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ALN-HTT02

Phase 1

Huntington's Disease | Small molecule | Rare Disease |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-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment66
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06585449A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's DiseasePHASE1 RECRUITING 66 — — Oct 14, 2024Jul 5, 2028May 14, 202619 Canada, Germany +1
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Study Endpoints
Primary Endpoints
Frequency of Adverse Events (AEs) in the Double-blind Part of the Study
Up to 12 months
Frequency of AEs in the Open-label Part of the Study
Up to 12 months
Frequency of AEs in the OLE Part of the Study
Up to 36 months
Secondary Endpoints
Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF)
Baseline up to Month 12 in the Double-blind Part of the study; Baseline up to Month 12 in the Open-label Part of the study ; Baseline up to Month 36 in the OLE Part of the study
Concentrations of ALN-HTT02 in Plasma
Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study; Up to Month 36 in the OLE Part of the study
Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF)
Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study ; Up to Month 36 in the OLE Part of the study
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-HTT02EXPERIMENTALParticipants will be administered a single dose of ALN-HTT02 during the Double-blind Part of the study. Participants who received ALN-HTT02 in the Double-blind part of the study will not receive an additional dose during the Open-label Part of the study. Participants have the option to receive repeat doses of ALN-HTT02 in the Open-label Extension (OLE) Part of the study.
Placebo + ALN-HTT02PLACEBO_COMPARATORParticipants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-label Part of the study. Participants have the option to receive repeat doses of ALN-HTT02 in the OLE Part of the study.
Interventions
NameTypeDescription
ALN-HTT02DRUGALN-HTT02 will be administered intrathecally
PlaceboDRUGPlacebo will be administered intrathecally
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Eligibility Criteria
Age Range25 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites19

Inclusion Criteria * Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS) Exclusion Criteria * Has significant structural or degenerative neurologic disease other than Huntington's Disease (HD) at screening * Has primary or second...

Countries:CanadaGermanyUnited Kingdom
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Competitive Landscape -Huntington's Disease 9 trials
CompanyTickerTrialsLead PhaseDrugs
Novartis AG Sponsored ADRNVS2PHASE3Votoplam
Neurocrine Biosciences, Inc.NBIX2PHASE3Valbenazine
Alnylam Pharmaceuticals, IncALNY1PHASE1ALN-HTT02
uniQure N.V.QURE2PHASE1intra-striatal rAAV5-miHTT
Sarepta Therapeutics, Inc.SRPT1PHASE1SRP-1005
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06585449primaryCompletionDate: changed
LOWMay 24, 2026NCT06585449studyFirstPostDate: changed