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ALN-6400

Phase 2

Von Willebrand Disease (VWD) | Small molecule | Hematology |Alnylam Pharmaceuticals, Inc.|Last Updated: May 14, 2026

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
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07575308HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMBPHASE2 NOT YET_RECRUITING 24May 22, 2026Nov 29, 2028May 14, 20261 United States
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Study Endpoints
Primary Endpoints
Frequency of Adverse Events
Up to Week 72
Secondary Endpoints
Change from Baseline in Plasminogen (PLG) Plasma Activity Levels
Screening and up to Week 72 postdose
Change from Baseline in PLG Plasma Protein Levels
Screening and up to Week 72 postdose
Change from Baseline in Menstrual Blood Loss Via Pictorial Blood Assessment Chart (PBAC)
Screening and up to Week 72 postdose
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-6400 (Treatment Group A)EXPERIMENTALParticipants will be administered multiple doses of ALN-6400.
ALN-6400 (Treatment Group B)EXPERIMENTALParticipants will be administered multiple doses of ALN-6400.
Interventions
NameTypeDescription
ALN-6400DRUGALN-6400 will be administered subcutaneously (SC).
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Eligibility Criteria
Age Range16 Years — 45 Years
SexFEMALE
Healthy VolunteersNo
Study Sites1

Inclusion Criteria - * Is an adolescent or adult patient with a diagnosis of VWD (including Type 1, Type 2, Type 3, and platelet-type VWD) and HMB for 2 cycles during screening Exclusion Criteria - * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2 times the u...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07575308primaryCompletionDate: changed
LOWMay 24, 2026NCT07575308studyFirstPostDate: changed
LOWMay 21, 2026NCT07575308NEW_TRIAL: changed
LOWMay 21, 2026NCT07575308NEW_TRIAL: changed