A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese Participants With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Phase: PHASE3
Conditions: Symptomatic Transthyretin Amyloid Cardiomyopathy
Sponsor: Alexion Pharmaceuticals, Inc.

Protocol Amendment 5.0 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 1 of 68 
Alexion Confidential 
TITLE PAGE 
Protocol Title: A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the 
Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese 
Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM). 
Protocol Number: ALXN2060-TAC-302 
Amendment Number: 5.0 
Compound: ALXN2060  
Study Phase: 3 
Short Title: A Phase 3 Study of ALXN2060 in Japanese Participants with Symptomatic 
ATTR-CM 
Sponsor Name: Alexion Pharmaceuticals, GK 
Legal Registered Address: Tamachi Station Tower N 3-1-1, Shibaura, Minato-ku, Tokyo 
108-0023, Japan
Regulatory Agency Identifier Number(s): NA
Approval Date: 11 Sep 2023
Sponsor Signatory:
Date 
Contact Personnel for this Protocol is based in Japan 
14-Sep-2023
NCT #: NCT04622046

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 2 of 68 
Alexion Confidential 
INVESTIGATOR’S AGREEMENT 
I have read the study protocol and agree to conduct the study in accordance with this 
protocol, all applicable government regulations, the principles of the ICH E6 Guidelines for 
Good Clinical Practice, and the principles of the World Medical Association Declaration of 
Helsinki. I also agree to maintain the confidentiality of all information received or developed 
in connection with this protocol. 
 
 
 
 
____________________________________ 
Printed Name of Investigator 
 
_____________________________________ 
Signature of Investigator 
 
_______________ 
Date

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 3 of 68 
Alexion Confidential 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE 
Amendment 5.0 (11-September-2023) 
Overall Rationale for the Amendment: 
The main purpose of this amendment is to incorporate the administrative changes from 
Administrative Letter 1.0 (13 Jul 2023) and clarify the definitions of terms. 
Other changes implemented through this Amendment constitute minor editorial corrections, 
fixing inconsistencies, and clarification. 
Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis, 4.1 Overall 
Design 
 
Deleted text stricken and additional text 
underlined: 
Following the completion of Month 30 
assessments in Part B, participants will be 
offered the opportunity to continue into 
the Extension Period which will last until 
ALXN2060 is approved or for up to 3024 
additional months or until ALXN2060 
can be provided via an Alexion post-
study access program (as allowed by local 
laws and regulations), whichever occurs 
first.  
To ensure treatment 
continuity for patients.  
1.1 Synopsis 
Approximately 32 months from 
Screening to Month 30 visit, and up to 
approximately 6056 months for 
participants who will continue treatment 
with ALXN2060 in the Extension Period. 
To align with the updated 
definition of the 
Extension Period. 
1.3 Schedule of Activities 
Table header and footnote d: Extended 
the duration from Month 24 to Month 30.  
To align with the updated 
definition of the 
Extension Period. 
6.7 Intervention After the 
End of the Study 
Eligible Participants will be offered the 
opportunity to continue receiving 
ALXN2060 800 mg bid. in the Extension 
Period which will last until ALXN2060 is 
approved in Japan or for up to 3024 
additional months or until ALXN2060 
can be provided via an Alexion post-
study access program (as allowed by local 
laws and regulations), whichever occurs 
first. 
To ensure treatment 
continuity for patients.  
8.2.2 Mortality and 
Cardiovascular-Related 
Hospitalization 
Hospitalizations that are submitted for 
adjudication and are deemed as not CV-
related by the CEC will be considered 
SAEs. Adverse events of special interest 
(AESIs, Section 8.4.5) are also 
considered as part of the efficacy 
endpoint of CV-related hospitalizations.  
To reflect Administrative 
Letter 1.0. 
To clarify the description 
in the CEC Charter as 
shown in Section 9.6 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 4 of 68 
Alexion Confidential 
Section # and Name 
Description of Change 
Brief Rationale 
8.6 Pharmacokinetics,  
8.7 Pharmacodynamics 
• Month 1 and every 63 months during 
the post Extension Period: Predose 
Correction.  
To align with Schedule of 
Activities. 
Throughout the document 
Minor editorial corrections and 
clarifications. 
For correction and/or 
clarification. 
 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 5 of 68 
Alexion Confidential 
TABLE OF CONTENTS 
TITLE PAGE ................................................................................................................ 1 
INVESTIGATOR’S AGREEMENT ............................................................................ 2 
PROTOCOL AMENDMENT SUMMARY OF CHANGES TABLE ......................... 3 
TABLE OF CONTENTS .............................................................................................. 5 
1. 
PROTOCOL SUMMARY ......................................................................... 9 
1.1. 
Synopsis ..................................................................................................... 9 
1.2. 
Schema ..................................................................................................... 13 
1.3. 
Schedule of Activities (SoA) ................................................................... 14 
2. 
INTRODUCTION ................................................................................... 17 
2.1. 
Study Rationale ........................................................................................ 18 
2.2. 
Background .............................................................................................. 18 
2.3. 
Benefit/Risk Assessment ......................................................................... 18 
2.3.1. 
Risk Assessment ...................................................................................... 18 
2.3.1.1. 
Coronavirus Disease 2019 ....................................................................... 19 
2.3.2. 
Benefit Assessment .................................................................................. 19 
2.3.3. 
Overall Benefit: Risk Conclusion ............................................................ 19 
3. 
OBJECTIVES AND ENDPOINTS ......................................................... 20 
4. 
STUDY DESIGN .................................................................................... 23 
4.1. 
Overall Design ......................................................................................... 23 
4.2. 
Scientific Rationale for Study Design ..................................................... 23 
4.2.1. 
Participant Input Into Design ................................................................... 23 
4.3. 
Justification for Dose ............................................................................... 23 
4.4. 
End of Study Definition ........................................................................... 24 
5. 
STUDY POPULATION .......................................................................... 25 
5.1. 
Inclusion Criteria ..................................................................................... 25 
5.2. 
Exclusion Criteria .................................................................................... 26 
5.3. 
Lifestyle Considerations .......................................................................... 27 
5.4. 
Screen Failures ......................................................................................... 27 
6. 
STUDY INTERVENTION ..................................................................... 28 
6.1. 
Study Intervention(s) Administered ........................................................ 28 
6.2. 
Preparation/Handling/Storage/Accountability ......................................... 28 
6.3. 
Measures to Minimize Bias: Randomization and Blinding ..................... 29 
6.4. 
Study Intervention Compliance ............................................................... 29 
6.5. 
Concomitant Therapy .............................................................................. 29 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 6 of 68 
Alexion Confidential 
6.5.1. 
Allowed Medicine and Therapy .............................................................. 29 
6.5.2. 
Disallowed Medicine and Therapy .......................................................... 29 
6.6. 
Dose Modification ................................................................................... 30 
6.7. 
Intervention After the End of the Study .................................................. 30 
7. 
DISCONTINUATION OF STUDY INTERVENTION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL .................... 31 
7.1. 
Discontinuation of Study Intervention ..................................................... 31 
7.1.1. 
Temporary Discontinuation ..................................................................... 31 
7.2. 
Participant Discontinuation/Withdrawal from the Study ........................ 31 
7.3. 
Lost to Follow up ..................................................................................... 32 
8. 
STUDY ASSESSMENTS AND PROCEDURES ................................... 33 
8.1. 
General Procedures .................................................................................. 33 
8.2. 
Efficacy Assessments .............................................................................. 33 
8.2.1. 
Six-Minute Walk Test (6MWT) .............................................................. 33 
8.2.2. 
Mortality and Cardiovascular-Related Hospitalization ........................... 34 
8.2.3. 
Kansas City Cardiomyopathy Questionnaire (KCCQ) ............................ 34 
8.2.4. 
EuroQoL-5 Dimensions (EQ-5D-5L) ...................................................... 34 
8.2.5. 
NT-proBNP and Troponin I ..................................................................... 35 
8.2.6. 
Cardiac MRI ............................................................................................ 35 
8.3. 
Safety Assessments .................................................................................. 35 
8.3.1. 
NewYork Health Assessment .................................................................. 35 
8.3.2. 
Physical Examinations ............................................................................. 35 
8.3.3. 
Vital Signs ............................................................................................... 36 
8.3.4. 
Electrocardiograms .................................................................................. 36 
8.3.5. 
Clinical Safety Laboratory Assessments ................................................. 36 
8.3.6. 
Pregnancy ................................................................................................ 37 
8.3.7. 
Prior and Concomitant Medications ........................................................ 37 
8.4. 
Adverse Events and Serious Adverse Events .......................................... 38 
8.4.1. 
Time Period and Frequency for Collecting AE and SAE Information .... 38 
8.4.2. 
Method of Detecting AEs and SAEs ....................................................... 38 
8.4.3. 
Follow-up of AEs and SAEs .................................................................... 38 
8.4.4. 
Regulatory Reporting Requirements for SAEs ........................................ 38 
8.4.5. 
Adverse Events of Special Interest .......................................................... 39 
8.5. 
Treatment of Overdose ............................................................................ 39 
8.6. 
Pharmacokinetics ..................................................................................... 39 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 7 of 68 
Alexion Confidential 
8.7. 
Pharmacodynamics .................................................................................. 40 
8.8. 
Exploratory Biomarkers ........................................................................... 40 
8.9. 
Immunogenicity Assessments ................................................................. 40 
8.10. 
Health Economics Data and Medical Resource Utilization .................... 40 
9. 
STATISTICAL CONSIDERATIONS .................................................... 41 
9.1. 
Statistical Hypotheses .............................................................................. 41 
9.2. 
Sample Size Determination ..................................................................... 41 
9.3. 
Populations for Analyses ......................................................................... 41 
9.4. 
Statistical Analyses .................................................................................. 42 
9.4.1. 
Efficacy Analyses .................................................................................... 42 
9.4.1.1. 
Analyses of Primary Efficacy Endpoint .................................................. 42 
9.4.1.2. 
Analyses of Secondary Efficacy Endpoint(s) .......................................... 42 
9.4.1.3. 
Pharmacokinetic/Pharmacodynamic Analysis ......................................... 42 
9.4.1.4. 
Analyses of Exploratory Endpoint(s) ...................................................... 42 
9.4.2. 
Safety Analyse(s) ..................................................................................... 43 
9.5. 
Interim Analyses ...................................................................................... 43 
9.6. 
Clinical Events Committee ...................................................................... 43 
10. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS ............................................................................... 44 
10.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight Considerations . 44 
10.1.1. 
Regulatory and Ethical Considerations ................................................... 44 
10.1.2. 
Financial Disclosure ................................................................................ 44 
10.1.3. 
Informed Consent Process ....................................................................... 44 
10.1.4. 
Data Protection ........................................................................................ 45 
10.1.5. 
Committees Structure .............................................................................. 45 
10.1.6. 
Dissemination of Clinical Study Data ..................................................... 46 
10.1.7. 
Data Quality Assurance ........................................................................... 46 
10.1.8. 
Source Documents ................................................................................... 46 
10.1.9. 
Study and Site Start and Closure ............................................................. 47 
10.1.10. 
Publication Policy .................................................................................... 47 
10.2. 
Appendix 2: Clinical Laboratory Tests .................................................... 49 
10.3. 
Appendix 3: Adverse Events: Definitions and Procedures for Recording, 
Evaluating, Follow-up, and Reporting ..................................................... 50 
10.3.1. 
Definition of AE ...................................................................................... 50 
10.3.2. 
Definition of SAE .................................................................................... 51 
10.3.3. 
Recording and Follow-Up of AE and/or SAE ......................................... 52 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 8 of 68 
Alexion Confidential 
10.3.4. 
Reporting of SAEs ................................................................................... 54 
10.4. 
Appendix 4: Contraceptive Guidance and Collection of Pregnancy 
Information .............................................................................................. 55 
10.4.1. 
Definitions ............................................................................................... 55 
10.4.2. 
Contraception Guidance: ......................................................................... 55 
10.4.2.1. 
Guidance for Female Participants ............................................................ 55 
10.4.2.2. 
Guidance for Male Participants ............................................................... 57 
10.4.3. 
Collection of Pregnancy Information ...................................................... 58 
10.4.3.1. 
Male Participants with Partners Who Become Pregnant ......................... 58 
10.4.3.2. 
Female Participants who Become Pregnant ............................................ 59 
10.5. 
Appendix 5: COVID-19 Risk Assessment .............................................. 60 
10.6. 
Appendix 6: Abbreviations ...................................................................... 62 
10.7. 
Appendix 7: Protocol Amendment History ............................................. 63 
11. 
REFERENCES ........................................................................................ 68 
 
LIST OF TABLES 
Table 1: 
Study Intervention, Dosage, and Mode of Administration ...................... 28 
Table 2: 
NYHA Class Assessment ........................................................................ 35 
Table 3: 
Protocol-Required Clinical Laboratory Assessments .............................. 49 
Table 4: 
Potential Risks and Mitigation Measures due to COVID-19 .................. 60 
 
LIST OF FIGURES 
Figure 1: 
Study Design ............................................................................................ 13 
 
 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 9 of 68 
Alexion Confidential 
1. 
PROTOCOL SUMMARY 
1.1. 
Synopsis 
Protocol Title: A Phase 3, Prospective, Multicenter, Open Label, 2-Part Study of the 
Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN2060 in Japanese 
Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM). 
Short Title: A Phase 3 Study of ALXN2060 in Japanese Participants with Symptomatic 
ATTR-CM 
Rationale: This prospective study is designed to evaluate the efficacy, safety, and 
tolerability, as well as to establish the pharmacokinetic (PK) and pharmacodynamic (PD) 
profile of ALXN2060 (previously AG10) in Japanese participants with symptomatic 
transthyretin amyloid cardiomyopathy (ATTR-CM), administered on a background of stable 
heart failure therapy. A multicenter, open label, 2-part design is considered to be the most 
appropriate study design for meeting this objective. Based on information gained from 
previous clinical experience with ALXN2060, a twice daily (bid) dose of 800 mg has been 
selected for this study to represent the optimal combination of potential efficacy, safety, and 
tolerability. 
Objectives and Endpoints 
Objective 
Endpoints 
Primary 
Part A:  
To determine the efficacy of orally administered 
ALXN2060 800 mg bid in the treatment of adult 
Japanese participants with symptomatic ATTR-CM 
as assessed by the 6MWT 
Part A:  
Change from Baseline to Month 12 of treatment in 
distance walked during the 6MWT  
Part B:  
To determine the efficacy of orally administered 
ALXN2060 800 mg bid in the treatment of adult 
Japanese participants with symptomatic ATTR-CM 
as assessed by all-cause mortality and 
cardiovascular-related hospitalization 
Part B: 
All-cause mortality and cardiovascular-related 
hospitalization over a 30-month period 
Secondary 
Part A:  
• 
To evaluate the effects of ALXN2060 800 mg 
bid on the 6MWT 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on health-related quality of life as 
measured by KCCQ-OS in adult Japanese 
participants with symptomatic ATTR-CM 
• 
To assess the safety and tolerability of 
ALXN2060 800 mg bid in adult Japanese 
participants with symptomatic ATTR-CM  
• 
To assess the PD effects of ALXN2060 by 
circulating serum TTR concentration as an in 
vivo biomarker of stabilization and using 
established ex vivo assays of TTR 
Part A:  
• 
Change from Baseline to Months 6 and 9 of 
treatment in distance walked during the 
6MWT  
• 
Change from Baseline to Months 6, 9, and 12 
of treatment in KCCQ-OS 
• 
Safety parameters to be assessed: treatment-
emergent SAEs and AEs, adverse events 
leading to treatment discontinuation, 
abnormal physical examination findings of 
clinical relevance, abnormal vital signs of 
clinical relevance, abnormal ECG parameters 
of clinical relevance, and changes in clinical 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 10 of 68 
Alexion Confidential 
Objective 
Endpoints 
stabilization (FPE); in addition, assess the 
correlation between TTR concentration/FPE 
and ALXN2060 concentration 
safety laboratory parameters of potential 
clinical concern 
• 
Change from Baseline in serum TTR 
concentration (an in vivo measure of TTR 
stabilization) and established ex vivo assays 
of TTR stabilization (FPE) at Days 14 and 28 
and subsequent visits  
Part B: 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on the 6MWT 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on health-related quality of life as 
measured by KCCQ-OS in adult Japanese 
participants with symptomatic ATTR-CM  
• 
To assess the safety and tolerability of 
ALXN2060 800 mg bid administered for 30 
months to adult Japanese participants with 
symptomatic ATTR CM 
• 
To assess the PD effects of ALXN2060 as 
assessed by circulating serum TTR 
concentration as an in vivo biomarker of 
stabilization and established ex vivo assays of 
TTR stabilization (FPE) and correlation 
between TTR concentration/FPE and 
ALXN2060 concentration 
 
Part B: 
• 
Change from Baseline to Months 18, 24, and 
30 of treatment in distance walked during the 
6MWT.  
• 
Change from Baseline to Months 18, 24, and 
30 of treatment in KCCQ-OS  
• 
Safety parameters: treatment emergent SAEs 
and AEs, adverse events leading to treatment 
discontinuation, abnormal physical 
examination findings of clinical relevance, 
abnormal vital signs of clinical relevance, 
abnormal ECG parameters of clinical 
relevance, and changes in clinical safety 
laboratory parameters of potential clinical 
concern 
• 
Change from Baseline in serum TTR 
concentration and established ex vivo assays 
of TTR stabilization (FPE) 
 
