An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst-sponsored Study
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Sponsor: BioCryst Pharmaceuticals

 
 
 
BIOCRYST 
PHARMACEUTICALS, INC. 
BCX9930 
PROTOCOL No. BCX9930-205 
EudraCT No. 2021-006776-17 
 
An Open-label Study to Evaluate the Long-term Safety of 
BCX9930 Monotherapy in Subjects with Paroxysmal 
Nocturnal Hemoglobinuria Who Previously Received 
BCX9930 in a BioCryst-sponsored Study 
 
 
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BioCryst Pharmaceuticals, Inc. 
4505 Emperor Boulevard, Suite 200 
Durham, North Carolina 27703, USA 
Phone: +1 919-859-1302 
Fax: +1 919-851-1416 
 
CONFIDENTIAL 
The information in this document contains proprietary and confidential information belonging to 
BioCryst Pharmaceuticals, Inc. As a result, no part of this document should be copied, referred 
to, released, published, or otherwise disclosed in any manner or media without prior written 
approval from BioCryst Pharmaceuticals, Inc. 
 

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CONFIDENTIAL 
SPONSOR SIGNATURE PAGE 
 
Protocol No. BCX9930-205 
An Open-label Study to Evaluate the Long-term Safety of BCX9930 
Monotherapy in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who 
Previously Received BCX9930 in a BioCryst-sponsored Study 
Version 4.0: 27 July 2023 
 
 
Sponsor’s Approval 
The protocol has been approved by BioCryst Pharmaceuticals, Inc. 
 
Senior Clinical Development Physician: 
 
 
 
 
Please see the last page for electronic signature 
 
 
 
BioCryst Pharmaceuticals, Inc. 
Date 
 
 
Sponsor’s Authorized Signatory: 
 
 
 
 
Please see the last page for electronic signature 
 
 
  
BioCryst Pharmaceuticals, Inc. 
Date 
PPD
PPD
PPD
PPD

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INVESTIGATOR AGREEMENT 
 
Protocol No. BCX9930-205 
An Open-label Study to Evaluate the Long-term Safety of BCX9930 
Monotherapy in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who 
Previously Received BCX9930 in a BioCryst-sponsored Study 
Version 4.0: 27 July 2023 
 
I have received and read the Investigator’s Brochure (IB) for BCX9930. 
I have carefully read this protocol and agree that it contains all of the necessary information 
required to conduct this study. I agree to conduct this study as described and according to the 
Declaration of Helsinki, International Council for Harmonisation guidelines for Good Clinical 
Practice, and all locally applicable regulations. 
I agree to maintain the confidentiality of all information received or developed in connection 
with this protocol. 
 
 
 
 
 
Printed Name of Investigator 
 
 
 
 
Signature of Investigator 
 
 
 
 
Date 
 

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investigator, have both benefited from treatment with BCX9930 and would benefit from continued 
treatment with BCX9930, who wish to continue treatment, and who do not have access to other 
effective treatment options. 
Methodology: 
Subjects who meet all of the inclusion and none of the exclusion criteria can be enrolled into 
Study 205. The last on-treatment visit completed for the prior study will also serve as the baseline 
visit for Study 205. If subjects have completed at least 24 weeks of BCX9930 treatment in the prior 
study, study visits will occur every 8 weeks to Week 48, then every 12 weeks until Week 96. Subjects 
who have not completed 24 weeks of treatment with BCX9930 prior to enrolling in Study 205 must 
return to the clinic every 4 weeks until they have completed 24 weeks of cumulative treatment with 
BCX9930. Subjects who discontinue treatment with BCX9930 for any reason (complete study visits 
or discontinue early), will be asked to complete an end-of-study or early termination visit, as 
applicable, approximately 3 weeks (± 3 days) after the last dose of BCX9930. 
Number of Subjects (Planned): 
Up to 30 subjects are planned. 
Diagnosis and Main Criteria for Inclusion: 
Subjects must meet all of the following inclusion criteria, as applicable, to be eligible for participation 
in this study: 
1. Able to provide written informed consent. 
2. Non-pregnant, non-lactating female subjects. 
3. Subjects with PNH who are participating in another BCX9930 clinical study, have completed 
at least 12 weeks treatment with BCX9930, and, in the opinion of the investigator, have 
benefited from treatment with BCX9930 and would benefit from continued treatment with 
BCX9930, wish to continue treatment, and do not have access to other effective treatment 
options. 
4. For female subjects:  
a. Must be of nonchildbearing potential, or 
b. If a woman of childbearing potential, must agree to use one of the following methods of 
highly effective contraception throughout the study and for a duration of 30 days after the 
last dose of study drug. Highly effective contraceptive methods are restricted to the 
following:  
o Implantable progestogen-only hormonal contraception associated with inhibition of 
ovulation: 
▪ 
For South Africa only: Progestogen-only hormonal contraception injection, 
with active monitoring of compliance, is allowed as a contraceptive method. 
o An intrauterine device 
o An intrauterine hormone-releasing system 
o A bilateral tubal occlusion  
o A vasectomized or sterile partner  

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Abstinence as a contraceptive method will not be an option regardless of lifestyle or 
sexual activity.  
5. Male subjects with a female partner of childbearing potential, unless vasectomized, must use 
condoms (with spermicide, where available) throughout the study and for a duration of 90 days 
after the last dose of study drug. Subjects with a pregnant female partner must use condoms to 
avoid potential exposure of the partner and fetus in utero through semen. Abstinence as a 
contraceptive method will not be an option regardless of lifestyle or sexual activity.  
6. In the opinion of the investigator, the subject is expected to adequately comply with all 
required study procedures and restrictions for the study. 
Subjects must not meet any of the following exclusion criteria to be eligible for participation in the 
study: 
1. Any clinically significant medical or psychiatric condition including alcohol or drug 
dependency that, in the opinion of the investigator or sponsor, would interfere with the 
subject’s ability to participate in the study or increase the risk of participation for that subject. 
2. An ongoing adverse event (AE), including a laboratory abnormality, or other unacceptable 
toxicity that, in the judgment of the investigator, compromises the ability of the subject to 
continue study-specific procedures or it is considered not to be in the subject’s best interest to 
continue or benefit-risk assessment is no longer in favor of the subject’s continued treatment. 
3. Daily use of medications listed in the currently applicable prohibited medications list. 
4. Known or suspected hypersensitivity to BCX9930 or any of its formulation excipients. 
(Note: prior drug rash is not exclusionary.) 
Investigational Product, Dosage, and Mode of Administration: 
Based on safety and efficacy data from previous studies, all subjects enrolled into this study will 
receive BCX9930 400 mg twice a day, with or without food. 
Duration of Treatment: 
Each subject will be eligible to receive study drug (BCX9930) under this protocol for up to 96 weeks 
as long as the investigator believes it is in the subject’s best interest to continue treatment, or until the 
subject has access to other therapy for PNH, whichever comes first. Treatment with BCX9930 will be 
discontinued for subjects who are deriving no meaningful clinical benefit, or who experience an 
unacceptable drug-related AE or are otherwise intolerant of study drug. 
Reference Therapy, Dosage and Mode of Administration: 
Not applicable. 
Criteria for Evaluation: 
Safety: Safety will be evaluated by TEAEs, laboratory analyses (clinical chemistry, hematology, 
coagulation, urinalysis including microscopy), vital signs, and physical examination findings. 
Statistical Methods: 
Data will be listed and summarized using descriptive statistics. 

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3. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF 
FIGURES 
TABLE OF CONTENTS 
1. 
TITLE PAGE ................................................................................................................1 
SPONSOR SIGNATURE PAGE ....................................................................................................2 
INVESTIGATOR AGREEMENT...................................................................................................3 
2. 
SYNOPSIS ...................................................................................................................4 
3. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF FIGURES .................7 
4. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS.............................11 
5. 
INTRODUCTION ......................................................................................................14 
5.1. 
Background .................................................................................................................14 
5.2. 
Rationale for Study .....................................................................................................15 
5.2.1. 
Rationale for Study Population and Design ................................................................15 
5.2.2. 
Rationale for Dose Selection ......................................................................................16 
5.3. 
Benefit-Risk Assessment ............................................................................................16 
6. 
STUDY OBJECTIVES ..............................................................................................18 
6.1. 
Objectives ...................................................................................................................18 
6.2. 
Endpoints ....................................................................................................................18 
7. 
INVESTIGATIONAL PLAN .....................................................................................18 
7.1. 
Overall Study Design ..................................................................................................18 
7.2. 
Number of Subjects ....................................................................................................18 
7.3. 
Treatment Dosing .......................................................................................................19 
7.4. 
Individual and Study Termination Criteria .................................................................19 
7.4.1. 
Subject Withdrawal from Study Treatment ................................................................19 
7.4.2. 
Criteria for Study Termination ...................................................................................20 
8. 
SELECTION AND WITHDRAWAL OF SUBJECTS ..............................................21 
8.1. 
Subject Inclusion Criteria ...........................................................................................21 
8.2. 
Subject Exclusion Criteria ..........................................................................................22 
8.3. 
Subject Withdrawal ....................................................................................................22 
8.4. 
End of Study Definition ..............................................................................................23 
9. 
TREATMENT OF SUBJECTS ..................................................................................23 
9.1. 
Description of Study Drug and Study Drug Product ..................................................23 

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9.2. 
Description of Study Drug Packaging, Labelling, and Storage ..................................23 
9.3. 
Randomization and Blinding of Study Treatment ......................................................23 
9.4. 
Study Drug Administration.........................................................................................23 
9.4.1. 
Treatment Interruption or Dose Reduction .................................................................23 
9.4.2. 
Dose Tapering .............................................................................................................24 
9.5. 
Treatment Compliance ................................................................................................24 
9.6. 
Missed Doses and Special Considerations for Dosing ...............................................25 
9.6.1. 
Missed Doses ..............................................................................................................25 
9.6.2. 
Special Considerations for Dosing, Including Inability to Take Medication 
Orally ..........................................................................................................................25 
9.7. 
Study Drug Accountability .........................................................................................25 
9.8. 
Prior and Concomitant Medications ...........................................................................26 
9.8.1. 
Prohibited and Restricted Medications .......................................................................26 
9.9. 
Vaccination Requirements, Vaccinations During Study, and Prophylactic 
Antibiotic Coverage ....................................................................................................27 
10. 
STUDY CONDUCT ...................................................................................................28 
10.1. 
Overview .....................................................................................................................28 
10.2. 
Schedule of Assessments ............................................................................................28 
10.3. 
Study Visits .................................................................................................................30 
10.3.1. 
Additional Assessments for Renal and Hepatic Safety Monitoring ...........................31 
11. 
STUDY ASSESSMENTS ..........................................................................................31 
11.1. 
Chronology of Assessments .......................................................................................31 
11.2. 
Demographic Information, Medical and Medication History ....................................31 
11.2.1. 
Contraception Requirements ......................................................................................31 
11.3. 
Virology Assessments ................................................................................................33 
11.4. 
Effectiveness Assessments .........................................................................................33 
11.5. 
Safety Assessments .....................................................................................................33 
11.5.1. 
Vital Signs ..................................................................................................................33 
11.5.2. 
Body Weight, Height, and Body Mass Index .............................................................34 
11.5.3. 
Physical Examination .................................................................................................34 
11.5.4. 
Clinical Chemistry, Hematology, Urinalysis, and Other Laboratory 
Assessments ................................................................................................................34 
11.5.4.1. 
Urine Collections for Urinalysis and Urine Microscopy ............................................36 

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11.5.5. 
Menopause and Pregnancy Testing ............................................................................36 
12. 
ADVERSE EVENTS..................................................................................................36 
12.1. 
Definition of Adverse Events .....................................................................................36 
12.1.1. 
Adverse Event .............................................................................................................36 
12.1.2. 
Serious Adverse Event ................................................................................................37 
12.1.3. 
Events of Special Monitoring .....................................................................................38 
12.2. 
Definition of Severity .................................................................................................38 
12.3. 
Relationship to Study Drug ........................................................................................39 
12.4. 
Method, Frequency, and Time Period for Detecting Adverse Events and 
Reporting Serious Adverse Events .............................................................................39 
12.4.1. 
Serious Adverse Events and Suspected Unexpected Serious Adverse 
Reactions .....................................................................................................................40 
12.4.2. 
Pregnancy ...................................................................................................................41 
12.5. 
Adverse Event Management .......................................................................................41 
12.5.1. 
Potential Drug-induced Liver Injury: Monitoring, Evaluation, and 
Discontinuation Criteria ..............................................................................................41 
12.5.2. 
Renal Events: Monitoring, Evaluation, and Discontinuation Criteria ........................42 
12.5.3. 
Risk of Hemolysis After Missed Doses, Treatment Interruption, or 
Permanent Discontinuation .........................................................................................43 
12.6. 
Overdose .....................................................................................................................44 
12.7. 
Data Monitoring Committees .....................................................................................44 
13. 
STATISTICS ..............................................................................................................45 
13.1. 
Sample Size Considerations .......................................................................................45 
13.2. 
Stratification ...............................................................................................................45 
13.3. 
Statistical Methods ......................................................................................................45 
13.3.1. 
Analysis Populations ..................................................................................................45 
13.3.1.1. 
Safety Population ........................................................................................................45 
13.3.2. 
General Considerations for Data Analysis .................................................................45 
13.3.3. 
Subject Demographic and Disposition Data ...............................................................45 
13.3.4. 
Analysis of Safety Variables ......................................................................................46 
14. 
STUDY ADMINISTRATION ...................................................................................46 
14.1. 
Study Monitoring ........................................................................................................46 
14.2. 
Audits and Inspections ................................................................................................47 
14.3. 
Ethics Committee ........................................................................................................47 

