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BCX10013

Phase 1

Paroxysmal Nocturnal Hemoglobinuria | Small molecule | Other |BioCryst Pharmaceuticals, Inc.|Last Updated: Jan 8, 2025

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment8
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06100900Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)PHASE1 COMPLETED 8 — — Oct 24, 2023Dec 11, 2024Jan 8, 20254 Malaysia, South Africa
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Study Endpoints
Primary Endpoints
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Graded Laboratory Abnormalities, and Changes From Baseline (CFB) in Laboratory Analytes, Vital signs, Electrocardiograms (ECGs), and Physical Examination Findings.
up to 52 weeks
Secondary Endpoints
CFB in Lactate Dehydrogenase
Baseline, Week 52
CFB in the Ratio of Total PNH Red Blood Cell Clone Size to PNH White Blood Cell Clone Size
Baseline, Week 52
CFB in Hemoglobin
Baseline, Week 52
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BCX10013EXPERIMENTALParticipants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur.
Interventions
NameTypeDescription
BCX10013DRUGMultiple dose levels may be tested in this study.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Key Inclusion Criteria: 1. Male or non-pregnant, non-lactating female adults ≥ 18 years old. 2. Documented diagnosis of PNH confirmed by flow cytometry. 3. Body mass index (BMI) ≤ 40 kg/m\^2. 4. Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with r...

Countries:MalaysiaSouth Africa
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