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ALN-6400

Phase 2

Von Willebrand Disease (VWD) | Small molecule | Hematology |Alnylam Pharmaceuticals, Inc.|Last Updated: Aug 12, 2026

Target and mechanism

Molecular targetPlasminogen
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment24

FDA Designations

No designations recorded

Clinical trial landscape

ALN-6400 · 2 trials · 3 indications

Phase 2 1Phase 1 1
NCT07575308HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMBVon Willebrand Disease (VWD)
RECRUITING24 Analytics
PHASE2RECRUITING
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
Von Willebrand Disease (VWD)Unlock trial analytics

Study Endpoints

Primary Endpoints

Frequency of Adverse Events
Up to Week 72
Part A: Frequency of Adverse Events
Up to Week 36
Part B: Frequency of Adverse Events
Up to Week 96

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.
Part A: ALN-6400EXPERIMENTALParticipants will be administered a single dose of ALN-6400.
Part A: PlaceboPLACEBO_COMPARATORParticipants will be administered a single dose of placebo.
Part B: ALN-6400EXPERIMENTALParticipants will be administered multiple doses of ALN-6400.
Part B: PlaceboPLACEBO_COMPARATORParticipants will be administered multiple doses of placebo.

Interventions

NameTypeDescription
ALN-6400DRUGALN-6400 will be administered subcutaneously (SC).
PlaceboDRUGPlacebo will be administered subcutaneously (SC)
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Eligibility Criteria

Age Range16 Years to 45 Years
SexFEMALE
Healthy VolunteersNo
Study Sites4

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 StatesAustraliaCanadaFranceGermanySpain
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Competitive Landscape -Von Willebrand Disease 8 trials (matched to "Von Willebrand Disease (VWD)")

Recent Changes (Last 90 Days)

LOWAug 12, 2026NCT07575308Status: NOT_YET_RECRUITING → RECRUITING
LOWAug 12, 2026NCT06659640lastUpdatePostDate: changed
LOWAug 12, 2026NCT07575308Status: NOT_YET_RECRUITING → RECRUITING
LOWAug 12, 2026NCT06659640lastUpdatePostDate: changed
LOWJul 14, 2026NCT07575308startDate: changed
HIGHJul 14, 2026NCT06659640Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWJul 14, 2026NCT07575308startDate: changed
HIGHJul 14, 2026NCT06659640Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWJun 16, 2026NCT07575308startDate: changed
LOWJun 16, 2026NCT07575308startDate: changed
LOWJun 16, 2026NCT07575308startDate: changed
LOWJun 16, 2026NCT07575308startDate: changed
LOWJun 12, 2026NCT06659640lastUpdatePostDate: changed
LOWJun 12, 2026NCT06659640lastUpdatePostDate: changed

Frequently asked questions about ALN-6400

What is ALN-6400 used for?

ALN-6400 is an investigational drug being studied for the treatment of Von Willebrand Disease (VWD) and Hereditary Hemorrhagic Telangiectasia (HHT). It is also being evaluated in patients with heavy menstrual bleeding associated with VWD. The drug is currently in clinical development and has not been approved by regulatory authorities.

What does ALN-6400 target?

ALN-6400 targets plasminogen, a protein involved in the breakdown of blood clots. By targeting plasminogen, the drug may help reduce bleeding episodes in conditions like Von Willebrand Disease and Hereditary Hemorrhagic Telangiectasia. The drug is being developed as a small molecule therapy.

Who is developing ALN-6400?

ALN-6400 is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company. Alnylam is conducting clinical trials to evaluate the safety and efficacy of ALN-6400 in patients with bleeding disorders. The company is listed on the stock exchange under the ticker symbol ALNY.

What phase is ALN-6400 in?

ALN-6400 is in Phase 1 and Phase 2 clinical development. A Phase 1 study is evaluating the drug in healthy volunteers and patients with Hereditary Hemorrhagic Telangiectasia. A Phase 2 study is recruiting female patients with Von Willebrand Disease and heavy menstrual bleeding. The drug is investigational and not yet approved.

What clinical trials is ALN-6400 in?

ALN-6400 is being studied in two clinical trials. NCT06659640 is a Phase 1 study in healthy volunteers and patients with Hereditary Hemorrhagic Telangiectasia, conducted in the United States, Australia, Canada, France, Germany, and Spain. NCT07575308 is a Phase 2 study in female patients with Von Willebrand Disease and heavy menstrual bleeding, conducted in the United States.

Is ALN-6400 the same as any other drug?

ALN-6400 is a distinct investigational drug being developed by Alnylam Pharmaceuticals. It is not known to be the same as any other marketed medication. The drug is being studied under its own name in clinical trials for bleeding disorders.