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tumors located on the nose or periorbital area at Weeks 6, 10, 14, 18, 22 and 26. 
• Number of new SEBs on the face at Weeks 6, 10, 14, 18, 22, and 26. 
• Proportion of facial BCCs that at Baseline and/or Week 2 were less than 5 mm in 
greatest diameter but by Weeks 6, 10, 14, 18, 22, and 26 visit have become greater 
than 5 mm in greatest diameter. 
• Proportion of baseline treatment targeted SEBs on the face that become less than 5 
mm in greatest diameter and non-facial baseline treatment targeted SEBs that become 
less than 9 mm in greatest diameter at Weeks 6, 10, 14, 18, 22, and 26. 
• The reduction in the HH signaling pathway after treatment with patidegib gel 2% or 
4% or vehicle applied twice daily for 6 weeks to treatment-targeted SEBs. 
Exploratory Efficacy Endpoints: 
• The proportion of baseline treatment targeted SEBs that are evaluated as being clear 
or almost clear at Weeks 6, 10, 14, 18, 22 and 26 based on the ISGTA. 
• DLQI and aBCCdex scores at Baseline and Weeks 14 and 26 as well as change from 
Baseline at Weeks 14 and 26. 
Efficacy Analyses: 
The sum of the greatest diameter of all treatment targeted SEBs for Baseline and post- 
baseline visits will be calculated. 
The primary endpoint and secondary endpoints of change in tumor size, reduction in the HH 
signaling pathway, and the exploratory endpoint of change in GLI1 mRNA levels will be 
evaluated with an analysis of covariance (ANCOVA) with factors of treatment group and 
baseline value as a covariate. For the change in tumor size analyses, the covariate will be the 
sum of the baseline diameters. Pairwise comparisons will be performed using contrasts 
within the ANCOVA. 
The number of new SEBs on the treated areas of the face will be analyzed with a Poisson 
Regression with factors of treatment group and the number of surgically eligible tumors at 
Baseline. Pairwise comparisons will be performed. 
The exploratory analysis of ISGTA will be analyzed with a Cochran-Mantel-Haenszel test. 
DLQI and aBCCdex scores will be summarized with descriptive statistics at Baseline and 
Weeks 14 and 26. Change from baseline will also be summarized at Weeks 14 and 26. DLQI 
responses of “not relevant” will be excluded from the change from baseline summaries. 
Safety: 
Safety Endpoints: 
• Adverse Events 
• Dermal Safety and Tolerability Including pain/burning, pruritus, erythema, edema, 
and scabbing/crusting. 
• Patidegib concentration levels in plasma 
• Clinical laboratory assessments 

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Safety Analyses: 
Subjects will be assessed for the occurrence of new and ongoing AEs. Descriptions of AEs 
will include the dates of onset and resolution (if resolved), maximum severity, seriousness, 
action taken regarding the study drug, corrective treatment, outcome, and Investigator’s 
assessment of causality. All AEs will be recorded and classified using terminology from the 
Medical Dictionary for Regulatory Activities (MedDRA). All reported treatment-emergent 
AEs (TEAEs), defined as any AE with an onset on or after the date of first study drug 
application, will be summarized by treatment group, the number of subjects reporting 
TEAEs, system organ class, preferred term, severity, and relationship to study drug. When 
summarizing TEAEs by severity or relationship to study drug, each subject will be counted 
only once within a system organ class or a preferred term using the event with the greatest 
severity or causality, respectively, within each category. All reported serious adverse events 
(SAEs) will be summarized by treatment group, the number of subjects reporting SAEs, 
system organ class, preferred term, severity, and relationship to study drug. 
All information pertaining to AEs noted during the study will be listed by subject and will 
include a verbatim description of the event as reported by the Investigator, as well as the 
preferred term, system organ class, start date, stop date (if stopped), seriousness, severity, 
action taken regarding the study drug, corrective treatment, outcome and relationship to the 
study drug. In addition, a listing of subjects who prematurely discontinue from the study due 
to AEs will be provided as well as a listing of subjects who reported an SAE. 
The frequency of dermal safety and tolerability assessments including pain/burning, pruritus, 
erythema, edema, and scabbing/crusting will be summarized by treatment group and visit. 
Plasma concentrations of patidegib will be summarized at Screening and Weeks 6, 14, and 
26 by treatment group. 
Changes from baseline in safety laboratory values and vital sign measurements will be 
summarized with descriptive statistics for each treatment group at all applicable study visits. 
Shift tables will be presented for changes in safety laboratory values to summarize 
laboratory test results collected at Baseline and Weeks 6, 14, and 26. Normal ranges 
established by the central laboratory will be used to determine the shifts. A listing of all out- 
of-range laboratory test results at any assessment time point will also be provided. 
Determination of clinical significance for all out-of-range laboratory values will be made by 
each Investigator and included in the listing. In addition, a listing of all clinically significant 
laboratory test results will be provided. 
Sample Size Calculations: 
Data from a trial of oral vismodegib (New England Journal of Medicine) given for 7 months 
resulted in a percent change in the sum of the greatest diameter of baseline treatment 
targeted SEBs of 69.2 and 13.7 with a standard deviation of 17 and 23 for treated and control 
subjects, respectively. A sample size of 6 subjects enrolled in each treatment group with a 
minimum of 5 SEBs will have greater than 95 percent power to detect a statistically 
significant difference at an alpha of 0.05. 

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2. TABLE OF CONTENTS 
1. 
SYNOPSIS ...............................................................................................................6 
2. 
TABLE OF CONTENTS .......................................................................................18 
3. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ........................21 
4. 
INTRODUCTION .................................................................................................23 
5. 
STUDY OBJECTIVES ..........................................................................................25 
6. 
INVESTIGATIONAL PLAN ................................................................................26 
6.1 
Overall Study Design and Plan ..................................................................26 
7. 
SELECTION AND WITHDRAWAL OF SUBJECTS .........................................33 
7.1 
Subject Inclusion Criteria ..........................................................................33 
7.2 
Subject Exclusion Criteria .........................................................................35 
7.3 
Subject Withdrawal Criteria ......................................................................36 
7.4 
Study Drug Discontinuation ......................................................................37 
7.5 
Discontinuation of the Study .....................................................................38 
8. 
TREATMENT PLAN ............................................................................................38 
8.1 
Methods of Assigning Subjects to Treatment Groups ...............................38 
8.2 
Randomization and Blinding .....................................................................38 
8.3 
Unblinding .................................................................................................39 
8.4 
Prior and Prohibited Concomitant Medication or Therapy ........................39 
8.5 
Treatment Compliance ...............................................................................40 
8.6 
Protocol Deviations and Violations ...........................................................40 
9. 
STUDY DRUG MATERIALS AND MANAGEMENT ......................................42 
9.1 
Packaging and Labeling .............................................................................43 
9.2 
Storage, Handling, and Disposal of Study Drug ........................................44 
9.3 
Administration ...........................................................................................44 
9.4 
Study Drug Accountability ........................................................................44 
10. 
STUDY PROCEDURES AND EVALUATIONS ................................................46 
10.1 
Schedule of Evaluations and Procedures ...................................................46 
10.1.1 Screening Visit (Day -42 to Day 0) ...............................................46 
10.1.2 Baseline Visit (Day 1) ....................................................................47 
10.1.3  Weeks 2, 10, 18, and 22 .................................................................48 
10.1.4  Week 6, 14, and 26 ........................................................................49 
10.1.5  Week 26 (Day 183 ± 3 Days) / Early Termination ........................50 
10.2 
Evaluation of Efficacy ...............................................................................51 
10.2.1 Assessment of BCCs ......................................................................52 

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10.2.2 Messenger RNA (mRNA)..............................................................52 
10.2.3 Investigator Global Static Tumor Assessment ...............................52 
10.2.4 Subject Questionnaires...................................................................53 
10.3 
Evaluation of Safety ...................................................................................53 
10.3.1 Dermal Safety and Tolerability ......................................................53 
10.3.2 Medical History and Abbreviated Physical Examination ..............54 
10.3.3 Safety Laboratory Tests .................................................................55 
10.3.4 Pregnancy Tests .............................................................................55 
10.3.5 Plasma Concentrations ...................................................................55 
10.3.6 Adverse Events ..............................................................................55 
10.3.6.1 Definition of Adverse Event .........................................55 
10.3.6.2 Documenting Adverse Experiences ..............................55 
10.3.6.3 Serious Adverse Events.................................................56 
10.3.6.4 Assessment of Severity .................................................57 
10.3.6.5 Assessment of Causality ...............................................57 
10.3.6.6 Reporting of Serious Adverse Events ...........................58 
10.3.6.7 Emergency Contact .......................................................59 
10.3.6.8 Expedited Serious Adverse Event Reports ...................59 
10.3.6.9 Other Required Safety Assessments .............................60 
11. 
STATISTICS .........................................................................................................61 
11.1 
Analysis Populations ..................................................................................61 
11.2 
Subject Disposition ....................................................................................61 
11.3 
Demographics and Baseline Characteristics ..............................................62 
11.4 
Protocol Deviations ....................................................................................62 
11.5 
Compliance ................................................................................................62 
11.6 
Interim Analyses ........................................................................................62 
11.7 
Assessment of Efficacy ..............................................................................62 
11.7.1 Efficacy Endpoints .........................................................................62 
11.7.1.1 Primary Efficacy Endpoints: .........................................62 
11.7.1.2 Secondary Efficacy Endpoints: .....................................62 
11.7.1.3 Exploratory Efficacy Endpoints: ...................................63 
11.7.2 Efficacy Analyses ..........................................................................63 
11.8 
Assessment of Safety .................................................................................64 
11.8.1 Dermal Safety and Tolerability ......................................................64 
11.8.2 Adverse Events ..............................................................................64 
11.8.3 Plasma Concentrations ...................................................................64 
11.8.4 Safety Laboratory Values and Vital Sign Measurements ..............65 

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11.8.5 Pregnancy Tests .............................................................................65 
11.8.6 Handling of Missing Data ..............................................................65 
11.8.7 Multicenter Issues ..........................................................................65 
11.8.8 Multiplicity Issues ..........................................................................65 
11.9 
Sample Size Determination........................................................................65 
12. 
QUALITY CONTROL AND QUALITY ASSURANCE .....................................66 
12.1 
Study Monitoring .......................................................................................66 
12.2 
Audits and Inspections ...............................................................................66 
12.3 
Data Quality Assurance .............................................................................67 
13. 
ETHICS AND ADMINISTRATIVE ISSUES ......................................................68 
13.1 
Ethical Conduct of the Study .....................................................................68 
13.2 
Ethics Review ............................................................................................68 
13.3 
Written Informed Consent .........................................................................68 
13.4 
Subject Data Protection..............................................................................68 
13.5 
Data Safety Monitoring Committee ...........................................................69 
13.6 
Financial Disclosure...................................................................................69 
13.7 
Investigator Obligations .............................................................................69 
13.8 
Changes to the Protocol .............................................................................69 
13.9 
Confidentiality/Publication of the Study ...................................................69 
14. 
DATA HANDLING AND RECORD KEEPING .................................................70 
14.1 
Inspection of Records ................................................................................70 
14.2 
Retention of Records..................................................................................71 
14.3 
Electronic Case Report Form Completion .................................................71 
15. 
REFERENCES ......................................................................................................72 
16. 
APPENDICES .......................................................................................................73 
16.1 
Body Diagram ............................................................................................73 
16.2 
Subject Questionnaire – DLQI...................................................................75 
16.3 
Subject Questionnaire – aBCCdex.............................................................76 
16.4 
Safety Laboratory Tests .............................................................................81 

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3. LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
AE 
ALT 
API 
AST 
Abbreviation or 
Specialist Term 
 
Definition or Explanation 
 
Adverse Event 
Alanine Aminotransferase 
Active Pharmaceutical Ingredient 
Aspartate Aminotransferase 
ANCOVA 
Analysis of Covariance 
BCC 
Basal Cell Carcinoma 
BCNS 
BUN 
Basal Cell Nevus Syndrome 
Blood Urea Nitrogen 
CBC/Diff 
Complete Blood Count with Differential 
CLL 
Chronic Lymphocytic Lymphoma 
DLQI 
Dermatology Life Quality Index 
eCRF 
Electronic Case Report Form 
ET 
Early Termination 
GCP 
Good Clinical Practice 
GLI 1 
Glioma-associated oncogene homolog 1 
GRAS 
Generally Recognized as Safe 
HCl 
Hydrogen Chloride 
HH 
Hedgehog 
ICF 
Informed Consent Form 
ICH 
International Conference on Harmonization 
IEC 
Independent Ethics Committee 
ISGTA 
Investigator Static Global Tumor Assessment 
ITT 
Intent-to-Treat 
LOCF 
Last Observation Carried Forward 
MedDRA 
MCH 
MCHC 
MCV 
Medical Dictionary for Regulatory Affairs 
Mean Corpuscular Hemoglobin 
Mean Corpuscular Hemoglobin Concentration 
Mean Corpuscular Volume 
MHRA 
Medicines and Healthcare Products Regulatory Agency 
mRNA 
Messenger Ribonucleic Acid 
PSCP 
Primary Skin Care Physician 
QOL 
RBC 
Quality of Life 
Red Blood Cell 
SAE 
Serious Adverse Event 
SAP 
SAS 
Statistical Analysis Plan 
Statistical Analysis System 
SEB 
Surgically Eligible Basal Cell Carcinoma 

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Abbreviation or 
Specialist Term 
Definition or Explanation 
 
SPF 
Sunscreen Protection Factor 
SAP 
Statistical Analysis Plan 
TEAE 
Treatment-Emergent Adverse Event 
UK 
United Kingdom 
UV 
WBC 
Ultraviolet 
White Blood Cell Count 
 