Exploratory  
• 
To determine the efficacy of orally 
administered ALXN2060 800 mg bid in the 
treatment of adult Japanese participants with 
symptomatic ATTR CM as assessed by all-
cause mortality and cardiovascular-related 
hospitalization (only Part A) 
• 
To explore the population PK of ALXN2060 
and its metabolites under the 800 mg orally 
administered bid dosing regimen 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on circulating biomarkers of myocardial 
wall stress and microvascular ischemia in 
Japanese participants with symptomatic 
ATTR-CM 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on EQ-5D-5L in Japanese participants 
with symptomatic ATTR-CM 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on cardiac MRI-derived measures of 
cardiac structure, function and tissue 
• 
All-cause mortality and cardiovascular-
related hospitalization over a 12-month 
period (only Part A) 
• 
Exploration of PK over 30 months for 
ALXN2060 and its metabolite  
• 
Changes from Baseline over Month 30 in NT-
proBNP and TnI  
• 
Change from Baseline in EQ-5D-5L over 
Month 30 
• 
Change from Baseline to Month 12, 24, and 
30 in cardiac MRI parameters (only at 
selected sites and consenting patients) 
Extension Period 
• 
Safety parameters same as the Intervention 
Period (Part A and Part B) are assessed in the 
Extension Period 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 11 of 68 
Alexion Confidential 
Objective 
Endpoints 
characterization, in Japanese participants with 
symptomatic ATTR-CM (only at selected 
sites and consenting patients) 
Extension Period 
• 
To assess the safety of ALXN2060 in 
Japanese participants with symptomatic 
ATTR-CM during the Extension Period 
• 
To evaluate the long-term efficacy of 
ALXN2060 in Japanese participants with 
symptomatic ATTR-CM during the 
Extension Period 
• 
To assess the PD effects of ALXN2060 
during the Extension Period  
• 
To characterize PK of ALXN2060 in 
Japanese participants with symptomatic 
ATTR-CM during the Extension Period 
• 
Efficacy endpoints same as the Intervention 
Period excluding cardiac MRI parameters are 
evaluated during the Extension Period 
• 
PD parameters (serum TTR concentration and 
TTR stabilization (FPE)) same as the 
Intervention Period are assessed in the 
Extension Period 
• 
Exploration of PK in the Extension Period for 
ALXN2060 and its metabolite 
Abbreviations: 6MWT = six-minute walk test; AE = adverse event; ATTR-CM = transthyretin amyloid cardiomyopathy; 
ECG = electrocardiography; EQ-5D-5L = European Quality of Life Health 5-item questionnaire dimensions 5 level 
questionnaire; FPE = fluorescent probe exclusion; KCCQ-OS = Kansas City Cardiomyopathy Questionnaire Overall Score; 
MRI = magnetic resonance imaging; NT-proBNP = N-terminal pro-brain-type natriuretic peptide; PD = pharmacodynamic; 
PK = pharmacokinetic; SAE = serious adverse event; TnI = troponin I; TTR = transthyretin. 
Overall Design 
This Phase 3, prospective, multicenter, open label, 2-part study will evaluate the efficacy, 
safety, tolerability, PK, and PD of ALXN2060 800 mg bid administered orally in Japanese 
participants with symptomatic ATTR-CM. 
Eligible Japanese participants with symptomatic ATTR-CM (either mutant or wild type) will 
be enrolled based on inclusion/exclusion criteria. In order to assess the efficacy of 
ALXN2060 800 mg bid as monotherapy, patients with ATTR-CM will be eligible to be 
enrolled as long as they were untreated with tafamidis for at least 14 days prior to the first 
dose of ALXN2060. In case new ATTR-CM therapies will become available during the 
course of the study, Alexion’s Medical Monitor in consultation and agreement with the 
Investigator will determine the pre-enrollment period needed to establish a proper TTR 
baseline. At the moment, the best assumptions would be a requirement of last dose 
administration of 90 days for patisiran and 180 days for inotersen prior to the first day of 
ALXN2060 dosing. 
A Screening Period of up to 35 days will confirm the participants’ diagnosis of ATTR-CM 
and suitability for a long-term investigation. This period will also cover the requirement of 
last dose of tafamidis administered at least 14 days prior to the first ALXN2060 dose. 
Following enrollment and screening, participants will receive ALXN2060 800 mg bid for 
12 months (Part A). Following the last visit (Month 12) of Part A, participants will continue 
the study in Part B, which will last for 18 months (30 months from Day 1). All participants 
will continue to receive oral treatment with ALXN2060 800 mg bid. 
Following the completion of Month 30 assessments in Part B, participants will be offered the 
opportunity to continue into the Extension Period for up to 30 additional months or until 
ALXN2060 can be provided via an Alexion post-study access program (as allowed by local 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 12 of 68 
Alexion Confidential 
laws and regulations), whichever occurs first. Participants will continue to receive oral 
treatment with ALXN2060 800 mg bid. 
The Study schema is presented in Section 1.2. 
Disclosure Statement: This is single-arm, open-label study with ALXN2060 800 mg bid. 
Number of Participants: Approximately 22 Japanese participants will be enrolled in this 
study. 
Intervention Groups and Duration: Approximately 32 months from Screening to Month 30 
visit, and up to approximately 60 months for participants who will continue treatment with 
ALXN2060 in the Extension Period.  
Data Monitoring Committee: No 

Protocol Amendment 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 13 of 68 
Alexion Confidential 
1.2. 
Schema 
 
Figure 1: 
Study Design 
 
 
Abbreviations: 6MWT = six-minute walk test; 30-D FU = 30-day follow up; CV = cardiovascular; EOST = end of study 
treatment. 
 
ALXN2060 
EOST 

Protocol Version 5.0 
 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 14 of 68 
Alexion Confidential 
1.3. 
Schedule of Activities (SoA) 
Procedure 
Screening  
(Up to 35 
Days Before 
Day 1) 
Intervention Period Day (D) and Month (M) 
Extension Period 
EDa/ 
EOST Follow-up 
(30 Days 
After Last 
Dose) 
(± 7 Days) 
Notes 
D1 
D14 
(± 3 
Days) 
D28 
(± 3 
Days) 
Monthly 
Phone 
Contact 
(± 7 Days) 
M3-27 
(Every 3 
Months) 
(± 7 Days) 
M12 
(± 7 
Days) 
M30b 
(± 7 
Days) 
M1 
(± 7 
Days) 
Quarterly 
Phone 
Contactc 
(± 7 days) 
M6-30 
(Every 6 
Months)d 
(± 7 Days) 
 
 
Informed consent 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
Inclusion and exclusion 
criteria reviewe 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
see Section 5.1 
and 5.2 
Patient registration (to 
IWRS) 
X 
X 
 
 
 
 
 
 
 
 
 
 
 
 
Medical/surgical 
history 
X 
 
 
 
 
 
 
 
 
 
 
 
 
 
NYHA class 
assessment 
X 
X 
 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
see Section 8.3.1 
Echocardiogramf 
X 
 
 
 
 
 
 
 
 
 
 
 
 
see Section 8.1  
Physical examinationg 
X 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
See Section 8.3.2 
Vital signs 
X 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
see Section 8.3.3  
Electrocardiograms 
X 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
see Section 8.3.4  
Laboratory assessments 
X 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
see Section 8.3.5  
PD blood samplingh 
 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
 
see Section 8.7  
PK blood samplingi 
 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
 
see Section 8.6  
6MWTj 
X 
 
 
 
X 
X 
X 
  
 
X 
X 
 
see Section 8.2.1 
KCCQk 
 
X 
 
 
 
X 
X 
X 
X 
 
X 
X 
 
see Section 8.2.3  
EQ-5D-5Lk 
 
X 
 
 
 
X 
X 
X 
X 
 
X 
X 
 
see Section 8.2.4   
Cardiac MRIl 
 
X 
 
 
 
X 
X 
X 
 
 
 
 
 
see Section 8.2.6 
Pregnancy testm 
X 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
X 
see Section 8.3.6  
Dispense/collect study 
drugn 
 
X 
X 
X 
 
X 
X 
X 
X 
 
X 
X 
 
 

Protocol Version 5.0 
 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 15 of 68 
Alexion Confidential 
Procedure 
Screening  
(Up to 35 
Days Before 
Day 1) 
Intervention Period Day (D) and Month (M) 
Extension Period 
EDa/ 
EOST Follow-up 
(30 Days 
After Last 
Dose) 
(± 7 Days) 
Notes 
D1 
D14 
(± 3 
Days) 
D28 
(± 3 
Days) 
Monthly 
Phone 
Contact 
(± 7 Days) 
M3-27 
(Every 3 
Months) 
(± 7 Days) 
M12 
(± 7 
Days) 
M30b 
(± 7 
Days) 
M1 
(± 7 
Days) 
Quarterly 
Phone 
Contactc 
(± 7 days) 
M6-30 
(Every 6 
Months)d 
(± 7 Days) 
 
 
Study drug compliance 
assessment 
 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
 
see Section 6.4 
Exploratory Biomarkers 
X 
 
 
 
 
 
 
 
 
 
 
X 
 
See Section 8.8  
Prior/concomitant 
drug/therapy 
 
Monitored continuously 
see Section 8.3.7  
Adverse eventso/Vital 
statusp 
 
Monitored continuously 
see Section 8.4  
a Participants who discontinue treatment with study drug before the end of scheduled treatment will be followed for 30 days post last dose 
b Day 1 visit of the Extension Period will occur on the same date as the Month 30 visit of the Intervention Period.  
c Telephone contact will occur quarterly during months without scheduled in-clinic visits (e.g., Months 3, 9, 15, 21) 
d In-clinic visits will occur at Month 1, Month 6, and every 6 months until Month 30. 
e Recheck clinical status before first dose of the study drug.  
f Resting transthoracic echocardiogram will be performed at Screening and read locally, if LV wall (interventricular septum or LV posterior wall) thickness is not documented in 
medical history based on echocardiogram or CMR. 
g Full physical examination with body weight measurement is performed at all visits. Height will be measured only at screening. 
h PD markers include TTR stabilization by FPE and serum TTR level (predose). PD samples will be collected at predose and 1-hour postdose at Day 28; predose at all other visits. 
Definition of the window of sample collection pre and 1 hour postdose (± x min): Predose = within 30 minutes; 1 hour postdose = ± 15 minutes. (Note that the exact time of 
sample collection will have to be recorded). PD backup samples may be used to measure exploratory biomarkers. 
i PK samples will be collected at predose and 1-hour postdose at Day 28; predose at all other visits. Definition of the window of sample collection pre and 1 hour postdose (± x 
min): Predose = within 30 minutes; 1 hour postdose = ± 15 minutes. (Note that the exact time of sample collection will have to be recorded). PK backup samples may be used to 
measure exploratory biomarkers. 
j Two baseline 6MWTs will be conducted > 24 hours to ≤ 3 weeks apart and prior to Day 1. 6MWT will be obtained on M6, M9, M12, M18, M24, and M30, and every 6 months 
during the Extension Period. 
k KCCQ and EQ-5D-5L will be obtained on Day 1, M6, M9, M12, M18, M24, M30, and Month 1 and every 6 months during the Extension Period 
l Cardiac MRI will be performed on Day 1, M12, M24 and M30. However, cardiac MRI will be conducted only at selected sites and consenting patients. 
m A pregnancy test (urinalysis β-HCG) must be performed on all women of child bearing potential at the specified time points and verified to have a negative result. A pregnancy 
test may also be performed at any visit at the Investigator’s discretion. 
n Study drug is not administered at the EOS if transitioning to commercially available drug. Day 1 is for dispense only and ED/EOST is for collection only. 
o If an AE occurs, the event will be followed up until it has stabilized or the participant has returned to the state they were in before administration of the study drug or the 
laboratory results return to baseline or normalize. All AEs must be managed and SAEs must be reported according to Section 8.4. 
p For a subject who discontinues IMP and study assessments, follow the subject for up to Month 30 by completing monthly contact for vital status (dead, alive, heart transplant and 
receiving CMAD) or until withdrawal of consent. 
Abbreviations: 6MWT = six-minute walk test; AE = adverse event; β-HCG = beta human chorionic gonadotropin; CMAD = Cardiac mechanical assist device; CMR = cardiac 
magnetic resonance; D = day; ED = early discontinuation; EOST = end of study treatment; EQ-5D-5L = EuroQol Health Outcomes Assessment tool; FPE = fluorescent probe 

Protocol Version 5.0 
 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 16 of 68 
Alexion Confidential 
exclusion; IWRS = Interactive Web Response System; KCCQ = Kansas City Cardiomyopathy Questionnaire; LV = left ventricular; M = month; MRI = magnetic resonance 
imaging; NYHA = New York Heart Association; PD = pharmacodynamic; PK = pharmacokinetic; SAE = serious adverse events; TTR = transthyretin.  
 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 17 of 68 
Alexion Confidential 
2. 
INTRODUCTION 
AG10 is a potent and selective stabilizer of transthyretin (TTR) that is being developed by Eidos 
Therapeutics, Inc (a subsidiary of BridgeBio Pharma Inc) for the treatment of TTR amyloidosis 
(ATTR), a progressive, fatal disease in which deposition of amyloid derived from either mutant 
or wild-type TTR causes severe organ damage and dysfunction (Rapezzi, 2010). Alexion 
received an exclusive license to develop and investigate AG10 (hereafter referred to as 
ALXN2060) in Japan from Eidos. 
Clinically, ATTR presents as either a cardiomyopathy (transthyretin amyloid cardiomyopathy 
[ATTR-CM]), an infiltrative, restrictive cardiomyopathy characterized by progressive left and 
right heart failure, or as a peripheral polyneuropathy (ATTR-PN), a length-dependent 
neurodegenerative disease affecting sensorimotor and autonomic functions (Rapezzi, 2010). 
Familial ATTR-CM (ATTRm-CM), or FAC, and familial ATTR-PN or FAP, are driven by 
pathogenic point mutations in the TTR gene; over 140 such mutations have been described 
(Rowczenio, 2019). In addition, older individuals may develop ATTR derived from wild-type 
TTR (ATTRwt, formerly called senile systemic amyloidosis or SSA). In ATTRwt, the major 
organ involved is the heart, although carpal tunnel syndrome and tendon involvement is also 
common (Ruberg, 2019). 
Destabilization, misfolding, and aggregation of TTR lead to deposition of TTR amyloid and 
tissue damage (Rapezzi, 2010). 
Previous studies reported that both in Japan and in the US, 12% to 25% of autopsied hearts from 
octogenarians showed TTR amyloid deposits (Cornwell, 1983), (Ueda, 2011). More recent 
autopsy reports suggest that the prevalence of TTR amyloid deposits in the myocardium of 
patients with heart failure and preserved ejection fraction increases significantly above 
the age of 80 (Mirzoyev, 2010). However, the prevalence of the overt manifestations of the 
disease is much lower and the nationwide survey on ATTRwt-CM conducted in 4,629 Japanese 
sites from 2012 to 2014 identified only 51 patients (Sekijima, 2019). This gap may be attributed 
to difficulty in diagnosis, but also highlights the existence of unknown factors that contribute to 
the disease (Rapezzi, 2010). 
The estimation of the number of ATTRm patients is about 700 based on a nationwide survey that 
identified 208 cases with a definite diagnosis, where the early onset variant of Val30Met (V30M) 
accounts for most cases (77%) and shows a predominantly neuropathic phenotype (Ando, 2016). 
This is consistent with the fact that Japan is among those countries in which ATTRm associated 
with V30M TTR variant is endemic. In line with this, the Transthyretin Amyloidosis Outcome 
Survey, a global, multi-center, longitudinal, observational survey of patients with both ATTRm 
and ATTRwt, enrolled 119 Japanese patients between 2007 and 2017, 112 of whom had ATTRm 
(Sekijima, 2019). Among symptomatic patients (n = 100), 90% were V30M carriers, the most 
common phenotype in symptomatic ATTRm subjects was neurologic (61 of 100; 61.0%), only 
10% of symptomatic patients had an exclusive cardiac phenotype, and 29% showed a mixed 
cardiac and neurologic phenotype (Sekijima, 2019)). 
Several small molecules have been shown to bind to and stabilize TTR, potentially preventing 
the initiation the amyloidogenesis cascade. Eidos’ therapeutic hypothesis is that a highly 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 18 of 68 
Alexion Confidential 
effective TTR stabilizer will halt or slow ATTR disease progression in ATTR-CM (both 
ATTRm and ATTRwt) and ATTR-PN. 
2.1. 
Study Rationale 
This prospective study is designed to evaluate the efficacy, safety and tolerability, as well as to 
establish the pharmacokinetic (PK) and pharmacodynamic (PD) profile of ALXN2060 in 
Japanese participants with symptomatic ATTR-CM, administered on a background of stable 
heart failure therapy. A multicenter, open label, 2-part design followed by an Extension Period is 
considered to be the most appropriate study design for meeting this objective. Based on 
information gained from previous clinical experience with ALXN2060, a twice daily (bid) dose 
of 800 mg has been selected for this study to represent the optimal combination of potential 
efficacy, safety, and tolerability. 
The dose proposed in this Phase 3 study on Japanese participants is the same as that used in the 
Global Phase 3 study (Eidos Study AG10-301). The chosen inclusion and exclusion criteria as 
well as the endpoints follow Eidos AG10-301 study as closely as possible. 
2.2. 
Background 
Currently, tafamidis is the only drug approved in Japan for the treatment of ATTR-CM. 
Transthyretin stabilization, as achieved by tafamidis, is proven to be beneficial to participants 
with symptomatic ATTR-CM (Maurer, 2018). As the main pathophysiological mechanism of 
ATTR involves the dissociation of the homo-tetrameric protein into monomers, TTR stabilizers 
that act by binding to TTR at the level of its binding site for thyroxine (T4), and therefore 
stabilizing the tetramer, prevent its dissociation and subsequent misfolding. Preventing the 
protein misfolding reduces the risk associated with the deposition of amyloid into various tissues, 
thereby enabling organ recovery and slowing the progress and/or arresting the progressive organ 
dysfunction (Maurer, 2018). 
ALXN2060 is a potent, highly selective, small molecule TTR stabilizer. It demonstrated ability 
to stabilize TTR in vivo following oral dosing to nonhuman mammals, in healthy volunteers, and 
in cardiomyopathy patients (Miller, 2018), (Fox, 2020), (Judge, 2019). 
2.3. 
Benefit/Risk Assessment 
2.3.1. 
Risk Assessment 
AG10 has been well-tolerated in toxicology studies at what are predicted to be supra-therapeutic 
exposures. Results from Phase 1 studies (AG10-001, AG10-003, AG10-004, and AG10-005) and 
Phase 2 (AG10-201) study to date, have shown that ALXN2060 was generally well tolerated 
without ALXN2060-related safety signals of potential clinical concern (Judge, 2019). The 
reported serious adverse events (SAEs) are consistent with those anticipated in this population. 
Currently, there are no important identified or potential risks for ALXN2060. More detailed 
information about the known and expected benefits and risks and reasonably expected adverse 
events (AEs) of AG10 (hereafter referred to as ALXN2060) may be found in the Investigator’s 
Brochure. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 19 of 68 
Alexion Confidential 
2.3.1.1. 
Coronavirus Disease 2019 
The coronavirus disease 2019 (COVID‑19) pandemic is active in Japan at the time of this 
protocol amendment. Given this unique circumstance, specific consideration has been given to 
the risks and benefits of the study as they relate to COVID-19, and the changes that exist as a 
result of the pandemic. This assessment is described in Section 10.5. 
2.3.2. 
Benefit Assessment 
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a serious, progressive, and life-
threatening disease associated with either wild-type TTR or, in the familial form of the disease, 
with pathogenic mutations in the TTR gene. While such loss-of-function mutations may 
accelerate the development and progression of ATTR-CM, age-related dissociation of the 
destabilized, native tetrameric form of the protein is the initiating pathophysiological event 
(Rapezzi, 2010). The degree to which individual mutations promote destabilization of the 
tetramer is associated with a higher degree of penetrance, an earlier onset of symptoms, and/or a 
more aggressive and rapidly progressive clinical course (Rapezzi, 2010). 
Dissociation of tetrameric TTR into intrinsically unstable monomeric TTR favors misfolding and 
aggregation into amyloidogenic precursors that are deposited in affected tissues and organs, 
leading to local cytotoxicity and both architectural and functional disruption. Small molecule 
stabilizers like ALXN2060 reduce the likelihood of tetramer dissociation and this mechanism 
provides the therapeutic rationale supporting their use in halting or slowing progression of the 
disease. The preclinical data and clinical efficacy and safety data collected to date support the 
continued clinical assessment of ALXN2060 in participants with symptomatic ATTR-CM. 
2.3.3. 
Overall Benefit: Risk Conclusion 
Alexion considers the benefit-risk balance to be favorable for the continuation of the 
development of ALXN2060 in participants with symptomatic ATTR-CM. 
 