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14.4. 
Serious Breaches of GCP ............................................................................................47 
15. 
QUALITY CONTROL AND QUALITY ASSURANCE .........................................47 
16. 
ETHICS ......................................................................................................................48 
16.1. 
Ethics Review .............................................................................................................48 
16.2. 
Ethical Conduct of the Study ......................................................................................48 
16.3. 
Written Informed Consent ..........................................................................................48 
17. 
DATA HANDLING AND RECORDKEEPING .......................................................49 
17.1. 
Inspection of Records .................................................................................................49 
17.2. 
Retention of Records ..................................................................................................49 
17.3. 
Confidentiality of Information and Data ....................................................................49 
18. 
PUBLICATION POLICY ..........................................................................................50 
19. 
LIST OF REFERENCES ............................................................................................51 
 
LIST OF TABLES 
Table 1: 
List of Abbreviations and Specialist Terms ................................................................11 
Table 2: 
Study BCX9930-205: Schedule of Study Assessments/Procedures ...........................29 
Table 3: 
Clinical Laboratory Evaluations .................................................................................35 
 

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4. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
The following abbreviations and specialist terms are used in this study protocol. 
Table 1: 
List of Abbreviations and Specialist Terms 
Abbreviation or 
Specialist Term 
Explanation 
ACIP 
Advisory Committee on Immunization Practices 
AE 
adverse event 
ALP 
alkaline phosphatase 
ALT 
alanine aminotransferase (also SGPT) 
AP 
alternative complement pathway 
ARC 
absolute reticulocyte count 
AST 
aspartate transferase (also SGOT) 
BCRP 
breast cancer resistance protein 
BID 
twice a day 
BMI 
body mass index 
C3 
complement component 3 
COVID-19 
coronavirus disease 2019 
CP 
classical complement pathway 
CTCAE 
Common Terminology Criteria for Adverse Events 
CYP 
cytochrome P450 
DDI 
drug-drug interaction 
DMC 
data monitoring committee 
eCRF 
electronic case report form 
EFD 
embryo-fetal development 
eGFR 
estimated glomerular filtration rate 
EOS 
end of study 
EOSM 
events of special monitoring 
ET 
early termination 
EU 
European Union 
EVH 
extravascular hemolysis 
FDA 
US Food and Drug Administration 
FIH 
first in human 
FSH 
follicle-stimulating hormone 
GCP 
Good Clinical Practice 
Hb 
hemoglobin 

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Abbreviation or 
Specialist Term 
Explanation 
HBV 
hepatitis B virus 
HCV 
hepatitis C virus 
HIV 
human immunodeficiency virus 
IB 
Investigator’s Brochure 
IC50 
50% of maximal inhibitory concentration 
ICF 
informed consent form 
ICH 
International Council for Harmonisation 
IMP 
investigational medicinal product 
INR 
international normalized ratio 
IUD 
intrauterine device 
IUS 
intrauterine hormone-releasing system 
IV 
intravenous 
IVH 
intravascular hemolysis 
LDH 
lactate dehydrogenase 
LP 
lectin complement pathway 
MAC 
membrane attack complex 
MAD 
multiple ascending dose 
MAVE 
major adverse vascular event 
MedDRA 
Medical Dictionary for Regulatory Activities 
N, n 
number of subjects or events (uppercase is total population; lowercase is subgroup 
population) 
NCI 
US National Cancer Institute 
NRMWG 
Nephrology Risk Mitigation Working Group 
PD 
pharmacodynamic(s) 
P-gp 
P-glycoprotein 
PK 
pharmacokinetic(s) 
PNH 
paroxysmal nocturnal hemoglobinuria 
RBC 
red blood cell 
SAE 
serious adverse event 
SAP 
statistical analysis plan 
SARS-CoV-2 
severe acute respiratory syndrome coronavirus 2 
sCr 
serum creatinine 
SUSAR 
suspected unexpected serious adverse reaction 
TEAE 
treatment-emergent adverse event 

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Abbreviation or 
Specialist Term 
Explanation 
TID 
three times a day 
uACR 
urine microalbumin to creatinine ratio 
ULN 
upper limit of normal (reference range) 
US 
United States 
WBC 
white blood cell 

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5. 
INTRODUCTION 
5.1. 
Background 
Complement activation is an innate defense mechanism that, when uncontrolled, leads to 
inflammation and local tissue damage. The complement system consists of 3 linked pathways: 
the classical pathway (CP), lectin pathway (LP), and alternative pathway (AP). Dysregulation of 
AP activity by germ line or somatic genetic mutations of complement regulatory proteins or 
enzymes, neutralizing antibodies to complement regulatory proteins, or stabilizing antibodies to 
complement enzyme complexes predisposes individuals to diverse disorders, including 
paroxysmal nocturnal hemoglobinuria (PNH), age-related macular degeneration, atypical 
hemolytic uremic syndrome, and complement 3 glomerulopathy (Holers 2008; Brodsky 2014; 
Zipfel et al. 2015; Ricklin et al. 2016). 
Factor D is the rate-limiting enzyme of the AP (Volanakis and Narayana 1996) and the AP 
amplification loop and is made constitutively by adipocytes (White et al. 1992). The only natural 
substrate for Factor D is complement Factor B bound to C3b (Volanakis and Narayana 1996). 
Factor D cleaves Factor B only after the latter is bound to C3b or C3(H2O) at the bond between 
Factor B amino acid residues 259 and 260, releasing the 30kD N-terminal Ba fragment and 
retention of the 60kD C-terminal Bb fragment that is required for production of the 
C3 convertases of the AP [ie, C3bBb and C3(H2O)Bb] (Torreira et al. 2009). In addition, 
Factor D is essential for the AP amplification loop following generation of C3b by the AP, LP, 
or CP (Lesavre and Muller-Eberhard 1978; Volanakis and Narayana 1996; Xu et al. 2001). 
Based on in vitro studies using human serum, this AP amplification loop appears to account for 
up to 80% of the C5a and membrane attack complex (MAC) generated from initial activation of 
complement by the CP or LP, or other proteolytic enzymes (eg, renin) in the plasma or other 
body fluids (Thurman and Holers 2006; Ekdahl et al. 2019) (BioCryst internal data on file). As 
such, Factor D plays a central role in the activation and amplification of the AP, as well as in the 
amplification of the LP and the CP. 
PNH is an acquired, rare, serious, and potentially life-threatening disorder characterized by 
destruction of red blood cells (RBCs) resulting from uncontrolled activity of complement. PNH 
is caused by a somatic mutation in the phosphatidylinositol glycan anchor biosynthesis class A 
(PIG-A) gene in hematopoietic stem cells that leads to a deficiency of the membrane-displayed 
complement regulatory proteins CD55 and CD59 that normally protect RBCs from effective 
opsonization and lysis by the complement system. These deficiencies result in unrestrained 
complement activity that attacks RBCs, leading to episodes of intravascular hemolysis (IVH), 
extravascular hemolysis (EVH), chronic hemolytic anemia, bone marrow failure (BMF), and 
thrombosis (Parker 2009; Pu and Brodsky 2011), and EVH in patients treated with C5 inhibitors. 
PNH affects men and women equally and is more common in people of Asian rather than 
Caucasian ethnicity (Hill et al. 2017; Jalbert et al. 2019). The onset of PNH can occur at any age. 
The median (interquartile range) age of patients with PNH at diagnosis reported in a large series 
was 44 years (30 to 60 years) (Curran et al. 2012; Ge et al. 2015; Mercuri et al. 2017; Narita et 
al. 2017; Cannizzo et al. 2019; Griesser et al. 2020).  
PNH is associated with significant morbidity and mortality (Harris et al. 1999; Rachidi et al. 
2010). Median survival in the era prior to use of C5 inhibitors was reported in one study as 
10 years (Hillmen et al. 1995); a retrospective analysis found that approximately 20% of patients 

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with only supportive care died within 6 years of diagnosis (Loschi et al. 2016). Thrombosis was 
the leading cause of death in PNH patients, accounting for up to 67% of (Loschi et al. 2016). 
While thrombosis is relatively rare as a presenting symptom, it has historically occurred in up to 
40% of patients during the course of disease (Pu and Brodsky 2011); a history of major adverse 
vascular events (MAVEs), including thromboembolism, was reported in 19% of patients with 
PNH in a large series (Schrezenmeier et al. 2020). Visceral thrombosis, cerebrovascular events, 
and pulmonary embolism predict a poor outcome (Ziakas et al. 2008). 
BioCryst Pharmaceuticals, Inc. (“BioCryst”) was developing BCX9930, a potent and selective, 
orally bioavailable, small-molecule inhibitor of complement Factor D, as a potential treatment 
for PNH and other complement-mediated diseases. Targeting Factor D with a pharmacologic 
inhibitor prevents both IVH and EVH in patients with PNH by blocking the formation of MAC 
and inhibiting the formation of C3 fragments. This represents a promising therapeutic strategy 
for the treatment of AP-mediated diseases such as PNH. The results of clinical studies and 
nonclinical pharmacology, pharmacokinetics (PK), and toxicology studies of BCX9930 can be 
found in the Investigator’s Brochure (IB).  
5.2. 
Rationale for Study 
On 15 December 2022, BioCryst announced that, based on new competitive data recently 
presented at the 2022 American Society of Hematology Annual Meeting, the company no longer 
believed that BCX9930 would be commercially competitive, and would, therefore, be 
discontinuing the development of BCX9930 (BioCryst Press Release 2022). Enrollment into the 
pivotal studies (Study 202 and Study 203) was halted permanently. Subjects already enrolled in 
the pivotal studies who were benefiting from treatment with BCX9930 were allowed to continue 
treatment. In addition, subjects in Study 202 randomized to continue C5 inhibitor therapy were 
offered the option to begin treatment with BCX9930. In Study 203, the treatment assignments of 
subjects was unblinded with subjects randomized to placebo given the option to transition to 
treatment with open-label BCX9930. Treatment with BCX9930 may be continued in the pivotal 
studies (and in ongoing rollover Study 201) until the subjects can be transitioned into this study, 
allowing those studies to be closed. 
As the development program is not being discontinued for safety reasons or a lack of efficacy, 
and the preliminary data available from the ongoing clinical studies in PNH do not change the 
current safety or efficacy profile for BCX9930, BioCryst remains committed to providing 
BCX9930 to those subjects participating in the ongoing studies who are currently receiving 
benefit from BCX9930 and wish to continue treatment. This study will be used to meet that 
commitment by allowing for continued access to treatment with BCX9930 for any subject with 
PNH currently receiving treatment with BCX9930 in a BioCryst-sponsored study who do not 
have access to other effective treatment options. As the development of BCX9930 is now 
terminated, treatment will be provided for a maximum of 96 weeks under this version of the 
protocol, as long as the investigator believes it is in the subject’s best interest to continue 
treatment, or until the subject has access to alternative therapy for PNH, whichever comes first. 
5.2.1. 
Rationale for Study Population and Design 
Subjects who are enrolled in a prior BCX9930 study for PNH (ie, Studies 201, 202, or 203), have 
completed at least 12 weeks of treatment with BCX9930 in the prior study, and, in the opinion of 