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4. INTRODUCTION 
Patients affected by the BCNS (Gorlin syndrome, nevoid basal cell carcinoma syndrome; OMIM 
#109400), a rare autosomal dominant inherited disorder, have a dramatically increased risk of 
developing BCCs (developing hundreds to thousands of BCCs) as well as an increased risk of 
developing certain extra cutaneous tumors (e.g. medulloblastomas and rhabdomyosarcomas). 
BCNS patients have multiple and frequent surgical removals of BCCs, with an average of 30 
surgeries per year.  Although this is problematic at all anatomical sites it is the greatest problem 
for facial tumors. While timely surgical removal of an individual BCC generally produces a cure 
and an acceptable cosmetic result, repeated surgeries can be uncomfortable, generally are 
disfiguring, and often cause functional impairment (e.g., of the  eyelid,  nose)  and  significant 
morbidity.  The sponsor plans to develop topical patidegib to slow the progression of BCC 
disease in BCNS patients. “Progression” is defined as the development of increasing numbers of 
BCCs that are of a surgically-eligible size. Our focus on facial BCCs is based on the patients’ 
need to minimize the number of facial surgeries with the consequent inevitable attendant scarring 
as well as to minimize impairment of function the lips, nose, eyelids, and ears. Surgical scarring 
on the trunk and extremities generally does not produce as much morbidity as does facial 
surgery. It may not be practical for patients to apply a topical product to their entire cutaneous 
surface, and treating the entire cutaneous surface would greatly increase the systemic exposure. 
Therefore in the present proof of concept trial non facial tumors will be treated only when 
needed to enable each patient to treat 5 baseline surgically eligible BCCs. SEBs are defined as 
tumors with a diameter of 3 mm or greater on the nose or periorbital skin, 5 mm or greater 
elsewhere on the face, and 9 mm or greater on non-facial skin in patients with BCNS. 
Illustrative of the potential of this pharmacologic inhibition of hedgehog signaling is a recently 
published trial of oral HH inhibitor vismodegib in which patients on active drug developed new 
SEBs at an annual median rate of 2 new SEBs and those on placebo at an annual median rate of 
29 new SEBs. 
Currently, 5-FU and Imiquimod are used as topical treatments for superficial BCCs. These are 
the easiest histologic subtype to treat but constitute only 20% of all BCCs. There are no topical  
drugs to prevent BCCs (except sunscreen) that are widely accepted. Oral retinoid treatment of 
BCNS patients can reduce their rate of development of BCCs but does so only at a dose that 
usually produces intolerable side effects. 
Inhibition of HH pathway with the oral HH inhibitor, vismodegib, has been shown to prevent 
BCC development and to shrink existing BCCs in BCNS patients as well as to control some 
advanced BCCs1. In addition sonidegib another oral HH inhibitor has recently been approved for 
the treatment of locally advanced BCCs. However, oral HH inhibitors produce class-specific side 
effects such as hair loss, taste loss, weight loss, fatigue, and intense muscle cramps. The severity 
of the adverse events (AEs) associated with vismodegib is illustrated by the fact that 54% of 

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BCNS patients in an oral vismodegib clinical trial discontinued treatment because of AEs, 
despite clear efficacy. 
PellePharm hopes to decrease the number of facial SEBs in BCNS patients by applying a topical 
HH inhibitor (patidegib) to produce good local effects without producing the side effects that 
occur with systemic administration of this class of drugs. PellePharm believes that there are two 
therapeutic effects that could decrease the patients’ number of SEBs, namely (i) shrinking 
preexisting tumors and (ii) preventing the development of new tumors. 
Patidegib is a semi-synthetic small molecule, and the topical drug product is manufactured with 
generally accepted, safe excipients. Oral patidegib has a good therapeutic effect on locally 
advanced and metastatic BCCs but produces the same types of adverse effects as do other 
systemic HH inhibitors. Topical patidegib has been shown to be stable in the developed gel 
formulation and deposits significant amounts of drug in the dermis after topical application to 
human cadaver skin in Franz chamber assays. Topical application of patidegib significantly 
reduces murine BCC tumor size in vivo and reduces GLI1 biomarker expression in vitro in 
human BCC tumors. 
The goal of the present trial is to evaluate topical patidegib’s safety, tolerability, and effects on 
the size of preexisting SEBs and on the development of new SEBs. If the efficacy with topical 
patidegib can approach the level of efficacy of oral HH inhibitors seen in BCNS patients while 
avoiding their systemic side effects, it would represent a major advance for BCNS patients. 

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5. STUDY OBJECTIVES 
The primary objectives of the study are to evaluate the following: 
1. The clinical efficacy of patidegib as defined by the percent decrease in greatest diameter 
of baseline treatment targeted Surgically Eligible basal cell carcinomas (SEBs) after 26 
weeks of treatment [SEBs are defined as clinically diagnosed basal cell carcinoma (BCC) 
5 mm or greater in diameter on the face excluding the nose and periorbital skin, and 9 
mm or greater at sites other than the face]. 
2. The molecular efficacy of treatment as defined by reduction in the hedgehog (HH) 
signaling pathway target gene GLI1 after treatment with patidegib gel 2% or 4% or 
vehicle applied twice daily for 6 weeks to treatment-targeted SEBs. 
3. The safety and tolerability of treatment with patidegib gel 2% or 4% or vehicle applied 
twice daily for 26 weeks. 
The secondary objectives of the study are to evaluate the following: 
1. The clinical efficacy of treatment as defined by percent decrease in greatest diameter of 
baseline central facial SEBs (BCCs 3 mm or greater in diameter at Baseline located on 
the nose or periorbital area). 
2. The frequency of new SEBs on the face. (A new SEB is defined as a SEB first noted at a 
visit after Week 2 and that developed at a site where there was no visible BCC of any size 
at the Baseline or the Week 2 visit.) 
3. The proportion of non-central facial BCCs that at Baseline and/or Week 2 visit were less 
than 5 mm in greatest diameter but by the Week 6, 10, 14, 18, 22, or 26 visit have 
become greater than 5 mm in greatest diameter. 
4. The proportion of baseline treatment targeted SEBs that at the end of 26 weeks of 
treatment are no longer large enough to be classified as SEBs (i.e., that is the proportion 
of baseline treatment targeted SEBs on the face that become less than 5 mm in greatest 
diameter and non-facial baseline treatment targeted SEBs that become less than 9 mm in 
greatest diameter). 
Exploratory objective: 
To evaluate the utility of an investigator static global tumor assessment (ISGTA) in assessing the 
proportion of baseline treatment targeted SEBs that are evaluated as being clear or almost clear. 

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6. INVESTIGATIONAL PLAN 
6.1 
Overall Study Design and Plan 
This is a multicenter, double-blind, randomized; vehicle-controlled designed to assess the 
efficacy and safety of patidegib gel, 2% and 4% in comparison with vehicle. To be eligible for 
the study, subjects must be at least 18 years of age and meet the diagnostic criteria for BCNS. 
 
Selection of dose levels and frequency of application for topically applied drug products is 
challenging. With systemic drugs it is possible to select the starting dose and frequency of dosing 
based on pharmacokinetics and pharmacodynamics obtained from animal studies and Phase 1 
normal volunteer studies. Such data is simply not available for topically applied drugs, owing to 
several technical challenges. Foremost is a lack of in-vivo pharmacokinetic and 
pharmacodynamics data in skin following topical application of a drug product. In addition, the 
solubility of a drug often sets the limit for the highest concentration as it is preferable not to work 
with topical drugs in suspension.   The reality is that the selection of doses for novel topical 
drugs is more an educated approximation than a calculation based on pharmacokinetics 
pharmacodynamics. 
First and foremost in selecting the dose were safety considerations. As detailed in our 
Investigator’s Brochure based on dermal application in mini pigs and oral administration in 
humans the proposed doses have a very large safety margin for both dermal tolerability and 
systemic safety. With these large safety margins the key question then in selecting the dose for 
this proof of concept clinical trial was choosing doses that had the greatest probability of 
showing biologic activity. 
 
The selection of doses for patidegib gel was informed by in vitro skin penetration studies, in 
vitro treatment of excised and explanted human BCCs, in vivo treatment of tumors in mice and 
the upper limit the solubility of the drug. The penetration of patidegib into and through human 
skin was studied in-vitro with multiple formulations and concentrations of patidegib in Franz 
cells experiments. In these experiments formulated drug is applied to cadaver skin and drug 
levels in the skin are then measured. Patidegib gel at 4.87%, 0.75%, and 0.1% applied 
continuously in drug excess resulted in drug levels in the epidermis of 758 µM, 315 µM, and 35 
µM and in the dermis of 341 µM, 59 µM, and 5 µM, respectively. BCCs are tumors of the 
epidermis that extend down into the dermis. While these levels represent only a small percentage 
of the applied dose they are well above the IC90 of 0.04 µM. It is not known how much of this 
drug is in the intracellular compartment and it should be noted that in Franz cell experiments 
formulated drug is applied in excess representing what is referred to as an infinite dose which is 
different than the clinical situation where a finite dose is applied. Because of these unknowns one 

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wants to be well above the IC 90 as long as there are no safety concerns. 
 
In trials of the oral formulation of patidegib for the treatment of BCC skin drug levels were 
measured. The levels of drug seen in vitro in the skin in Franz cells were 20 times higher than the 
skin levels seen in the skin following oral administration of 130 mg of patidegib to patients with 
BCCs. In mice with BCC allographs and in genetically modified mice with BCCs patidegib gel 
at 1% and 4% applied twice daily decreased tumor size and inhibited the GL1 biomarkers levels 
comparable to oral vismodegib. It is well known that drugs penetrate mouse skin much better 
than human skin. This is thought to be due to mouse skin being thinner and having far more large 
terminal hair follicles than human skin. In vitro in explanted human BCC tumors 5% patidegib 
gel was able to inhibit the GL1 biomarker after 5 days of treatment to levels comparable to those 
seen with oral vismodegib. In the process of developing the clinical formulation of patidegib the 
gel formation which showed the best in vitro skin penetration the solubility of the drug limited 
the highest concentration to 4%. 
 
Based on the above observations PellePharm concluded that patidegib in the gel formulation at 
2% and 4% applied twice daily would have a good probability of being effective treatment 
regimens. These concentrations also provide a large safety margin for both dermal and systemic 
safety. Assuming that this proof of concept trail demonstrates clinically meaningful activity 
future clinical trials will refine the treatment regimen in terms of drug concentration and/or 
frequency of application. 
 
Approximately 18 subjects who meet the study entry criteria will be randomized in a 1:1:1 ratio 
to receive patidegib gel 2%, patidegib gel 4%, vehicle gel. One or two tubes of the assigned 
study drug will be dispensed to the subject at the Baseline visit. The study drug will be applied 
topically to the entire face as well as to treatment-targeted SEBs at other anatomical sites twice 
daily for 26 weeks of treatment. Subjects will apply their twice daily treatments at home as 
explained by the study coordinator or designee at each study center. The subject will apply the 
initial application under the supervision of the study staff. If a subject has a treatment targeted 
SEB on an anatomical location that the subject cannot reach such as the back, a friend or family 
member may apply the treatment. In this case the friend or family member applying the gel 
cannot be a woman of childbearing potential, and the person should apply the gel using a latex or 
vinyl glove. As BCNS patients are routinely advised, all subjects will be instructed to avoid 
exposure to direct sunlight and to continue their use of sunscreens to minimize their exposure to 
ultraviolet (UV) radiation. 
 
At each post-baseline visit during the treatment period, (Weeks 2, 6, 10, 14, 18, 22, and 26) the 
subjects will be asked to return their tubes of study drug which will be evaluated for drug usage 

 
PellePhan,nInc. 
Proof of Concept Study in Godin Syndrome Patients 
 
compliance. It has been estimated that subjects will require 330 mg of gel to adequately cover 
their face and 20 mg of gel to adequately cover each non-facial SEBs. The Study Coordinator or 
designee at each study center will dispense new tube(s) of study drng to each subject at study 
center visits at Baseline through Week 22. The goal is that each subject will have an adequate 
supply of gel to last until the next clinic visit plus some extra in case of application enors and or 
delay in study visits. While individual usage rates of topical products have individual variability 
the use of dosing card should help limit the variation in dosing inherent with topical products. 
PellePhaim estimates that subjects ti·eating only the face, 1, 2, 3, 4, or 5 non-facial SEBs will 
consume 19.8 gm, 21 gm, 22.2 gm, 23.4 gm, 24.6 gm, and 25.8 gm of gel over the course of a 30 
day month. 
 
Table 1: Estimated Quantity of Study Drug Usage 
 
Area Treated 
Single 
Dose 
Daily Dose 
60 Doses 
Face and SEB 60 Doses 
Face 
330mg 
660mg 
19.8 gm 
Non-Facial SEB 
0 
19.8 gm 
1 
20mg 
40mg 
1.2 gm 
21.0 gm 
2 
40mg 
80mg 
2.4 gm 
22.2 gm 
3 
60mg 
120mg 
3.6 gm 
23.4 gm 
4 
80mg 
160mg 
4.8 gm 
24.6 gm 
5 
100mg 
200mg 
6.0 gm 
25.8 gm 
 
Upon completion of the 26-week ti·eatment period, all subjects will be asked to return to the 
investigational center for final evaluation. During the study, subjects will be allowed to use 
moisturizers and emollients and sunscreen. The Investigator will assess the areas affected at each 
study visit. 
 