 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 20 of 68 
Alexion Confidential 
3. 
OBJECTIVES AND ENDPOINTS 
Objective 
Endpoints 
Primary 
Part A: 
To determine the efficacy of orally administered 
ALXN2060 800 mg bid in the treatment of adult 
Japanese participants with symptomatic ATTR-CM 
as assessed by the 6MWT 
Part A: 
Change from Baseline to Month 12 of treatment in 
distance walked during the 6MWT  
Part B: 
To determine the efficacy of orally administered 
ALXN2060 800 mg bid in the treatment of adult 
Japanese participants with symptomatic ATTR-CM 
as assessed by all-cause mortality and 
cardiovascular-related hospitalization 
Part B: 
All-cause mortality and cardiovascular-related 
hospitalization over a 30-month period 
Secondary 
Part A:  
• 
To evaluate the effects of ALXN2060 800 mg 
bid on the 6MWT 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on health-related quality of life as 
measured by KCCQ-OS in adult Japanese 
participants with symptomatic ATTR-CM 
• 
To assess the safety and tolerability of 
ALXN2060 800 mg bid in adult Japanese 
participants with symptomatic ATTR-CM  
• 
To assess the PD effects of ALXN2060 by 
circulating serum TTR concentration as an in 
vivo biomarker of stabilization and using 
established ex vivo assays of TTR 
stabilization (FPE); in addition, assess the 
correlation between TTR concentration/FPE 
and ALXN2060 concentration 
Part A:  
• 
Change from Baseline to Months 6 and 9 of 
treatment in distance walked during the 
6MWT  
• 
Change from Baseline to Months 6, 9, and 12 
of treatment in KCCQ-OS 
• 
Safety parameters to be assessed: treatment-
emergent SAEs and AEs, adverse events 
leading to treatment discontinuation, 
abnormal physical examination findings of 
clinical relevance, abnormal vital signs of 
clinical relevance, abnormal ECG parameters 
of clinical relevance, and changes in clinical 
safety laboratory parameters of potential 
clinical concern 
• 
Change from Baseline in serum TTR 
concentration (an in vivo measure of TTR 
stabilization) and established ex vivo assays 
of TTR stabilization (FPE) at Days 14 and 28 
and subsequent visits  

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 21 of 68 
Alexion Confidential 
Objective 
Endpoints 
Part B: 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on the 6MWT 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on health-related quality of life as 
measured by KCCQ-OS in adult Japanese 
participants with symptomatic ATTR-CM  
• 
To assess the safety and tolerability of 
ALXN2060 800 mg bid administered for 30 
months to adult Japanese participants with 
symptomatic ATTR-CM 
• 
To assess the PD effects of ALXN2060 as 
assessed by circulating serum TTR 
concentration as an in vivo biomarker of 
stabilization and established ex vivo assays of 
TTR stabilization (FPE) and correlation 
between TTR concentration/FPE and 
ALXN2060 concentration 
Part B: 
• 
Change from Baseline to Months 18, 24, and 
30 of treatment in distance walked during the 
6MWT.  
• 
Change from Baseline to Months 18, 24, and 
30 of treatment in KCCQ-OS  
• 
Safety parameters: treatment emergent SAEs 
and AEs, adverse events leading to treatment 
discontinuation, abnormal physical 
examination findings of clinical relevance, 
abnormal vital signs of clinical relevance, 
abnormal ECG parameters of clinical 
relevance, and changes in clinical safety 
laboratory parameters of potential clinical 
concern 
• 
Change from Baseline in serum TTR 
concentration and established ex vivo assays 
of TTR stabilization (FPE) 
Exploratory  
• 
To determine the efficacy of orally 
administered ALXN2060 800 mg bid in the 
treatment of adult Japanese participants with 
symptomatic ATTR CM as assessed by all-
cause mortality and cardiovascular-related 
hospitalization (only Part A) 
• 
To explore the population PK of ALXN2060 
and its metabolites under the 800 mg orally 
administered bid dosing regimen 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on circulating biomarkers of myocardial 
wall stress and microvascular ischemia in 
Japanese participants with symptomatic 
ATTR-CM 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on EQ-5D-5L in Japanese participants 
with symptomatic ATTR-CM 
• 
To evaluate the effects of ALXN2060 800 mg 
bid on cardiac MRI-derived measures of 
cardiac structure, function and tissue 
characterization, in Japanese participants with 
symptomatic ATTR-CM (only at selected 
sites and consenting patients) 
Extension Period 
• 
All-cause mortality and cardiovascular-
related hospitalization over a 12-month 
period (only Part A) 
• 
Exploration of PK over 30 months for 
ALXN2060 and its metabolite  
• 
Changes from Baseline over Month 30 in NT-
proBNP and TnI  
• 
Change from Baseline in EQ-5D-5L over 
Month 30 
• 
Change from Baseline to Month 12, 24, and 
30 in cardiac MRI parameters (only at 
selected sites and consenting patients) 
Extension Period 
• 
Safety parameters same as the Intervention 
Period (Part A and Part B) are assessed in the 
Extension Period 
• 
Efficacy endpoints same as the Intervention 
Period excluding cardiac MRI parameters are 
evaluated during the Extension Period 
• 
PD parameters (serum TTR concentration and 
TTR stabilization (FPE)) same as the 
Intervention Period are assessed in the 
Extension Period 
• 
Exploration of PK in the Extension Period for 
ALXN2060 and its metabolite 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 22 of 68 
Alexion Confidential 
Objective 
Endpoints 
• 
To assess the safety of ALXN2060 in 
Japanese participants with symptomatic 
ATTR-CM during the Extension Period 
• 
To evaluate the long-term efficacy of 
ALXN2060 in Japanese participants with 
symptomatic ATTR-CM during the 
Extension Period 
• 
To assess the PD effects of ALXN2060 
during the Extension Period  
• 
To characterize PK of ALXN2060 in 
Japanese participants with symptomatic 
ATTR-CM during the Extension Period 
Abbreviations: 6MWT = six-minute walk test; AE = adverse event; ATTR-CM = transthyretin amyloid cardiomyopathy; 
ECG = electrocardiography; EQ-5D-5L = European Quality of Life Health 5-item questionnaire dimensions 5 level 
questionnaire; FPE = fluorescent probe exclusion; KCCQ-OS = Kansas City Cardiomyopathy Questionnaire Overall Score; 
MRI = magnetic resonance imaging; NT-proBNP = N-terminal pro-brain-type natriuretic peptide; PD = pharmacodynamic; 
PK = pharmacokinetic; SAE = serious adverse event; TnI = troponin I; TTR = transthyretin.  