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the investigator, have both benefited from that treatment and would continue to benefit from 
treatment, and who do not have access to other effective treatment options will be eligible for 
this study. Most participants in Studies 201 and 203 do not have access to approved therapies for 
PNH. Participants in Study 202 had access to one or both of the approved C5 inhibitors, but had 
an inadequate response to those therapies. Requiring subjects to complete a minimum of 
12 weeks of treatment in the prior study will allow adequate time to confirm tolerability to the 
recommended 400 mg twice a day (BID) BCX9930 dose regimen, including completing any 
enhanced safety monitoring needed to monitor for potential renal toxicity, before entering into 
this study. The study features (eg, endpoints, withdrawal criteria, assessments, etc.) were selected 
to align closely with previous PNH studies of BCX9930 and will allow for the continued 
monitoring of subject safety and the ongoing assessment of risks and benefits in subjects 
previously exposed to BCX9930 continuing treatment in this study. 
5.2.2. 
Rationale for Dose Selection 
Based on safety and efficacy data from previous studies, all subjects enrolled into Study 205 will 
receive BCX9930 at a dose of 400 mg BID, which is the dose regimen that was being evaluated 
in the pivotal studies prior to the decision to discontinue further development of the drug. 
5.3. 
Benefit-Risk Assessment 
Subjects participating in this study will have already been administered BCX9930 in a previous 
study; thus, the benefit-risk profile of initial treatment with BCX9930 will previously have been 
assessed by the investigator in each subject eligible for Study 205. The benefit-risk profile of 
long-term treatment with BCX9930 will be evaluated throughout the duration of this study. 
Treatment with BCX9930 will be discontinued for subjects who are deriving no meaningful 
clinical benefit, or who experience an unacceptable drug-related adverse event (AE) or are 
otherwise intolerant of study drug. 
Potential risks associated with complement inhibitors for the treatment of PNH, as well as 
potential risks associated with BCX9930 based on nonclinical and clinical data to date, are 
described in detail in the IB for BCX9930.  
A significant risk associated with complement inhibitor therapies is the risk of life-threatening or 
fatal meningococcal infections (Figueroa and Densen 1991; Hillmen et al. 2006). As BCX9930 
blocks the AP of complement, subjects may have increased susceptibility to bacterial infections, 
especially infections with encapsulated organisms such as Neisseria meningitidis, Streptococcus 
pneumoniae, and Haemophilus influenzae, but also with unencapsulated strains of 
N. meningitidis and other bacteria such as Neisseria gonorrhoeae (Ram et al. 2010). Study 
subjects receiving BCX9930 will be monitored closely for signs and symptoms of infection; 
infection risk will be mitigated by requiring subjects to have up-to-date vaccinations against 
appropriate bacterial strains. If required on an individual basis, prophylactic antibiotic 
administration will be allowed.  
Like other complement inhibitor therapies, discontinuation or interruption of BCX9930 may 
result in increased risk of hemolysis of PNH RBCs (Soliris-USPI 2020; Empaveli-USPI 2021; 
Ultomiris-USPI 2022). Subjects should be instructed to immediately contact the investigative site 
if they miss any BCX9930 doses and experience new or worsening symptoms of hemolysis. A 
suggested dose tapering schedule is provided in Section 9.4.2 for subjects who discontinue study 

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6. 
STUDY OBJECTIVES 
6.1. 
Objectives 
The objectives of this study are: 
• To provide continued access to BCX9930 for subjects with PNH who have benefited 
from treatment with BCX9930 in another BioCryst-sponsored study and who, in the 
opinion of the investigator, would benefit from continued treatment with BCX9930, 
and who do not have access to other effective treatment options 
• To monitor the safety of BCX9930 in subjects continuing to receive BCX9930 for the 
treatment of PNH 
6.2. 
Endpoints 
The endpoints of this study are: 
• Subject incidence of graded TEAEs, laboratory abnormalities, changes to vital signs, 
and physical examination findings 
7. 
INVESTIGATIONAL PLAN 
7.1. 
Overall Study Design 
This study is designed to provide continued access to BCX9930 for subjects currently receiving 
treatment with BCX9930 in another BioCryst-sponsored clinical study for PNH (ie, Studies 201, 
202, or 203, hereafter referred to as the “prior study”) who, in the opinion of the investigator, 
have both benefited from treatment with BCX9930 and would benefit from continued treatment 
with BCX9930, who wish to continue treatment, and who do not have access to other effective 
treatment options. 
Subjects will be eligible to receive study drug (BCX9930) under this version of the protocol for 
up to 96 weeks for as long as the investigator believes it is in the subject’s best interest to 
continue treatment, or until the subject has access to another therapy for PNH, whichever comes 
first. Treatment with BCX9930 will be discontinued for subjects who are deriving no meaningful 
clinical benefit, or who experience an unacceptable drug-related AE or are otherwise intolerant 
of study drug. 
An independent BCX9930 program-wide DMC will provide oversight of the safety of subjects 
receiving BCX9930 in this study. 
7.2. 
Number of Subjects 
The number of subjects will depend on the number of potentially eligible subjects still 
participating in prior studies, estimated to be up to 30 subjects. 

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BioCryst reserves the right to discontinue the study (eg, if alternative therapy for PNH becomes 
available) but intends only to exercise this right for valid scientific, medical, or administrative 
reasons. If BioCryst does discontinue the study, the investigator must contact all participating 
subjects immediately after notification of study termination. 
If the study is halted due to safety concerns, the study will not be re-started. 
8. 
SELECTION AND WITHDRAWAL OF SUBJECTS 
8.1. 
Subject Inclusion Criteria 
Subjects must meet all of the following inclusion criteria, as applicable, to be eligible for 
participation in this study: 
1. Able to provide written informed consent. 
2. Non-pregnant, non-lactating female subjects. 
3. Subjects with PNH who are participating in another BCX9930 clinical study, have 
completed at least 12 weeks treatment with BCX9930, and, in the opinion of the 
investigator, have benefited from treatment with BCX9930 and would benefit from 
continued treatment with BCX9930, wish to continue treatment, and do not have access 
to other effective treatment options. 
4. For female subjects:  
a. Must be of nonchildbearing potential, or 
b. If a woman of childbearing potential, must agree to use one of the following methods 
of highly effective contraception throughout the study and for a duration of 30 days 
after the last dose of study drug. Highly effective contraceptive methods are restricted 
to the following:  
o Implantable progestogen-only hormonal contraception associated with inhibition 
of ovulation 
▪ For South Africa only: Progestogen-only hormonal contraception 
injection, with active monitoring of compliance, is allowed as a 
contraceptive method.  
o An intrauterine device (IUD) 
o An intrauterine hormone-releasing system (IUS) 
o A bilateral tubal occlusion  
o A vasectomized or sterile partner 
Abstinence as a contraceptive method will not be an option regardless of lifestyle or 
sexual activity. 
5. Male subjects with a female partner of childbearing potential, unless vasectomized, must 
use condoms (with spermicide, where available) throughout the study and for a duration 
of 90 days after the last dose of study drug. Subjects with a pregnant female partner must 
use condoms to avoid potential exposure of the partner and fetus in utero through semen. 
Abstinence as a contraceptive method will not be an option regardless of lifestyle or 
sexual activity.  

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6. In the opinion of the investigator, the subject is expected to adequately comply with all 
required study procedures and restrictions for the duration of the study. 
Additional details are provided in Section 11.2.1. 
8.2. 
Subject Exclusion Criteria 
Subjects must not meet any of the following exclusion criteria to be eligible for participation in 
the study: 
1. Any clinically significant medical or psychiatric condition including alcohol or drug 
dependency that, in the opinion of the investigator or sponsor, would interfere with the 
subject’s ability to participate in the study or increase the risk of participation for that 
subject. 
2. An ongoing AE, including a laboratory abnormality, or other unacceptable toxicity that, 
in the judgment of the investigator, compromises the ability of the subject to continue 
study-specific procedures or it is considered not to be in the subject’s best interest to 
continue or benefit-risk assessment is no longer in favor of the subject’s continued 
treatment. 
3. Daily use of medications listed in the currently applicable prohibited medications list. 
4. Known or suspected hypersensitivity to BCX9930 or any of its formulation excipients. 
(Note: prior drug rash is not exclusionary.) 
8.3. 
Subject Withdrawal 
Participation in the study is strictly voluntary; a subject may withdraw consent to contribute 
additional study information at any point. Subjects have the right to withdraw from the study at 
any time and for any reason. In addition, a subject’s participation may be terminated for any of 
the reasons described in Section 7.4.1. Whenever possible, the investigator will consult with the 
sponsor medical monitor before halting a subject’s participation in the study. 
Although a subject may withdraw from the study at any time without specifying a reason for 
withdrawal, the reason for withdrawal, if provided by the subject, will be recorded in the 
subject’s medical records (source documents) and also in the eCRF. If the reason for subject 
withdrawal is not known, attempts to contact the subject must be documented to establish 
whether the reason was due to an AE, and if so, this must be reported in accordance with the 
procedures outlined in Section 12.4.1. Vigorous attempts will be made for follow-up of all 
subjects who miss a study visit, given the potential for hemolysis if BCX9930 dosing is 
interrupted. If at any point in the study the clinic is unable to contact the subject after appropriate 
attempts have been made, the subject will be considered lost to follow-up. If a subject’s 
participation in this study is terminated, the responsible investigator/clinical staff member will 
document termination in the source documents. 
Unless consent is withdrawn, subjects who discontinue study treatment will be monitored for 
potential hemolysis and may be required to return to the clinic for an additional visit(s) to assess 
for acute symptomatic hemolysis, if and when symptoms occur. A dose tapering schedule should 
be considered (Section 9.4.2). An early termination (ET) visit will be completed approximately 
3 weeks after the date of last dose of BCX9930 (see Table 2). 

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Once subjects have withdrawn from the study, the sponsor will no longer provide treatment 
through the study. 
8.4. 
End of Study Definition 
The end of the study (EOS) will be defined as the date when the last subject completes the last 
protocol scheduled visit. 
9. 
TREATMENT OF SUBJECTS 
9.1. 
Description of Study Drug and Study Drug Product 
BCX9930 is a small-molecule inhibitor of Factor D and will be supplied by BioCryst as 100-mg 
tablets for oral administration. The active ingredient is BCX9930 drug substance. 
BCX9930 tablets contain the active ingredient blended with the excipients 
 
 
 
 
Additional details of the chemical and physical characteristics of BCX9930 may be found in the 
IB for BCX9930 and the investigational medicinal product (IMP) manual for the study. Any 
changes to the formulation will be detailed in the IMP manual. 
9.2. 
Description of Study Drug Packaging, Labelling, and Storage 
BCX9930 tablets will be packaged in an appropriate size bottle. Each bottle will be labeled with 
the information required per local law, which may include sponsor name, study protocol number, 
description of the contents, a statement regarding the investigational (clinical study) use of the 
study drug, and expiry date. 
The bottles of BCX9930 tablets should be stored at 15°C to 25°C (59ºF to 77ºF). 
Additional details on the packaging, labeling, shipment, storage, and dispensing of BCX9930 
tablets will be provided in the IMP manual for the study. 
9.3. 
Randomization and Blinding of Study Treatment 
This is an open-label, non-randomized study. Study treatment will not be blinded or masked. 
9.4. 
Study Drug Administration 
BCX9930 will be supplied as 100-mg tablets. Subjects will take four BCX9930 100-mg tablets 
orally BID, approximately 12 hours apart. BCX9930 may be taken with or without food. 
Subjects should be counseled to maintain adequate hydration throughout the treatment period to 
prevent the formation of highly concentrated urine. 
9.4.1. 
Treatment Interruption or Dose Reduction  
Treatment interruption or dose reduction as a result of investigator management of AEs 
potentially related to study drug is permissible with appropriate monitoring for potential 
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hemolysis as described in Section 12.5.3. Any treatment interruption or dose reduction will be 
recorded in the eCRF and source documents, including the reason for the interruption or 
reduction. Resumption of study drug administration (up to the recommended 400 mg BID dose 
regimen) is also permissible upon resolution of the event, as assessed by the investigator, with a 
plan for monitoring of the subject for recurrence of the AE, as appropriate. Dose adjustment to a 
dose regimen > 400 mg BID is not permitted. 
See Section 9.4.2 for potential dose reduction options. 
9.4.2. 
Dose Tapering 
In the event of permanent discontinuation of BCX9930, 
 
 
 
 
 
