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Dose 1 of BMN 331

Phase 1

Hereditary Angioedema | Gene therapy | Other |BioMarin Pharmaceutical Inc.|Last Updated: May 16, 2024

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 Enrollment44
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary AngioedemaPHASE1 ACTIVE NOT_RECRUITING 44 — — Feb 15, 2022Nov 1, 2028May 16, 202416 United States, Australia +1
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Study Endpoints
Primary Endpoints
Number of participants with treatment-emergent adverse events following a single IV administration of BMN 331
At 5 years
Secondary Endpoints
Time-normalized number of investigator-confirmed HAE attacks
At 5 years
Time-normalized number of investigator-confirmed HAE attacks by severity (mild, moderate, severe)
At 5 years
Time-normalized use of HAE-specific medication
At 5 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BMN 331EXPERIMENTALAAV Gene Therapy Infusion
Interventions
NameTypeDescription
Dose 1 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 2 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 3 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 4 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 5 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 6 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 7 of BMN 331GENETICBMN 331 AAV Gene Therapy
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: 1. Female or male adults ( ≥ 18 years old) 2. Part A only: Confirmed diagnosis of Type I HAE due to C1-INH deficiency confirmed by genotyping of the SERPING1 gene Part B only: Confirmed diagnosis of Type I or II HAE due to C1-INH deficiency confirmed by genotyping of the SERPING...

Countries:United StatesAustraliaSpain
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Competitive Landscape -Hereditary Angioedema 10 trials
CompanyTickerTrialsLead PhaseDrugs
Intellia Therapeutics, Inc.NTLA3PHASE3NTLA-2002, Normal Saline Administration, Biological NTLA-2002
BioCryst Pharmaceuticals, Inc.BCRX2PHASE3Berotralstat, berotralstat
Ionis Pharmaceuticals, Inc.IONS1PHASE3Donidalorsen
KalVista Pharmaceuticals, Inc.KALV1PHASE3KVD900, Drug: KVD900
Pharvaris N.V.PHVS1PHASE2deucrictibant
BioMarin Pharmaceutical Inc.BMRN1PHASE1Dose 1 of BMN 331
Astria Therapeutics, Inc.ATXS1PHASE2STAR-0215
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05121376primaryCompletionDate: changed
LOWMay 24, 2026NCT05121376studyFirstPostDate: changed