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 23 of 68 
Alexion Confidential 
4. 
STUDY DESIGN 
4.1. 
Overall Design 
This Phase 3, prospective, multicenter, open label, 2-part study will evaluate the efficacy, safety, 
tolerability, PK, and PD of ALXN2060 800 mg bid administered orally in Japanese participants 
with symptomatic ATTR-CM. The Study schema is presented in Section 1.2. 
Eligible Japanese participants with symptomatic ATTR-CM (either mutant or wild type) will be 
enrolled based on inclusion/exclusion criteria. In order to assess the efficacy of ALXN2060 
800 mg bid as monotherapy, patients with ATTR-CM will be eligible to be enrolled as long as 
they were untreated with tafamidis for at least 14 days prior to first dose of ALXN2060. In case 
new ATTR-CM therapies will become available during the course of the study, Alexion’s 
Medical Monitor in consultation and agreement with the Investigator will determine the pre-
enrollment period needed to establish a proper TTR baseline. At the moment, the best 
assumptions would be a requirement of last dose administration of 90 days for patisiran and 180 
days for inotersen prior to first day of ALXN2060 dosing. 
A Screening Period of up to 28 ± 7 days will confirm the participants’ diagnosis of ATTR-CM 
and suitability for a long-term investigation. This period will also cover the requirement of last 
dose of tafamidis administered at least 14 days prior to the first ALXN2060 dose. 
Following enrollment and screening, participants will receive ALXN2060 800 mg bid for 
12 months (Part A). Following the last visit (Month 12) of Part A, participants will continue the 
study in Part B, which will last for 18 months (30 months from Day 1). All participants will 
continue to receive oral treatment with ALXN2060 800 mg bid. 
Following the completion of Month 30 assessments in Part B, participants will be offered the 
opportunity to continue into the Extension Period for up to 30 additional months or until 
ALXN2060 can be provided via an Alexion post-study access program (as allowed by local laws 
and regulations), whichever occurs first. Participants will continue to receive oral treatment with 
ALXN2060 800 mg bid. 
4.2. 
Scientific Rationale for Study Design 
A multicenter, open label, 2-part design is considered to be the most appropriate study design for 
meeting the study objectives. Based on information gained from previous clinical experience 
with ALXN2060 (see Section 2), a dose of 800 mg bid has been selected for this study to 
represent the optimal combination of potential efficacy, safety, and tolerability. 
4.2.1. 
Participant Input Into Design 
There were no inputs from participants into the study design. 
4.3. 
Justification for Dose 
The dose selected for this study (ALXN2060 800 mg bid) is based on the results of 2 Phase 1 
studies in healthy participants: a single and multiple ascending dose Phase 1 study (Study AG10-
001) and a Phase 1 bridging study assessing comparability of exposure in Japanese and 
non-Japanese healthy volunteers (Study AG10-004), and a Phase 2 study in patients with 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 24 of 68 
Alexion Confidential 
symptomatic ATTR-CM (Study AG10-201). In addition, the dose selected for this study is the 
same dose and regimen as in the ongoing global Phase 3 study in participants with symptomatic 
ATTR-CM (Study AG10-301). 
The first-in-human study (Study AG10-001) was conducted in healthy volunteers and evaluated 
4 single oral doses of AG10 (50, 150, 300, and 800 mg) in a sequential manner and 3 multiple 
oral doses of AG10 (100, 300, and 800 mg every 12 hours for a total of 12 days). In this study 
AG10 was well tolerated, with no SAEs or AEs, or any safety signals of potential clinical 
concern considered related to the administration of AG10. 
In the Phase 1 PK bridging study (Study AG10-004), Japanese and non-Japanese healthy 
volunteers were administered 400 or 800 mg of AG10 and showed similar exposure and PK 
parameters. 
In the Phase 2 study (Study AG10-201), 49 adults with symptomatic ATTR-CM were 
randomized to oral doses of placebo (n = 17), AG10 400 mg (n = 16), or 800 mg (n = 16) bid. 
The study showed that AG10 was well tolerated and there were no AG10-related safety signals 
of potential clinical concern. 
Transthyretin stabilization as measured by fluorescent probe exclusion (FPE) demonstrated 
complete stabilization at peak and > 90% stabilization at trough at the higher dose with low 
inter-subject variability. Average change from Baseline in serum TTR concentrations, a direct in 
vivo reflection of TTR stabilization, showed a clear dose response: after 28 days of treatment, 
serum TTR concentrations increased on average by 36% in the 400 mg bid group and by 51% in 
the 800 mg bid group; serum TTR decreased on average by 7% in patients administered with 
placebo. 
4.4. 
End of Study Definition 
A participant is considered to have completed the study if he/she has completed both Part A and 
Part B of the study including the last scheduled procedure shown in the Schedule of Activities 
(SoA) (Section 1.3). 
The end of study for each participant is defined as his/her last visit in the study. The end of the 
study is defined as the last participant’s last visit in the study. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 25 of 68 
Alexion Confidential 
5. 
STUDY POPULATION 
Prospective approval of protocol deviations to recruitment and enrollment criteria, also known as 
protocol waivers or exemptions, is not permitted. 
5.1. 
Inclusion Criteria 
Male or female participants are eligible to be included in the study only if all of the following 
criteria apply: 
Age 
1. Male or females ≥ 20 and ≤ 90 years of age, inclusive, at the time of signing the informed 
consent. 
Type of Participant and Disease Characteristics 
2. Have an established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR 
genotype (confirmed by genotyping) based on either (a) endomyocardial biopsy with 
confirmatory TTR amyloid typing by either immunohistochemistry, mass spectrometry, 
or immunoelectron microscopy, or (b) positive technetium-99m (99mTc)-pyrophosphate or 
bisphosphonate (DPD or HMPD) scan, combined with accepted laboratory criteria 
excluding a diagnosis of light-chain (AL) amyloidosis (based on both immunofixation 
electrophoresis [IFE] of serum and urine, and serum free light chain [sFLC] analysis) and 
extracardiac biopsy (eg, abdominal fat aspiration) documenting TTR amyloid deposits; 
participants with concurrent monoclonal gammopathy of undetermined significance 
(MGUS) may require confirmation of the diagnosis of ATTR-CM by tissue biopsy with 
confirmatory TTR amyloid typing by either immunohistochemistry, mass spectrometry, 
or immunoelectron microscopy. 
3. Have (a) a history of heart failure evidenced by at least one prior hospitalization for heart 
failure or (b) clinical evidence of heart failure without prior heart failure hospitalization 
manifested by signs or symptoms of volume overload or elevated pressures (eg, elevated 
jugular venous pressure, shortness of breath or signs of pulmonary congestion on x-ray or 
auscultation, or peripheral edema), or (c) heart failure symptoms that required or requires 
ongoing treatment with a diuretics. 
4. Have New York Heart Association (NYHA) Class I-III symptoms due to ATTR-CM. 
5. Participants taking cardiovascular (CV) medical therapy, with the exception of diuretic 
drugs, must be on stable doses (defined as no greater than 50% dose adjustment and no 
categorical changes of medications) for at least 2 weeks prior to Screening. 
6. Have completed ≥ 150 m on the six-minute walk test (6MWT) on at least 2 tests > 24 
hours to ≤ 3 weeks apart and prior to Day 1. The distance walked must be within 15% on 
2 tests. If one of the first 2 tests is not ≥ 150 m or the first 2 tests are not within 15% of 
distance walked, a third test must be conducted ≤ 3 weeks of the first test. The patient 
will be ineligible if the third test is still not ≥ 150 m or within 15% of one of the first 2 
tests. 
7. Have left ventricular (LV) wall (interventricular septum or LV posterior wall) thickness 
≥ 12 mm as measured by transthoracic echocardiogram (ECHO) or cardiac magnetic 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 26 of 68 
Alexion Confidential 
resonance (CMR) documented in medical history within 10 years of Screening or at 
Screening ECHO or CMR. 
8. Have biomarkers of myocardial wall stress: N-terminal pro-brain-type natriuretic peptide 
(NT-proBNP) level ≥ 300 pg/mL at Screening 
Sex 
9. Women of childbearing potential (WOCBP) who engage in heterosexual intercourse must 
agree to use highly effective methods of contraception beginning with Screening and 
continuing for 30 days after the last dose of the study drug as detailed in Section 10.4 . A 
man who is sexually active with WOCBP and has not had a vasectomy must agree to use 
highly effective methods of contraception from Day 1 to at least 90 days after the last 
dose of the study drug as detailed in Section 10.4. 
Informed Consent 
10. Able to understand and sign a written Informed Consent Form (ICF), which must be 
obtained prior to initiation of study procedures. Participants must be capable of giving 
signed informed consent as described in Appendix 1 which includes compliance with the 
requirements and restrictions listed in the ICF and in this protocol. 
5.2. 
Exclusion Criteria  
Participants are excluded from the study if any of the following criteria apply: 
Medical Conditions 
1. Had an acute myocardial infarction, acute coronary syndrome, or coronary 
revascularization within 90 days prior to Screening. 
2. Experienced stroke or transient ischemic attack (TIA) within 90 days prior to Screening. 
3. Have CV hemodynamic instability at Screening that, in the judgment of the Investigator, 
would pose too great a risk for participation in the study. 
4. Are likely to undergo heart transplantation within a year of screening. 
5. Have known hypersensitivity to ALXN2060, its metabolites, or formulation excipients. 
6. Females who are pregnant or breastfeeding. Lactating females must agree to discontinue 
nursing before study drug is administered. A negative urine pregnancy test at Screening 
and at Day 1 are required for WOCBP. 
7. In the judgment of the Investigator or Medical Monitor, have any clinically important 
ongoing medical condition or laboratory abnormality or condition that might jeopardize 
the patient’s safety, increase their risk from participation, or interfere with the study. 
Prior/Concomitant Therapy 
8. Are receiving treatment for ATTR-CM with tafamidis, with marketed drug products 
lacking a labeled indication for ATTR-CM (eg, diflunisal, doxycycline); or with natural 
products or derivatives used as unproven therapies for ATTR-CM (eg, the green tea 
extract, tauroursodeoxycholic acid [TUDCA]/ursodiol) within 14 days prior to dosing; 
treatment with patisiran, inotersen, or any other gene silencing agent: within 90 days for 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 27 of 68 
Alexion Confidential 
patisiran and 180 days for inotersen, and 5 half-lives for any other gene silencing agent, 
prior to dosing. 
9. Requires treatment with calcium channel blockers with conduction system effects (eg, 
verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. 
The use of digitalis will only be allowed if required for management of atrial fibrillation 
with rapid ventricular response. 
Prior/Concurrent Clinical Study Experience 
10. Are participating in another investigational drug or investigational device study, within 
30 days prior to the first day of ALXN2060 dosing, with potential residual effects that 
might confound the results of this study. 
Diagnostic Assessments 
11. Have confirmed a diagnosis of AL amyloidosis. 
12. Have abnormal liver function tests at Screening, defined as alanine aminotransferase 
(ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) or total 
bilirubin > 3 × ULN. 
13. Have NT-ProBNP ≥ 8,500 pg/mL at Screening. 
14. Have an estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2 as calculated 
by Modification of Diet in Renal Disease (MDRD) formula at Screening. 
Other Exclusions 
15. Have any condition that, in the opinion of the Investigator or Medical Monitor would 
preclude compliance with the study protocol such as a history of substance abuse, 
alcoholism or a psychiatric condition. 
5.3. 
Lifestyle Considerations  
No specific restrictions are applicable for this study. 
5.4. 
Screen Failures 
Screen failures are defined as participants who consent to participate in the clinical study but did 
not meet eligibility criteria. A minimal set of screen failure information is required to ensure 
transparent reporting of screen failure participants and to respond to queries from regulatory 
authorities. Minimal information includes demography, screen failure details (eg, failed 
eligibility criteria), and any AEs, including any SAEs and any concomitant medication, during 
the Screening Period. 
Individuals who do not meet the criteria for participation in this study (screen failure) due to a 
reason that is expected to resolve or has resolved, may be rescreened based on discussion and 
agreement between the Investigator and the Medical Monitor. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 28 of 68 
Alexion Confidential 
6. 
STUDY INTERVENTION 
Study intervention is defined as any investigational intervention(s), marketed product(s), 
placebo, or medical device(s) intended to be administered to a study participant according to the 
study protocol. 
6.1. 
Study Intervention(s) Administered 
The study intervention is ALXN2060 800 mg bid administered orally (Table 1). 
Table 1: 
Study Intervention, Dosage, and Mode of Administration 
Study Drug Name 
ALXN2060  
Type  
Drug  
Dose Formulation 
Film-coated Tablet  
Unit Dose Strength(s) 
400 mg ALXN2060  
Dosage Level(s) 
800 mg bid  
Route of Administration 
Oral  
Use 
Experimental  
Sourcing 
Provided centrally by Alexion 
Packaging and Labeling 
Study Intervention will be provided in bottles. Each bottle 
will be labeled as required per country requirement 
6.2. 
Preparation/Handling/Storage/Accountability 
1. The Investigator or designee must confirm appropriate temperature conditions have been 
maintained during transit for all study intervention received and any discrepancies are 
reported and resolved before use of the study intervention. 
2. Only participants enrolled in the study may receive study intervention and only 
authorized site staff may supply or administer study intervention. All study intervention 
must be stored in a secure, environmentally controlled, and monitored (manual or 
automated) area in accordance with the labeled storage conditions with access limited to 
the Investigator and authorized site staff. 
3. The Investigator, institution, or the head of the medical institution (where applicable) is 
responsible for study intervention accountability, reconciliation, and record maintenance 
(ie, receipt, reconciliation, and final disposition records). 
a. This responsibility includes the reporting of any product complaints to 
productcomplaints@alexion.com within 1 business day. A product complaint is 
defined as any written, electronic, or oral communication that alleges deficiencies 
related to the identity, quality, durability, reliability, usability, safety, effectiveness, or 
performance of a product or clinical trial material and/or its packaging components 
after it is has been released for distribution to an end customer that affects the 
performance of such product. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 29 of 68 
Alexion Confidential 
4. Further guidance and information for the final disposition of unused study interventions 
are provided in the Study Reference Manual. 
6.3. 
Measures to Minimize Bias: Randomization and Blinding 
There is no randomization or blinding in this single-arm, open-label study of approximately 
22 participants. 
6.4. 
Study Intervention Compliance 
When participants self-administer study intervention(s) at home, compliance with study 
intervention will be assessed at each visit. Compliance will be assessed by counting returned 
tablets during the site visits and documented in the source documents and electronic case report 
form (eCRF). Deviation(s) from the prescribed dosage regimen should be recorded in the eCRF. 
Record of the doses dispensed to and taken by each participant must be maintained and 
reconciled with study intervention and compliance records. Intervention start and stop dates, 
including dates for planned and unplanned intervention delays and/or dose reductions will also 
be recorded in the eCRF. 
For additional information on study intervention compliance and management, refer to the 
Pharmacy Manual. 
6.5. 
Concomitant Therapy 
As stated in Section 5.1, participants taking CV medical therapy, with the exception of diuretic 
drugs, must be on stable doses (defined as no greater than 50% dose adjustment and no 
categorical changes of medications) for at least 2 weeks prior to Screening. 
The Medical Monitor should be contacted if there are any questions regarding concomitant or 
prior therapy. 
6.5.1. 
Allowed Medicine and Therapy 
Participants may continue medications required to treat existing medical conditions as long as 
they are not anticipated to interfere with the conduct or outcome of the study. Participants should 
avoid starting or stopping medications and all potential medication changes for CV medical 
therapy should be reviewed by the Investigator and consulted with the Medical Monitor to ensure 
changes are not expected to interfere with the outcome of the study. 
6.5.2. 
Disallowed Medicine and Therapy 
The following is prohibited as listed in Section 5.2: 
• Use of patisiran, inotersen, tafamidis or any other approved or investigational agent 
for the treatment of ATTR-CM is prohibited during the study. 
• Use of marketed drug products lacking a labeled indication for ATTR-CM (e.g., 
diflunisal, doxycycline) or of natural products or derivatives used as unproven 
therapies for ATTR-CM (e.g., green tea extract, tauroursodeoxycholic acid 
[TUDCA]/ursodiol) is prohibited. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 30 of 68 
Alexion Confidential 
• Use of calcium channel blockers with conduction system effects (eg, verapamil, 
diltiazem) is prohibited. Use of dihydropyridine calcium channel blockers is allowed. 
The use of digitalis will only be allowed if required for management of atrial 
fibrillation with rapid ventricular response. 
6.6. 
Dose Modification 
Dose modification is not applicable to this study. 
6.7. 
Intervention After the End of the Study 
Eligible Participants will be offered the opportunity to continue receiving ALXN2060 800 mg 
bid. in the Extension Period for up to 30 additional months or until ALXN2060 can be provided 
via an Alexion post-study access program (as allowed by local laws and regulations), whichever 
occurs first. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 31 of 68 
Alexion Confidential 
7. 
DISCONTINUATION OF STUDY INTERVENTION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL 
7.1. 
Discontinuation of Study Intervention 
In rare instances, it may be necessary for a participant to permanently discontinue (definitive 
discontinuation) study intervention. If study intervention is definitively discontinued, the 
participant should remain in the study to be evaluated for safety follow-up. See the SoA 
(Section 1.3) for data to be collected at the time of discontinuation of study intervention and 
follow-up and for any further evaluations that need to be completed. 
Participants should be considered for discontinuation from intervention if any of the following 
occur during the study: 
1. Serious hypersensitivity reaction; 
2. Use of disallowed medication as defined in Section 6.5 
3. Received heart or liver transplant or received Cardiac mechanical assist device (CMAD) 
at any time during the trial 
4. Pregnancy or planned pregnancy (Section 10.4.3); or 
5. Alexion or the Investigator deems it is necessary for the participant. 
7.1.1. 
Temporary Discontinuation 
If considered necessary, as per Investigator’s discretion, IMP may be interrupted (e.g., in case of 
AE, hospitalization or procedure), however IMP should be resumed as soon as practically 
possible, unless there are safety concerns. Investigators are encouraged to discuss such cases 
with a Medical Monitor. 
7.2. 
Participant Discontinuation/Withdrawal from the Study 
• All efforts should be made to ensure participants are willing to comply with study 
participation prior to conducting the screening procedures. The study staff should 
notify Alexion and their site monitor of all study withdrawals as soon as possible. The 
reason for participant discontinuation must be recorded in the source documents and 
eCRF 
• A participant may withdraw from the study with or without agreement for follow-up 
at any time at his/her own request, or may be withdrawn at any time at the discretion 
of the Investigator for safety, behavioral, compliance, or administrative reasons. 
Note: If a subject discontinues IMP and study assessments, all efforts must be made 
to continue to follow the subject for up to Month 30 by completing monthly contact 
for vital status (dead, alive, heart transplant and receiving CMAD) or until withdrawal 
of consent.  
• At the time of discontinuing from the study, if possible, an Early Discontinuation 
Visit should be conducted, as shown in the SoA (Section 1.3) for data to be collected 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 32 of 68 
Alexion Confidential 
at the time of study discontinuation and follow-up and for any further evaluations that 
need to be completed. 
• The participant will be permanently discontinued both from the study intervention 
and from the study at that time. 
• If the participant withdraws consent for disclosure of future information, Alexion may 
retain and continue to use any data collected before such a withdrawal of consent. 
Alexion will obtain final vital status for such patients. 
7.3. 
Lost to Follow up 
A participant will be considered lost to follow-up if he or she repeatedly fails to return for 
scheduled visits and is unable to be contacted by the study site. 
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit: 
• The site must attempt to contact the participant and reschedule the missed visit as 
soon as possible and counsel the participant on the importance of maintaining the 
assigned visit schedule and ascertain whether or not the participant wishes to and/or 
should continue in the study. 
• Before a participant is deemed lost to follow up, the Investigator or designee must 
make every effort to regain contact with the participant (where possible, 3 telephone 
calls and, if necessary, a certified letter to the participant’s last known mailing 
address or local equivalent methods). These contact attempts should be documented 
in the participant’s medical record. 
• Should the participant continue to be unreachable, he/she will be considered to have 
withdrawn from the study. 
Discontinuation of specific sites or of the study as a whole are handled as part of Section 10.1.9. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 33 of 68 
Alexion Confidential 
8. 
STUDY ASSESSMENTS AND PROCEDURES 
8.1. 
General Procedures 
• Study procedures and their timing are summarized in the SoA (Section 1.3). Protocol 
waivers or exemptions are not allowed. 
• Immediate safety concerns should be discussed with Alexion immediately upon 
occurrence or awareness to determine if the participant should continue or 
discontinue study intervention. 
• Adherence to the study design requirements, including those specified in the SoA, is 
essential and required for study conduct. 
• All inclusion (Section 5.1) and exclusion (Section 5.2) criteria must be reviewed by 
the Investigator or qualified designee to ensure the patient qualifies for study 
participation. 
• All screening evaluations must be completed and reviewed to confirm that potential 
participants meet all eligibility criteria. The Investigator will maintain a screening log 
to record details of all participants screened and to confirm eligibility or record 
reasons for screening failure, as applicable. Resting transthoracic ECHO will be 
performed at Screening and read locally, if LV wall (interventricular septum or LV 
posterior wall) thickness not documented in medical history within 10 years of 
Screening based on transthoracic ECHO or CMR 
• Procedures conducted as part of the participant’s routine clinical management (eg, 
blood count) and obtained before signing of the ICF may be utilized for screening or 
baseline purposes provided the procedures met the protocol-specified criteria and 
were performed within the time frame defined in the SoA. 
8.2. 
Efficacy Assessments 
8.2.1. 
Six-Minute Walk Test (6MWT) 
Prior to Day 1, at least two 6MWTs will be conducted > 24 hours to ≤ 3 weeks apart. The 
walking distances must be ≥ 150 meters and the distance walked must be within 15% on two 
tests on different days. If one of the first 2 tests is not ≥ 150 m or the first 2 test results are not 
within the 15% of distance walked, a third test must be conducted ≤ 3 weeks of the first test. If 
the third test is still not ≥ 150 m or within 15% of one of the first two tests, the subject will not 
be eligible for participation. 
If the subject has a need for a walking aid (eg, cane) or supplemental oxygen at Baseline, it must 
be consistently used at each subsequent 6MWT throughout the study. 
The 6MWT should be conducted after completion of the Kansas City Cardiomyopathy 
Questionnaire Overall Score (KCCQ) and European Quality of Life Health 5-item questionnaire 
dimensions 5 level questionnaire (EQ-5D-5L) at the visits where required. 
The 6MWT with Borg Scale will be conducted based on the guidelines of the American Thoracic 
Society with appropriate modifications for the patient population. Complete details on the 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 34 of 68 
Alexion Confidential 
procedures for the 6MWT are provided in the Study Procedures Manual. The 6MWT will be 
obtained on M6, M9, M12, M18, M24, and M30, and every 6 months during the Extension 
Period. 
8.2.2. 
Mortality and Cardiovascular-Related Hospitalization 
Mortality (including CMAD and heart transplant) and CV-related hospitalizations are endpoints 
for the trial but the events that lead to death or hospitalization will be reported as AEs to the 
Sponsor throughout the trial. These events will be reviewed and adjudicated by an independent 
Clinical Events Committee (CEC) and determined whether reason for hospitalization and cause 
of death meet the definition of protocol-specified efficacy endpoints (Section 9.6). 
Hospitalizations that are submitted for adjudication and are deemed as not CV-related by the 
CEC will be considered SAEs. Adverse events of special interest (AESIs, Section 8.4.5) are also 
considered as part of the efficacy endpoint of CV-related hospitalizations. 
The Investigator is responsible for ensuring potential study endpoints, including dates of 
admissions and discharge, are collected and documented; for providing Investigator assessment 
whether the hospitalization is CV-related; and for submitting Adverse Event notification for all 
AEs that result in deaths or hospitalizations. 
8.2.3. 
Kansas City Cardiomyopathy Questionnaire (KCCQ) 
The KCCQ is a 23-item questionnaire developed to measure health status and health-related 
quality of life in participants with heart failure. Items include heart failure symptoms, impact on 
physical and social functions, and how their heart failure impacts their quality of life. The KCCQ 
questionnaire is completed during Part A and Part B (Day 1, M6, M9, M12, M18, M24, and 
M30) and during the Extension Period (every 6 months) (Section 1.3). Complete details are 
provided in the Study Procedures Manual. 
8.2.4. 
EuroQoL-5 Dimensions (EQ-5D-5L) 
The EQ-5D-5L is a brief, self-administered generic health status instrument that takes about 5 
minutes to complete and should be conducted after completion of the KCCQ. The instrument 
includes two parts. In the first part, respondents are asked to rate their current health state on 5 
dimensions (mobility, self-care, usual activities, pain or discomfort, and anxiety or depression) 
with each dimension having five levels of function (1-no problem, 2-slight problem, 3-moderate 
problem, 4-severe problem, and 5-extreme problem). The second part is a respondents self-rating 
of current health status on a Visual Analog Scale (EQ VAS) with endpoints labeled “best 
imaginable health state” (score of 100) and “worst imaginable health state” (score of 0). The 
scores from the 5 dimensions may be used to calculate a single index value, also known as a 
utility score. Complete details of administration and scoring are provided in the Study 
Procedures Manual. 
Scores for EQ-5D-5L will be obtained on during Part A and Part B (Day 1, M6, M9, M12, M18, 
M24, and M30) and during the Extension Period (every 6 months) (Section 1.3). 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 35 of 68 
Alexion Confidential 
8.2.5. 
NT-proBNP and Troponin I 
Exploratory assessments of NT-proBNP and Tn I evaluation effects of ALXN2060 on circulating 
biomarkers of myocardial wall stress and microvascular ischemia will be performed by 
standardized methods at time points for laboratory tests specified in Section 1.3, 
8.2.6. 
Cardiac MRI 
The cardiac MRI parameters will include structural measures, functional measures, and tissue 
characterization. The cardiac MRI will be performed on Day 1, M12, M24, and M30 (only at 
selected sites and consenting patients). Complete details are provided in the Study Procedures 
Manual.  
8.3. 
Safety Assessments 
Planned time points for all safety assessments are provided in the SoA (Section 1.3). 
8.3.1. 
NewYork Health Assessment 
The NYHA classification is employed to classify patient’s heart failure according to the severity 
of their symptoms. Physicians will assess and place participants in one of four categories based 
on their limitation during physical activity according to Table 2 below. Planned time points for 
NYHA assessments are provided in the SoA (Section 1.3). 
Table 2: 
NYHA Class Assessment 
Class 
Patient Symptoms 
I 
No limitation of physical activity. Ordinary physical activity 
does not cause undue fatigue, palpitation, dyspnea (shortness 
of breath) 
II 
Slight limitation of physical activity. Comfortable at rest. 
Ordinary physical activity results in fatigue, palpitation, 
dyspnea (shortness of breath) 
III 
Marked limitation of physical activity. Comfortable at rest. 
Less than ordinary activity causes fatigue, palpitation, or 
dyspnea 
IV 
Unable to carry on any physical activity without discomfort. 
Symptoms of heart failure at rest. If any physical activity is 
undertaken, discomfort increases 
8.3.2. 
Physical Examinations 
• Physical examination with body weight measurement is performed at all visits. 
Height will be measured only at screening. 
• Investigators should pay special attention to clinical signs related to previous serious 
illnesses. 
• For consistency, all efforts should be made to have the physical examination 
performed by the same qualified study staff at each study visit. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 36 of 68 
Alexion Confidential 
• Additional physical examinations can be performed as medically indicated during the 
study at the Investigator’s discretion. 
8.3.3. 
Vital Signs 
• Pulse rate, respiratory rate, and systolic and diastolic blood pressure (mm Hg) will be 
assessed predose after a 5-minute rest. 
• Blood pressure and pulse measurements will be assessed with a completely 
automated device. Manual techniques will be used only if an automated device is not 
available. 
8.3.4. 
Electrocardiograms  
• A standard 12-lead ECG will be conducted at timings specified in the SoA (see 
Section 1.3). 
• Electrocardiograms will be performed in the supine position after a 5-minute rest at 
predose. The 1-hour postdose ECG will be conducted on Day 28. 
• The Investigator or Sub-Investigator will be responsible for reviewing the ECG to 
assess whether the ECG is within normal limits and determine the clinical 
significance of the results. These assessments will be recorded in the source 
documents (ECG results will be confirmed centrally). 
8.3.5. 
Clinical Safety Laboratory Assessments 
• See Section 10.2 for the list of clinical laboratory tests to be performed and to the 
SoA for the timing and frequency. 
• The Investigator must review the laboratory report, document this review, and record 
any clinically relevant changes occurring during the study in the AE section of the 