The decision to taper the dose and duration of any taper should be based on the investigator’s 
medical judgement, taking into account the reason for discontinuation of the drug, the severity of 
any reported signs or symptoms, and/or any reported worsening of signs or symptoms. The 
tapering schedule should be discussed with the sponsor medical monitor (or designee), ideally 
prior to implementation, and adjusted, as appropriate, for the individual subject. 
9.5. 
Treatment Compliance 
All doses can be taken at home; subjects do not need to withhold any doses on clinic days or take 
a dose at the clinic, unless the clinic visit falls during the subject’s normal time of dosing. 
Subjects will be provided instructions for taking the twice-daily doses of BCX9930 at home, 
including frequency and time of administration. Where possible, the BCX9930 doses should be 
taken at the same times each day as close as possible to 12 hours apart (eg, 8:00 AM and 
8:00 PM). 
Subject compliance with the BID dosing schedule for BCX9930 is critical for optimizing 
suppression of the AP and minimizing the risk of hemolysis. Missing more than 2 consecutive 
doses of BCX9930 increases the risk of subjects experiencing a hemolytic episode. Therefore, 
participants must be re-educated about and understand the importance of compliance with the 
dosing schedule, be reminded about the risks associated with breakthrough hemolysis, and the 
need to promptly report new or worsening signs and symptoms of potential hemolysis to staff, 
especially when associated with missed doses. Once enrolled in this study, each participant will 
need to be responsible for taking BCX9930 twice a day, at approximately the same times and as 
close as possible to 12 hours apart. BioCryst will provide materials that can be used to 
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communicate the importance of compliance. Subjects will be required to bring the bottles of 
BCX9930 to clinic visits, including any unscheduled visit to assess for potential hemolysis, when 
possible. Investigators should remind participants who regularly miss doses of BCX9930 about 
the importance of treatment compliance. 
9.6. 
Missed Doses and Special Considerations for Dosing 
If BCX9930 needs to be withheld (eg, to assess a potentially drug-related TEAE), the 
investigator should consider the potential for hemolysis as described in Section 12.5.3. 
9.6.1. 
Missed Doses 
Ideally, BCX9930 doses should be taken 12 hours apart at the same times each day without 
missing doses. If a subject does not take a BCX9930 dose at the scheduled time, the missed dose 
can be taken up to 6 hours after the scheduled dose time. If more than 6 hours have passed, the 
missed dose should not be taken. Regardless of whether the missed dose was made up, dosing 
should resume with the next dose taken at the scheduled time. For example, a subject who 
normally takes the BCX9930 doses at 8:00 AM/PM each day can take a missed morning dose as 
late as 2:00 PM with the evening dose administered on schedule at 8:00 PM. A missed evening 
dose can be taken as late as 2:00 AM the next day, with the next dose taken on schedule at 
8:00 AM. 
Subjects should be instructed to immediately contact the investigator if they miss any BCX9930 
doses and have new or worsening signs or symptoms consistent with acute symptomatic 
hemolysis (see Section 12.5.3). 
9.6.2. 
Special Considerations for Dosing, Including Inability to Take Medication 
Orally 
It is imperative that study subjects take study drug as scheduled without missing doses. Under 
certain circumstances, when a subject cannot take or absorb BCX9930 (including acute illness 
with vomiting, dysphagia due to symptomatic hemolysis, and unplanned circumstances requiring 
hospitalization), there are provisions that can be taken. 
For acute vomiting, treatment of nausea and/or vomiting with an oral, orally dissolving, or rectal 
suppository anti-emetic 30 minutes to an hour (as a suggestion) before attempting to swallow the 
BCX9930 tablets could be attempted. If a subject vomits within 5 minutes after taking a dose of 
BCX9930 or intact tablets or pieces of the tablets are seen in the vomitus (vomit contents), the 
subject may be re-dosed (ie, take new tablets). 
Please refer also to the IMP manual for specific instructions. 
9.7. 
Study Drug Accountability 
Accountability of BCX9930 dispensed and returned (as applicable) will be performed at Day 1 
and at each subsequent study visit (including at unscheduled visits, when possible). Returned 
study drug bottles should be retained and reviewed during monitoring visits by the study 
monitor. 
The investigator or designee (eg, pharmacist) must maintain accurate records of the disposition 
of all study drugs received from the sponsor, directly administered to the subject (if applicable) 

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or dispensed to the subject. At the end of the study, information describing study drug 
accountability must be maintained at the site. 
All study drug not dispensed to the subject and any returned study drug (including empty bottles) 
will be returned to the sponsor or destroyed on site as instructed by the sponsor following IMP 
accountability by the study monitor, abiding by appropriate standard operating procedure(s) at 
the participating institution. 
9.8. 
Prior and Concomitant Medications 
All concomitant medication use, beginning with the baseline visit, will be recorded in the source 
documentation and eCRF. 
During the course of the study, the investigator should review the subject’s medication list for 
potentially nephrotoxic medications and consider, when medically feasible, whether these 
medications may be stopped or substituted with non- or less nephrotoxic medications. Caution 
should be exercised with the chronic use of non-steroidal anti-inflammatory drugs while taking 
BCX9930. With the exception of any changes to nephrotoxic medications and prohibited and 
restricted medications detailed below (Section 9.8.1), subjects should continue their normal 
medications. 
Prophylactic use of antibacterial and antiviral medications, other than those expressly prohibited 
on the prohibited and restricted medication list, may be considered if clinically indicated and 
after discussion with the sponsor medical monitor (or designee), with consideration of the list of 
prohibited and restricted medications separate from this protocol (see Section 9.8.1). 
Please refer to Section 9.9 for vaccination requirements. 
9.8.1. 
Prohibited and Restricted Medications 
Drug interactions with concomitant medications (including supplements) may affect plasma 
concentrations of that medication or of BCX9930. Adjustments in the dosing regimen of 
concomitant medications and/or increased safety monitoring may be necessary based on the 
potential for interaction. Additional details regarding potential for interaction with specific 
medications may be found in the prohibited and restricted medications list. 
All subjects should observe the prohibitions and restrictions on concomitant use described in 
Section 8.2 and in the following text. 
Use of the following medications is currently excluded while subjects are receiving treatment 
with BCX9930, as is the planned initiation of treatment with such medications during the study: 
 
 
 
  
 
 
 
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The sponsor will provide a list separate from the protocol of prohibited and restricted 
medications for which there may be a drug interaction with BCX9930 based upon regulatory 
labeling for individual medications, regulatory guidance, peer-reviewed literature, and regularly 
updated drug interaction reference databases (eg, University of Washington School of Pharmacy 
Drug Interaction Database, Pharmapendium). The sponsor may modify this list of prohibited 
medications based upon available PK and safety data on concomitant medications, ongoing 
clinical findings, and continued review of labeling, regulatory guidance, literature, and reference 
databases. A memorandum of any such changes to this list will be provided to all clinical sites. 
Subjects should inform all treating physicians of their participation in this study and the 
possibility of potential interactions with BCX9930. 
Throughout the course of the study, investigators should inquire about newly initiated, daily, 
chronic-use medications; the investigator should compare these medications to the prohibited 
medication list. Please contact the sponsor medical monitor for specific questions regarding 
prohibited and restricted medications if needed. 
9.9. 
Vaccination Requirements, Vaccinations During Study, and 
Prophylactic Antibiotic Coverage 
Subjects participating in this study will have been required to meet the vaccine requirements for 
entry into the prior study. In this study, booster vaccinations will be maintained according to 
either the US Centers for Disease Control and Prevention Advisory Committee on Immunization 
Practices (ACIP) recommendations for meningococcal vaccination and ACIP guidance for 
altered immunocompetence (including complement inhibition therapy) or to local guidelines, 
whichever are more stringent (Mbaeyi et al. 2020; Kroger et al. 2021). 
Investigators will review each subject’s vaccination status at periodic intervals to ensure that any 
needed booster vaccinations are administered at the optimal timing during the study. Site 
personnel should contact the sponsor medical monitor or designee for any questions. 
If a live vaccine needs to be given during the study, the site should contact the sponsor medical 
monitor or designee for the most up-to-date information. 
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Prophylactic antibiotic therapy may be administered at the discretion of the investigator. If 
administered, local treatment guidelines for patients receiving treatment with complement 
inhibitors should be followed. Before administration of antibiotics, the investigator must check 
the prohibited medication list. 
10. 
STUDY CONDUCT 
10.1. 
Overview 
Subjects who meet all of the inclusion and none of the exclusion criteria can be enrolled into 
Study 205. Prospective subjects should have completed at least 12 weeks treatment with 
BCX9930 in the prior study. The last on-treatment visit completed for the prior study will also 
serve as the baseline visit for Study 205. Therefore, procedures at the last on-treatment visit in 
the prior study and any specific assessments for Study 205 that may be needed will be conducted 
at this visit. (Note: With the expected overlap in study procedures, it is anticipated that few, if 
any, specific procedures for Study 205 will need to be completed. Please contact the sponsor 
medical monitor [or designee] if this is unclear). If subjects have completed at least 24 weeks of 
BCX9930 treatment in the prior study, study visits will occur every 8 weeks to Week 48, then 
every 12 weeks until Week 96. Subjects who have not completed 24 weeks of treatment with 
BCX9930 prior to entering this study must return to the clinic every 4 weeks until they have 
completed 24 weeks of cumulative treatment with BCX9930. More frequent monitoring may be 
required for subjects with any ongoing safety concern, including 
 
Additional visits may be required for subjects who experience hemolysis on study or who require 
unscheduled visits for additional or repeat evaluations. Additional visits will be required in the 
case of 
 for additional detail). 
Subjects who discontinue dosing with BCX9930 for any reason (complete study visits or 
discontinue early), will be asked to complete an EOS or ET visit, as applicable, approximately 
3 weeks (± 3 days) after last dose of BCX9930. 
10.2. 
Schedule of Assessments 
The schedule of study assessments/procedures for this study is presented in Table 2. 
Assessments in the study are intended to be conducted in clinic; they may be conducted remotely 
under extenuating circumstances (eg, COVID-19 restrictions), which will be determined 
individually for each site and/or subject. 
 
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Table 2: 
Study BCX9930-205: Schedule of Study Assessments/Procedures 
Assessment/Procedure 
Baseline 
Day 1a 
Treatment Period 
(Weeks 8, 16, 24, 32, 40, 
48, 60, 72, 84, and 96b) 
EOS/ETc 
Written informed consent 
X 
 
 
Review eligibility criteria 
X 
 
 
Medical & medication historyd 
X 
 
 
HBV, HCV, HIV viral load testinge 
 
 
 
Height, body weight & BMI 
X 
 
 
Symptom-directed physical examinationf 
X 
X 
X 
Vital signsg 
X 
X 
X 
Clinical chemistryh/hematologyi/coagulation/ 
urinalysis including microscopyj 
X 
X 
X 
Pregnancy testk 
X 
X 
X 
FSH testl 
X 
X 
X 
Study drug administrationm 
 
 
 