Non-facial baseline ti·eatment-tai·getedSEBs ai·e defined as SEBs identified at Baseline as 
ti·eatment tai·geted SEBs on skin other than skin of the face. While eyelids can be u-eated as paii 
of the applicationto the whole face, subjects need to be cautioned not to apply the gel to their 
eyes or the inner eyelids. Infonnation on repo1i ed and observed AEs will be obtained at each 
visit. An abbreviated physical exainination will be perfo1m ed at Baseline, Week 14, and Week 
26. 
 
At Baseline and Weeks 6, 10, 14, 18, 22, and 26, all visible BCCs will be identified by the 
Investigator, circled in ink, measured, and recorded on a body diagram. Treatment tai·geted SEBs 
 
 
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PellePharm, Inc. 
Proof of Concept Study in Gorlin Syndrome Patients 
 
are defined as SEBs on the face and/or other anatomical areas identified at Baseline as SEBs and 
which were treated during the 6 month treatment phase. 
 
Biomarkers: A single baseline SEB designated as a treatment targeted tumor at Baseline will be 
biopsied with a 2 mm punch biopsy and at the end of 6 weeks of treatment for determination of 
GLI1 mRNA levels. In addition and whenever possible non-treatment targeted tumors or any 
tumor treated by surgical removal during the treatment phase of the trial will also be evaluated 
for GLI1 mRNA levels. 
 
Blood samples for complete blood count and serum chemistry and urine for urinalysis will be 
collected from subjects at Screening, Week 6, Week 14, and Week 26. Plasma samples will also 
be collected at Screening and Weeks 6, 14, and 26 to determine plasma concentrations of 
patidegib and relevant metabolites. 
 
Subjects who terminate study participation early will be asked to complete all Week 26 
assessments, as appropriate, prior to commencement of any alternative therapy for BCC (if 
possible). Subjects who discontinue from the study during the treatment period will not be 
replaced. 
 
If signs or symptoms develop in the treatment areas during the treatment period that restrict daily 
activities or make continued application of the study drug difficult due to discomfort, the 
Investigator may instruct the subject to temporarily interrupt use of the study drug and to resume 
application of the study drug once the signs/symptoms have subsided. The Investigator should 
try to minimize study drug interruptions; and if needed, make best efforts to limit a “drug 
holiday” to 7 days. If the study drug interruption does exceed 4 consecutive days, the 
Investigator should consult with the Medical Monitor to determine a course of action. If the 
study drug is interrupted, discontinued, or a concomitant medication is used to treat a sign or 
symptom, an AE shall be recorded. 
 
Subjects who discontinue from the study, due to clinically significant laboratory abnormalities or 
AEs will be asked to complete all Week 26 evaluations. Any subject who has an AE during the 
treatment period will be monitored by the Investigator until resolution (return to normal or to the 
baseline state) or stabilization, as determined by the Investigator. 
 
In addition, application of study drug may be delayed or halted at any time if ongoing safety data 
evaluations raise concern for subject safety. If the subject participation is suspended, all of the 
subject’s safety data will be reviewed by the Medical Monitor in conjunction with the 
Investigator to determine course of action. 
 
 
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Protocol Pelle-926-201 
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Table 2: Schedule of Assessments 
 
Assessments 
Screening 
Baseline 
(Within 6 weeks 
of Screening) 
Week2 
Week6 
Week 10 
Week 14 
Week 18 
Week22 
Week26/ ET3 
Day 
-42 to 0 
1 
15 
43 
71 
99 
127 
155 
183 
Window (days) 
±2 
± 3 
±3 
±3 
± 3 
± 3 
±3 
Info1med Consentb 
X 
Eligibility Confnmation 
X 
X 
Demographics 
X 
Medical Histo1y 
X 
X: 
Heigh t, We ight 
X 
Xd 
X 
X 
X 
X 
X 
X 
X 
Physical Examination (vital 
signs) 
X 
X 
X 
Laborato1y Testing (blood and 
urinee) 
X 
X 
X 
X 
Semm Pregnancy Test 
X 
X 
X 
X 
Randomization 
X 
Quality of Life Questionnaires 
X 
X 
X 
Tasks to be Done by Physician
Identify BCC to be Biopsied 
X 
X 
Identify 5 SEBs 
X 
X 
Application Site Evaluation 
X 
X 
X 
X 
X 
X 
X 
X 
ISGTA 
X 
X 
X 
X 
X 
X 
X 
X 
Measure and map visible 
BCCsf 
X 
X 
X 
X 
X 
X 
X 
X 
Dete1mine Clinical Tumor 
Type 
X 
Imagingg 
X 
X 
X 
X 
X 
X 
X 
X 
Syste mic Symptoms 
X 
X 
X 
X 
X 
X 
X 
X 
Patidegib Plasma Levels 
X 
X 
X 
X 

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ProtocloPelle-92-6201 
PellePham1, Inc. 
 
 
 
Assess ments 
Screening 
Baseline  
(Within 6 weeks 
of Screening) 
Week 2 
Week6 
Week 10 
Week 14 
Week 18 
Week 22 
Week 26 / ET 
Day 
-42 to 0 
1 
15 
43 
71 
99 
127 
155 
183 
Window (days) 
± 2 
± 3 
±3 
± 3 
± 3 
± 3 
±3 
Patidegib N ehicle 
Applicationsh 
X 
X 
X 
X 
X 
X 
X 
X 
S tu dy chug review/In structions 
Dispensa tionh 
X 
X 
X 
X 
X 
X 
X 
Co n co mitant Medications 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Adverse Events 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Review Test Article 
Compliance/Accountability1 
X 
X 
X 
X 
X 
X 
X 
X 
 
BCC= basal cell carcinoma; ET = early termination; ISGTA = investigator static global tumor assessment; SEB = surgically eligib le basal cell carcinoma 
 
• For subjects who discontinue early dming the treatment period, all procedures outlined for the ET visit should be completed at the time of discontinuation. 
b Must be signed prior to any study procedures. 
c Medical histo1ywill be updated at Baseline visit. 
d Height will be measured at Baseline only. 
e The following blood samples for laborato1ytests will be collected: Alanine aminotransferase (ALT), albumin, alkaline phosphatase, aspartate aminotransferase 
(AST), total bilimbin, blood urea nitrogen (BUN), calcium, carbon dioxide, chloride, creatinine, glucose, potassium, protein, sodium, white blood cell (WBC), red 
blood cell (RBC), hemoglobin, hematocrit, Mean Corpuscular Volume (MCV), Mean corpuscular hemoglobin (MCH), Mean corpuscular hemoglobin concentration 
(MCHC) Red Cell Distribution Width, Platelets, Mean Platelet Volumes, Absolute/Percent Neutrophil Count, Absolute/PercentLymphocyte count, 
Absolute/Percent Monocyte count, Absolute/Percent Eosinophil Count, Absolute/Percent Basophil Count. Urinalysis will include reflex microscopic examination. 
Clinically significant laborato1yfindings at Week 6, Week 14, or Week 26 will be repeated at the discretion of the Investigator, and the subjectwill be followed until 
resolution (retum to normal or to the baseline state) or until clinically stable as detennined by the Investigator. 
 
f T umor s to be mea s ured and mapped include the 5 baseline treatment-targeted tumors as well as all other facial tumors including those on the eyelids and the nose. 
In addition, up to 10 non treatment-targeted non-facial tumors will also be measured and mapped. 

 
Protocol Pelle-926-201 
PellePharm, Inc. 
g Imaging requirements are provided in the imaging manual. 
h Subjects will be trained on how to administer the study drug at the Baseline visit and retrained at subsequent visits, if necessary. The study drug may be applied at 
the study visit. Subjects will record any missed doses and provide to the study site for review. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
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7. SELECTION AND WITHDRAWAL OF SUBJECTS 
7.1 
Subject Inclusion Criteria 
Subjects meeting all of the following criteria will be eligible for study entry: 
 
1. The subject is from 18 to 85 years of age, inclusive. 
2. The subject must provide written informed consent prior to any study procedures. 
3. The subject must meet diagnostic criteria for BCNS including major criterion #3a plus 
one additional major criterion or major criterion #3a plus two of the minor criteria 
outlined below: 
 
Major Criteria: 
a. More than 2 histologically confirmed BCCs or one under the age of 20 years 
b. Odontogenic keratocysts of the jaw proven by histology 
c. Three or more palmar and/or plantar pits 
d. Bilamellar calcification of the falx cerebri (if less than 20 years old) 
e. Fused, bifid, or markedly splayed ribs. 
f. First degree relative with basal cell nevus syndrome (BCNS) 
g. PTCH1 gene mutation in normal tissue 
Minor Criteria 
a. Macrocephaly 
b. Congenital malformations: cleft lip or palate, frontal bossing, “coarse face”, 
moderate or severe hypertelorism 
c. Skeletal abnormalities: sprengel deformity, marked pectus deformity, or 
marked syndactyly of the digits 
d. Radiological abnormalities: bridging of the sella turcica, vertebral anomalies 
such as hemivertebrae, fusion or elongation of the vertebral bodies, modeling 
defects of the hands and feet, or flame shaped lucencies of the hands or feet 
e. Ovarian fibroma 
f. 
Medulloblastoma 
(modification of criteria outlined by Kimonis et al, Am J Med Genetics 69:299- 308, 

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1997) 
4. The subject must have a history of at least 10 BCCs in toto present at Baseline and/or 
treated within 24 months prior to screening. 
5. The subject has at Baseline a total of at least 5 previously untreated SEBs (greatest 
diameter 5 mm or greater on the face excluding the nose and periorbital skin, 9 mm or 
greater on non-facial areas excluding the skin below the knees), as documented clinically 
by the Investigator at Baseline. Untreated is define as no previous surgical or topical or 
intralesional drug treatment. Previous treatment with systemically administered drugs 
more than 6 months prior to Baseline is not considered previous treatment as long as 
there was no clinical evidence of resistance to oral HH (e.g., vismodegib, patidegib, and 
sonidegib) inhibitors. Baseline treatment targeted SEBs must not exceed a diameter of > 
2cm. At least one of these tumors must be appropriate for a 2 mm punch biopsy for 
biomarker analysis at Baseline and Week 6 visits. If a subject has 5 or more facial, 
excluding periorbital and nasal skin, SEBs at Baseline, non-facial SEBs will not be 
treatment targeted SEBs. 
6. The subject is willing to have SEBs biopsied for biomarkers and plasma to be collected to 
measure drug levels as required in the protocol. 
7. The subject is willing to abstain from application of non-study topical prescription and 
over the counter medications to facial skin and within 5 cm of treatment targeted SEBs at 
other anatomical areas for the duration of the study except as prescribed by the 
Investigator. Moisturizers and emollients are allowable. Subjects will be encouraged to 
use sunscreen with a sunscreen protection factor (SPF 15 or higher) at least once daily on 
all exposed skin sites. 
8. Female subjects must have a negative serum pregnancy test at Screening. 
9. If the subject is a male with a female sexual partner who is of childbearing potential the 
couple is willing to use two effective methods of birth control during the duration of the 
trial and for one month after the last application of the gel. A female of childbearing 
potential is a sexually mature woman who: 1) has not undergone a hysterectomy or 
bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 
consecutive months (i.e., has had menses at any time in the preceding 24 consecutive 
months, must agree to use two effective methods of contraception for the duration of the 
study and at least 1 month after the last study drug application. The two forms of birth 
control authorized are defined as the use of a barrier method of contraception (condom 
with spermicide) in association with one of the following methods of birth control: 
bilateral tubal ligation; combined oral contraceptives (estrogens and progesterone) or 
implanted or injectable contraceptives with a stable dose for at least 1 month prior to 
Baseline; hormonal intra-uterine device (IUD) inserted at least 1 month prior to Baseline. 

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10. The subject is willing to contact the study center after each primary skin care physician 
(PSCP) visit to provide the study center details of the visit and any treatment of skin 
tumors. 
11. The subject is willing to forego treatment of the treatment targeted baseline SEBs except 
when the Investigator and/or PSCP believes that delay in treatment potentially might 
compromise the health of the subject. 
7.2 
Subject Exclusion Criteria 
Subjects meeting any of the following criteria will be excluded from the study: 
1. The subject is a woman of childbearing potential. This proscription is based on the key 
role of the HH pathway in embryogenesis, the known preclinical teratogenic effects of 
systemic cyclopamine, a naturally occurring inhibitor of SMO, and the unknown level of 
systemic exposure following topical application in humans. A female of childbearing 
potential is a sexually mature woman who: 1) has not undergone a hysterectomy or 
bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 
consecutive months (i.e., has had menses at any time in the preceding 24 consecutive 
months. 
2. The subject has used topical products to the face or within 5 cm of a treatment targeted 
SEB or systemic therapies that might interfere with the evaluation of the study 
medication during the study. Specifically these include the use of: 
a. Topical glucocorticoids 30 days prior to screening 
b. Retinoids (e.g., etretinate, isotretinoin, tazarotene, tretinoin, adapalene) 
systemically or topically or > 5% of an alphahydroxy acid (e.g., glycolic acid, 
lactic acid) or 5-fluorouracil or imiquimod (except as topical treatment to discrete 
BCCs) systemically or topically to the skin during the six months prior to entry. 
c. Systemic chemotherapy within one year prior to screening. (Note: field therapy 
with topically applied treatments can be done as long as they are not applied 
within 5 cm of a treatment targeted tumor). 
d. Known inhibitors of the HH signaling pathway (e.g., vismodegib, patidegib, and 
sonidegib) topically or systemically within 6 months of entry into the study. 
3. The subject has a history of hypersensitivity to any of the ingredients in the study 
medication formulation. 
4. The subject is unable or unwilling to make a good faith effort to return for all follow-up 
visits and tests. 
5. The subject has uncontrolled systemic disease. 