eCRF. The laboratory reports must be filed with the source documents. Clinically 
significant abnormal laboratory findings are those which are not associated with the 
underlying disease, unless judged by the Investigator to be more severe than expected 
for the participant's condition. 
• All laboratory tests with values considered clinically significantly abnormal during 
participation in the study or within 30 days after the last dose of study intervention 
should be repeated until the values return to normal or baseline or are no longer 
considered clinically significant by the Investigator or Medical Monitor. 
− If such values do not return to normal/baseline within a period of time judged 
reasonable by the Investigator, the etiology should be identified, and Alexion 
notified. 
− All protocol-required laboratory assessments, as defined in Section 10.2, must be 
conducted in accordance with the laboratory manual and the SoA.  
− If laboratory values from non-protocol specified laboratory assessments 
performed at the institution’s local laboratory require a change in participant 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 37 of 68 
Alexion Confidential 
management or are considered clinically significant by the Investigator (eg, SAE 
or AE or dose modification), then the results must be recorded in the eCRF.  
8.3.6. 
Pregnancy 
• Pregnancy data from WOCBP and female spouses/partners of male participants will be 
collected from the signing of the ICF until the Follow-up Visit (see Section 1.3). 
Additional pregnancy tests may be performed at any visit at the Investigator’s discretion 
• Any female participant who becomes pregnant while participating in the study will be 
discontinued from study intervention. The Investigator must immediately inform Alexion 
within 24 hours awareness of the pregnancy and follow the procedures outlined in 
Section 10.4 
• For all Alexion products, both in development or postapproval, exposure during 
pregnancy must be recorded and the pregnancy followed until the outcome of the 
pregnancy is known (ie, spontaneous miscarriage, elective termination, normal birth, or 
congenital abnormality), even if the participant discontinues study intervention or 
withdraws from the study. The corresponding infant must be followed for 3 months 
postpartum. 
• Pregnancy is not considered an AE (Section 10.4.3) unless there is a suspicion that the 
study intervention may have interfered with the effectiveness of a contraceptive 
medication. However, complications of pregnancy and abnormal outcomes of pregnancy 
are AEs and may meet the criteria for an SAE (eg, ectopic pregnancy, spontaneous 
abortion, intrauterine fetal demise, neonatal death, or congenital anomaly) (Section 8.4). 
Elective abortions without complications should not be reported as AEs. 
8.3.7. 
Prior and Concomitant Medications 
Medications taken as discussed in the exclusion criteria (Section 5.2) before the start of the first 
dose of study drug, will be recorded in the eCRF. 
Concomitant medications (defined in Section 6.5) are those received on or after the first dose of 
the study drug (Day 1), including those started before Day 1 and continued after Day 1. At each 
study visit, participants should be questioned about any new medication or non-drug therapies or 
changes to concomitant medications and nondrug therapies since the last visit. Concomitant 
medications and non-drug therapies should be recorded in the source documents and the patient’s 
eCRF. 
Concomitant medications must be recorded in the patient’s source document/medical chart and 
eCRF along with: 
• Reason for use 
• Dates of administration including start and end dates 
• Dosage information including dose and frequency 
The Medical Monitor should be contacted if there are any questions regarding concomitant or 
prior therapy. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 38 of 68 
Alexion Confidential 
8.4. 
Adverse Events and Serious Adverse Events 
The definitions of AEs and SAEs can be found in Section 10.3. 
AEs will be reported to the Investigator or qualified designee by the participant (or, when 
appropriate, by a caregiver, surrogate, or the participant's legally authorized representative). 
The Investigator and any qualified designees are responsible for detecting, documenting, and 
recording events that meet the definition of an AE or SAE and remain responsible for following 
up until resolution or until AE is deemed stable, or the laboratory results return to baseline or 
normalize. AEs that are serious, considered related to the study intervention or study procedures, 
or that have caused the participant to discontinue the study (see Section 7). 
Procedures for recording, evaluating, follow-up, and reporting AEs and SAEs are outlined in 
Section 10.3 (Appendix 3). 
8.4.1. 
Time Period and Frequency for Collecting AE and SAE Information 
All AEs and SAEs will be collected from the signing of the ICF until the Follow-up Visit 
specified in the SoA (Section 1.3). 
All SAEs will be recorded and reported to Alexion or designee immediately and under no 
circumstance should this exceed 24 hours, as indicated in Section 10.3 Appendix 3. The 
Investigator will submit any updated SAE data to Alexion within 24 hours of it being available. 
Investigators are not obligated to actively seek AE or SAE after conclusion of the study 
participation. However, if the Investigator learns of any SAE, including a death, at any time after 
a participant has been discharged from the study, and he/she considers the event to be reasonably 
related to the study intervention or study participation, the Investigator must promptly notify 
Alexion. 
8.4.2. 
Method of Detecting AEs and SAEs 
The method of recording, evaluating, and assessing causality of AE and SAE and the procedures 
for completing and transmitting SAE reports are provided in Section 10.3. 
Care will be taken not to introduce bias when detecting AEs and/or SAEs. Open-ended and 
non-leading verbal questioning of the participant is the preferred method to inquire about 
AE occurrences. 
8.4.3. 
Follow-up of AEs and SAEs 
After the initial AE/SAE report, the Investigator is required to proactively follow each 
participant at subsequent visits/contacts. All SAEs will be followed until resolution, stabilization, 
the event is otherwise explained, or the participant is lost to follow-up (as defined in 
Section 7.3). Further information on follow-up procedures is provided in Section 10.3. 
8.4.4. 
Regulatory Reporting Requirements for SAEs  
• Prompt notification by the investigator to Alexion of a SAE is essential so that legal 
obligations and ethical responsibilities towards the safety of participants and the 
safety of a study intervention under clinical investigation are met. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 39 of 68 
Alexion Confidential 
• Alexion has a legal responsibility to notify both the local regulatory authority and 
other regulatory agencies about the safety of a study intervention under clinical 
investigation. Alexion will comply with country-specific regulatory requirements 
relating to safety reporting to the regulatory authority, Institutional Review Boards 
(IRB)/Independent Ethics Committees (IEC), and Investigators. 
•  Suspected unexpected serious adverse reactions (SUSAR) must be reported 
according to local regulatory requirements and Alexion policy and forwarded to 
Investigators as necessary. 
• An Investigator who receives an Investigator safety report describing a SAE or other 
specific safety information (eg, summary or listing of SAEs) from Alexion will 
review and then file it along with the Investigator’s Brochure and will notify the 
IRB/IEC, if appropriate according to local requirements. 
8.4.5. 
Adverse Events of Special Interest  
Adverse events of special interest are defined as medical visits (eg, emergency department/ward, 
urgent care clinic, day clinic, etc) of less than 24 hours where diagnosis and interventions 
indicate that the purpose of the visit was for intravenous diuretic therapy for management of 
decompensated heart failure. 
8.5. 
Treatment of Overdose 
For this study, any dose of ALXN2060 greater than that specified in the protocol will be 
considered an overdose. 
Alexion does not recommend specific treatment for an overdose. 
Overdoses are medication errors that are not considered AEs unless there is an untoward medical 
occurrence resulting from the overdose 
In the event of an overdose, the Investigator should: 
1. Contact the Medical Monitor immediately 
2. Closely monitor the participant for any AE/SAE 
3. Obtain a plasma sample for PK analysis if requested by the Medical Monitor (determined 
on a case-by-case basis) 
Decisions regarding dose interruptions or modifications will be made by the Investigator in 
consultation with the Medical Monitor based on the clinical evaluation of the participant. 
8.6. 
Pharmacokinetics 
In a subgroup of participants, PK samples will be collected at the following times to determine 
ALXN2060 plasma concentrations: 
• Day 1, Day 14 and at every 3 months study visits during Part A and B: Predose 
• Day 28: Predose and at 1-hour postdose 
• Month 1 and every 6 months during the Extension Period: Predose 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 40 of 68 
Alexion Confidential 
• Early Discontinuation 
PK backup samples may be used to measure exploratory biomarkers. 
8.7. 
Pharmacodynamics 
In a subgroup of participants, PD properties of ALXN2060 will be assessed by established 
assays of TTR stabilization, including FPE assay. Sampling will be done at the following times 
to perform these PD assays: 
• Day 1, Day 14, and at every 3 months study visits during Part A and B: Predose 
• Day 28: Predose and at 1-hour postdose 
• Month 1 and every 6 months during the Extension Period: Predose 
• Early Discontinuation 
PD backup samples may be used to measure exploratory biomarkers. 
8.8. 
Exploratory Biomarkers 
Blood samples will be collected for assessing exploratory biomarkers for potential evaluation of 
TTR stabilization effects and analysis of TTR variants evaluation effects at screening and EOST 
or ED (Section 1.3). 
8.9. 
Immunogenicity Assessments 
Immunogenicity assessments are not performed in this study. 
8.10. 
Health Economics Data and Medical Resource Utilization 
These assessments are not performed in this study. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 41 of 68 
Alexion Confidential 
9. 
STATISTICAL CONSIDERATIONS 
9.1. 
Statistical Hypotheses 
The primary objective of this study is to assess the efficacy of ALXN2060 for the parameters 
listed in section 3. No formal statistical hypothesis is defined for this study. The success criteria 
based on both Part A and Part B of the study are defined in section 9.4. The sample size is set to 
detect the change in 6MWT at 12 months with certain precision (Section 9.2) as part of the 
success criteria in Part A. 
9.2. 
Sample Size Determination 
A total of 22 Japanese participants are planned for enrollment in this study. The primary 
endpoint in Part A is the change from Baseline to Month 12 in the distance achieved in the 
6MWT, which is an important indicator to investigate functional improvement. 
Assuming similarity with the tafamidis ATTR-ACT study (Maurer, 2018), where the mean 
change in 6MWT distance from Baseline to Month 12 in placebo group was estimated as -60 m, 
which could be considered as the minimum threshold for the changes to be clinically relevant for 
this study; and also assuming the mean change (standard deviation [SD]) from Baseline to Month 
12 in ALXN2060 group was -25 (50) m, an estimated sample size of 19 will provide over 80% 
power for the lower limit of a 95% confidence interval (CI) of change from Baseline to Month 
12 in 6MWT to be over -60 m. 
The primary endpoints in Part B are all-cause mortality and CV-related hospitalizations over a 
30-month period. A sample size of 19 will have over 80% probability to show consistency 
between this study and AG10-301 study with respect to the two primary endpoints in Part B, 
respectively, based on simulations. 
Considering a missing rate of 10% to 15% for 6MWT at Month 12, the target sample size of 22 
is set for Study ALXN2060-TAC-302. 
9.3. 
Populations for Analyses 
The following populations are defined: 
Population 
Description 
Enrolled Set  
All consented participants meeting eligibility criteria.  
Safety Set (SS)  
All participants who have received at least 1 dose of ALXN2060. 
Full Analysis Set (FAS)  
All participants who have received at least 1 dose of ALXN2060.  
Per-Protocol Set (PPS)  
All FAS participants without major protocol deviations that may affect 
the primary efficacy endpoint.  
Pharmacokinetic Analysis 
Set (PKAS) 
All participants who have received at least 1 dose of ALXN2060 and 
who have at least one evaluable ALXN2060 concentration.  
Pharmacodynamic Analysis 
Set (PDAS) 
All participants who have received at least 1 dose of ALXN2060 and 
who have evaluable pharmacodynamics (PD) data.  
 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 42 of 68 
Alexion Confidential 
9.4. 
Statistical Analyses 
Statistical methods described in this section will be further elaborated in a separate Statistical 
Analysis Plan (SAP). 
All analyses will be performed using SAS® release, version 9.4 or higher (SAS Institute Inc, 
Cary, NC, USA) or other validated statistical software. 
Continuous variables will be summarized using descriptive statistics, including number of 
observations and mean, SD, median, minimum, and maximum values. Categorical variables will 
be summarized by frequency counts and percentage of participants. 
All data and all outcomes derived from the data will be presented in detailed data listings or 
summary tabulations. Graphical displays may also be provided when appropriate. 
9.4.1. 
Efficacy Analyses 
9.4.1.1. 
Analyses of Primary Efficacy Endpoint 
The primary endpoints are the change from Baseline to Month 12 of treatment in distance 
walked during the 6MWT (Part A) and all-cause mortality and CV-related hospitalizations over a 
30-months’ period (Part B). Descriptive statistics will be presented for all the endpoints. The 
mean and its 95% CI will be presented for the change in 6MWT from Baseline to Month 12. In 
addition, summary statistics from the Kaplan-Meier analysis of all-cause mortality at Month 12 
and Month 30 will be provided as point estimates along with 95% CIs. The Kaplan-Meier curve 
will also be plotted. Cumulative frequency of CV-related hospitalization will be estimated using 
negative binomial regression analysis with no covariates but an offset term equal to log of each 
subject’s study duration included in the model. The CV-related hospitalization per person per year at 
Month 30 will be presented with 95% CI. 
Success criterion for Part A is defined as the lower bound of the 95% CI of the change in 6MWT 
from Baseline to Month 12 is greater than -60 m. 
Success criterion for Part B is defined as the estimated survival probability at Month 30 is greater 
than that for placebo patients in Study AG10-301. The success criterion for Part B is based on 
all-cause mortality only, because it is considered more clinically important than CV-related 
hospitalizations. 
9.4.1.2. 
Analyses of Secondary Efficacy Endpoint(s) 
Changes from Baseline in 6MWT and KCCQ-OS will be analyzed using mixed model repeated 
measures (MMRM) model adjusting for baseline measures and visits. 
9.4.1.3. 
Pharmacokinetic/Pharmacodynamic Analysis 
Descriptive statistics will be provided, PK/PD correlation and population PK analyses will be 
explored on ALXN2060 and its metabolite. 
9.4.1.4. 
Analyses of Exploratory Endpoint(s) 
Statistical analysis of the endpoints will depend on the nature of the endpoint and will be 
described in the Statistical Analysis Plan. All-cause mortality at Month 12 will be analyzed using 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 43 of 68 
Alexion Confidential 
Kaplan-Meier method. Summary statistics will be provided as point estimates with 95% CIs. The 
Kaplan-Meier curve will also be plotted. The CV-related hospitalization over 12 months will be 
estimated using negative binomial regression analysis with no covariates. Changes from Baseline 
in NT-proBNP, TnI, cardiac MRI parameters and EQ-5D-5L will be analyzed by MMRM model 
adjusting for baseline measures and visits. 
For the extension period, endpoints will be analyzed from the baseline of the Intervention Period 
to the end of the extension period to assess long term efficacy. 
9.4.2. 
Safety Analyse(s) 
All safety analyses will be made on the Safety Set. The safety and tolerability of ALXN2060 in 
participants with ATTR-CM will be assessed based on AEs, ECG abnormalities, clinical 
laboratory data, physical examinations, and vital sign measurements, and will be presented using 
descriptive statistics. No formal hypothesis testing will be performed for the safety parameters. 
9.5. 
Interim Analyses  
No formal interim analyses are planned for decision making on the continuity of the study. 
Month 12 (Part A) data will be analyzed together at the time of Part B analysis as described in 
Section 9.4. 
9.6. 
Clinical Events Committee 
An independent CEC will review and adjudicate Investigator-reported events of death and CV-
related hospitalizations to determine whether reason for hospitalization and cause of death meet 
the definition of protocol-specified efficacy endpoints for Part B. Investigators will follow the 
SAE notification process (Section 8.4.4). Additional details regarding the responsibilities of the 
CEC are available in the CEC Charter. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 44 of 68 
Alexion Confidential 
10. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS 
10.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight 
Considerations 
10.1.1. 
Regulatory and Ethical Considerations 
• This study will be conducted in accordance with the protocol and with the following: 
− Consensus ethical principles derived from international guidelines including the 
Declaration of Helsinki and Council for International Organizations of Medical 
Sciences (CIOMS) International Ethical Guidelines 
− Applicable ICH Good Clinical Practice (GCP) Guidelines 
− Applicable laws and regulations 
• The protocol, protocol amendments, ICF, Investigator Brochure, and other relevant 
documents (eg, advertisements) must be submitted to an IRB/IEC by the Investigator 
and reviewed and approved by the IRB/IEC before the study is initiated. 
• Any amendments to the protocol will require IRB/IEC approval before 
implementation of changes made to the study design, except for changes necessary to 
eliminate an immediate hazard to study participants. 
• The Investigator will be responsible for the following: 
− Providing written summaries of the status of the study to the IRB/IEC annually or 
more frequently in accordance with the requirements, policies, and procedures 
established by the IRB/IEC 
− Notifying the IRB/IEC of SAEs or other significant safety findings as required by 
IRB/IEC procedures 
− Providing oversight of the conduct of the study at the site and adherence to 
requirements of ICH guidelines, the IRB/IEC, Pharmaceutical and Medical 
Devices (PMD) Act, and all other applicable local regulations 
10.1.2. 
Financial Disclosure 
Investigators and Sub-Investigators will provide Alexion with sufficient, accurate financial 
information as requested to allow Alexion to submit complete and accurate financial certification 
or disclosure statements to the appropriate regulatory authorities. Investigators are responsible 
for providing information on financial interests during the course of the study and for 1 year after 
completion of the study. 
10.1.3. 
Informed Consent Process 
• It is the responsibility of the Investigator to obtain signed (written or electronic 
signature) informed consent from all study participants prior to any study-related 
procedures including screening assessments. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 45 of 68 
Alexion Confidential 
• The Investigator or his/her representative will explain the nature of the study 
(including but not limited to the objectives, potential benefits and risk, 
inconveniences, and the participant’s rights and responsibilities) to the participant or 
his/her legally authorized representative, defined according to local and country 
regulations where the study is taking place, and answer all questions regarding the 
study. 
• Participants must be informed that their participation is voluntary. Participants or 
their legally authorized representative will be required to sign a statement of informed 
consent that meets the requirements of local regulations and ICH guidelines 
requirements, where applicable, and the IRB/IEC or study center. 
• The medical record must include a statement that signed (written or electronic) 
informed consent was obtained before the participant was screened in the study and 
the date the written consent was obtained. The authorized person obtaining the 
informed consent must also sign the ICF(s). 
• Participants must be re-consented to the most current version of the ICF(s) during 
their participation in the study. 
• A copy of the signed (written or electronic) informed consent documentation (ie, a 
complete set of participant information sheets and fully executed signature pages) 
must be provided to the participant or the participant’s legally authorized 
representative, as applicable. This document may require translation into the local 
language. Signed (written or electronic) consent forms must remain in each 
participant’s study file and must be available for verification at any time. 
Participants who are rescreened are required to sign a new ICF (see Section 5.4). 
10.1.4. 
Data Protection 
• Participants will be assigned a unique identifier by Alexion. Any participant records 
or datasets that are transferred to Alexion will contain the identifier only; participant 
names or any information which would make the participant identifiable will not be 
transferred. 
• The participant must be informed that his/her personal study-related data will be used 
by Alexion in accordance with local data protection law. The level of disclosure must 
also be explained to the participant who will be required to give consent for their data 
to be used as described in the informed consent.  
• The participant must be informed that his/her medical records may be examined by 
Clinical Quality Assurance auditors or other authorized personnel appointed by 
Alexion, by appropriate IRB/IEC members, and by inspectors from regulatory 
authorities. 
10.1.5. 
Committees Structure 
The charter for 1 committee (CEC, see Section 9.6) included in this study is available. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 46 of 68 
Alexion Confidential 
10.1.6. 
Dissemination of Clinical Study Data 
Study-related information and study results may be posted on publicly accessible clinical study 
databases (eg, Clinicaltrials.gov. and Japic CTI), as appropriate, and in accordance with national, 
regional, and local regulations. 
10.1.7. 
Data Quality Assurance 
• All participant data relating to the study will be recorded on eCRF unless transmitted 
to Alexion or designee electronically (eg, laboratory data). The Investigator is 
responsible for verifying that data entries are accurate and correct by physically or 
electronically signing the eCRF. 
• The Investigator must maintain accurate documentation (source data) that supports 
the information entered in the eCRF. 
• The Investigator must permit study-related monitoring, audits, IRB/IEC review, and 
regulatory agency inspections and provide direct access to source data documents. 
• Alexion or designee is responsible for the data management of this study including 
quality checking of the data. 
• Study monitors will perform ongoing source data verification to confirm that data 
entered into the eCRF by authorized site personnel are accurate, complete, and 
verifiable from source documents; that the safety and rights of participants are being 
protected; and that the study is being conducted in accordance with the currently 
approved protocol and any other study agreements, ICH GCP, and all applicable 
regulatory requirements. 
• Records and documents, including signed ICFs, pertaining to the conduct of this 
study must be retained by the Investigator for 8 to 10 years after the last marketing 
application approval, or if not approved, 3 years following the discontinuance of the 
study intervention, unless local regulations or institutional policies require a longer 
retention period. No records may be destroyed during the retention period without the 
written approval of Alexion. No records may be transferred to another location or 
party without written notification to Alexion. 
10.1.8. 
Source Documents 
• Source documents provide evidence for the existence of the participant and 
substantiate the integrity of the data collected. The Investigator or designee will 
prepare and maintain adequate and accurate source documents (eg, medical records, 
ECGs, AE and concomitant medication reporting, raw data collection forms) 
designed to record all observations and other pertinent data for each participant. 
Data reported on the eCRF that are transcribed from source documents must be consistent with 
the source documents or the discrepancies must be explained. The Investigator may need to 
request previous medical records or transfer records, depending on the study. Also, current 
medical records must be available. Source documents are filed at the Investigator’s site. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 47 of 68 
Alexion Confidential 
10.1.9. 
Study and Site Start and Closure 
The study start date is the date on which the first participant is consented. 
Alexion reserves the right to close the study site or terminate the study at any time for any reason 
at the sole discretion of Alexion. Study sites will be closed after the study is completed or 
following the decision to close or terminate the study. A study site is considered closed when all 
participants have completed the end of study or early discontinuation visit, all data has been 
collected, entered and cleaned in EDC, all required documents and study supplies have been 
collected, and a study-site closure visit has been performed. 
The Investigator may initiate study-site closure at any time, provided there is reasonable cause 
and sufficient notice is given in advance of the intended termination. 
Reasons for the early closure of a study site by Alexion or Investigator may include but are not 
limited to: 
• Failure of the Investigator to comply with the protocol, the requirements of the 
IRB/IEC or local health authorities, Alexion's procedures, or GCP guidelines 
• Inadequate recruitment of participants by the Investigator 
• Discontinuation of further study intervention development 
If the study is prematurely terminated or suspended, Alexion shall promptly inform the 
Investigators, the IECs/IRBs, the regulatory authorities, and any contract research 
organization(s) used in the study of the reason for termination or suspension, as specified by the 
applicable regulatory requirements. The Investigator shall promptly inform the participant and 
should assure appropriate participant therapy and/or follow-up. 
10.1.10. 
Publication Policy 
• Where possible, primary manuscripts reporting results of the primary efficacy 
endpoint or the final results will be submitted for publication within 12-18 months of 
the primary evaluation date or end of study, whichever is earlier. 
• Investigators who participate as authors in manuscripts derived from Alexion-
sponsored studies will agree to the prerequisites as outlined in the Alexion author 
engagement agreement prior to engaging in manuscript development. 
• The Investigator agrees to submit proposals for new manuscripts (whether or not the 
proposed analyses are derived from protocol-specified endpoints) to Alexion for 
review and consideration. All manuscripts or abstracts emanating from approved 
proposals are to be submitted to Alexion for review before submission to the 
journal/society. Alexion will ask Eidos, the licensor of ALXN2060 (Code number at 
Eidos: AG10), for the review of it based on the licensing agreement between them. 
This allows Alexion and Eidos to protect proprietary information and to provide 
comments. 
− The proprietary nature of some development work may preclude publication. In 
some cases, it may be necessary to delay a publication to allow Alexion to ensure 
protection of intellectual property. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 48 of 68 
Alexion Confidential 
• In general, primary publications, including congress and journal publications, 
containing the protocol-specified results of a study should occur prior to the 
publication of individual study site results or case reports. Alexion’s policy prohibits 
duplicate publication, whereby the same results must not be published in multiple 
peer-reviewed journal manuscripts. 
− Encore congress publications may be appropriate to allow communication of 
research findings to relevant audience and geographical regions. 
• Alexion will comply with the requirements for publication of study results. In 
accordance with standard editorial and ethical practice, Alexion will generally 
support publication of multicenter studies only in their entirety and not as individual 
site data. In this case, a coordinating Investigator will be designated by mutual 
agreement. 
• Authorship will be determined by mutual agreement and in line with International 
Committee of Medical Journal Editors authorship requirements and per the Alexion 
Publication Policy. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 49 of 68 
Alexion Confidential 
10.2. 
Appendix 2: Clinical Laboratory Tests 
• The tests detailed below will be performed by the central laboratory. 
• Protocol-specific requirements for inclusion or exclusion of participants are detailed 
in Section 5. 
• Additional tests may be performed at any time during the study as determined 
necessary by the Investigator or required by local regulations. 
• Pregnancy Testing: WOCBP should only be enrolled after a negative urine pregnancy 
test result at screening. Additional urine pregnancy testing will be standard for the 
protocol unless serum testing is required by site policies, local regulation, or IRB/IEC 
and should be performed per the time points specified in the SoA (Section 1.3).] 
Table 3: 
Protocol-Required Clinical Laboratory Assessments  
Laboratory 
Assessments 
Parameters 
Hematology 
Hemoglobin, hematocrit, white blood cell (WBC) count, platelet count, complete 
blood count (CBC), and differential 
Chemistry 
Sodium, potassium, chloride, carbon dioxide (bicarbonate), glucose, blood urea 
nitrogen (BUN), creatinine, aspartate aminotransferase (AST), alanine 
aminotransferase (ALT), total protein, albumin, prealbumin, retinol-binding 
protein (RBP), free thyroxine (FT4), alkaline phosphatase, calcium, phosphorus, 
total and fractionated (indirect or direct) bilirubin, uric acid, thyroid-stimulating 
hormone (TSH), troponin I (Tn I), creatine kinase (CK), CK-MB, and N-terminal 
pro-brain-type natriuretic peptide (NT-proBNP) 
Urinalysis 
Complete urinalysis (specific gravity, pH, glucose, protein, hemoglobin, leukocyte 
esterase, and nitrite. Additionally, albumin to creatinine ratio and a microscopic 
urinalysis will be performed on every specimen and will specifically look for 
casts, bacteria, WBCs, epithelial cells, and red blood cells 
Others 
Follicle-stimulating hormone (FSH) only to confirm post-menopausal status at 
Screening in female subjects who do not have menses for at least 12 months and 
are not using hormonal contraception or hormone replacement therapy. A 
postmenopausal state is defined as no menses for 12 months without an alternative 
medical cause 
Pregnancy test 
Highly sensitive urine test at all visits as outlined in the Schedule of Assessments 
(female subjects of childbearing potential only) 
Investigators must document their review of each laboratory safety report. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 50 of 68 
Alexion Confidential 
10.3. 
Appendix 3: Adverse Events: Definitions and Procedures for 
Recording, Evaluating, Follow-up, and Reporting 
10.3.1. 
Definition of AE 
AE Definition 
• 
An AE is any untoward medical occurrence in a participant, temporally associated with the 
use of study intervention, whether or not considered related to the study intervention. 
• 
NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal 
laboratory finding), symptom, or disease (new or exacerbated) temporally associated with 
the use of study intervention. 
 