Study drug accountability 
X 
X 
X 
Record AEs, concomitant medications & 
transfusions 
X 
X 
X 
Abbreviations: AE = adverse event; BID = twice daily; BMI = body mass index; EOS = end of study; ET = 
early termination; FSH = follicle-stimulating hormone; HBV = hepatitis B virus; HBC = hepatitis C virus; 
HIV = human immunodeficiency virus; ICF = informed consent form; PNH = paroxysmal nocturnal 
hemoglobinuria; RBC = red blood cell; WBC = white blood cell. 
a The last on-treatment visit completed for the prior study will also serve as the baseline visit for Study 205. 
Therefore, procedures at the last on-treatment visit in the prior study and any specific assessments for Study 
205 will be conducted at this visit. If there will be a lapse between the end of the prior study and rollover into 
Study 205 such that the baseline visit for Study 205 is not conducted coincident with the last on-treatment visit 
in the prior study, the site must contact the sponsor medical monitor (or designee) to determine whether the 
subject will be eligible to participate in Study 205. If eligible and subsequently enrolled into Study 205, the 
subjects must complete the Study 205 baseline visit. The sponsor medical monitor (or designee) will 
recommend which procedures need to be performed at that visit. 
b Visits may be performed within a window of ± 6 days. Subjects who have not completed 24 weeks of 
treatment with BCX9930 prior to entering this study must return to the clinic every 4 weeks until they have 
completed 24 weeks of cumulative treatment. Subjects with 
 will be required to complete 
additional assessments 
at the investigational site, or at a remote 
laboratory more convenient for the subject as indicated in Section 10.3.1 and Section 12.5.2. Additional visits 
may be required for subjects who experience hemolysis on study or following the permanent discontinuation 
of BCX9930, or who require unscheduled visits for additional or repeat evaluations. Subjects do not need to 
withhold any BCX9930 doses on clinic days or take a dose in the clinic, unless the clinic visit falls during the 
subject’s normal time of dosing. 
c Subjects who discontinue dosing with BCX9930 for any reason (complete study visits or discontinue early), 
will be asked to complete an EOS or ET visit, as applicable, approximately 3 weeks (± 3 days) after last dose 
of BCX9930. 
d Medical and medication history will be obtained from the prior study. 
e For those who entered the prior study with positive serology for HIV or active infection with HBV or HCV, 
the appropriate testing for viral load should be repeated approximately yearly from the date of first viral load 
assessment. If positive, contact the medical monitor to discuss the best option for the subject. 
f Symptom-directed physical examinations will be performed at all regularly scheduled visits. 
g To include blood pressure, pulse rate, and temperature. Prior to obtaining blood pressure and pulse rate, 
subjects should rest quietly for a brief period. 
h Cholesterol and triglycerides will be measured every 6 months (ie, Weeks 24, 48, 72, and 96). 
i PNH RBC and WBC clone size included. 
j Urine microscopy will be performed at the central laboratory as reflex testing in the event of an abnormal 
urinalysis. 
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k For women of childbearing potential, a urine pregnancy test will be assessed at baseline, at onsite visits during 
the treatment period, and at the EOS/ET visit. Women of childbearing potential will be provided with urine 
pregnancy test kits for home testing once a month. 
l FSH may be assessed at any visit if menopausal status changes to confirm menopausal status. The subject 
must have a negative urine pregnancy test as per protocol until menopausal status is confirmed. 
m BCX9930 will be administered orally at a total dose of 400 mg BID without regard to food. 
10.3. 
Study Visits 
Unless indicated otherwise, all subjects are expected to complete all scheduled study 
assessments/procedures. 
Written informed consent must be obtained from each subject before initiation of any 
assessments or procedures conducted specifically for Study 205. Where practical to do so, 
informed consent should be obtained a minimum of 4 weeks prior to the end of the prior 
study to allow the sponsor adequate time to provide new study drug and other supplies to the 
site. 
The current study will consist of the following visits: 
• Baseline Visit (Day 1) 
• On-study Visits 
− Occurring in clinic every 8 weeks at Week 8 (Day 57 ± 6 days), Week 16 
(Day 113 ± 6 days), Week 24 (Day 169 ± 6 days), Week 32 (Day 225 ± 6 
days), Week 40 (Day 281 ± 6 days), until Week 48 (Day 337 ± 6 days), then 
every 12 weeks at Week 60 (Day 421 ± 6 days), Week 72 (Day 505 ± 6 days), 
Week 84 (Day 589 ± 6 days), until Week 96 (Day 673 ± 6 days). Subjects who 
have not completed 24 weeks of treatment with BCX9930 prior to enrolling in 
this study must return to the clinic every 4 weeks until they have completed 
24 weeks of cumulative treatment. 
• Unscheduled Visit 
− Subjects should be instructed to immediately contact the investigator if they 
have new or worsening symptoms of hemolysis. Following the permanent 
discontinuation of BCX9930, subjects may be required to complete an 
additional visit(s) to assess for acute symptomatic hemolysis if and when 
symptoms occur (see Section 12.5.3). 
 
 
 
• EOS/ET Visit 
- Subjects who discontinue dosing with BCX9930 for any reason (complete 
study visits or discontinue early), will be asked to complete an EOS or ET 
visit, as applicable, approximately 3 weeks (± 3 days) after last dose of 
BCX9930. 
For the assessments to be performed at each visit, see Table 2. 
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10.3.1. 
Additional Assessments for Renal and Hepatic Safety Monitoring  
 
 The minimum procedures to be completed 
at each assessment will depend on whether the subject is assessed at the investigative site or 
remotely. 
For subjects assessed at the investigational site: 
• Review/record AEs, concomitant medication use, and blood transfusions  
• Vital signs (resting blood pressure, pulse rate, and temperature)  
• Blood collection for clinical laboratory evaluations  
- Clinical chemistry 
• Urine collection for clinical laboratory evaluations: 
- Urinalysis including microscopy 
- Urine albumin to creatinine ratio (uACR) 
For subjects assessed remotely: 
• Blood collection for clinical laboratory evaluations: 
- Clinical chemistry (to include at a minimum: sCr, eGFR, blood urea nitrogen, 
ALT, AST, ALP, and bilirubin [total and direct]) 
• Urine collection for clinical laboratory evaluations: 
- Urinalysis (to include at a minimum specific gravity, pH, protein, and blood) 
- uACR 
11. 
STUDY ASSESSMENTS 
11.1. 
Chronology of Assessments 
Clinic procedures, such as physical examinations, and vital signs measurements, should be 
completed prior to any blood collection. 
11.2. 
Demographic Information, Medical and Medication History 
Demographic information and medical and medication history will be obtained from the prior 
study. Medication review and review of inclusion and exclusion criteria and prohibited and 
restricted medications will also be updated and rechecked at baseline (Day 1). For subjects 
completing a Study 205-specific baseline visit, demographic information and medical and 
medication history will be rechecked at baseline (Day 1) and updated. 
A PNH history will have been taken in the prior study to document PNH clinical 
characteristics and disease burden. 
11.2.1. 
Contraception Requirements 
The following represents the minimum contraception that should be used by study 
participants and their partners. Additional contraceptive requirements (eg, requiring the 
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female partners of male subjects to additionally use highly effective contraception) may be 
required by local site practice and/or the governing ethics committee. All participants must 
follow all country, regional, and local contraceptive guidelines. It is anticipated that that not 
all contraceptive methods may be available in all countries/regions, so the list should be 
modified accordingly. 
For the purposes of this study, females are considered fertile following menarche and until 
becoming postmenopausal, unless permanently sterile (ie, premenopausal with one of the 
following: documented hysterectomy, documented bilateral salpingectomy, or documented 
bilateral oophorectomy). Documentation can come from site personnel’s review of the 
subject’s medical records, medical examination, or medical history interview. 
Female subjects must continue to meet at least one of the following requirements: 
1. Be a woman of nonchildbearing potential, either postmenopausal (defined as without 
menses for ≥ 12 months without an alternative medical cause with a follicle-
stimulating hormone [FSH] > 40 mIU/mL) or had a hysterectomy, bilateral 
salpingectomy, or bilateral oophorectomy 
2. Be a woman of childbearing potential (defined as a female following menarche and 
prior to becoming postmenopausal who has not had a hysterectomy, bilateral 
salpingectomy, or bilateral oophorectomy) who agrees to use an acceptable highly 
effective contraceptive method while enrolled in the study and for a duration of 
30 days after last dose of study drug.  
Acceptable highly effective contraceptive methods are restricted to the following 
methods with low user dependency: 
− surgical sterilization (ie, bilateral tubal occlusion or vasectomy of the sole 
male partner and the vasectomized partner has received medical assessment of 
surgical success) 
− IUD or IUS 
− implantable progestogen-only hormonal contraception associated with 
inhibition of ovulation  
Exception for South Africa only: Progestogen-only hormonal contraception injection, 
with active monitoring of compliance, is allowed as a contraceptive method. 
Female subjects must abstain from egg donation throughout the study and for a duration of 
30 days after last dose of study drug. 
Male subjects with a female partner of childbearing potential, unless vasectomized, must use 
condoms (with spermicide, where available) for the purposes of contraception throughout the 
study and for a duration of 90 days after the last dose of study drug. It is not known if 
BCX9930 or BCX13559, the major metabolite of BCX9930, are present in human semen. 
Therefore, subjects with a pregnant female partner must use condoms to avoid potential 
exposure of the partner and fetus in utero through semen. 
Male subjects must abstain from sperm donation throughout the study and for a duration of 
90 days after last dose of study drug. 
Abstinence as a contraceptive method will not be an option for subjects regardless of lifestyle 
or sexual activity.  

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Per European Clinical Trials Facilitation and Coordination Group guidance, contraception 
and pregnancy testing requirements must remain in place through the end of relevant 
systemic exposure (CTFG 2020). Based on data generated in healthy subjects in Study 101, 
the geometric mean half-life of BCX9930 was ≤ 17.35 hours (suggesting that 5 half-lives are 
elapsed within approximately 3.6 days). Further, the human mass balance study (Study 102) 
identified BCX13559 (an acyl glucuronide) as the only major metabolite of BCX9930. 
Clearance of BCX13559 was determined to be formation-rate limited. Study 102 also showed 
all subjects achieved > 90% recovery of the administered radioactive dose within 12 days 
(single dose). Together, these findings suggest that the end of relevant systemic exposure for 
BCX9930 and BCX13559 occurs well before the ET or EOS visit performed approximately 
3 weeks after the last dose of BCX9930. 
Methods of contraception, as applicable, for both male and female participants should be 
documented in the source documentation. Investigators will also review each subject’s 
continued use of contraception at periodic intervals to ensure compliance with the protocol 
contraception requirements, including reminding subjects about the need to renew their 
contraception. 
11.3. 
Virology Assessments 
Antibodies to varicella-zoster virus were tested at the screening visit in the prior study. For 
this study, any subject who has an exposure to someone with primary varicella (chickenpox) 
or herpes-zoster (shingles), or who has recently received a live varicella vaccine, should 
contact the investigator, and administration of varicella zoster immune globulin or other 
medications should be considered where available. 
For those who entered the prior study with positive serology for HIV or active infection with 
HBV or HCV, the appropriate testing for viral load should be repeated approximately yearly 
from the date of first viral load assessment. If positive, contact the medical monitor to discuss 
the best option for the subject. 
11.4. 
Effectiveness Assessments 
As currently amended, the protocol no longer requires specific effectiveness assessments to 
be performed. The clinical benefit of treatment with BCX9930 will be assessed for each 
individual subject based on the medical judgement of the investigator and the standard-of-
care practices for the treatment and management of PNH disease at his or her institution. 
Common laboratory measurements of PNH will continue to be assessed and reported in the 
safety testing (eg, Hb, LDH, absolute reticulocyte count, AST, bilirubin, etc.) performed by 
the designated clinical safety laboratory. Any other laboratory testing should be performed 
and monitored per appropriate medical judgement. This information will be used to assess the 
ongoing benefit-risk of continued treatment with BCX9930. 
11.5. 
Safety Assessments 
All assessments will be performed as indicated in the schedule of assessments (Table 2). 
11.5.1. 
Vital Signs 
Measurements of vital signs will include blood pressure, pulse rate, and temperature and 
should be obtained after the subject has rested for at least 5 minutes in a quiet room at a 
comfortable temperature, with the subject’s arm unconstrained by clothing or other material. 
Blood pressure measurements will be obtained with the appropriate cuff size, with the 

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subject’s arm supported at the level of the heart, while the subject is resting in a semi-supine 
position. 
11.5.2. 
Body Weight, Height, and Body Mass Index 
Body weight, height, and body mass index (BMI) will be recorded at the last on-treatment 
visit in the prior study/baseline. For determination of height and body weight, subjects should 
be clothed with shoes removed. 
11.5.3. 
Physical Examination 
A targeted (ie, symptom driven) physical examination will be performed at all regularly 
scheduled assessments to assess for any changes from the previous examination, including, at 
a minimum, evaluation of new or worsening signs or symptoms. 
11.5.4. 
Clinical Chemistry, Hematology, Urinalysis, and Other Laboratory 
Assessments 
The following text assumes the use of a central laboratory to support the clinical laboratory 
testing for safety monitoring, including testing for laboratory measurements of the 
effectiveness of PNH treatment. However, at investigational sites where it is no longer 
considered necessary and/or practical to use a central laboratory for the safety oversight of 
clinical study subjects, local laboratory services may be used. Data will continue to be 
reported and available to the sponsor to meet ongoing safety review commitments. 
Blood and urine samples will be collected during the study. Individual laboratory tests are 
specified in Table 3. In general, all laboratory samples collected at visits to the investigational 
site should be collected using kits provided by the designated central laboratory. However, at 
the investigator’s discretion, local laboratories may be used in place of the central laboratory 
(eg, for logistical reasons such as delays in transport due to COVID-19) or for analysis of 
samples collected for assessment of possible AEs (eg, when emergent safety concerns require 
expedited turnaround times for safety laboratory assessments). The additional renal and 
hepatic safety assessments may be performed at the investigational site or remotely at a 
location more convenient for the subject. The results from laboratory testing performed at a 
local or remote laboratory must be provided to the investigational site. The use of a local or 
remote laboratory in this manner, and any differences in analyte panels with the central 
laboratory, based on the availability of testing at the local or remote laboratory, will not be 
considered protocol deviations for the purposes of this protocol. Reference ranges for each 
local or remote laboratory used will be provided to the sponsor and included in data listings. 
Results from the laboratory values should be reviewed as received by the investigator. 
Evidence of this review should be provided in the source records and may include printing of 
the laboratory reports with a signature attesting to a review. For out-of-range laboratory 
findings, the interpretation of clinically significant or not clinically significant should be 
denoted in the source records. Clinically significant laboratory findings, as assessed by the 
investigator, should be recorded as AEs as described in the protocol (Section 12.1). 