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6. The subject has clinically important history of liver disease, including viral hepatitis, 
current alcohol abuse, or cirrhosis. 
7. The subject has any condition or situation which in the Investigator’s opinion may put the 
subject at significant risk, could confound the study results, or could interfere 
significantly with the subject’s participation in the study. This includes history of other 
skin conditions or diseases, metabolic dysfunction, physical examination findings, or 
clinical laboratory findings giving reasonable suspicion of a disease or condition that 
contraindicates use of an investigational drug or that might affect interpretation of the 
results of the study or render the patient at high risk from treatment complications. 
8. The subject has a history of invasive cancer within the past five years excluding non- 
melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of breast, or 
chronic lymphocytic lymphoma (CLL) (Stage 0). 
9. The subject has current, recent (within 4 weeks of Baseline visit), or planned 
participation in an experimental drug study while enrolled in this study. 
10. Female sexual partner(s) of male subjects are unwilling or unable to comply with 
pregnancy prevention measures. 
7.3 
Subject Withdrawal Criteria 
Participation in the study is strictly voluntary. A subject has the right to withdraw from the 
study at any time for any reason and is under no obligation to disclose the reason. If a subject 
withdraws, the Investigator is to be informed immediately. 
The Investigator has the right to terminate participation of a subject at any time for any of the 
following: 
• Intake of non-permitted concomitant medication 
• Lack of patient compliance 
• Protocol violation - Contact PellePharm or designee before making decision. 
• Disease progression 
• Worsening of any condition - Subject requires alternate treatment before the end 
of the study and the Investigator determines it is not due to lack of efficacy. 
• A perceived safety risk 
• Adverse Event – Complete AE form 
• Lost to Follow-Up – Document with at least 2 phone calls and a certified letter 
• Subject Request – Consent withdrawal, subject moved, schedule conflicts 

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• Pregnancy – Subject will discontinue study drug immediately, but will be 
followed until delivery. Complete pregnancy form 
• Lack of Efficacy – Subject requires alternate treatment after at least 2 weeks of 
study drug treatment and the risk of continuing the subject in the study outweighs 
the benefit as determined by the Investigator. However, if treatment targeted 
SEBs requires treatment because of increase in size these tumors can be treated 
and the subject can remain in the trial. 
• Other – Specify in comments section of electronic case report form (eCRF) 
If a study subject experiences disease progression or begins another treatment for his/her 
disease, follow-up per protocol will no longer be required. 
Subjects who terminate treatment early will be asked to complete all Week 26 assessments 
prior to commencement of any alternative therapy (if possible). Subjects who discontinue 
from the study during the treatment period will not be replaced. 
All subjects are free to withdraw from participating in this study at any time and for whatever 
reason, specified or unspecified, and without prejudice. No constraints will be placed on 
ordinary subject management, and subjects, when appropriate, will be placed on other 
conventional therapy upon request or whenever clinically necessary as determined by their 
physician. 
All premature discontinuations and their reasons must be carefully documented by the 
Investigator on the final eCRF, and, if need be, on the AE form. In any case, no subject who 
has been included and has a study number assigned can be replaced by another if they 
discontinue prematurely for whatever reason. 
If, for any reason, a subject is dropped during the treatment period prior to Week 26, all end 
of treatment study (i.e., Week 26) evaluations should be performed at the time of early 
termination and the reason for termination will be recorded in the end of study source 
documentation. All data gathered on the subject prior to termination will be made available 
to PellePharm. Subjects who discontinue during the post-treatment follow-up period should 
have the assessments performed and reported on the eCRFs for that corresponding visit. 
7.4 
Study Drug Discontinuation 
The Investigator may discontinue study drug administration for any patient at any time. The 
study drug administration must be discontinued for any of the following: 
• Occurrence of an exclusion criterion that is clinically relevant and affects the patient's 
safety, if discontinuation is considered necessary by the Investigator. 

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• Occurrence of AEs, if discontinuation of study drug is desired or considered 
necessary by the Investigator or patient. 
• Pregnancy 
• Disease progression 
 
7.5 
Discontinuation of the Study 
Study discontinuation is at the discretion of PellePharm or the Investigator in any of, but not 
limited to, the following events: 
• Occurrence of unusual AEs in terms of their nature, severity, duration, or unexpected 
incidence. 
• Medical or ethical reasons affecting the continued performance of the study. 
• Difficulties in the recruitment of the patients. 
 
8. TREATMENT PLAN 
8.1 
Methods of Assigning Subjects to Treatment Groups 
This is a double-blinded study, in which the identity of the study drug will be unknown to 
Investigator and subjects, as well as to all individuals closely associated with the study. 
Subjects will be randomized to 1 of the 3 study drug groups in a ratio of 1:1:1 (patidegib gel, 
2%, patidegib gel, 4%, vehicle gel). Each screened subject will be assigned a unique 5-digit 
study subject number assigned by the investigational center, which will consist of the 2 digit 
investigational center number and the 3 digit chronological screening order number, starting 
with 001 (e.g., 01001, 01002). The study drug kit will be assigned to subjects based on a 
randomization code, and kits will be dispensed to the subjects at Baseline in the order that 
they are enrolled by taking the lowest numbered kit available in inventory at the 
investigational site. A study drug accountability log will document the inventory and 
dispensing of study drug at each investigational center. 
8.2 
Randomization and Blinding 
The study drugs will be packaged and labeled identically, and the study drug kits will be 
numbered sequentially and dispensed randomly to the subjects entering the study within each 
investigational center. Study drug supplies will be distributed to the investigational centers to 
maintain the randomization ratio within each investigational center. 
As a double-blinded study, the Investigators, the site staff, PellePharm, and the Clinical 
Monitors will not be aware of the treatment assigned to the individual study subjects. 

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Delegated staff members at each investigational center will dispense the study drugs and will 
collect and weigh all used and unused study drug tubes as scheduled. 
8.3 
Unblinding 
The treatment assignments for all enrolled subjects will be unblinded only after the 
conclusion of the treatment phase of the study. Specifically, the blind will be broken only 
after all data through Week 26 are verified, entered into the database, and validated; subject 
evaluability assessments are performed and entered into the database; and the database is 
locked. 
In the case of a medical emergency, the Investigator can break the blind for the subject 
involved. After the code is broken the Investigator will contact the PellePharm representative 
for unblinding information. The Investigator will record the code break in the subject’s 
source documents. 
8.4 
Prior and Prohibited Concomitant Medication or Therapy 
Subjects must not have used topical products to the face or within 5 cm of a treatment 
targeted SEB or systemic therapies that could interfere with the evaluation of the study 
medication during the study. Specifically, these include use of the following: 
• Topical glucocorticoids 30 days prior to Screening 
• Retinoids (e.g., etretinate, isotretinoin, tazarotene, tretinoin, adapalene) systemically 
or topically or > 5% of an Alphahydroxy acid (e.g., glycolic acid, lactic acid) or 5- 
fluorouracil or imiquimod (except as topical treatment to discrete BCCs) systemically 
or topically to the skin during the six months prior to entry. 
• Treatment with systemic chemotherapy within one year prior to screening. 
• Known inhibitors of the HH signaling pathway (e.g., vismodegib, patidegib, and 
sonidegib) topically or systemically within 6 months of entry into the study. 
Subjects will not be allowed to use topical or systemic therapies that may affect treatment 
targeted SEBs and/or their development of new SEBs. Tumors that the Investigator or PSCP 
determines require treatment during the trial can be removed by the modality selected by 
their PSCP. 
During the study, subjects will be allowed to use moisturizers and emollients and will be 
encouraged to use sunscreen of SPF at least 15 at least once daily on all exposed skin sites. 
Subjects using concomitant therapies during the course of the study that could interfere with 
the interpretation of the study results (including but not limited to those listed above) should 

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not be withdrawn, but the use of the concomitant product should be discontinued. No other 
topical treatment (except as noted above) other than the study drug will be permitted. 
Information on concomitant therapies will be recorded in the source document. Any therapy 
used by the subject will be considered concomitant therapy (e.g., aspirin, 
paracetamol/acetaminophen, vitamins, moisturizers, sunscreens). Every attempt should be 
made to keep concomitant therapy dosing constant during the study. Any change to 
concomitant therapy should be noted on the source document and eCRF. 
8.5 
Treatment Compliance 
Each subject will be instructed on the importance of returning the study drug at each 
applicable study visit. If a subject does not return his or her study drug, he or she will be 
instructed to return it as soon as possible. The subjects will bring the tubes dispensed at each 
treatment visit to the next subsequent study visit. Each tube will be weighed by the Study 
Coordinator or designee prior to dispensation and after collection. The subject will be asked 
to keep a record of missed doses. A subject who deviates significantly from the prescribed 
application amount will be counseled. Any missed applications of study drug will be noted in 
the appropriate source document. Missed applications will be documented in the eCRF. 
The Investigator will record the time and dose of all administrations in the eCRF. Any 
reasons for non-compliance will also be documented including: 
• Missed visits 
• Interruptions in the schedule of administration 
• Non-permitted medications 
 
8.6 
Protocol Deviations and Violations 
The Investigators must read the protocol thoroughly and must follow the instructions exactly. 
Whenever possible, any planned deviations should be agreed to by prior discussion the 
Medical Monitor and the Investigator, with appropriate documentation of Medical Monitor’s 
approval prior to effecting the changes agreed upon. 
A deviation from the protocol is an unintended and/or unanticipated departure from the 
procedures and/or processes approved by PellePharm and the Institutional Review Board 
(IRB) or Independent Ethics Committee (IEC) and agreed to by the Investigator. Deviations 
usually have an impact on individual patients or a small group of patients and do not involve 
eligibility or primary endpoint criteria. 
A protocol violation occurs when there is nonadherence to the protocol that results in a 
significant, additional risk to the patient, when the subject or Investigator has failed to adhere 

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to significant protocol requirements (eligibility criteria) and the patient was enrolled without 
prior PellePharm approval, or when there is nonadherence to MHRA or local authority 
regulations and/or ICH GCP guideline. 
The Investigator or designee must document and explain in the patients’ source 
documentation any deviation from the approved protocol. The Investigator may implement a 
deviation from, or a change of, the protocol to eliminate an immediate hazard to study 
patients without prior IRB/IEC approval. As soon as possible after such an occurrence, the 
implemented deviation or change, the reasons for it, and any proposed protocol 
amendment(s) should be submitted to the IRB/IEC for review and approval, to PellePharm 
for agreement, and to the regulatory authorities, if required. 

 
Protocol Pelle-926-201 
PellePham1, Inc . 
 
 
9. 
S T UDY DRUG MATERIALS AND MANAGEMENT 
 
The study drng will be dispensed by a trained qualified member of the study staff assigned by 
the Investigator to this task 
Patidegib is generated from a plant source by the extraction of crnde cyclopamine. 
Cyclopamine is can ied through a series of synthetic steps to yield the well-characterized 
staii ing material for GMP conversion to patidegib. 
Patidegib HCl active ingredient is found as a white free-flowingc1ystalline solid. Patidegib 
topical gel is a smooth clear viscous hydro alcoholic gel for topical administration. The gel is 
non-in itatingand easy to spread on the lesions as directed. 
The following excipients are used in the fonnulation of patidegib topical gel: All components 
of patidegib gel meet standai·d US or international compendial standai·ds and are generally 
recognized as safe (GRAS). 
Table 3: Excipients of Patidegib Topical Gel 
 
Component 
Monoeraph 
Patidegib 
NI A 
Transcutol P 
USP, NF, PH. Eur. 
Ethanol 
USP,NF,JP, Ph. Eur. 
Proovlene Glycol 
USP, NF, Ph. Eur. 
Purified Water * 
USP, Ph. Eur. 
Boric Acid * 
Ph. Eur. 
SodilllllHydroxide * 
NF, BP, JP, Ph. Eur. 
Phenoxvethanol 
Ph. Eur. 
Hvdroxypropylcellulose HF 
Ph. Eur. 
*Components for pH 7.5 borate buffer 
 
 
Table 4: Specific Compositions of the Vehicle, 2% and 4% Patidegib Gels 
 
Comoonent 
4%* 
2%* 
Placebo 
Patidegib 
4.97 
2.49 
0.00 
TranscutolP 
18.80 
18.80 
18.80 
Ethanol 
23.50 
23.50 
23.50 
Propylene Glycol 
18.80 
18.80 
18.80 
pH 7.5 Borate buffer 
30.93 
33.41 
35.90 
Phenoxvethanol 
1.00 
1.00 
1.00 
HPC-HF** 
1.00 
1.00 
1.00 
TOTAL 
100.00 
100.00 
100.00 
 
 
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*The label strength of patidegib topical gels is on a free base adjusted for HCl, trace 
solvents, moisture etc.; while compounding is on the as-is bases corrected by the potency 
of the particular active pharmaceutical ingredient (API) lot 
**Hydroxypropylcellulose –HF 
 
9.1 
Packaging and Labeling 
Study drug will be packaged in tubes and provided in study drug kits. Each kit will contain 
18 tubes, each containing 15 grams of study material. The subjects will be dispensed study 
medication as needed at Baseline and at all subsequent study visits, through Week 22. The 
tubes will be weighed prior to dispensing. The subject will bring the tubes to the next study 
visit, where they will be collected and weighed; partially used tubes can be re-dispensed 
along with new tubes. If the subject loses a tube (lost or damaged tube), another tube will be 
dispensed. The goal is to ensure that the subject has an adequate supply of gel to able to 
administer all scheduled treatments between clinic visits. Each drug dispensation will be 
documented on the drug accountability log. Labels on the tubes will contain the following 
information: 
• Protocol Number 
• Name, address and telephone number of the sponsor, contract research 
organization or Investigator (the main contact for information on the product, 
clinical trial and emergency unblinding); 
• Batch number 
• Directions for use 
• Period of use (expiry date), in month/day/year format and in a manner that avoids 
any ambiguity. 
• Subject Number 
• Space for entry of the subject initials 
• Space for entry of date dispensed 
• A statement reading, “For external use only. Avoid contact with eyes and lips” 
• A statement reading, “Store at controlled room temperature 59°F to 86°F (15°C to 
30°C). Do not refrigerate” 
• A statement indicating the sponsor, PellePharm Inc. 
• A statement indicating the quantity of product (15 grams) 

