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BCX9930

Phase 1

Paroxysmal Nocturnal Hemoglobinuria | Small molecule | Other |BioCryst Pharmaceuticals, Inc.|Last Updated: Feb 17, 2022

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
PLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment168
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04330534First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNHPHASE1 COMPLETED 168Mar 3, 2020Jan 25, 2021Feb 17, 20224 Austria, South Africa +1
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Study Endpoints
Primary Endpoints
Incidence of graded treatment-emergent adverse events
Part 1: Day 16
Incidence of graded laboratory chemistry abnormalities
Part 1: Day 16
Incidence of graded urinalysis abnormalities
Part 1: Day 16
Incidence of graded coagulation abnormalities
Part 1: Day 16
Incidence of graded hematology abnormalities
Part 1: Day 16
Change from baseline in blood pressure
Part 1: Day 16
Change from baseline in temperature
Part 1: Day 16
Change from baseline in heart rate
Part 1: Day 16
Change from baseline in respiratory rate
Part 3: Day 44 or Week 50 (South Africa only)
Change in Electrocardiogram (PR interval)
Part 1: Day 16
Change in Electrocardiogram (QRS interval)
Part 1: Day 16
Change in Electrocardiogram (RR interval)
Part 1: Day 16
Change in Electrocardiogram (QT interval)
Part 1: Day 16
Secondary Endpoints
Plasma BCX9930 Cmax
plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 Tmax
plasma PK parameters are based on blood sampling through Day 4 for Part 1; through Day 6, 14 or 18 for Part 2 (Day depends on dosing duration); and through Day 28 for Part 3
Plasma BCX9930 AUCinf
plasma PK parameters are based on blood sampling through Day 4 for Part 1
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BCX9930EXPERIMENTALParts 1, 2 and 3
PlaceboPLACEBO_COMPARATORParts 1 and 2 only
Interventions
NameTypeDescription
BCX9930DRUGBCX9930 capsules for oral administration
PlaceboDRUGplacebo to match BCX9930 capsules for oral administration
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersYes
Study Sites4

Key Inclusion Criteria (Parts 1, 2, and 3): * Able to provide written informed consent * Acceptable birth control measures for male subjects and women of childbearing potential * Is expected to adequately comply with required study procedures and restrictions Key Inclusion Criteria (Parts 1 and 2)...

Countries:AustriaSouth AfricaUnited Kingdom
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