Events Meeting the AE Definition  
• 
Any abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other 
safety assessments (eg, ECG, radiological scans, vital signs measurements), including those 
that worsen from baseline, considered clinically significant in the medical and scientific 
judgment of the Investigator (ie, not related to progression of underlying disease). 
• 
Exacerbation of a chronic or intermittent pre-existing condition including either an increase in 
frequency and/or intensity of the condition. 
• 
New conditions detected or diagnosed after study intervention administration even though it 
may have been present before the start of the study. 
• 
Signs, symptoms, or the clinical sequelae of a suspected drug-drug interaction. 
• 
Signs, symptoms, or the clinical sequelae of a suspected overdose of either study intervention 
or a concomitant medication. Overdose per se will not be reported as an AE/SAE unless it is 
an intentional overdose taken with possible suicidal/self-harming intent. Such overdoses 
should be reported regardless of sequelae. 
• 
“Lack of efficacy” or “failure of expected pharmacological action” per se will not be reported 
as an AE or SAE. Such instances will be captured in the efficacy assessments. However, the 
signs, symptoms, and/or clinical sequelae resulting from lack of efficacy will be reported as 
AE or SAE if they fulfil the definition of an AE or SAE.  
 
Events NOT Meeting the AE Definition  
• 
Medical or surgical procedure (eg, endoscopy, appendectomy): the condition that leads to the 
procedure is the AE. Situations in which an untoward medical occurrence did not occur (eg, 
hospitalization for elective surgery if planned before the signing the ICF, admissions for 
social reasons or for convenience). 
• 
Anticipated day-to-day fluctuations of pre-existing disease(s) or condition(s) present or 
detected at the start of the study that do not worsen. 
• 
A medication error (including intentional misuse, abuse, and overdose of the product) or use 
other than what is defined in the protocol is not considered an AE unless there is an untoward 
medical occurrence as a result of a medication error. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 51 of 68 
Alexion Confidential 
Events NOT Meeting the AE Definition  
• 
Cases of pregnancy that occur during maternal or paternal exposure to study intervention are 
to be reported within 24 hours of Investigator/site awareness. Data on fetal outcome and 
breastfeeding will be collected for regulatory reporting and safety evaluation. 
• 
Any clinically significant abnormal laboratory findings or other abnormal safety assessments 
which are associated with the underlying disease, unless judged by the Investigator to be more 
severe than expected for the participant’s condition. 
• 
The disease/disorder being studied or expected progression, signs, or symptoms of the 
disease/disorder being studied, unless more severe than expected for the participant’s 
condition. 
• 
Situations in which an untoward medical occurrence did not occur (social and/or convenience 
admission to a hospital). 
10.3.2. 
Definition of SAE 
If an event is not an AE per definition above, then it cannot be an SAE even if serious conditions 
are met (eg, hospitalization for signs/symptoms of the disease under study, death due to 
progression of disease). 
A SAE is defined as any untoward medical occurrence that, at any dose: 
1. Results in death 
2. Is life-threatening 
The term 'life-threatening' in the definition of 'serious' refers to an event in which the participant 
was at risk of death at the time of the event. It does not refer to an event, which hypothetically 
might have caused death, if it were more severe. 
3. Requires inpatient hospitalization or prolongation of existing hospitalization 
In general, hospitalization signifies that the participant has been detained (usually involving at 
least an overnight stay) at the hospital or emergency ward for observation and/or treatment that 
would not have been appropriate in the physician’s office or outpatient setting. Complications 
that occur during hospitalization are AEs. If a complication prolongs hospitalization or fulfills 
any other serious criteria, the event is serious. When in doubt as to whether “hospitalization” 
occurred or was necessary, the AE should be considered serious. 
Hospitalization for elective treatment of a pre-existing condition that did not worsen from 
baseline is not considered an AE. 
4. Results in persistent disability/incapacity 
• 
The term disability means a substantial disruption of a person’s ability to conduct normal life 
functions. 
• 
This definition is not intended to include experiences of relatively minor medical 
significance such as uncomplicated headache, nausea, vomiting, diarrhea, influenza, and 
accidental trauma (eg, sprained ankle) which may interfere with or prevent everyday life 
functions but do not constitute a substantial disruption. 
5. Is a congenital anomaly/birth defect 
6. Other situations: 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 52 of 68 
Alexion Confidential 
A SAE is defined as any untoward medical occurrence that, at any dose: 
• 
Medical or scientific judgment should be exercised in deciding whether SAE reporting is 
appropriate in other situations such as important medical events that may not be immediately 
life-threatening or result in death or hospitalization but may jeopardize the participant or 
may require medical or surgical intervention to prevent one of the other outcomes listed in 
the above definition. These events should usually be considered serious. 
• 
Examples of such events include invasive or malignant cancers, intensive treatment in an 
emergency room or at home for allergic bronchospasm, blood dyscrasias or convulsions that 
do not result in hospitalization, or development of drug dependency or drug abuse. 
10.3.3. 
Recording and Follow-Up of AE and/or SAE 
AE and SAE Recording 
• 
When an AE/SAE occurs, it is the responsibility of the Investigator to review all 
documentation (eg, hospital progress notes, laboratory reports, and diagnostics reports) 
related to the event. 
• 
The Investigator will then record all relevant AE/SAE information in the eCRF. 
• 
It is not acceptable for the Investigator to send photocopies of the participant's medical 
records to Alexion in lieu of completion of the AE/SAE eCRF page. 
• 
There may be instances when copies of medical records for certain cases are requested by 
Alexion. In this case, all participant identifiers, with the exception of the participant number, 
will be redacted on the copies of the medical records before submission to Alexion. 
• 
The Investigator will attempt to establish a diagnosis of the event based on signs, symptoms, 
and/or other clinical information. Whenever possible, the diagnosis (not the individual 
signs/symptoms) will be documented as the AE/SAE. 
Assessment of Intensity 
The Investigator will make an assessment of intensity for each AE and SAE reported during the 
study and assign it to one of the following categories from National Cancer Institute Common 
Terminology Criteria for Adverse Events (CTCAE) v5.0, published 27 Nov 2017:  
• 
Grade 1: Mild (awareness of sign or symptom, but easily tolerated) 
• 
Grade 2: Moderate (discomfort sufficient to cause interference with normal activities) 
• 
Grade 3: Severe (incapacitating, with inability to perform normal activities) 
• 
Grade 4: Life-threatening 
• 
Grade 5: Fatal 
 