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• A statement reading, “for clinical trial use only” 
• A statement reading, “Keep from breastfeeding or childbearing potential women” 
• A statement reading, “Keep out of Reach and Sight of Children” 
 
9.2 
Storage, Handling, and Disposal of Study Drug 
The tubes should be stored under controlled room temperature at 20° – 25° C (68° – 77° F). 
Normal variations in temperatures are expected and acceptable around these target 
temperatures for patient’s storage of dispensed tubes. 
9.3 
Administration 
The investigational center staff member will instruct the subject to apply the study drug to 
the affected treatment areas identified at the Baseline visit by the Investigator. The staff 
member will instruct the subject on the proper use of laminated dosing cards during the 
Baseline visit. In addition to the verbal instructions given during the visit, the subjects will be 
provided with written instructions. 
Subjects will be instructed to apply a thin layer of study drug to the entire selected treatment 
area(s) as indicated on the body diagram twice daily up to the Week 26 visit. Subjects will be 
advised to avoid or minimize exposure to direct sunlight while in the study and to wash their 
hands before and after application of the study drug. 
Subjects will be instructed to store their test article in a secure location away from children. 
Women of child bearing potential should not come in contact with the gel. 
The amount of study drug used by the subjects will be monitored by weighing each newly 
dispensed study drug tube and re-weighing each returned study drug tube at all applicable 
study visits. 
9.4 
Study Drug Accountability 
Upon receipt of the study drug, the Investigator is responsible for ensuring that the 
designated study drug staff will conduct a complete inventory of study materials and assume 
responsibility for their storage and dispensing. The Investigators must agree to keep all study 
materials in a secure location with restricted access. The Investigator will keep a record of 
the inventory and dispensing of all study drugs. This record will be made available to the 
Clinical Monitor for the purpose of accounting for all clinical supplies. Any significant 
discrepancy and/or deficiency must be recorded with an explanation. 
All supplies sent to the investigators will be accounted for and, in no case, used in any 
unauthorized situation. Each tube will be weighed (with the cap on) before dispensing to and 

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upon return by the subjects, and weights will be recorded on the pharmacy log and 
appropriate eCRF. All supplies will be returned to the sponsor for destruction at the 
conclusion of the study. 

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10. STUDY PROCEDURES AND EVALUATIONS 
The study will be conducted as outlined in the Schedule of Assessments (Table 2). 
All subject information and data obtained during the study visits will be recorded in the 
source documents, applicable study logs, and eCRFs. 
Investigators must have appropriate, documented experience and training, or obtain approval 
from PellePharm based on experience (or through additional training organized by 
PellePharm). 
At each study visit, every attempt should be made to ensure that the same investigator 
assesses the same subject. The sponsor realizes that the conduct of a clinical trial involves a 
team of study staff who perform a variety of functions under the supervision of the Principal 
Investigator. Tasks and responsibilities are delegated by the Principal Investigator as dictated 
by local laws and institutional regulation. There are specific tasks that PellePharm requires be 
done by a physician. These include identification of SEBs, identification of the treatment- 
targeted tumor to be biopsied for biomarkrs, measuring and mapping of tumors, 
determination of clinical tumor type, recording application site reactions, and the 
determination of the ISGTA. 
PellePharm also realizes that scheduling visits can be challenging and has allowed for study 
windows. It should be noted that the indicated visit day is in reference to the Baseline visit. 
For example the Week 6 (Day 43) visit is intended to be Day 43 after the Baseline visit. 
10.1 
Schedule of Evaluations and Procedures 
10.1.1 
Screening Visit (Day -42 to Day 0) 
Following written informed consent from each subject, the Investigator will determine 
whether subjects are eligible to participate in the study by performing screening tests and 
evaluations. 
At the Screening visit, continuous monitoring of concomitant medications and therapies 
(including prophylactic treatments and medical interventions) and AEs throughout the study 
period will begin. 
Screen failure information will be maintained at the site to document specified information, 
including but not limited to, reason for failure. 
The following procedures will be conducted at this visit: 
1. Obtain verbal and written informed consent from the subject prior to performing any 
study related procedures. Give a signed copy to the subject. 

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2. Review and explain the nature of the study and what will be expected at each visit to 
ensure subject can meet the requirements and has adequate transportation. 
3. Assign the subject a 5-digit subject number, which will consist of the 2-digit site 
number and the 3-digit chronological screening order number, starting with 001 
(e.g., 01 001, 01 002). 
4. Record the subject’s demographic information. 
5. Record the subject’s medical history. 
6. Record all medications for BCC used during the prior year in the eCRF. Include all 
medications used in the past 30 days and any therapy that requires a washout prior to 
Baseline. 
7. Record any prescription or over-the-counter therapies that are being used 
concomitantly in the eCRF. 
8. Perform a brief physical exam including vital signs and weight. 
9. The Investigator will identify 5 previously untreated non central facial SEBs one of 
which will be acceptable for biopsy for biomarker. The biomarker designated tumor 
will be biopsied with a 2 mm punch biopsy at Baseline and Week 6. 
10. Verify that the subject meets the applicable inclusion/exclusion criteria as outlined in 
Sections 7.1 and 7.2. 
11. Discuss the use of moisturizers and emollients with the subject. 
12. Collect blood samples for routine laboratory analysis [complete blood 
count/differential (CBC/Diff), urinalysis, serum chemistry and serum pregnancy test 
on all female subjects] and plasma drug concentrations. 
13. Record any AEs related to screening procedures on the AE eCRF. 
14. Schedule subject to return for the Baseline/Day 1 visit. If the subject requires a 
washout, schedule the Baseline/Day 1 visit to occur after the washout is complete. 
10.1.2 
Baseline Visit (Day 1) 
The following procedures will be conducted at this visit: 
1. Record any changes in medical history since Screening. 
2. Record changes in any previous BCC medications since the previous visit in eCRF. 
Check for prohibited concomitant therapies and confirm any therapy that requires a 
washout prior to Baseline as per Section 8.4. 
3. Record changes in any concomitant medications since the previous visit in eCRF. 
Check for prior and concomitant therapies as per Section 8.4. 
4. Verify that the subject continues to meet the applicable study eligibility criteria as 
outlined in Sections 7.1 and 7.2. 

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5. An abbreviated physical examination including measurements of height, weight, and 
vital signs (blood pressure, heart rate, respiration rate, and oral temperature). 
6. Have the subject complete the DLQI and aBCCdex questionnaires. 
7. The Investigator will perform the clinical evaluation to identify BCCs including the 5 
baseline treatment-targeted tumors, as well as, all other facial tumors (including those 
on the eyelids and the nose). In addition, up to 10 non treatment-targeted non-facial 
tumors will also be clinically classified as superficial, nodular, infiltrative, morpheic 
or sclerosing, pigmented, or micronodular/morpheaform, circle each tumor in ink, 
photographed, measured, and recorded on a body diagram. The 5 baseline treatment- 
targeted tumors will also be evaluated based on the ISGTA. 
8. Obtain 2 mm punch biopsy from the baseline treatment targeted SEB that has been 
designated for biomarker evaluation. The 2 mm punch biopsies can be performed by 
the Investigator or designee as allowed by the clinical site’s normal policies and 
procedures. 
9. The Investigator or designee will assess each of the areas to be treated by 
observations and questioning the subjects as necessary for the signs and symptoms of 
irritation including pain/burning, pruritus, erythema, edema, and scabbing/crusting. 
Each of the 5 treatment targeted-tumors, as well as the face in general, will be 
evaluated separately. 
10. The Investigator will query the patient about the symptoms of any abnormalities in 
taste, frequency and severity of muscle cramps, recent changes in quality or quantity 
of hair in treated and untreated areas and any change in frequency of shaving of 
treated or untreated areas, any change in frequency of obtaining haircuts. 
11. Randomize the subject to a treatment group and record the assigned kit number in the 
source document and in the eCRF. 
12. The designated study drug staff will weigh each tube within the assigned kit and 
dispense them to the subject. 
13. The Study Coordinator or designee will instruct the subject on the proper application 
procedure for the study drug. For the first application, the subject will apply the study 
drug at the investigational center under the direction of the Study Coordinator or 
designee. The study drug should be applied after all clinical assessments. The 
subjects will be asked to avoid exposure to direct sunlight on the initial application 
day and thereafter. The Study Coordinator or designee will instruct the subjects to 
apply the study drug twice daily at home. 
14. Record any AEs reported spontaneously by the subject. 
15. Schedule the next study visit at Week 2 (Day 15 ± 2 days). 
10.1.3 
Weeks 2, 10, 18, and 22 
The following procedures will be conducted at this visit: 

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1. Record changes in any concomitant medications since the previous visit in the eCRF. 
Check for prior and concomitant therapies as per Section 8.4. 
2. Record any new AEs reported spontaneously by the subject or changes in any 
ongoing AEs. 
3. Record subject weight. 
4. The Investigator will perform the clinical evaluation of the 5 baseline treatment- 
targeted tumors, as well as, all other facial tumors (including those on the eyelids and 
the nose). In addition, up to 10 non treatment-targeted non-facial tumors will be 
circled in ink, photographed, measured, and recorded on a body diagram. The 5 
baseline treatment-targeted tumors will be evaluated based on the ISGTA. 
5. The Investigator or designee will assess each of the treated areas by observations and 
questioning the subjects as necessary for the signs and symptoms of irritation 
including pain/burning, pruritus, erythema, edema, and scabbing/crusting. Each of the 
5 treatment-targeted tumors, as well as the face in general, will be evaluated 
separately. 
6. The Investigator will question the subject about any concurrent illness or systemic 
sign or symptom including but not limited to fatigue, loss or change in taste, nausea, 
vomiting, diarrhea, muscle spasms or cramps, changes in quality or quantity of hair in 
treated areas and any change in frequency of shaving. 
7. The Study Coordinator or designee will collect and weigh the previously dispensed 
study drug tube. The study coordinator or designee will weigh and dispense a new 
study drug tube from the subject assigned kit. 
8. Any missed doses or deviations should be reported. 
9. The Study Coordinator or designee will remind the subject of the proper technique for 
application of the study drug. If needed, the subject can apply the study drug at the 
investigational center during the day under the direction of the Study Coordinator or 
designee to confirm proper technique. 
10. 
Schedule the next study visit. 
10.1.4 
Week 6, 14, and 26 
The following procedures will be conducted at this visit: 
1. Record changes in any concomitant medications since the previous visit in the eCRF. 
Check for prior and concomitant therapies as per Section 8.4. 
2. Record any new AEs reported spontaneously by the subject or changes in any 
ongoing AEs. Record subject weight. 
3. Perform a brief physical exam including vital signs and weight (Weeks 14 and 26 
only). 
4. Collect blood samples for routine laboratory analysis (CBC/Diff and serum 
chemistry) and plasma concentrations. 

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5. Perform a serum pregnancy test for all female subjects. 
6. Have the subject complete the DLQI and aBCCdex questionnaires (Weeks 14 and 
26). 
7. Obtain a biopsy with a 2 mm punch at Week 6 from the single SEB designated as the 
biomarker treatment targeted tumor and previously biopsied at baseline. The 2 mm 
punch biopsies can be performed by the Investigator or designee as allowed by the 
clinical site’s normal policies and procedures. 
8. The Investigator will perform the clinical evaluation of the 5 baseline treatment- 
targeted tumors, as well as, all other facial tumors (including those on the eyelids and 
the nose). In addition, up to 10 non treatment-targeted non-facial tumors will be 
circled in ink, photographed, measured and recorded on a body diagram. The 5 
baseline treatment-targeted tumors will be evaluated based on the ISGTA. 
9. The Investigator or designee will assess each of the treated areas by observations and 
questioning the subjects as necessary for the signs and symptoms of irritation 
including pain/burning, pruritus, erythema, edema, and scabbing/crusting. Each of the 
5 treatment-targeted tumors, as well as the face in general, will be evaluated 
separately. 
10. The Investigator will question the subject about any concurrent illness or systemic 
sign or symptom including but not limited to fatigue, loss or change in taste, nausea, 
vomiting, diarrhea, muscle spasms or cramps, changes in quality or quantity of hair in 
treated areas and any change in frequency of shaving. 
11. The Study Coordinator or designee will collect and weigh the previously dispensed 
study drug tube. The study coordinator or designee will weigh and dispense a new 
study drug tube from the subject assigned kit. 
12. The Study Coordinator will verify study drug compliance. Any missed doses or 
deviations should be reported. 
13. The Study Coordinator or designee will remind the subject of the proper technique for 
application of the study drug. If needed, the subject can apply the study drug at the 
investigational center during the day under the direction of the Study Coordinator or 
designee to confirm proper technique. The study drug should be applied after all 
clinical assessments. 
14. Schedule the next study visit (Weeks 6 and 14 only). 
10.1.5 
Week 26 (Day 183 ± 3 Days) / Early Termination 
The following procedures will be conducted at this visit: 
1. Record changes in any concomitant medications since the previous visit in the eCRF. 
Check for prior and concomitant therapies as per Section 8.4. 
2. Record any new AEs reported spontaneously by the subject or changes in any 
ongoing AEs. Record subject weight. 

