Huntington's disease drug pipeline

Upcoming readouts & recent results

• 2024-11-20ReportedDalzanemdorTopline datamissedNCT05107128

  Phase 2 DIMENSION topline: dalzanemdor missed the primary endpoint (change from baseline in SDMT at Day 84 vs placebo; n=189) with no statistically significant or clinically meaningful secondary-endpoint differences; Sage ends all development of dalzanemdor and closes the Phase 3 open-label PURVIEW study. ↗

• 2024-06-11ReportedDalzanemdorTopline datamixedNCT05358821

  Phase 2 SURVEYOR topline: primary endpoint met — a statistically significant baseline difference on the HD-CAB composite between HD participants and healthy volunteers — but only a small numerical dalzanemdor-placebo difference; well tolerated, no new safety signals. ↗

See the full catalyst calendar →

Compounds in development (2)

Trials recruiting now (12)

12 drug-interventional huntington's disease trials recruiting now, of 141 registered interventional trials. Browse all on ClinicalTrials.gov →

• Phase 3Pridopidine Phase 3 Study in Huntington's Disease

  Pridopidine, Placebo

  Prilenian=400Started Jun 2026NCT07609108
• Phase 3A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

  Votoplam (blinded), Placebo

  Novartis Pharma AGn=770Canada, China +5 · 27 sitesStarted Mar 2026NCT07326709
• Phase 3Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in Canada

  Valbenazine

  Neurocrine Biosciences, Inc.n=7Canada · 3 sitesStarted Apr 2024NCT06312189
• Phase 2/3An Extension of SKY-0515 in Participants With Huntington's Disease

  SKY-0515

  Skyhawk Therapeutics, Inc.n=500Australia · 4 sitesStarted Apr 2026NCT07537075
• Phase 2/3Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease

  SKY-0515, SKY-0515 Placebo

  Skyhawk Therapeutics, Inc.n=400Argentina, Brazil +3 · 9 sitesStarted Jan 2026NCT07378644
• Phase 2A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers

  NAC, Placebo

  Western Sydney Local Health Districtn=160Australia · 5 sitesStarted Jun 2024NCT05509153
• Phase 2Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease

  Moderate doses of Thiamine y Biotin, High doses of Thiamine y Biotin

  Fundación Pública Andaluza para la gestión de la Investigación en Sevillan=24Spain · 3 sitesStarted Apr 2023NCT04478734
• Phase 1/2A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD

  VO659

  Vico Therapeutics B. V.n=68Denmark, France +4 · 14 sitesStarted Feb 2023NCT05822908
• Phase 1A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease

  SRP-1005, Placebo

  Sarepta Therapeutics, Inc.n=32New Zealand · 2 sitesStarted May 2026NCT07536061
• Phase 1Mass Balance Study of [14C] LPM3770164 in Healthy Participants

  [14C] LPM3770164

  Luye Pharma Group Ltd.n=8China · 1 siteStarted Apr 2026NCT07516899
• Phase 1A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

  RG6496, Placebo

  F. Hoffmann-La Roche Ltdn=40Argentina, Australia +1 · 4 sitesStarted Nov 2025NCT07246941
• Phase 1A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease

  ALN-HTT02, Placebo

  Alnylam Pharmaceuticalsn=66Canada, Germany +1 · 19 sitesStarted Oct 2024NCT06585449