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Table 3: 
Clinical Laboratory Evaluations 
Serum Chemistry 
Urinalysis with Microscopya 
Albumin 
Alanine aminotransferase (ALT) (SGPT) 
Alkaline phosphatase (ALP) 
Amylaseb 
Aspartate aminotransferase (AST) (SGOT) 
Bilirubin (total and direct) 
Blood urea nitrogen (BUN) 
Calcium 
Chloride 
Cholesterol (total, high- and low-density 
lipoprotein) 
Creatine (phospho)kinase (C[P]K) 
Creatinine (sCr) 
Glucose 
Haptoglobin 
Lactate dehydrogenase (LDH) 
Magnesium 
Potassium 
Sodium 
Protein (total) 
Triglycerides 
Uric acid 
Albumin 
Bilirubin 
Blood 
Creatinine 
Glucose 
Ketones 
Leukocytes 
Nitrites 
pH 
Protein 
Specific gravity 
Urine albumin to creatinine ratio (uACR) 
Urobilinogen 
Pregnancy/Menopause 
Follicle-stimulating hormone (FSH) 
Urine pregnancy testc 
Coagulation/Fibrinolysis 
Activated partial thromboplastin time (aPTT) 
Prothrombin time (PT) 
International normalized ratio (INR) 
Thrombin time 
Hematology 
Hemoglobin (Hb) 
Hematocrit (Hct) 
Mean corpuscular hemoglobin (MCH) 
Mean corpuscular hemoglobin concentration 
(MCHC) 
Mean corpuscular volume (MCV) 
Platelet count (PLT) 
Red blood cell (erythrocyte) count (RBC) 
Reticulocyte count 
White blood cell count (WBC) with differential 
Other Testing 
Estimated glomerular filtration rate(eGFR)d 
PNH RBC clone size (total) 
PNH WBC clone size 
Varicella-zoster virus (VZV) immunoglobulin G 
(IgG) titer (where appropriate) 
Hepatitis B virus (HBV), hepatitis C virus (HCV), 
human immunodeficiency virus (HIV) viral load 
(where appropriate) 
Abbreviations: DNA = deoxyribonucleic acid; PNH = paroxysmal nocturnal hemoglobinuria; 
RNA = ribonucleic acid; SGOT = serum glutamic-oxaloacetic transaminase; SGPT = serum glutamic-pyruvic 
transaminase. 
Note: Some analytes may not be analyzed, depending on the availability of validated assays at the designated 
regional central laboratory 
a Reflex to microscopy if dipstick is abnormal. For local laboratory testing, urinalysis analytes may be different.  
b If amylase is elevated, reflex to lipase. 
c For women of childbearing potential (or as required by local or institutional practice). 
d To be calculated by the designated central reference laboratory using the Chronic Kidney Disease 
Epidemiology Collaboration (CKD-EPI) equation using the serum creatinine method (Levey and Stevens 
2010). 

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11.5.4.1. Urine Collections for Urinalysis and Urine Microscopy 
Investigators should emphasize to staff and the subjects the importance of collecting fresh, 
high-quality urine specimens (clean-catch, mid-void, etc.) and ensuring appropriate handling 
and prompt processing of the samples for urinalysis. Urine microscopy will be performed at 
the central laboratory as a reflex test if the urinalysis testing is abnormal. For subjects with 
 sites are encouraged to perform urine microscopy using their local laboratory, 
where appropriate on-site testing capabilities are available, to assess for crystals and/or casts 
(see also Section 12.5.2). 
11.5.5. 
Menopause and Pregnancy Testing 
FSH may be assessed at any visit if menopausal status changes to confirm menopausal status. 
The subject must have a negative urine pregnancy test as per protocol until menopausal status 
is confirmed. 
For women of childbearing potential, a urine pregnancy test will be assessed at baseline, at 
monthly intervals during the treatment period, and at the EOS/ET visit, or as required per 
normal site practice. A serum pregnancy test should immediately be drawn and sent for 
analysis for any positive urine pregnancy test. Where required, pregnancy testing may be 
performed using the local laboratory, and using serum testing in lieu of the urine pregnancy 
tests. 
Women of childbearing potential will be provided with urine pregnancy test kits for home 
testing once a month. 
12. 
ADVERSE EVENTS 
12.1. 
Definition of Adverse Events 
12.1.1. 
Adverse Event 
An AE is any untoward medical occurrence in a clinical study subject. No causal relationship 
with the study drug/IMP or with the clinical study itself is implied. An AE may be an 
unfavorable and unintended sign, symptom, syndrome, or illness that develops or worsens 
during the clinical study. Clinically relevant abnormal results of diagnostic procedures 
including abnormal laboratory findings (eg, requiring unscheduled diagnostic procedures or 
treatment measures, or resulting in withdrawal from the study) are considered to be AEs. 
Abnormal results of diagnostic procedures that are incidental or not clinically relevant are not 
reportable as AEs. If the diagnostic procedure prompts no additional treatment, visits, or 
monitoring, it will not meet the definition of an AE. 
This includes the following: 
• AEs not previously observed in the subject that emerge during the protocol-specified 
AE reporting period (see Section 12.4.1). 
• Findings from protocol-mandated interventions. This can include laboratory 
assessments performed in the course of the clinical study. AEs should be reported 
only if the abnormalities are changes from baseline and are clinically significant as 
described above. 
• Pre-existing medical conditions (other than the condition being studied) judged by the 
investigator to have worsened in severity or frequency or changed in character during 
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the protocol-specified AE reporting period. When recording such events on an 
AE/SAE eCRF page, it is important to convey the concept that the pre-existing 
condition has changed by including applicable descriptors (eg, “more frequent 
headaches”). 
An adverse reaction is defined in International Council for Harmonisation (ICH) E6 guideline 
as follows: all untoward and unintended responses to a study drug/IMP related to any dose 
administered. The definition covers also medication errors and uses outside what is foreseen 
in the protocol, including misuse and abuse of the product. The definition implies a 
reasonable possibility of a causal relationship between the event and the study drug/IMP. 
This means that there are facts (evidence) or arguments to suggest a causal relationship.  
Hospitalization scenarios do not require reporting as an SAE where there is no occurrence of 
an AE. These scenarios include a planned hospitalization or prolonged hospitalization to: 
• Perform a routine control screening for a pre-existing illness or to diagnose a 
suspected illness. In the case of the latter, the symptomatology should be reported as 
an AE and amended if a diagnosis is confirmed. 
• Undergo a diagnostic or elective surgical procedure for a pre-existing medical 
condition that has not changed (eg, a joint replacement for which the subject was on a 
waiting list). 
• Undergo medical observation without the occurrence of an AE due to standard of care 
in the region or hospital. 
Surgical procedures should not be reported as AEs. The condition for which the surgery is 
required should be reported as the AE if it occurs or is detected during the study period. 
Planned surgical measures permitted by the clinical study protocol and the conditions(s) 
leading to these measures are not AEs if the condition(s) was (were) known before the start of 
study treatment. In the latter case, the condition should be reported as medical history. 
AEs are designated as “nonserious” or “serious”. 
12.1.2. 
Serious Adverse Event  
A serious adverse event (SAE) is an AE/reaction that results in any of the following 
outcomes: 
• Death 
• Is life-threatening (subject is at immediate risk of death at the time of the event; it 
does not refer to an event that hypothetically might have caused death if it were more 
severe) 
• Requires subject hospitalization (formal admission to a hospital for medical reasons) 
or prolongation of existing hospitalization 
• Results in persistent or significant disability/incapacity (ie, there is a substantial 
disruption of a person’s ability to carry out normal life functions) 
• Is a congenital anomaly/birth defect 
Important medical events that may not result in death, are not life-threatening, or do not 
require hospitalization may be considered SAEs when, based upon appropriate medical 
judgment, they may jeopardize the subject’s health or may require medical or surgical 
intervention to prevent one of the outcomes listed in this definition. Examples of such events 

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may include allergic bronchospasm requiring intensive treatment in an emergency room or at 
home, blood dyscrasias, or convulsions that do not result in subject hospitalization. 
In addition, the sponsor considers any abortions (elective or spontaneous), fetal demise, and 
still birth to be SAEs for reporting purposes (see Section 12.4.2). 
Some hospitalization scenarios, as outlined in Section 12.1.1, do not require reporting as 
SAEs. 
Overdose will be considered an SAE only if any of the seriousness criteria are met. Any 
clinical complication in association with the overdose should be reported as an AE or SAE 
(as applicable) along with the overdose (see Section 12.6). Details of signs or symptoms, 
clinical management, and outcome should be reported, if available. Overdose without 
associated signs or symptoms should not be recorded as AEs but should be recorded as 
protocol deviations. 
12.1.3. 
Events of Special Monitoring 
Events of special monitoring (EOSM) are AEs or safety topics for which special monitoring, 
additional data collection activities, and/or enhanced signal detection activities within 
BioCryst are considered appropriate for this population and study. Identified EOSMs can be 
of particular concern based on findings from the IMP clinical program to date, potential 
and/or known risks generally associated with the underlying disease, or comorbidities and 
risk factors prevalent in the study population. 
EOSM for this study include 
 
12.2. 
Definition of Severity 
All AEs will be assessed (graded) for severity by the investigator and classified using the US 
National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 
(Version 5.0, published 27 November 2017). The NCI CTCAE is a descriptive terminology 
that can be used for AE reporting. The CTCAE displays Grades 1 through 5 with unique 
clinical descriptions of severity for each AE based on the following general guideline: 
Mild: 
(Grade 1): Mild; asymptomatic or mild symptoms; clinical or diagnostic 
observations only; intervention not indicated. 
Moderate: 
(Grade 2): Moderate; minimal, local or noninvasive intervention 
indicated; limiting age-appropriate instrumental ADLa. 
Severe: 
(Grade 3): Severe or medically significant but not immediately 
life-threatening; hospitalization or prolongation of hospitalization 
indicated; disabling; limiting self-care ADLb. 
Life-threatening: 
(Grade 4): Life-threatening consequences; urgent intervention indicated. 
Death 
(Grade 5): Death related to AEc. 
Abbreviations: ADL = activities of daily living; AE = adverse event. 
a Instrumental ADL refer to preparing meals, shopping for groceries or clothes, using the telephone, managing 
money, etc. 
b Self-care ADL refer to bathing, dressing and undressing, feeding self, using the toilet, taking medications, and 
not bedridden. 
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c Grade 5 (Death) is not appropriate for some AEs and therefore is not an option. 
It is important to distinguish between serious and severe AEs. Severity is a measure of 
intensity, as defined above, whereas seriousness is defined by the criteria under 
Section 12.1.2. An AE of severe intensity may or may not be considered serious. 
12.3. 
Relationship to Study Drug 
An investigator who is qualified in medicine must make the determination of relationship to 
the study drug/IMP for each AE (not related, unlikely related, possibly related, probably 
related, definitely related). The investigator should decide whether, in his or her medical 
judgment, there is a reasonable possibility that the event may have been caused by the 
investigational product. If no valid reason exists for suggesting a relationship, then the AE 
should be classified as “not related”. If there is any valid reason, even if undetermined, for 
suspecting a possible cause-and-effect relationship between the study drug/IMP and the 
occurrence of the AE, then the AE should be considered “related”. 
Not Related: 
The event can be readily explained by other factors such as the subject’s 
underlying medical condition, concomitant therapy, or accident, and no 
temporal relationship exists between the study drug/IMP and the event. 
Unlikely 
Related: 
The event does not follow a reasonable temporal sequence from drug 
administration and is readily explained by the subject’s clinical state or by 
other modes of therapy administered to the subject. 
Possibly 
Related: 
There is some temporal relationship between the event and the administration 
of the study drug/IMP and the event is unlikely to be explained by the subject’s 
medical condition, other therapies, or accident. 
Probably 
Related: 
The event follows a reasonable temporal sequence from study drug/IMP 
administration, abates upon discontinuation of the study drug/IMP, and cannot 
be reasonably explained by the known characteristics of the subject’s clinical 
state. 
Definitely 
Related: 
The event follows a reasonable temporal sequence from study drug/IMP 
administration, follows a known or suspected response pattern to the study 
drug/IMP, is confirmed by improvement upon stopping the study drug/IMP 
(dechallenge), and reappears upon repeated exposure (rechallenge, if 
rechallenge is medically appropriate). 
If the relationship between the (S)AE and the investigational product is determined to be 
“possible”, “probable”, or “definite”, the event will be considered to be related to the study 
drug/IMP for the purposes of expedited regulatory reporting (as applicable per country-
specific regulatory requirements). 
The sponsor may upgrade causality if deemed appropriate. 
12.4. 
Method, Frequency, and Time Period for Detecting Adverse 
Events and Reporting Serious Adverse Events 
Reports of all AEs and SAEs, regardless of investigator attribution, are to be collected from 
the time of enrollment through to the last study visit (ie, through the post-treatment follow-up 