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3. Perform a brief physical exam including vital signs and weight. 
4. Collect blood samples for routine laboratory analysis (CBC/Diff and serum 
chemistry) and plasma concentrations. 
5. The Investigator or designee will assess each of the treated areas by observations and 
questioning the subjects as necessary for the signs and symptoms of irritation 
including pain/burning, pruritus, erythema, edema, and scabbing/crusting. Each of the 
5 treatment-targeted tumors, as well as the face in general, will be evaluated 
separately. 
6. The Investigator will perform the clinical evaluation of the 5 baseline treatment- 
targeted tumors, as well as, all other facial tumors (including those on the eyelids and 
the nose). In addition, up to 10 non treatment-targeted non-facial tumors will be 
circled in ink, photographed, measured and recorded on a body diagram. The 5 
baseline treatment-targeted tumors will be evaluated based on the ISGTA. The 
Investigator or designee will assess the treated areas by observations and questioning 
the subjects as necessary for the signs and symptoms of irritation including 
pain/burning, pruritus, erythema, edema, and scabbing/crusting. 
7. The Investigator will question the subject about any concurrent illness or systemic 
sign or symptom including but not limited to fatigue, loss or change in taste, nausea, 
vomiting, diarrhea, muscle spasms or cramps, changes in quality or quantity of hair in 
treated areas and any change in frequency of shaving. 
8. The Study Coordinator or designee will collect and weigh the previously dispensed 
study drug tube. 
9. Any missed doses or deviations should be reported. 
10. Exit the subject from the study and complete the end of study eCRFs. 
10.2 
Evaluation of Tumors 
Clinical Descriptions of Basal Cell Carcinoma 
Nodular basal cell carcinoma 
A pearly, waxy, semi-translucent nodule sometimes forming a central depression that may or 
not be ulcerated or crusted. There can be overlying telangiectasias and characteristically 
there is a “rolled border”. 
Superficial basal cell carcinoma 
An erythematous scaly thin plaque or patch often with areas of hypopigmentation and 
superficial scarring, occasionally with a thin crust. Careful examination will often show a 
subtle raised border. 

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Infiltrative basal cell carcinoma 
An erythematous, frequently ulcerated or crusted ill-defined plaque. Occasionally it can be 
quite indurated or elevated. 
Morpheic or sclerosing basal cell carcinoma 
An ill-defined hypopigmented sclerotic plaque, sometimes with subtle telangiectasias. 
Rarely ulcerated or crusted. They can present with an atrophic or depressed appearance 
centrally often mimicking an old scar. 
Micronodular basal cell carcinoma 
A dome-shaped sclerotic hypopigmented or flesh colored nodule or plaque with undermining 
borders where a mass-like effect is palpable below normal appearing skin. 
Pigmented basal cell carcinoma 
Each of the classic variants described above may be pigmented, particularly in Types III-V 
skin. These will have blue, black, brown pigmentation usually involving part or most of the 
tumor with more classic or characteristic features often noted elsewhere. 
10.2.1 
Assessment of BCCs 
At Baseline and at Weeks 6, 10, 14, 18, 22, and 26, all visible BCCs will be identified by the 
Investigator. The Investigator will circle in ink, photograph, measure the greatest diameter, 
and record the BCCs on a body diagram. 
Treatment targeted SEBs will be identified at Baseline on the face and/or other anatomical 
areas and will be treated during the 26 week treatment phase. The diameter of each SEB will 
be reported on the eCRF. New SEBs on the face will be reported on the eCRF at Weeks 2, 6, 
10, 14, 18, 22, and 26. 
10.2.2 
Messenger RNA (mRNA) 
A single SEB designated as a treatment targeted tumor at Baseline will be biopsied with a 2 
mm punch biopsy at Baseline as well as at the end of Week 6 for determination of GLI1 
mRNA levels. It is preferable that the tumor selected for biopsy has the clinical features of a 
nodular BCC. 
10.2.3 
Investigator Global Static Tumor Assessment 
The Investigator will assess and record each baseline treatment targeted SEBs at Weeks 6, 
10, 14, 18, 22, and 26. 

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Table 5: Investigator Static Global Tumor Assessment 
 
0 
Clear - no evidence of residual tumor is seen or palpated, faint ill-defined macular 
erythema may be present; n01m al skin markings are seen 
1 
Almost clear - residual macular e1ythema is clearly seen but there is no palpable tumor, 
scale, erosions, ulcerations; n01mal skin markings are seen 
2 
Minimal residual tumor - macular e1ythemais clearly seen and slight palpable tumor with 
or without scale, erosions, ulcerations; n01m al skin markings are not clearly visible 
3 
Clearly visible tumor - is seen and felt on palpation with or without rolled borders erosion, 
ulceration, or scale; n01mal skin markings not seen 
 
 
10.2.4 
Subject Questionnaires 
 
Subjects will complete the DLQI and aBCCdex questionnaires at Baseline and Weeks 14 and 
26. The DLQI and aBCCdex questionnaires can be found in Appendix 16.2 and 16.3, 
respectively. 
 
 
 
10.3 
Evaluation of Safety 
 
10.3.1 
Dermal Safety and Tolerability 
 
Safety and tolerability will be evaluated through assessments of selected local signs and 
symptoms (pain / bmnin g, pmritus, e1yt hema, edema, and scabbing / cmsting). Each of the 5 
treatment-targeted tumors, as well as the face in general, will be evaluated separately. Any 
local skin reaction requiring use of a concomitant therapy or is a cause for study diug 
intenu ption or discontinuation should be repo1i ed as an AE. The scales to be used for 
assessing local skin reactions follow: 
Table 6: Dermal Safety and Tolerability Scales 
 
Score Grade 
Description 
Pain/Burning: as reported by the subject as being the greatest intensity they have 
experienced at the application site within the last 24 hours at Baselineor since the last 
visit at subsequent visits. 
0 
None 
No pain/bmning 
1 
Mild 
Slight bmning/stinging sensation; not really bothersome 

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2 
Mode rat e Definite wa1m, bmning/stinging that is somewhat bothersome 
3 
Severe 
Hot bmning/stinging sensation that causes definite discomfo1i and 
may intenupt daily activities and/or sleep 
Pruritus: as reported by the subject as being the greatest intensity they have experienced 
at the application site within the last 24 hours at Baseline or since the last visit at 
subsequent visits. 
0 
None 
No prnritus 
1 
Mild 
Slight prnritus, not really bothersome 
2 
Moderate Definite prnritus that is somewhat bothersome 
3 
Severe 
Intense prnritus that may intenu pt daily activities and/or sleep 
E1ythema: as assessed by the Investigator at each site 
0 
None 
No e1yt hema present 
1 
Mild 
Slight pink coloration 
2 
Moderate Definite redness 
3 
Severe 
Marked e1yht ema, bright red to dusky dark red in color 
Edema: as assessed by the Investigatorat each site 
0 
None 
No edema 
1 
Mild 
Slight, but definite edema 
2 
Moderate Definite edema 
3 
Severe 
Marked edema 
Scabbing/Crusting:as assessed by the Investigator at each site 
0 
None 
No scabbing/crnsting 
1 
Mild 
Slight, but definite scabbing/crnsting 
2 
Moderate Definite scabbing/crnsting 
3 
Severe 
Marked scabbing/crnsting 
 
 
10.3.2 
Medical History and Abbreviated Physical Examination 
 
A medical histo1ywill be taken at Screening, and confnmed and revised if needed, at 
Baseline. Medical conditions that resolved 2 or more years before baseline need not be 
collected unless considered relevant by the Investigator. 

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An abbreviated physical examination including measurements of height, weight and vital 
signs (blood pressure, heart rate, respiration rate, and oral temperature) will be performed at 
Baseline, Week 14, and Week 26. Height will be measured at Baseline only. 
10.3.3 
Safety Laboratory Tests 
Routine safety laboratory tests as per Appendix 16.4 will be performed at Screening, Week 6, 
Week 14, and Week 26. Any out-of-range laboratory result that is considered clinically 
significant by the Investigator will be recorded as an AE and should be accessed for 
reproducibility by repeat testing at the discretion of the Investigator. Clinically significant 
laboratory abnormalities at any visit will be followed to resolution (return to normal or to the 
Baseline state) or until clinically stable as determined by the Investigator. 
10.3.4 
Pregnancy Tests 
All female subjects will have a pregnancy test performed at specified visits during the study. 
A serum pregnancy test will be performed at Screening and Weeks 6, 14, and 26. 
10.3.5 
Plasma Concentrations 
A blood sample will be collected at Screening, Weeks 6, 14, and 26 to assess patidegib levels 
in plasma. 
10.3.6 
Adverse Events 
10.3.6.1 
Definition of Adverse Event 
An AE is any untoward medical occurrence in a patient or clinical investigation subject 
administered a medicinal product and which does not necessarily have a causal relationship 
with the study drug. AEs include any unfavorable and unintended illness, sign (e.g., 
including an abnormal laboratory finding), symptom, clinically significant laboratory test 
abnormality, or disease temporally associated with the use of a medicinal product that has 
appeared or worsened during the course of the clinical trial, regardless of causal relationship 
to the study drug(s) under study. 
10.3.6.2 
Documenting Adverse Experiences 
It is the responsibility of the Investigator to document all AEs that occur during the course of 
the study. The AEs should be documented as a single medical diagnosis. When this is not 
possible, the AE should be documented in terms of signs and/or symptoms observed by the 
Investigator or reported by the subject at each study visit. 

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All AEs occurring after the subject signs the informed consent through the last study visit 
must be reported, regardless of whether or not the AEs are considered drug-related. All AEs, 
whether in response to a query, observed by the study site personnel, or reported 
spontaneously by the subject, will be recorded. 
At each visit during the study, the subject will be assessed for the occurrence of new and 
ongoing AEs. Dermal safety and tolerability that result in the subject’s requiring a 
concomitant therapy or discontinuation from the study will be reported as an AE. The 
following data will be collected on all AEs and recorded on the appropriate CRF: 
• Event name (diagnosis preferred, if unknown, record the signs/symptoms) 
• Onset date and end date 
• Maximum intensity (severity) 
• Seriousness 
• Action taken regarding study drug 
• Corrective treatment, if given 
• Outcome 
In addition, the Investigator’s assessment of causality will be recorded. 
Vital sign abnormalities are to be recorded as AEs only if they are clinically significant (for 
example: are symptomatic, requiring corrective treatment, leading to discontinuation or 
fulfilling a seriousness criterion). 
Subjects will be questioned about any concurrent illness or systemic sign or symptom 
including but not limited to fatigue, loss or change in taste, nausea, vomiting, alopecia, 
diarrhea, and muscle spasms. Any incidence of these systemic signs or symptoms will be 
reported on the AE eCRF. 
10.3.6.3 
Serious Adverse Events 
All AEs will be assessed as either serious or nonserious. 
An SAE or serious adverse reaction is defined as any untoward medical occurrence that at 
any dose: 
• Results in death 
• Is immediately life threatening, (the term "life threatening" in the definition of 
"serious" refers to an event in which the subject is at risk of death at the time of 

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the event; it does not refer to an event which hypothetically might have caused 
death if it were more severe) 
• Requires in patient hospitalization or prolongation of existing hospitalization 
(hospitalization for elective surgery for a baseline condition is not considered an 
AE) 
• Results in persistent or significant disability/incapacity (permanent or substantial 
disruption of a person’s ability to conduct normal life functions) 
• Is a congenital anomaly/birth defect 
• Is a medically important event that may not be immediately life threatening or 
result in death or hospitalization, but may jeopardize the subject and may require 
medical or surgical intervention to prevent 1 of the above listed outcomes 
Note: A spontaneous abortion will be considered an SAE, and must be reported per 
Reporting of SAEs under Section 10.3.6.6. 
10.3.6.4 
Assessment of Severity 
The severity assigned to an AE should be determined by the maximum severity of the AE. 
The categories described below should be used to estimate the severity of AEs: 
• Mild: Transient or mild discomfort; no limitation in activity; no medical 
intervention/therapy required 
• Moderate: Mild to moderate limitation in activity; some assistance may be 
needed; no or minimal medical intervention/therapy required 
• Severe: Marked limitation in activity; some assistance usually required; medical 
intervention/therapy required; hospitalization or prolongation of current 
hospitalization possible; may be incapacitating or life threatening 
10.3.6.5 
Assessment of Causality 
The Investigator should assess the relationship of the AE, if any, to the study drug. The 
following should be taken into account when assessing SAE causality: 
• Positive temporal relationship to study drug, such as if the study drug was 
withdrawn and the SAE resolved or the event recurred after re-introduction. 
• If there is a reasonable possibility that the AE is associated with an underlying or 
concomitant illness. 
• Possible association with previous or concomitant therapy. 