Assessment of Causality 
• 
The Investigator is obligated to assess the relationship between the study intervention and 
each occurrence of each AE or SAE. An Investigator causality assessment must be provided 
for all AEs (both nonserious and serious). This assessment must be recorded in the eCRF and 
on any additional forms, as appropriate. The definitions for the causality assessments are as 
follows: 
− 
Not related: There is no reasonable possibility the study intervention caused the 
adverse event.  

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 53 of 68 
Alexion Confidential 
Assessment of Causality 
 
The adverse event has a more likely alternative etiology; it may be due to 
underlying or concurrent illness, complications, concurrent treatments, or 
effects of another concurrent drug. 
 
The event does not follow a reasonable temporal relationship to 
administration of the study intervention. 
− 
Related: There is a reasonable possibility the study intervention caused the 
adverse event. 
 
The adverse event has a temporal relationship to the administration of the 
study intervention. 
 
The event does not have a likely alternative etiology. 
 
The event corresponds with the known pharmaceutical profile of the 
study intervention. 
 
There is improvement on discontinuation and/or reappearance on 
rechallenge. 
• 
The Investigator will use clinical judgment to determine the relationship. 
• 
Alternative causes, such as underlying disease(s), concomitant therapy, and other risk 
factors, as well as the temporal relationship of the event to study intervention administration 
will be considered and investigated. 
• 
The Investigator will also consult the Investigator’s Brochure (IB) and/or Product 
Information, for marketed products, in his/her assessment. 
• 
For each AE/SAE, the Investigator must document in the medical notes that he/she has 
reviewed the AE/SAE and has provided an assessment of causality. 
• 
There may be situations in which an SAE has occurred, and the Investigator has minimal 
information to include in the initial report. However, it is very important that the Investigator 
always make an assessment of causality for every event before the initial transmission of the 
SAE data. 
• 
The Investigator may change his/her opinion of causality in light of follow-up information 
and send a SAE follow-up report with the updated causality assessment. 
• 
The causality assessment is one of the criteria used when determining regulatory reporting 
requirements. 
Follow-up of AEs and SAEs 
• 
The Investigator is obligated to perform or arrange for the conduct of supplemental 
measurements and/or evaluations as medically indicated or as requested by Alexion to 
elucidate the nature and/or causality of the AE or SAE as fully as possible. This may 
include additional laboratory tests or investigations, histopathological examinations, or 
consultation with other health care professionals. 
• 
If a participant dies during participation in the study or during a recognized follow-up 
period, the Investigator will provide Alexion with a copy of any post-mortem findings 
including histopathology.  
• 
New or updated information will be recorded in the originally completed eCRF. 
• 
The Investigator will submit any updated SAE data to Alexion within 24 hours of receipt of 
the information. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 54 of 68 
Alexion Confidential 
10.3.4. 
Reporting of SAEs  
SAE Reporting to Alexion via an Electronic Data Collection Tool 
• 
All SAEs will be recorded and reported to Alexion or designee immediately and within 24 
hours of awareness, 
• 
The primary mechanism for reporting an SAE to Alexion will be the electronic data 
collection tool. 
• 
If the electronic system is unavailable at the time that the Investigator or site becomes 
aware of an SAE, the site will use the paper Contingency Form for SAE reporting via fax or 
email. Facsimile transmission or email may be used in the event of electronic submission 
failure. 
− 
Email: clinicalsae@alexion.com or Fax: + 1.203.439.9347 
• 
The site will enter the SAE data into the electronic data capture (EDC) system as soon as it 
becomes available. 
• 
When further information becomes available, the EDC system should be updated within 24 
hours with the new information and an updated SAE report should be submitted to Alexion 
global drug safety (GDS) via the RAVE Safety Gateway. 
• 
After the study is completed at a given site, the electronic data collection tool will be taken 
off-line to prevent the entry of new data or changes to existing data. 
• 
If a site receives a report of a new SAE from a study participant or receives updated data on 
a previously reported SAE after the electronic data collection tool has been taken off-line, 
then the site can report this information on a paper SAE form or to Alexion GDS by 
telephone. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 55 of 68 
Alexion Confidential 
10.4. 
Appendix 4: Contraceptive Guidance and Collection of Pregnancy 
Information 
10.4.1. 
Definitions 
Woman of Childbearing Potential (WOCBP) 
A woman is considered fertile following menarche and until becoming post-menopausal unless 
permanently sterile (see below). 
If fertility is unclear (eg, amenorrhea in adolescents or athletes) and a menstrual cycle cannot be 
confirmed before first dose of study intervention, additional evaluation should be considered. 
Women in the following categories are not considered WOCBP 
1. Premenarchal 
2. Premenopausal female with 1 of the following: 
• Documented hysterectomy 
• Documented bilateral salpingectomy 
• Documented bilateral oophorectomy 
For individuals with permanent infertility due to an alternate medical cause other than the above, 
(eg, mullerian agenesis, androgen insensitivity), Investigator discretion should be applied to 
determining study entry. 
Note: Documentation can come from the site personnel’s: review of the participant’s medical 
records, medical examination, or medical history interview. 
3. Postmenopausal female 
• A postmenopausal state is defined as no menses for 12 months without an alternative 
medical cause. 
− A high follicle stimulating hormone (FSH) level in the postmenopausal range may 
be used to confirm a postmenopausal state in women not using hormonal 
contraception or hormonal replacement therapy (HRT). However, in the absence 
of 12 months of amenorrhea, confirmation with more than one FSH measurement 
is required. 
• Females on HRT and whose menopausal status is in doubt will be required to use one 
of the non-estrogen hormonal highly effective contraception methods if they wish to 
continue their HRT during the study. Otherwise, they must discontinue HRT to allow 
confirmation of postmenopausal status before study enrollment. 
10.4.2. 
Contraception Guidance: 
10.4.2.1. Guidance for Female Participants 
Female participants of non-child bearing potential are exempt from contraception requirements. 
Non-child bearing potential for female participants is defined as any of the following: 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 56 of 68 
Alexion Confidential 
1. Prior to first menses 
2. Postmenopausal, as documented by amenorrhea for at least 1 year prior to the Day 1 visit 
and follicle stimulating hormone (FSH) serum levels consistent with postmenopausal 
status 
3. Permanent sterilization at least 6 weeks prior to the Day 1 visit: 
4. Hysteroscopic sterilization 
5. Bilateral tubal ligation or bilateral salpingectomy 
6. Hysterectomy 
7. Bilateral oophorectomy 
Female participants of child-bearing potential must use a highly effective method of 
contraception, including at least one of the following: 
1. Intrauterine device (without copper) in place for at least 6 weeks 
2. Progestogen-only oral hormonal contraception for at least 6 weeks 
3. Intrauterine progestogen releasing system for at least 6 weeks 
4. Bilateral tubal occlusion for at least 6 weeks 
5. Combined (estrogen and progestogen containing) hormonal oral contraception for at least 
6 weeks. Estrogen-containing hormonal contraception is acceptable only if it has been 
used for at least 6 weeks immediately prior to the Day 1 visit. Estrogen-containing 
hormonal contraception may not be initiated during the study period; 
6.  Surgical sterilization of the male partner (medical assessment of azoospermia is required 
if vasectomy was performed within the prior 6 months) 
7. Sexual abstinence for female participants 
a. Sexual abstinence is considered a highly effective method only if defined as 
refraining from heterosexual intercourse. In this study, abstinence is only acceptable 
if consistent with the participant’s preferred and usual lifestyle. Abstinent female 
participants who wish to initiate a highly effective method of contraception during the 
study must refrain from heterosexual intercourse. 
b. Periodic abstinence (eg, calendar, symptothermal, or post-ovulation methods) is not 
considered a highly effective method of contraception for female participants. 
Other methods of contraception that are not considered highly effective for female participants: 
1. Barrier methods, such as male or female condoms, diaphragm, or cervical cap, used alone 
or in combination, are not acceptable. 
2. Spermicides or spermicidal sponges, used alone or in combination with barrier methods, 
are not acceptable. 
Withdrawal (coitus interruptus) is not acceptable. 
Lactational amenorrhea is not acceptable. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 57 of 68 
Alexion Confidential 
Female participants must not donate ova from the Day 1 up to 30 days after the last dose of the 
study drug. 
10.4.2.2. Guidance for Male Participants 
Contraception is the responsibility of the heterosexually active male participants, regardless of 
his female partner’s method of contraception. 
Male participants who have not had a vasectomy must agree to use a barrier method of birth 
control. 
10.4.2.2.1. Sexual Abstinence for Male Participants 
Sexual abstinence is considered a highly effective method only if defined as refraining from 
heterosexual intercourse. In this study, abstinence is only acceptable if consistent with the 
participants’ s preferred and usual lifestyle. Abstinent male participants who become 
heterosexually active must use a condom and spermicide during intercourse. 
Periodic abstinence (eg, calendar, symptothermal, or post-ovulation methods for a female 
partner) is not considered a highly effective method of contraception for male participants. 
Male participants must not donate sperm from the Day 1 to at least 90 days after the last dose of 
study drug. 
CONTRACEPTIVESa ALLOWED DURING THE STUDY INCLUDE: 
Highly Effective Methodsb That Have Low User Dependency Failure rate of < 1% per year when used 
consistently and correctly. 
Implantable progestogen-only hormone contraception associated with inhibition of ovulation 
Intrauterine device (IUD) 
Intrauterine hormone-releasing system (IUS) 
Bilateral tubal occlusion 
• Vasectomized partner 
(Vasectomized partner is a highly effective contraceptive method provided that the partner is the sole sexual 
partner of the woman of childbearing potential and the absence of sperm has been confirmed. If not, an 
additional highly effective method of contraception should be used. Spermatogenesis cycle is approximately 
90 days.) 
• 
Highly Effective Methods b That Are User Dependent Failure rate of < 1% per year when used 
consistently and correctly. 
• 
Combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of 
ovulation 
• 
Oral 
• 
Progestogen-only hormone contraception associated with inhibition of ovulation 
• 
Oral 
• 
Sexual abstinence 
(Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual 
intercourse during the entire period of risk associated with the study intervention. The reliability of sexual 
abstinence needs to be evaluated in relation to the duration of the study and the preferred and usual lifestyle of 
the participant.) 
• 
a) Contraceptive use by men or women should be consistent with local regulations regarding the use of 
contraceptive methods for those participating in clinical studies. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 58 of 68 
Alexion Confidential 
• 
b) Failure rate of < 1% per year when used consistently and correctly. Typical use failure rates differ from 
those when used consistently and correctly. 
Note: Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), 
spermicides only, and lactational amenorrhoea method (LAM) are not acceptable methods of contraception. 
Male condom and female condom should not be used together (due to risk of failure with friction) 
10.4.3. 
Collection of Pregnancy Information 
Pregnancy data will be collected from Day 1 through 30 days after the last dose of study drug for 
all female participants and female spouses/partners of male participants. Exposure during 
pregnancy (also referred to as exposure in utero) can be the result of either maternal exposure or 
transmission of drug product via semen following paternal exposure. If a female participant or a 
male participant’s female partner becomes pregnant during the conduct of this study, the 
Investigator must submit the “Pregnancy/Breastfeeding Reporting and Outcome Form to Alexion 
GDS via facsimile or email. When the outcome of the pregnancy becomes known, the form 
should be updated and submitted to Alexion GDS. If additional follow-up is required, the 
Investigator will be requested to provide the information. 
Exposure of an infant to an Alexion product during breastfeeding must also be reported (via the 
Pregnancy/Breastfeeding Reporting and Outcome Form) and any AEs experienced by the infant 
must be reported to Alexion GDS or designee via email or facsimile. 
Pregnancy is not regarded as an AE unless there is a suspicion that the study intervention may 
have interfered with the effectiveness of a contraceptive medication. However, complications of 
pregnancy and abnormal outcomes of pregnancy are AEs and may meet the criteria for an SAE 
(eg, ectopic pregnancy, spontaneous abortion, intrauterine fetal demise, neonatal death, or 
congenital anomaly). Elective abortions without complications should not be reported as AEs. 
Any female participant who becomes pregnant while participating in the study will be 
discontinued from study intervention. 
10.4.3.1. Male Participants with Partners Who Become Pregnant 
The Investigator will attempt to collect pregnancy information on any male participant’s female 
partner who becomes pregnant while the male participant is in this study. This applies only to 
male participants who receive ALXN2060. 
• After obtaining the necessary signed informed consent from the pregnant female 
partner directly, the Investigator will record pregnancy information on the appropriate 
Pregnancy/Breastfeeding Reporting and Outcome Form and submit it to Alexion 
within 24 hours of learning of the partner’s pregnancy. The female partner will also 
be followed to determine the outcome of the pregnancy. Information on the status of 
the mother and child will be forwarded to Alexion. Generally, the follow-up will be 3 
months following the estimated delivery date. Any termination of the pregnancy will 
be reported regardless of fetal status (presence or absence of anomalies) or indication 
for the procedure. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 59 of 68 
Alexion Confidential 
10.4.3.2. Female Participants who Become Pregnant 
• The Investigator will collect pregnancy information on any female participant who 
becomes pregnant while participating in this study. The initial Information will be 
recorded on the appropriate form and submitted to Alexion within 24 hours of 
learning of a participant's pregnancy. 
• The participant will be followed to determine the outcome of the pregnancy. The 
Investigator will collect follow-up information on the participant and the neonate and 
the information will be forwarded to Alexion. Generally, follow-up will be 3 months 
following the estimated delivery date. Any termination of pregnancy will be reported, 
regardless of fetal status (presence or absence of anomalies) or indication for the 
procedure.  
• While pregnancy itself is not considered to be an AE or SAE, any pregnancy 
complication or elective termination of a pregnancy for medical reasons will be 
reported as an AE or SAE. A spontaneous abortion (occurring at < 22 weeks 
gestational age) or still birth (occurring at > 22 weeks gestational age) is always 
considered to be an SAE and will be reported as such. Any post-study pregnancy 
related SAE considered reasonably related to the study intervention by the 
Investigator will be reported to Alexion as described in Section 8.4.4. While the 
Investigator is not obligated to actively seek this information in former study 
participants, he or she may learn of an SAE through spontaneous reporting. 
 
 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 60 of 68 
Alexion Confidential 
10.5. 
Appendix 5: COVID-19 Risk Assessment 
ATTR-CM can cause irreversible morbidity and even mortality, if untreated. As such, and due to 
the limited number of available treatment options, the benefit a participant may receive from 
joining an investigational study with a therapeutic treatment is potentially significant. In this 
particular case, the fact that the study is open-label and every participant is treated with the study 
intervention also contributes to the potential benefit a participant may derive from partaking in 
the study. The site Investigator will therefore balance the risk/benefit considerations in the study 
participant taking these factors into account.  
The potential risks identified and mitigation measures put in place in light of the COVID-19 
pandemic are provided in Table 4. 
Table 4: 
Potential Risks and Mitigation Measures due to COVID-19 
Risks category 
Summary of Data/ 
Rationale for Risk 
Mitigation Strategy 
Potential risks 
Healthcare 
institution 
availability for 
non-COVID-19 
related activities  
COVID-19 pandemic may impact the workload 
of healthcare institutions globally and may 
reduce staff availability to perform non-urgent 
activities and non-COVID-19 related activities. 
 