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visit). If AEs/SAEs occur after the subject has signed the informed consent form (ICF) for 
Study 205 but while the subject is still enrolled in the prior study, the AEs/SAEs will be 
collected according to the procedures specified in the prior study protocol. All AEs and SAEs 
are to be reported on the AE eCRF. 
AEs should be documented on the eCRFs as investigators become aware of them. AEs are to 
be followed until the event resolves. If an event is ongoing at the last follow-up visit, Grade 1 
and Grade 2 events do not need to be followed if the event is deemed unlikely to be related or 
not related to study drug/IMP (see Section 12.2 for AE grading). For all Grade 3 and Grade 4 
events or events deemed possibly, probably, or definitely related to use of study drug/IMP, 
the event should be followed until the AE is resolved or the subject is in a clinically stable 
condition with regard to the AE. 
The investigator shall report all SAEs immediately and no later than 24 hours of their 
knowledge of the event to the sponsor by communicating with the medical monitor (phone or 
email) and by entering the event into the AE eCRF and by completion of the SAE eCRF. The 
SAE eCRF is an additional form to the AE eCRF that provides important details on the SAE. 
The investigator should follow all unresolved SAEs observed during the study until they are 
resolved, or are judged medically stable, or are otherwise medically explained. 
The investigator should attempt, if possible, to establish a diagnosis based on the presenting 
signs and symptoms. In such cases, the diagnosis should be documented as the AE and not 
the individual sign/symptom. If a clear diagnosis cannot be established, each sign and 
symptom must be recorded individually. Once a diagnosis is made during evaluation or 
treatment, the investigator will update the AE record with this diagnosis and delete the 
previously reported signs and symptoms. The rapid reporting of SAEs ensures that the 
sponsor shall have the necessary information to continuously assess the benefit-risk profile of 
the study drug in clinical studies.  
12.4.1. 
Serious Adverse Events and Suspected Unexpected Serious Adverse 
Reactions 
The investigator must report all SAEs immediately and in no case later than within 24 hours 
of their knowledge of the event. Investigators should adhere to their country or region 
requirements for the reporting timeframe, which may not allow any delay. SAEs should be 
reported to the sponsor medical monitor and via the AE and SAE eCRFs. The SAE eCRF is 
an additional form to the AE eCRF that provides important details on the SAE. All additional 
follow-up evaluations of the SAE must be reported to BioCryst or its designee as soon as they 
are available by amending these eCRFs. 
In the event the eCRF system is not functioning, the reporting of an SAE must not be 
delayed. Sites will have SAE report forms (electronic Word document) that can be completed 
and emailed to the below recipient. As soon as the eCRF system is functioning, that particular 
SAE must be entered into the AE eCRF. The eCRF system automatically sends SAE 
notifications to the following email address: safety@biocryst.com. 
Immediate reporting should allow BioCryst to take the appropriate measures to address 
potential new risks in a clinical study. Therefore, the initial report should be submitted by the 
investigator within a very short period of time and under no circumstances should this period 
exceed 24 hours following awareness of the SAE. 
The follow-up report should allow BioCryst to determine whether the SAE requires a 
reassessment of the benefit-risk profile of the study drug/IMP in clinical study, if the relevant 
information was not already available and provided in the initial report. 

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Country-specific rules and requirements for safety management will be described in the 
Safety Management Plan prepared for this study. 
Investigators or designees at each site are responsible for retaining copies of all suspected 
unexpected serious adverse reaction (SUSAR) reports (initial and follow-up) and other safety 
information (eg, revised IB) in the investigator site files. 
BioCryst shall ensure that all relevant information about SUSARs that are fatal or 
life-threatening is recorded and reported as soon as possible to all competent authorities, and 
to the applicable ethics committee in any case no later than 7 calendar days after knowledge 
by BioCryst of such a case, and that relevant follow-up information is subsequently 
communicated within an additional 8 days. All other SUSARs shall be reported to the 
competent authorities concerned and to the ethics committees, as applicable, according to 
local regulations, as soon as possible but in no case later than 15 calendar days of first 
knowledge by BioCryst. BioCryst or designee shall also inform the investigator. 
 
 
12.4.2. 
Pregnancy 
Any female subject who becomes pregnant during the course of the study should have 
BCX9930 discontinued immediately and must be followed through the end of the pregnancy. 
While pregnancy is not considered an AE, all cases of fetal drug exposure via the parent as a 
study participant (including partners of study participants) are to be reported immediately to 
BioCryst or its designee. Consent from study partners who become pregnant will be obtained 
prior to reporting any details of the pregnancy. Information related to the pregnancy must be 
given on a “Pregnancy Notification and Outcome” form that will be provided by the sponsor 
or its designee so that the pregnancy may be followed, and an outcome determined. Any AEs 
or SAEs experienced by a pregnant subject are to be reported as directed in Section 12.4.1. 
Any complications reported in a subject’s pregnant partner should be reported on the 
Pregnancy Notification and Outcome form. All pregnancies must be followed to outcome, 
which occurs when an infant is delivered (live or still born), there is fetal demise, or there is 
an abortion (spontaneous or induced). Abortion (spontaneous or induced), fetal demise, and 
still birth, along with congenital abnormalities in the newborn, should be reported as separate 
SAEs. 
12.5. 
Adverse Event Management 
The investigator (or qualified designee) will grade clinically significant events and laboratory 
abnormalities (if considered AEs) as detailed in Section 12.2. Grade 3 and Grade 4 clinically 
significant laboratory abnormalities should be confirmed by repeat testing and before any 
contemplated study drug/IMP discontinuation, unless such a delay is not consistent with good 
medical practice. 
12.5.1. 
Potential Drug-induced Liver Injury: Monitoring, Evaluation, and 
Discontinuation Criteria 
All treatment-emergent ALT elevations > 3 × ULN should be confirmed, preferably within 
72 hours, with repeat assessment of ALT, AST, total bilirubin, ALP, prothrombin time/INR, 
and complete blood count with differential. These may be repeated at a local laboratory as 
long as the results are reported to the investigator when available and the investigative site 
contacts the subject to ascertain any symptoms. 
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Subjects with ALT elevation > 3 × ULN must be assessed to determine whether study drug 
must be withheld or discontinued. In addition, any potentially contributing medications, 
supplements, or over-the-counter medications that are not medically necessary should be 
discontinued if medically feasible. 
Discontinuation of treatment should be considered for any of the following, and should be 
discussed with the sponsor medical monitor prior to BCX9930 discontinuation, considering 
the benefit-risk of discontinuing BCX9930: 
• ALT or AST > 8 × ULN 
• ALT or AST > 5 × ULN for more than 2 weeks 
• ALT or AST > 3 × ULN and (total bilirubin > 2 × ULN [unless there is evidence 
that the increase in bilirubin is due to hemolysis or Gilbert’s syndrome] or 
INR > 1.5 × ULN in the absence of warfarin therapy) 
• ALT or AST > 3 × ULN with the appearance of fatigue (over baseline fatigue), 
nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or 
eosinophilia (> 5%) 
If subjects are asymptomatic with no other pertinent laboratory abnormality, study drug may 
be continued under close observation. Transaminases, total bilirubin, ALP, and INR should 
be repeated at least twice weekly. Frequency of retesting can decrease to once per week or 
less if abnormalities stabilize or the study drug has been discontinued and the subject is 
asymptomatic. Additionally, several other items should be considered, including obtaining a 
more detailed history of symptoms and prior or concurrent diseases; obtaining a history of 
concomitant use of nonprescriptive medications, herbal and dietary supplements, alcohol 
intake, recreational drug use, or special diets; evaluation for acute viral hepatitis A, B, C, D, 
and E and potentially other serologies; evaluation for others as clinically indicated including 
alcoholic and autoimmune hepatitis, non-alcoholic steatohepatitis (NASH), and biliary tract 
disease. 
The above criteria will be applied to increases in AST unless there is evidence that the 
increase is due to hemolysis. If both ALT and AST are elevated, subjects will be managed 
based on their ALT level, regardless of whether their AST increase is thought to be due to 
hemolysis. 
If Hy’s Law criterion is met and is assessed as probably or definitely related per treatment 
discontinuation criterion in Section 7.4.1, then study drug must be discontinued. Prior to 
discontinuation from study drug/BCX9930, the investigator should contact the sponsor 
medical monitor for a discussion regarding the event and assessment. If medically indicated, 
the investigator can discontinue the subject immediately and follow up with the sponsor 
within 24 hours. 
12.5.2. 
Renal Events: Monitoring, Evaluation, and Discontinuation Criteria 
There are multiple potential reasons that sCr can increase in those with PNH, including 
hemolysis. For subjects who have a confirmed treatment-emergent increase in sCr, 
evaluations to investigate potential etiology should be chosen considering baseline renal 
function, history of prior renal events including those due to PNH, other comorbidities, 
concomitant medications, and other relevant factors. In addition, the following 
recommendations are made: 

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12.5.3. 
Risk of Hemolysis After Missed Doses, Treatment Interruption, or 
Permanent Discontinuation 
Investigators should remind subjects of the potential risk of hemolysis following missed 
doses (particularly multiple consecutive missed doses) and treatment interruption, and the 
definite risk after permanent discontinuation of BCX9930. By inhibiting the AP of 
complement, BCX9930 may improve Hb levels and increase circulating RBC clone size by 
reducing the hemolysis of PNH RBCs. Missed doses and treatment interruption of BCX9930 
may lead to hemolysis. Subjects are at risk of severe hemolysis when BCX9930 is 
permanently discontinued. Based on available data regarding the PK and PD of BCX9930, 
severe hemolysis, if it occurs, would be expected to present within days after discontinuation 
of therapy.  
Monitoring for hemolysis should be performed based on the medical judgement of the 
investigator. However, when subjects permanently discontinue BCX9930 at any time during 
the study, the following recommendations are made: 
• If the subject is asymptomatic, it is recommended that contact with the subject be 
maintained on a daily basis for a minimum of 7 days. Contact may be conducted 
by telephone, email, or other means, including in person, if preferred. When any 
sign or symptom of hemolysis occurs, the subject should be promptly evaluated. 
Additional monitoring following the first contact after permanent discontinuation 
should be guided by the medical judgement of the investigator. 
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• For subjects who are already symptomatic when treatment is discontinued, 
whether the subject should be assessed in person or can be followed with daily 
contact will be based on the investigator’s medical judgement taking into account 
the severity of the reported signs or symptoms and/or any reported worsening of 
the signs or symptoms. When possible, subjects should return to the investigative 
center; however, when this is not possible, arrangements may be made for the 
subject to be assessed by his or her local hematologist and a local laboratory used 
for clinical laboratory testing. When hemolysis occurs after the permanent 
discontinuation of BCX9930, treatment should be provided according to the 
judgement of the investigator. 
• Subjects should be asked to contact the investigator if they experience any new or 
worsening signs or symptoms of hemolysis. Subjects should be evaluated per the 
investigator’s medical judgement. A review of dosing compliance to ensure that 
the subject is taking the drug should be performed, regardless of the method used 
to evaluate the subject (ie, in-person or remote assessment). It should also be 
determined whether the subject has had any recent complement-activating 
conditions (eg, infection, trauma, or surgery) that may have contributed to the 
hemolytic event. 
• Treatment with BCX9930 should not be interrupted or withheld unless there is an 
absolute contraindication for continued dosing. Suggestions for anti-emetic 
therapy for subject with active nausea and vomiting when tablet contents are 
vomited are given in Section 9.6.2. 
The sponsor medical monitor should be notified in the event of hemolysis symptoms, any 
planned treatment interruption, or permanent discontinuation of BCX9930. Any missed 
doses, treatment interruption, or permanent discontinuation must be recorded in the eCRF and 
source documents, including the reason for the interruption, missed doses, or discontinuation. 
12.6. 
Overdose 
To date, there is no experience with overdose of BCX9930. Healthy subjects received 
BCX9930 at a dose of 2000 mg administered as single doses and as multiple once-daily doses 
for 3 days in Study 101 with no clinically significant safety concerns. If study personnel 
become aware of an overdose of BCX9930 that is associated with an AE, both the overdose 
and the resultant event should be reported as AEs. Overdose without any symptoms (ie, AEs) 
does not need to be reported as an AE. If overdose occurs with or without associated AEs, 
subjects should undergo clinical and laboratory monitoring, as appropriate, for their clinical 
condition and, if indicated, should receive clinically indicated supportive therapy. See also 
Section 12.1.2. 
12.7. 
Data Monitoring Committees 
A DMC has been established for ongoing safety monitoring. The specific responsibilities and 
composition of the DMC are outlined in a separate DMC charter. In addition, the details of 
outputs provided for the meetings will be referenced in this charter. 
 