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• No temporal relationship to the study drug and/or a more likely alternative 
etiology exists. 
• If the AE is directly related to study procedures or a lack of efficacy. 
The following classifications should be used when evaluating the relationship of AEs and 
SAEs to the investigational drug. 
1. None: No relationship between the experience and the administration of study drug; 
related to other etiologies such as concomitant medications or patient’s clinical state. 
2. Unlikely: The current state of knowledge indicates that a relationship is unlikely. 
3. Possibly: A reaction that follows a plausible temporal sequence from administration of 
the study drug and follows a known response pattern to the suspected study drug. The 
reaction might have been produced by the patient’s clinical state or other modes of 
therapy administered to the patient. 
4. Probably: A reaction that follows a plausible temporal sequence from administration of 
the study drug and follows a known response pattern to the suspected study drug. The 
reaction cannot be reasonably explained by the known characteristics of the patient’s 
clinical state or other modes of therapy administered to the patient. 
5. Definitely: A reaction that follows a plausible temporal sequence from administration of 
the study drug and follows a known response pattern to the suspected study drug and can 
be confirmed with a positive re-challenge test or supporting laboratory data 
10.3.6.6 
Reporting of Serious Adverse Events 
When new significant information is obtained as well as when the outcome of an event is 
known, the Investigator should record the information on a new SAE form. If the subject 
was hospitalized, a copy of the discharge summary must be included as part of the subject’s 
medical file. In all instances, the Investigator should follow up with subjects until the 
outcome of the SAE is known. 
All AEs spontaneously reported by the subject and/or in response to an open question from 
study personnel or revealed by observation, physical examination or other diagnostic 
procedures will be recorded on the AE eCRF. Any clinically relevant change in laboratory 
assessments or other clinical findings is considered an AE and must be recorded on the AE 
eCRF. All AEs are to be followed until the event resolves or the clinical course is stabilized. 
For SAEs, a SAE form must be completed with as much information as possible and 
submitted in the time frame described below. 
PellePharm must be notified of all SAEs (regardless of casual relationship to study drug) 
within 24 hours of first knowledge of the event by the Investigator or other study personnel 
by faxing a completed SAE form to the contact information below. 

 
ProtocloPelle-92-6201 
PellePham1, Inc. 
 
 
Safety Reporting Contact 
Name: 
Premier Research Phan nacovigilance 
Facsimile: 
+42 1268203713 
Email 
GlobalPV-Slovakia@12r e mi e r - r ese arch.com 
 
 
If there are serious, unexpected AEs associated with the use of the study diug, PellePhaim 
will notify the appropriate regulato1yagency(ies) and all appropriate pa1iies on an expedited 
basis. It is the responsibility of the Investigator to promptly notify the IRB/IEC of all 
unexpected SAEs involving risk to human patients. 
The Investigator should take all appropriate measures to ensure the safety of the subjects, 
notably and should follow a subject with an SAE until the event has resolved or the condition 
has stabilized. This may imply that follow-up will continue after the subject has left the 
study, and that additional investigations may be requested by PellePhaim. 
 
10.3.6.7 
Emergency Contact 
 
In the event of a medical emergency (i.e., an event that requires immediate attention 
regai·ding the treatm ent of a patient, operat ion of the clinical study , and/or the use of 
investigational di11g), investigational site personnel must immediately contact the Medical 
Monitor. 
 
Medical Monitor Contact 
Name: 
I 
rPD 
T e lephon 
e : 
rPD
I 
F a cs imil e : 
rPD
I 
E m a il 
rPD
I 
 
 
10.3.6.8 
Expedited Serious Adverse Event Reports 
 
An AE, whether serious or nonserious, is designated unexpected (unlabeled) if it is not 
repo1i ed in the clinical safety section of the Investigator Brochure or if the event is of greater 
frequency, specificity or severity. 
Expedited SAE reports ai·e those that document AEs that ai·e both unexpected based on the 
reference document (Investigator Brochure) and ai·e related (i.e ., the relations hip cannot be 
rnled out) to the study diug. These expedited repo1is ai·e s ubject to repo 1i ing timelines of 7 
 
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(SAEs) and/or 15 (non SAE) calendar days to the regulatory reporting agency(ies). 
PellePharm will notify regulatory authorities of these AEs and all participating 
investigational centers in writing for submission by the Investigator to the IRB/IEC. This 
notification will be in the form of a Safety Update to the Investigator Brochure (i.e., “15-day 
letter”). 
Upon receiving such notices, the Investigator must review and retain the notice with the 
Investigator Brochure and immediately submit a copy of this information to the responsible 
IRB/IEC according to local regulations. The Investigator and IRB/IEC will determine if the 
informed consent requires revision. The Investigator should also comply with the IRB/IEC 
procedures for reporting any other safety information. 
10.3.6.9 
Other Required Safety Assessments 
A clinically significant worsening from Baseline of any abnormal study assessment, such as 
laboratory test, physical examination, or vital signs, should be considered an AE and 
recorded accordingly. If possible, a diagnosis for the clinically significant study assessment 
should be provided by the Investigator (e.g., urinary tract infection or anemia). In the 
absence of a diagnosis, the abnormal study assessment itself should be listed as the AE (e.g., 
bacteria in urine or decreased hemoglobin). 
An abnormal study assessment is considered clinically significant if the patient has one or 
more of the following related to the abnormal study assessment: 
1. 
Concomitant clinical signs or symptoms 
2. 
Further diagnostic testing or medical/surgical intervention 
3. 
A change in the dose of study drug or is discontinued from the study 
Repeat testing to determine whether the result is abnormal, in the absence of any of the above 
criteria, does not necessarily meet clinically significant criteria. 

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11. STATISTICS 
All statistical processing will be performed using Statistical Analysis System (SAS®) unless 
otherwise stated. If determined appropriate by the Sponsor, limited efficacy analysis on 
tumor shrinkage and biomarkers may be performed after the last subject completes 14 weeks 
of treatment. Except where noted, all statistical tests will be two-sided and will be performed 
at the 0.05 level of significance. 
Descriptive statistics will be used to provide an overview of the efficacy and safety results. 
For categorical parameters, the number and percentage of subjects in each category will be 
presented. For continuous parameters, descriptive statistics will include n (number of 
subjects), mean, and standard deviation, median, minimum, and maximum. Appropriate 
inferential statistics will be used for the primary and secondary efficacy variables. 
The last observation carried forward method (LOCF) will be used to impute missing efficacy 
data (i.e., the last available on-therapy observation for a subject will be used to estimate 
subsequent missing data points). No imputations will be made for missing safety data. 
No adjustments will be made for multiplicity. 
If determined appropriate by the Sponsor, limited efficacy analysis on tumor shrinkage and 
biomarkers may be performed after the last subject completes 14 weeks of treatment. 
Shortly after the last subject completes 14 weeks of treatment an independent data safety 
management board (DSMB) will evaluate all safety data as detailed in the DSMB charter. 
A statistical analysis plan (SAP), describing all statistical analyses will be provided as a 
separate document. The SAP will be finalized prior to unblinding of the study treatments. 
11.1 
Analysis Populations 
Efficacy analyses will be performed using the intent-to-treat (ITT) population. Safety 
analyses will be performed using the safety population. 
All subjects who are randomized and dispensed study drug will be included in the ITT 
analysis set. 
All subjects who are randomized, receive at least 1 confirmed dose of study drug and have at 
least one post-baseline safety assessment will be included in the safety analysis set. 
11.2 
Subject Disposition 
A tabulation of subject disposition will be provided. The tabulation will include the numbers 
of subjects who enter the study, complete the study, and discontinue the study. The reasons 
for discontinuation will be included. 

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11.3 
Demographics and Baseline Characteristics 
Subject demographic data and baseline characteristics will be summarized by treatment 
group using descriptive statistics for the ITT and Safety populations. 
11.4 
Protocol Deviations 
All protocol deviations will be reported to the PellePharm and recorded throughout the study. 
A tabulation of protocol deviations will be included in the final study report. 
11.5 
Compliance 
No formal evaluations of compliance are planned. 
 
11.6 
Interim Analyses 
If determined appropriate by the Sponsor, limited efficacy analysis on tumor shrinkage and 
biomarkers may be performed after the last subject completes 14 weeks of treatment. 
11.7 
Assessment of Efficacy 
11.7.1 
Efficacy Endpoints 
The efficacy endpoints are intended to compare twice daily application of patidegib gel, 2%, 
patidegib gel, 4%, and vehicle gel. Efficacy assessments will be summarized descriptively by 
treatment group and visit. 
11.7.1.1 
Primary Efficacy Endpoints: 
The primary efficacy endpoints include: 
• Decrease in SEB size defined as percent decrease in the sum of the greatest diameter 
of baseline treatment targeted SEBs at the Week 26 visit. 
• Change in GLI1 mRNA levels in drug-treated versus placebo-treated tumors after 6 
weeks of treatment. 
11.7.1.2 
Secondary Efficacy Endpoints: 
The secondary efficacy endpoints include: 
• Change in tumor size defined as percent decrease in the sum of the greatest diameter 
of baseline treatment targeted SEBs at Weeks 6, 10, 14, 18, and 22. 

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• Change in tumor size defined as percent change in greatest diameter from Baseline of 
tumors located on the nose or periorbital area at Weeks 6, 10, 14, 18, 22 and 26. 
• Number of new SEBs on the face at Weeks 6, 10, 14, 18, 22, and 26. 
• Proportion of facial BCCs that at Baseline and/or Week 2 were less than 5 mm in 
greatest diameter but by Weeks 6, 10, 14, 18, 22, and 26 visit have become greater 
than 5 mm in greatest diameter. 
• Proportion of baseline treatment targeted SEBs on the face that become less than 5 
mm in greatest diameter and non-facial baseline treatment targeted SEBs that become 
less than 9 mm in greatest diameter at Weeks 6, 10, 14, 18, 22, and 26. 
• The reduction in the HH signaling pathway after treatment with patidegib gel 2% or 
4% or vehicle applied twice daily for 6 weeks to treatment-targeted SEBs. 
11.7.1.3 
Exploratory Efficacy Endpoints: 
• The proportion of baseline treatment targeted SEBs that are evaluated as being clear 
or almost clear at Weeks 6, 10, 14, 18, 22 and 26 based on the ISGTA. 
• DLQI and aBCCdex scores at Baseline and Weeks 14 and 26 as well as change from 
Baseline at Weeks 14 and 26. 
11.7.2 
Efficacy Analyses 
The sum of the greatest diameter of all treatment targeted SEBs for Baseline and post- 
baseline visits will be calculated. 
The primary endpoint and the secondary endpoints of change in tumor size, reduction in the 
HH signaling pathway, and the exploratory endpoint of change in GLI1 mRNA levels will be 
evaluated with an analysis of covariance (ANCOVA) with factors of treatment group and 
baseline value as a covariate. For the change in tumor size analyses, the covariate will be the 
sum of the baseline diameters. Pairwise comparisons will be performed using contrasts 
within the ANCOVA. 
The number of new SEBs on the treated areas of the face will be analyzed with a Poisson 
Regression with factors of treatment group and the number of surgically eligible tumors at 
Baseline. Pairwise comparisons will be performed. 
The exploratory analysis of ISGTA will be analyzed with a Cochran-Mantel-Haenszel test. 

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DLQI and aBCCdex scores will be summarized with descriptive statistics at Baseline and 
Weeks 14 and 26. Change from baseline will also be summarized at Weeks 14 and 26. DLQI 
responses of “not relevant” will be excluded from the change from baseline summaries. 
11.8 
Assessment of Safety 
11.8.1 
Dermal Safety and Tolerability 
The frequency of dermal safety and tolerability assessments including pain/burning, pruritus, 
erythema, edema, and scabbing/crusting will be summarized descriptively by treatment group 
and visit. 
11.8.2 
Adverse Events 
Subjects will be assessed for the occurrence of new and ongoing AEs. Descriptions of AEs 
will include the dates of onset and resolution (if resolved), maximum severity, seriousness, 
action taken regarding the study drug, corrective treatment, outcome, and Investigator’s 
assessment of causality. All AEs will be recorded and classified using terminology from the 
Medical Dictionary for Regulatory Activities (MedDRA). All reported treatment-emergent 
AEs (TEAEs), defined as any AE with an onset on or after the date of first study drug 
application, will be summarized by treatment group, the number of subjects reporting 
TEAEs, system organ class, preferred term, severity, and relationship to study drug. When 
summarizing TEAEs by severity or relationship to study drug, each subject will be counted 
only once within a system organ class or a preferred term using the event with the greatest 
severity or causality, respectively, within each category. All reported SAEs will be 
summarized by treatment group, the number of subjects reporting SAEs, system organ class, 
preferred term, severity, and relationship to study drug. 
All information pertaining to AEs noted during the study will be listed by subject and will 
include a verbatim description of the event as reported by the Investigator, as well as the 
preferred term, system organ class, start date, stop date (if stopped), seriousness, severity, 
action taken regarding the study drug, corrective treatment, outcome and relationship to the 
study drug. In addition, a listing of subjects who prematurely discontinue from the study due 
to AEs will be provided as well as a listing of subjects who reported an SAE. 
11.8.3 
Plasma Concentrations 
Plasma concentrations of patidegib and any major metabolites will be summarized 
descriptively at Screening and Weeks 6, 14, and 26 by treatment group. 

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11.8.4 
Safety Laboratory Values and Vital Sign Measurements 
Changes from Baseline in safety laboratory values and vital sign measurements will be 
summarized with descriptive statistics for each treatment group at all applicable study visits. 
Shift tables will be presented for changes in safety laboratory values to summarize laboratory 
test results collected at Baseline and Weeks 6, 14, and 26. Normal ranges established by the 
central laboratory will be used to determine the shifts. A listing of all out-of-range laboratory 
test results at any assessment time point will also be provided. Determination of clinical 
significance for all out-of-range laboratory values will be made by each Investigator and 
included in the listing. In addition, a listing of all clinically significant laboratory test results 
will be provided. 
11.8.5 
Pregnancy Tests 
Pregnancy test results will presented in a data listing. 
 
11.8.6 
Handling of Missing Data 
The last observation carried forward (LOCF) method will be used to impute missing efficacy 
data (i.e., the last available on-therapy observation for a subject will be used to estimate 
subsequent missing data points). No imputations will be made for missing safety data. 
11.8.7 
Multicenter Issues 
The study will be conducted at multiple investigational centers in the United Kingdom with 
the intention of pooling the results for analysis. 
11.8.8 
Multiplicity Issues 
Since this is a Phase 2 study, no adjustments have been made for multiplicity. 
 