During the time that the 
COVID-19 pandemic is 
active, Alexion will not open 
study sites or enroll new 
participants at sites unless 
the sites have the resourcing 
and capabilities to implement 
the study per protocol. 
Data quality and 
integrity 
Lack of availability of site personnel to perform 
study assessments and capture study specific 
data in a timely manner and to maintain 
adequate quality standards. 
Lack of availability of site personnel to ensure 
adequate and continuous chain of custody, 
storage conditions, and monitoring for 
investigational product and biological samples. 
Inability of study monitors and quality 
personnel to conduct in-person visits to exercise 
adequate oversight of study execution at 
investigational sites. 
Missing data (COVID-19 pandemic may impact 
study visit schedules, and increase missed visits 
and/or participant study discontinuations 
inadvertently resulting in missing data [eg, for 
protocol-specified procedures]). 
During the time that the 
COVID-19 pandemic is 
active, Alexion will only 
open study sites that report 
enough personnel capacity to 
sufficiently conduct clinical 
study-related activities.  
Each site is also evaluated 
for the capacity to perform 
remote monitoring visits and 
remote source data 
verification.  
During the time that the 
COVID-19 pandemic is 
active, it will be important to 
capture specific information 
in the eCRF that explains the 
reason the data is missing 
(eg, missed study visits or 
participant study 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 61 of 68 
Alexion Confidential 
Table 4: 
Potential Risks and Mitigation Measures due to COVID-19 
Risks category 
Summary of Data/ 
Rationale for Risk 
Mitigation Strategy 
discontinuations due to 
COVID-19). 
Abbreviation: COVID-19 = coronavirus disease 2019. 
 
 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 62 of 68 
Alexion Confidential 
10.6. 
Appendix 6: Abbreviations 
 
Abbreviation 
Definition 
6MWT 
six-minute walk test 
AE 
adverse event 
AL amyloidosis 
light-chain amyloidosis 
ATTR 
TTR amyloidosis 
ATTR-CM 
transthyretin amyloid cardiomyopathy 
ATTRm 
mutated ATTR 
ATTR-PN 
ATTR peripheral polyneuropathy 
ATTRwt 
wild-type ATTR 
CEC 
Clinical Events Committee 
CI 
confidence interval 
CMR 
cardiac magnetic resonance 
COVID-19 
coronavirus disease 2019 
ECHO 
echocardiogram  
eCRF 
electronic case report form 
EQ-5D-5L 
European Quality of Life Health 5-item questionnaire dimensions 5 level 
FPE 
fluorescent probe exclusion  
HRT 
hormonal replacement therapy  
ICF 
Informed Consent Form  
IEC 
Independent Ethics Committee 
IRB 
Institutional Review Board 
KCCQ-OS  
Kansas City Cardiomyopathy Questionnaire Overall Score 
LV 
left ventricular 
MRI 
magnetic resonance imaging 
NT-proBNP 
N-terminal pro-brain-type natriuretic peptide 
NYHA 
New York Heart Association  
PD 
Pharmacodynamic 
PK 
Pharmacokinetic 
SAE 
serious adverse event 
TIA 
transient ischemic attack  
TTR 
transthyretin  
WOCBP 
women of childbearing potential  

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 63 of 68 
Alexion Confidential 
10.7. 
Appendix 7: Protocol Amendment History 
The Protocol Amendment Summary of Changes Table for the current amendment is located 
directly before the Table of Contents (TOC). 
Amendment 4.0 (12-January-2023) 
Overall Rationale for the Amendment: 
The main purpose of this amendment was to align the terminology definitions with Study AG10-
301 Protocol Amendment 6.0 and to align the timing of study visits in the Extension Period with 
Study AG10-304.  
Other changes implemented through this Amendment constituted minor editorial corrections, 
fixing inconsistencies, and clarification. 
Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis, 3 Objectives 
and Endpoints 
Clarified the Extension Period objectives 
and endpoints in the table.  
For clarification. 
1.3 Schedule of Activities 
Added quarterly phone contacts to the 
Extension Period. 
Changed the visit timing in the Extension 
Period from every 3 months to every 6 
months. 
Added KCCQ and EQ-5D-5L 
assessments to Month 1. 
To align with Study AG10-
304. 
1.3 Schedule of Activities 
Added a footnote for vital status to be 
collected during follow-up. 
To align with Study AG10-
301. 
6.5.2 Disallowed Medicine 
and Therapy 
Updated text to prohibit the use of 
tafamidis and other drugs for the 
treatment of ATTR-CM during the study. 
For clarification. 
7.1.1 Temporary 
Discontinuation 
Added a section for IMP interruption. 
To align with Study AG10-
301. 
7.2 Participant 
Discontinuation/Withdrawal 
from the Study 
Added text that vital status to be collected 
include dead, alive, heart transplant, 
receiving CMAD.  
To align with Study AG10-
301. 
8.2.2 Mortality and 
Cardiovascular-Related 
Hospitalization 
Added text to indicate mortality includes 
CMAD and heart transplant. 
To align with Study AG10-
301. 
9.4.1.1 Analyses of Primary 
Efficacy Endpoint 
Added the following text for analysis plan 
clarification:  
Cumulative frequency of CV-related 
hospitalization will be estimated using 
negative binomial regression analysis 
with no covariates but an offset term 
equal to log of each subject’s study 
duration included in the model. The CV-
For clarification. 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 64 of 68 
Alexion Confidential 
Section # and Name 
Description of Change 
Brief Rationale 
related hospitalization per person per year 
at Month 30 will be presented with 95% 
CI. 
9.4.1.2 Analyses of 
Secondary Efficacy 
Endpoint(s) 
Updated the following text for analysis 
plan clarification: 
Changes from Baseline in 6MWT and 
KCCQ-OS will be analyzed using mixed 
model repeated measures (MMRM) 
model adjusting for baseline measures 
and visits. 
For clarification. 
9.4.1.4 Analyses of 
Exploratory Endpoint(s) 
Updated the following text for analysis 
plan clarification: 
The CV-related hospitalization over 12 
months will be estimated using negative 
binomial regression analysis with no 
covariates. Changes from Baseline in NT-
proBNP, TnI, cardiac MRI parameters 
and EQ-5D-5L will be analyzed by 
MMRM model adjusting for baseline 
measures and visits. 
Added the following text for clarification:  
For the extension period, endpoints will 
be analyzed from the baseline of the 
Intervention Period to the end of the 
extension period to assess long term 
efficacy. 
For clarification. 
9.5 Interim Analyses 
Clarified that Part A analysis will be 
performed at the same time as Part B 
analysis.  
To accurately reflect the 
current plan.  
Amendment 3 (15-February-2021) 
Overall Rationale for the Amendment: 
The main purpose of this amendment was to add an exploratory endpoint (cardiac MRI) and to 
add text referring to COVID-19 considerations. 
Other changes implemented through this Amendment constituted minor editorial corrections and 
fixing inconsistencies. 
Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis 
Changed from 28days +-7 to 35 days 
To correct the text 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 65 of 68 
Alexion Confidential 
Section # and Name 
Description of Change 
Brief Rationale 
1.1 Synopsis 
1.3 Schedule of Activities 
3 OBJECTIVES AND 
ENDPOINTS 
8.2.6 Cardiac MRI 
9.4.1.4 Analysis of 
Exploratory Endpoint(s) 
 
Added an explanation of cardiac MRI. 
Measurement of cardiac 
MRI will be added as an 
exploratory endpoint to 
evaluate the effects of 
ALXN2060 on cardiac 
MRI- derived measures of 
cardiac structure, function 
and tissue characterization.  
1.3 Schedule of Activities 
8.6 Pharmacokinetics 
8.7 Pharmacodynamics 
Added text referring to PK/PD backup 
samples as exploratory biomarkers. 
PK/PD backup samples may 
be used to measure 
exploratory biomarkers. 
2.3.1.1 Coronavirus 
Disease 2019 
10.5 Appendix 5: COVID- 
19 Risk Assessment 
Added text referring to COVID-19 
considerations. 
To allow treatment 
continuity and ensure 
patients safety during 
COVID-19 pandemic 
10.2 Appendix 2: Clinical 
Laboratory Tests 
Changed from “serum” to “urine” 
To correct the text 
10.4.2.2 Guidance for 
Male Participants 
Changed from “have had a vasectomy” to 
“have not had a vasectomy”  
To correct the text 
Amendment 2 (24-July-2020) 
Overall Rationale for the Amendment: 
The main purpose of this amendment was minor changes for consistency. 
Section # and Name 
Description of Change 
Brief Rationale 
TITLE PAGE 
Removed “(previously AG10)” 
Cosmetic change 
1.3 Schedule of Activities 
(SoA) 
“Dispense participant safety card” was 
deleted 
Not to use “Dispense 
participant safety card” in 
this study 
5.1 Inclusion Criteria 9. 
Corrected description of period of 
contraception for female 
To correct description 
Amendment 1 (20-July-2020) 
Overall Rationale for the Amendment: 
The main purpose of this amendment was to respond to inquiries from PMDA on contraception 
guidance for male and TTR genotyping. 
In addition, minor editorial changes were implemented for consistency. 
Section # and Name 
Description of Change 
Brief Rationale 
1.2 Schema 
“AG10 800 mg b.i.d” was changed to 
“ALXN2060 800 mg b.i.d”. 
To keep consistency of the 
study drug name 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 66 of 68 
Alexion Confidential 
Section # and Name 
Description of Change 
Brief Rationale 
1.3 Schedule of Activities 
(SoA) 
7.2 Participant 
Discontinuation/Withdrawal 
from the Study 
Added “Note: If a subject discontinues 
IMP and study assessments, all efforts 
must be made to continue to follow the 
subject for up to Month 30 by completing 
monthly contact for vital status or until 
withdrawal of consent. 
Added “Vital status” after “Adverse 
event” 
To align with CRF and 
protocol of AG10-301 
study. 
1.3 Schedule of Activities 
(SoA) 
8.8 Genetics 
Removed section 8.8 Genetics and 
“confirm genotype” in SoA. 
TTR genotyping is 
conducted in all study sites 
to establish ATTR-CM 
diagnosis before informed 
consent. 
1.3 Schedule of Activities 
(SoA) 
6MWT evaluation timepoint was 
corrected as the approved PCS. 
For consistency with the 
actual procedure as well as 
with AG-301 study 
1.3 Schedule of Activities 
(SoA) 
In the footnote, the sample collection 
timeframe for PD was added. 
To clarify the timeframe 
5.1 Inclusion Criteria 
“the first dose of ALXN2060” was 
changed to “Day 1” 
To keep consistency with 
SoA footnote and actual 
procedure 
5.1 Inclusion Criteria 
Corrected description of the methods of 
contraception for a male 
To keep consistency with 
section 10.4 
5.1 Inclusion Criteria 
Added “from Day 1 to at least 90 days 
after the last dose of the study drug” 
To clarify the guidance for 
Male participants in 
response to PMDA 
inquiries 
8.2.3 Kansas City 
Cardiomyopathy 
Questionnaire (KCCQ) 
“KCQG” was corrected to “KCCQ” 
Typo 
8.3.4 Electrocardiograms 
Added “(ECG results will be confirmed 
centrally)” 
To clarify and make it 
consistent with actual 
procedure 
8.6 Pharmacokinetics 
8.7 Pharmacodynamics 
Added “Day 14” 
To keep consistency with 
SoA footnote and actual 
procedure 
10.1.3 Informed Consent 
Process 
Deleted “[or assent]” 
It does not apply to this 
study. 
10.1.10 Publication Policy 
“AG10” was changed to “ALXN2060 
(Code number at Eidos: AG10)” 
To be consistent 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 67 of 68 
Alexion Confidential 
Section # and Name 
Description of Change 
Brief Rationale 
10.3.1 Definition of AE 
Deleted “• 
The signs, symptoms, 
and/or clinical sequelae resulting from 
lack of efficacy will be reported as AE or 
SAE if they fulfil the definition of an AE 
or SAE.” 
To correct overlapping with 
the latter half of the 
previous bullet 
10.4.2.2.1 Guidance for 
Female Participants 
Deleted superscript “c” 
No corresponding footnote 
10.5 Appendix 5 
“Appendix 11” was corrected to 
“Appendix 5”. 
Typo 
 
DOCUMENT HISTORY 
Document 
Date 
Overall Rationale for the Amendment 
Amendment 5 
11-September-
2023 
The main purpose of this amendment is to incorporate the 
changes from Administrative Letter 1.0 and to clarify the 
terminology definitions.  
Amendment 4 
12-January-2023 The main purpose of this amendment was to align the 
terminology definitions with Study AG10-301 Protocol 
Amendment 6.0 and to align the timing of study visits in the 
Extension Period with Study AG10-304. 
Amendment 3 
15-February-
2021 
The main purpose of this amendment was to add an exploratory 
endpoint (cardiac MRI) and to add text referring to COVID-19 
considerations. 
Amendment 2 
24-July-2020 
The purpose of this amendment was minor changes for 
consistency. 
Amendment 1 
20-July-2020 
The main purpose of this amendment was to respond to 
inquiries from PMDA on contraception guidance for male and 
TTR genotyping. 
Original protocol 
19-June-2020 
- 

Protocol Version 5.0 
 
Alexion Pharmaceuticals, Inc 
ALXN2060-TAC-302 
Page 68 of 68 
Alexion Confidential 
11. 
REFERENCES 
Ando Y, Ikeda S, Sekijima Y, et al. National Epidemiological Survey of Hereditary 
Transthyretin Amyloidosis; Report of Research Group on Amyloidosis (Report in Japanese). 
Health and Labor Science Research Grant. 2016. Available: https://mhlw-
grants.niph.go.jp/niph/search/NIDD00.do?resrchNum=201610022B [Accessed: 12-Jun-2020] 
 
Cornwell GG, 3rd, Murdoch WL, Kyle RA, Westermark P, Pitkanen P. Frequency and 
distribution of senile cardiovascular amyloid. A clinicopathologic correlation. Am J Med. 
1983;75(4):618-623. 
 
Fox JC, Hellawell JL, Rao S, et al. First-in-Human Study of AG10, a Novel, Oral, Specific, 
Selective, and Potent Transthyretin Stabilizer for the Treatment of Transthyretin Amyloidosis: A 
Phase 1 Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Adult 
Volunteers. Clin Pharmacol Drug Dev. 2020;9(1):115-129. 
 
Judge DP, Heitner SB, Falk RH, et al. Transthyretin Stabilization by AG10 in Symptomatic 
Transthyretin Amyloid Cardiomyopathy. J Am Coll Cardiol. 2019;74(3):285-295. 
Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis Treatment for Patients with 
Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018;379(11):1007-1016. 
 
Miller M, Pal A, Albusairi W, et al. Enthalpy-Driven Stabilization of Transthyretin by AG10 
Mimics a Naturally Occurring Genetic Variant That Protects from Transthyretin Amyloidosis. J 
Med Chem. 2018;61(17):7862-7876. 
 
Mirzoyev SA, Edwards WD, Mohammed SF, et al. Abstract 17926. Circulation. 2010;122. 
Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related amyloidoses and the heart: a clinical 
overview. Nat Rev Cardiol. 2010;7(7):398-408. 
 
Rowczenio D, Quarta CC, Fontana M, et al. Analysis of the TTR gene in the investigation of 
amyloidosis: A 25-year single UK center experience. Hum Mutat. 2019;40(1):90-96. 
 
Ruberg FL, Grogan M, Hanna M, Kelly JW, Maurer MS. Transthyretin Amyloid 
Cardiomyopathy: JACC State-of-the-Art Review. J Am Coll Cardiol. 2019;73(22):2872-2891. 
 
Sekijima Y, Mundayat R, Ishii T, Ando Y. The current status of the Transthyretin Amyloidosis 
Outcomes Survey (THAOS) in Japan. Amyloid. 2019;26(sup1):61-62. 
 
Ueda M, Horibata Y, Shono M, et al. Clinicopathological features of senile systemic 
amyloidosis: an ante- and post-mortem study. Mod Pathol. 2011;24(12):1533-1544. 
 
 
 

ALXN2060-TAC-302 Protocol Amendment
5.0_11Sep2023_final
Final Audit Report
2023-09-15
Created:
2023-09-15 (Japan Standard Time)
By:
Status:
Signed
Transaction ID:
CBJCHBCAABAAsVqud0fy9SuxhHxgvEeHF2H3WUCPyeqS
"ALXN2060-TAC-302 Protocol Amendment 5.0_11Sep2023_fina
l" History
Document created by 
2023-09-15 - 0:04:20 AM GMT+9
Document emailed to 
 for signature
2023-09-15 - 0:06:49 AM GMT+9
Email viewed by 
2023-09-15 - 8:36:27 AM GMT+9
authenticated with Adobe Acrobat Sign.
Challenge: The user opened the agreement.
2023-09-15 - 8:37:37 AM GMT+9
 authenticated with Adobe Acrobat Sign.
Challenge: The user completed the signing ceremony by clicking on 'Click to Sign' button.
2023-09-15 - 8:38:41 AM GMT+9
Document e-signed by 
Signing reason: I approve this document
Signature Date: 2023-09-15 - 8:38:42 AM GMT+9 - Time Source: server
Agreement completed.
2023-09-15 - 8:38:42 AM GMT+9