 
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13. 
STATISTICS 
13.1. 
Sample Size Considerations 
No sample size calculations were conducted for this open-label, long-term safety study. Up to 
30 subjects may be enrolled in this study, to allow continued access to BCX9930 following a 
subject’s participation in another BCX9930 study.  
13.2. 
Stratification 
Not applicable. 
13.3. 
Statistical Methods 
A detailed statistical analysis plan (SAP) will be developed to describe the methods of 
analyses and summaries, including all endpoints, time points, populations, missing data, etc. 
Deviations from the analyses outlined in the SAP will be described in the clinical study 
report. 
13.3.1. 
Analysis Populations 
The analysis populations for the study are defined below. 
13.3.1.1. Safety Population 
The safety population is defined as all subjects who receive at least 1 dose of study drug. This 
population will be used for all analyses of accountability, demographics, and safety. 
13.3.2. 
General Considerations for Data Analysis 
In general, descriptive summaries will include the sample size (n), mean, standard deviation 
(SD) and/or standard error (SE), median, minimum, and maximum for continuous variables 
and n and percentage for categorical variables. Data will be summarized by prior study and 
overall. Summaries will be presented by study visit where appropriate (eg, laboratory data) 
until subjects complete or withdraw from the study. No missing value imputation will be 
done. No statistical testing is anticipated as there are no a priori hypotheses stated for this 
study. 
All individual subject data will be listed as measured. All statistical summaries and analyses 
will be performed using SAS® software (SAS Institute, Cary, North Carolina, USA), using 
version 9.4 or higher. 
13.3.3. 
Subject Demographic and Disposition Data 
Demographic data and baseline characteristics including age, gender, race or ethnicity, 
height, body weight, BMI, and PNH history will be summarized by prior study and overall. 
Subject disposition will be presented for all subjects and by prior study. The number of 
subjects who completed the study and those that discontinued from the study will be 
provided. The reasons for early discontinuation will be presented. A tabulation of the number 
of subjects exposed to study drug and duration of exposure will also be presented. Treatment 
adherence, dose interruptions, and reason for dose interruptions will be provided as 
summaries or listed, as appropriate. 

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13.3.4. 
Analysis of Safety Variables 
Safety endpoints will be summarized and will include, at a minimum, the proportion of 
subjects 1) with TEAEs; 2) who discontinue due to a TEAE; 3) with treatment-emergent 
SAE; 4) with a Grade 3 or 4 TEAE; and 5) with Grade 3 or 4 laboratory abnormalities. 
AEs will be mapped to a Medical Dictionary for Regulatory Activities (MedDRA, Version 
24.0) preferred term and system organ classification. The occurrence of TEAEs will be 
summarized using MedDRA preferred terms, system organ classifications, and severity. In 
addition to severity, AEs and SAEs will also be summarized based on investigator assessment 
of relationship to study drug. 
For all analyses of change from baseline, the baseline is the baseline value for the current 
study. 
Descriptive statistics for vital signs, weight, and clinical laboratory results will be presented 
by study visit. Laboratory abnormalities will be graded according to CTCAE scales (Version 
5.0, 27 November 2017). The number and percentage of subjects experiencing treatment-
emergent graded laboratory toxicities will be summarized. Laboratory toxicity shifts from 
baseline to the worst post-baseline value as well as to the last visit will be summarized. 
The number and percentage of subjects having elevations in ALT, AST, or bilirubin 
abnormalities in relation to fold above ULN will be summarized according to the US FDA’s 
Premarketing Clinical Evaluation on Drug-Induced Liver Injury Guidance for Industry (FDA 
2009). 
14. 
STUDY ADMINISTRATION 
14.1. 
Study Monitoring 
Before an investigational site can enter a patient into the study, a representative(s) of 
BioCryst or its designee will assess the investigational study site to: 
• Determine continued adequacy of the facilities 
• Discuss with the investigator(s) and other personnel their responsibilities with regard 
to protocol adherence, and the responsibilities of BioCryst or its designee. This will 
be documented in a Clinical Study Agreement between BioCryst and the investigator. 
During study conduct, BioCryst or its designee will conduct periodic monitoring visits to 
ensure that the protocol and Good Clinical Practice (GCP) are being followed. The monitor(s) 
will review source documents to confirm that the data recorded on eCRFs are accurate. The 
investigator and institution will allow the representative(s) of BioCryst or its designee access 
to source documents to perform this verification. 
It is important that the investigator(s) and relevant personnel are available during the 
monitoring visits and that sufficient time is devoted to the process. 
During the study, a representative(s) from BioCryst or its designee will have regular contacts 
with the investigational site personnel for the following: 
• Provide information and support to the investigator(s) 
• Confirm that facilities remain acceptable 

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• Confirm that the investigational team is adhering to the protocol, that data are being 
accurately recorded in the eCRFs, and that IMP accountability checks are being 
performed 
• Perform source data verification. This includes a comparison of the data in the eCRFs 
with the subject’s medical records at the hospital or practice, and other records 
relevant to the study. This will require direct access to all original records for each 
subject (eg, clinic charts). 
• Record and report any protocol deviations not previously sent to BioCryst or its 
designee 
• Confirm AEs and SAEs have been properly documented on the eCRFs and confirm 
any SAEs have been forwarded to BioCryst or its designee and those SAEs that met 
criteria for reporting have been forwarded to the ethics committee 
The representative(s) of BioCryst or its designee will be available between visits if the 
investigator(s) or other staff needs information or advice. 
14.2. 
Audits and Inspections 
Authorized representatives of BioCryst or its designee, US FDA and other regulatory 
authorities, and/or ethics committee may visit the site to perform audits or inspections, 
including source data verification. The purpose of a BioCryst audit or inspection is to 
systematically and independently examine all study-related activities and documents to 
determine whether these activities were conducted, and data were recorded, analyzed, and 
accurately reported according to the protocol, standard operating procedures, ICH GCP 
guidelines, and any applicable regulatory requirements. The investigator should contact 
BioCryst immediately if contacted by a regulatory agency about an inspection. 
It is important that the investigator and relevant personnel are available during the possible 
audits or inspections and that sufficient time is devoted to the process. 
14.3. 
Ethics Committee  
The investigator must obtain ethics approval for the investigation. Initial ethics approval and 
all materials approved by the ethics committee, including the ICF and any recruitment 
materials, must be maintained by the investigator and made available for inspection. 
14.4. 
Serious Breaches of GCP 
It is the responsibility of the sponsor to notify the competent authority of any serious breach 
of GCP that is likely to affect, to a significant degree, the safety or mental integrity of the 
subjects of the study or the scientific value of the study. All serious breaches will be notified 
to the relevant competent authority in accordance with locally applicable regulations. The 
reporting to the sponsor will be performed by the party who suspects the serious breach. 
15. 
QUALITY CONTROL AND QUALITY ASSURANCE 
During study conduct, BioCryst or its designee will conduct periodic monitoring visits to 
ensure that the protocol and GCPs are being followed as described in Section 14.1. 
To ensure compliance with GCP and all applicable regulatory requirements, BioCryst or its 
designee may conduct a quality assurance audit. Please see Section 14.2 for more details 

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regarding the audit process. The investigator agrees to allow the auditors to inspect the drug 
storage area, study drug stocks, drug accountability records, subject charts and study source 
documents, and other records relative to study conduct.  
16. 
ETHICS 
16.1. 
Ethics Review 
The final study protocol and the final version of the ICF must be approved or given a 
favorable opinion in writing by an ethics committee, as appropriate. The investigator must 
submit written approval from the ethics committee to BioCryst before he or she can enroll 
any subject into the study. 
The ethics committee will be informed of any amendment to the protocol in accordance with 
local requirements. In addition, the ethics committee must approve any advertising used to 
recruit subjects for the study. The protocol must be re-approved by the ethics committee upon 
receipt of amendments and annually, as local regulations require. 
The ethics committee will be provided with reports of any reportable serious adverse drug 
reactions from any other study conducted with the investigational product, in accordance with 
local regulations. BioCryst will provide this information to the investigator. 
Progress reports and notifications of serious adverse drug reactions will be provided to the 
ethics committee according to local regulations and guidelines. 
16.2. 
Ethical Conduct of the Study 
The study will be performed in accordance with ethical principles that have their origin in the 
Declaration of Helsinki and are consistent with ICH GCP, applicable regulatory 
requirements, and BioCryst’s policies. 
16.3. 
Written Informed Consent 
In accordance with applicable national or local law, and current institutional practice, written 
informed consent to participate in the study will be obtained from each subject prior to 
conducting any study-related assessments/procedures. 
A signed ICF must be obtained from each subject prior to performing any study-related 
procedures. Each subject should be given both oral and written information describing the 
nature, purpose, and duration of the study. Subjects will be informed that they are free not to 
participate in the study and that they may withdraw consent to participate at any time. They 
will be told that refusal to participate in the study will not prejudice future treatment. They 
will also be told that their records may be examined by competent authorities and authorized 
persons, but that personal information will be treated as strictly confidential and will not be 
publicly available. The informed consent process should take place under conditions where 
the subject has adequate time to consider the risks and benefits associated with participation 
in the study. Subjects must be given the opportunity to ask questions. Subjects will not be 
screened or treated until the subject has signed an approved ICF written in a language in 
which the subject is fluent. 
The ICF that is used must be approved both by BioCryst and by the governing ethics 
committee. The ICF should be in accordance with the current revision of the Declaration of 
Helsinki, current ICH and GCP guidelines, and BioCryst policies. 

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The subject should receive a signed and dated copy of the ICF. The original signed ICF 
should be retained in the study files. 
The investigator shall maintain a log of all subjects for whom consent was signed and 
indicate if the subject was enrolled into the study or reason for non-enrollment. 
17. 
DATA HANDLING AND RECORDKEEPING 
17.1. 
Inspection of Records 
BioCryst or designee will be allowed to conduct site visits to the investigation facilities for 
the purpose of monitoring any aspect of the study. The investigator agrees to allow the 
monitor to inspect the drug storage area, study drug stocks, drug accountability records, 
subject charts and study source documents, and other records relative to study conduct. 
17.2. 
Retention of Records 
The investigator will maintain adequate records for the study, including the identity of all 
participating subjects (sufficient information to link records, eCRFs, and medical/hospital 
records), all original signed ICFs, all original signed assents (if applicable), all eCRFs, drug 
dispensing and disposition records, safety reports, information regarding participants who 
discontinued, and other pertinent data. 
All essential documents should be retained by the investigator according to local regulations 
or as specified in the Clinical Study Agreement, whichever is longer. It is the responsibility 
of BioCryst to inform the investigator/institution as to when these documents no longer need 
to be retained. It is the investigator’s responsibility to notify BioCryst if they relocate or retire 
so that document storage can be addressed. The investigator must obtain BioCryst’s written 
permission before disposing of any records and must notify BioCryst before transferring any 
records to another facility. 
All correspondence related to records retention, destruction, or transfer of study documents, 
should be sent directly to BioCryst study personnel, copying the email 
archives@biocryst.com. 
17.3. 
Confidentiality of Information and Data  
BioCryst affirms the subject’s right to protection against invasion of privacy and secure 
maintenance of the confidential nature of his/her personal data. Only a subject identification 
number and subject identifiers permitted by local regulation will identify subject data 
retrieved by BioCryst. However, in compliance with federal regulations, BioCryst requires 
the investigator to permit BioCryst’s representatives and, when necessary, representatives of 
the FDA or other regulatory authorities to review and/or copy any medical records relevant to 
the study, maintaining pseudo-anonymity. 
All parties will abide by all applicable laws and regulations regarding subject privacy and 
confidentiality, including the Health Insurance Portability and Accountability Act (HIPAA), 
where this rule is applicable, and the requirements of the General Data Protection Regulation 
(GDPR) in the EU, where applicable. A valid authorization and consent must meet the 
specifications of the applicable laws and regulations relating to such personal data and health 
information. It is the responsibility of the investigator and institution to obtain such 

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waiver/authorization in writing from the appropriate individual. HIPAA authorizations are 
required for US sites only. 
18. 
PUBLICATION POLICY 
All data generated from this study are the property of BioCryst and shall be held in strict 
confidence along with all information furnished by BioCryst. Except as provided through 
written agreement between BioCryst, independent analysis and/or publication of these data 
by the investigator or any member of his/her staff is not permitted without prior written 
consent of BioCryst. Such consent will not be withheld unreasonably. BioCryst is in 
agreement with the principle of full disclosure of clinical study results. 

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Signature Page for VV-CLIN-004627 v1.0
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