11.9 
Sample Size Determination 
Data from a trial of oral vismodegib1 given for 7 months resulted in a percent change in the 
sum of the greatest diameter of baseline treatment targeted SEBs of 69.2 and 13.7 with a 
standard deviation of 17 and 23 for treated and control subjects, respectively. A sample size 
of 6 subjects enrolled in each treatment group with a minimum of 5 SEBs will have greater 
than 95 percent power to detect a statistically significant difference at an alpha of 0.05. 

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12. QUALITY CONTROL AND QUALITY ASSURANCE 
12.1 
Study Monitoring 
An Investigator Meeting or an initiation visit will be conducted with the Principal 
Investigator and Study Coordinators by PellePharm and/or its designee. During this meeting, 
an extensive review and discussion of the protocol, the role of the study technician, all study 
procedures, source documents, and eCRFs will be conducted. Evaluation procedures will be 
reviewed extensively and documentation of training will be recorded for training of sponsor- 
approved evaluators. 
The study monitors/clinical research associates will be trained prior to study initiation. 
Following this training, an overview of the study disease and study material background will 
be understood. Specific monitoring guidelines and procedures to be followed during 
monitoring visits will also be utilized. During the course of the study, all data will be 100% 
source document verified by the monitors. All subject source records must be made available 
to the monitors. 
The conduct of the study will be closely monitored by the sponsor following GCP guidelines. 
The reports of these verifications will also be archived with the study report. In addition, 
inspections or on site audits may be carried out by local authorities or by the sponsor's 
Quality Assurance Department. The Investigators will allow the sponsor's representatives and 
any regulatory agency to examine all study records, corresponding subject medical records, 
clinical dispensing records and storage area, and any other documents considered source 
documentation. The Investigators agree to assist the representative, if required. 
12.2 
Audits and Inspections 
The study will be conducted under the sponsorship of PellePharm in conformation with all 
appropriate legal regulations, as well as ICH guidelines. Interim and end of study audits of 
raw data, study files, and final report may be conducted by PellePharm’s Quality Assurance 
Department or designee. 
The sponsor is responsible for implementing and maintaining quality assurance and quality 
control systems to ensure that studies are conducted and data are generated, documented, and 
reported in compliance with the protocol, GCP, and applicable regulatory requirements. In 
addition, the sponsor will be responsible for securing agreement from all involved parties to 
ensure direct access to all study related investigational centers, source data/documents, 
eCRFs, and reports for the purpose of monitoring and auditing by the sponsor, and inspection 
by domestic and foreign regulatory authorities. 

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12.3 
Data Quality Assurance 
All assessments performed will be accurately documented in the subject’s source documents 
and eCRFs. The Investigator or designee will enter the information required by the protocol 
into the source documents and eCRFs provided by the sponsor or designee. Subjects will be 
identified in the eCRFs by their assigned subject number and initials only. 
The Investigators must read the protocol thoroughly and must follow the instructions exactly. 
Any deviations should be agreed to by prior discussion between the sponsor and the 
Investigator, with appropriate written protocol amendments made prior to implementing the 
agreed changes. Any amendment containing major modifications (particularly if it may 
involve an increased risk to the subjects) will be approved by the IRB before it may be 
implemented. No change in the conduct of the study can be instituted without written 
approval from the sponsor. 

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13. ETHICS AND ADMINISTRATIVE ISSUES 
13.1 
Ethical Conduct of the Study 
This study will be conducted in accordance with the ethical principles originating from the 
Declaration of Helsinki, ICH guidelines, GCP, and in compliance with local regulatory 
requirements. The Investigator agrees, when signing the protocol, to adhere to the 
instructions and procedures described in it and thereby to adhere to the principles of GCP. 
13.2 
Ethics Review 
This protocol, proposed informed consent form and other information to subjects, and all 
appropriate amendments will be properly reviewed and approved by the IRB/IEC. A signed 
and dated notification of the IRB/IEC approval will be provided to the sponsor and 
Investigator prior to study initiation. The name and occupation of the chairman and members 
of the IRB/IEC will be supplied to the sponsor. The Investigator will provide required 
progress reports and report all SAEs to the IRB/IEC as required by the IRB/IEC. 
13.3 
Written Informed Consent 
Written informed consent is required from each subject prior to any testing under this 
protocol, including screening tests and evaluations. The informed consent form (ICF), as 
specified by the investigational site’s IRB/IEC, must follow the Protection of Human 
Subjects regulations listed in 21 CFR Part 50. 
The background of the proposed study and the benefits and risks of the procedures and study 
must be explained to the subjects. It is the responsibility of the Investigator to obtain consent 
and to provide the subject with a copy of the signed and dated ICF. Confirmation of a 
subject’s informed consent must also be documented in the subjects’ medical record prior to 
any testing under this protocol, including screening tests and evaluations. 
All ICFs used in this study must be approved by the appropriate IRB/IEC and by PellePharm 
or designee. The ICF must not be altered without the prior agreement of the relevant 
IRB/IEC and PellePharm. 
13.4 
Subject Data Protection 
Subject data will be protected by ensuring that no captured data contain subject names, 
addresses, telephone numbers, email addresses, or other direct personally identifying 
information. It is acknowledged that subject initials, demographics (including birthdate), 
medical histories, and prior concomitant medication uses, along with the name and address of 
the enrolling investigator may allow for personal identification of study participants. Other 

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than where necessary to meet regulatory requirements, all data collected in this study will be 
presented in tabulated (i.e., aggregate) form and listings containing information that could be 
used to identify an individual subject will not be included in any public disclosures of the 
study data or the study results. 
13.5 
Data Safety Monitoring Committee 
The Data Safety Monitoring Committee (DSMB) will meet after subjects complete 10 weeks 
of treatment, and on an as needed basis, to review and evaluate the ongoing study safety data. 
Details for the Data Safety Monitoring Committee can be found in the DSMB charter. 
 
13.6 
Financial Disclosure 
Financial disclosures will be obtained from all investigators in order to document any 
potential conflicts of interest. 
13.7 
Investigator Obligations 
The Investigator agrees, when signing the protocol, to adhere to the instructions and 
procedures described in it and thereby to adhere to the principles of Good Clinical Practice 
(GCP). 
13.8 
Changes to the Protocol 
The Investigators must read the protocol thoroughly and must follow the instructions exactly. 
Whenever possible, any planned deviations should be agreed to by prior discussion between 
the sponsor and the Investigator, with appropriate documentation of sponsor approval prior to 
effecting the changes agreed upon. Any amendment to the protocol containing major 
modifications (particularly if it may involve an increased risk to the subjects) will be 
approved by the IRB before it may be implemented. No change in the conduct of the study 
can be instituted without written approval from the sponsor. 
13.9 
Confidentiality/Publication of the Study 
All the data furnished to the Investigator and his/her staff and all data obtained through this 
protocol will be regarded as confidential and proprietary in nature and will not be disclosed 
to any third party, except for the MHRA or other regulatory body, without written consent 
from the sponsor. 

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14. DATA HANDLING AND RECORD KEEPING 
14.1 
Inspection of Records 
Investigators must maintain detailed records on all study subjects who are enrolled in the 
study or undergo screening. Data will be recorded in the subject’s source documents and in 
applicable study logs provided by the sponsor. Source documents include subject medical 
records, hospital charts, clinic charts, investigator subject study files, as well as the results of 
diagnostic tests (e.g., laboratory tests). All required data should be recorded in the study 
documentation completely for prompt data review. Upon study completion or at any other 
time specified by the sponsor or designee, the appropriate study documents must be 
submitted. 
The Investigator must keep accurate separate records (source documentation) of all subject 
visits, being sure to include all pertinent study related information. At a minimum, this 
includes the following information: 
• A statement indicating that the subject has been enrolled in the study and the 
subject number 
• Date that informed consent was obtained 
• Evidence that the subject meets study eligibility requirements (e.g., medical 
history, screening evaluations) 
• Dates of all study related visits and results of any evaluations/procedures 
performed, including who performed each assessment at each visit 
• Use of any concurrent medications during the study 
• Documentation of study drug accountability 
• Any and all side effects and AEs must be thoroughly documented to conclusion 
• Results of any diagnostic tests conducted during the study 
• The date the subject exited the study and a statement indicating that the subject 
completed the study or was discontinued early, including the reason for 
discontinuation 
Notes describing telephone conversations and all electronic mail with the subject or the 
sponsor (sponsor’s designee) concerning the study must be recorded or kept on file. All 
source documents must be made available to the sponsor and the sponsor’s designated 
monitor upon request. 

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14.2 
Retention of Records 
The Investigator should properly store and maintain all study records in accordance with 
sponsor directives. All records relating to the conduct of this study are to be retained by the 
Investigator until notified by the sponsor in writing that the records may be destroyed. 
The Investigator will allow representatives of the sponsor’s monitoring team, the governing 
IRB/IEC, the MHRA, or other applicable local authorities to inspect all study records, CRFs, 
and corresponding portions of the subject’s clinic and/or hospital medical records at regular 
intervals throughout the study. These inspections are for the purpose of verifying adherence 
to the protocol, completeness and accuracy of the data being entered onto the CRF, and 
compliance with MHRA or other local authority regulations. 
14.3 
Electronic Case Report Form Completion 
eCRFs will be completed for each enrolled patient. It is the Investigator’s responsibility to 
ensure the accuracy, completeness, and timeliness of the data reported in the subject’s eCRF. 
Source documentation supporting the eCRF data should indicate the subject’s participation in 
the study and should document the dates and details of study procedures, AEs, and patient 
status. 
Investigators will maintain copies of the eCRFs at the clinical site. For subjects who 
discontinue or terminate from the study, the eCRFs will be completed as much as possible, 
and the reason for the discontinuance or termination clearly and concisely specified on the 
appropriate eCRF. 

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15. REFERENCES 
Tang, J.T., et al. Inhibiting the hedgehog pathway in patients with the basal-cell nevus syndrome. 
2012. N. Engl. J. Med. 366(23):2180-2188. 

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16. APPENDICES 
16.1 
Body Diagram 
 
 
 
Appendix: Photographs of tumors and sequence of regional photos 
1. Identify the 5 treatment targeted SEBs with labels 
2. Measure greatest diameter of each tumor, perform ISTA and determine clinical type. 
3. Identify all other tumors measure and record their greatest diameter and the clinical 
type of each tumor. 
4. Circle all tumors both treatment targeted tumors and other tumors: 
6 quadrants on face/neck: 
1. R forehead 
2. R cheek 
3. R neck 
4. L forehead 
5. L cheek 
6. L neck 

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20 quadrants on body: 
1. R chest 
2. L chest 
3. R abdomen 
4. L abdomen 
5. R upper arm 
6. R lower arm 
7. L upper arm 
8. L lower arm 
9. R anterior thigh 
10. L anterior thigh 
11. R anterior leg 
12. L anterior leg 
13. R upper back 
14. L upper back 
15. R lower back 
16. L lower back 
17. R posterior thigh 
18. L posterior thigh 
19. R posterior leg 
20. L posterior leg 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
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16.2 
Subject Questionnaire- DLQI 
 
 
 
Hosp ital No: 
N am e: 
Add r ess : 
DERMATOLOGY LI FE QUALIT Y INDE X 
 
Date: 
 
Dia gnos is : 
 
 
 
Score: 
 
DLQI 
D 
 
 
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Pelle -926 -201 , VS , 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 75 of81 
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Protocol Pelle-926-201 
PellePharm, Inc. 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 76 of 81 
 
 
 
16.3 
Subject Questionnaire – aBCCdex 
General Instructions: 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
© Genentech Inc 2013 
PPD 

Protocol Pelle-926-201 
PellePharm, Inc. 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 77 of 81 
 
 
 
PPD 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
© Genentech Inc 2013 

Protocol Pelle-926-201 
PellePharm, Inc. 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 78 of 81 
 
 
 
 
 
PPD 

Protocol Pelle-926-201 
PellePharm, Inc. 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 79 of 81 
 
 
 
 
 
 
 
PPD 
© Genentech Inc 2013 

Protocol Pelle-926-201 
PellePharm, Inc. 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 80 of 81 
 
 
 
 
 
PPD 

Protocol Pelle-926-201 
PellePharm, Inc. 
Pelle-926-201, V5, 18AUG2016 
PROPRIETARY AND CONFIDENTIAL 
Page 81 of 81 
 
 
 
16.4 
Safety Laboratory Tests 
The following blood samples for laboratory tests will be collected: 
• Alanine aminotransferase (ALT) 
• Albumin, alkaline phosphatase 
• Aspartate aminotransferase (AST) 
• Total bilirubin 
• Blood urea nitrogen (BUN) 
• Calcium, carbon dioxide, chloride 
• Creatinine 
• Glucose 
• Potassium 
• Protein 
• Sodium 
• White blood cell (WBC) 
• Red blood cell (RBC) 
• Hemoglobin 
• Hematocrit 
• Mean Corpuscular Volume (MCV) 
• Mean corpuscular hemoglobin (MCH) 
• Mean corpuscular hemoglobin concentration (MCHC) 
• Red Cell Distribution Width 
• Platelets, Mean Platelet Volumes 
• Absolute/Percent Neutrophil Count 
• Absolute/Percent Lymphocyte count 
• Absolute/Percent Monocyte count 
• Absolute/Percent Eosinophil Count 
• Absolute/Percent Basophil Count 
Urinalysis with reflex microscopic examination.