VivoSeq
Neurology

Huntington's Disease

2

Pipeline Programs↗

9

Active Trials↗

16

Companies Involved↗

Huntington's Disease is an early-stage landscape with 2 programs across 16 companies. The most active players are Roche, Eli Lilly, Neurocrine Biosciences. 1 therapy is approved.

Intelligence

Pipeline Metrics

Competitive Density

30/100

Pipeline Velocity

11/100

Mech. Concentration

100/100

Completion Rate

79/100

Attrition Rate

17/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Roche logo

Roche

1 program

Phase 1Eli Lilly logo

Eli Lilly

1 program

Phase 1Neurocrine Biosciences logo

Neurocrine Biosciences

1 program

ApprovedSupernus Pharmaceuticals, Inc. logo

Supernus Pharmaceuticals, Inc.

0 programs

PreclinicalPfizer logo

Pfizer

0 programs

PreclinicalNovartis logo

Novartis

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical
No programs
Phase 11

RG6496

Roche

Phase 2
No programs
Phase 3
No programs
Approved1

Valbenazine

Neurocrine Biosciences

Analysis

Trial Status

72

clinical trials

Completed44 · 61%
Terminated12 · 17%
Unknown status7 · 10%
Recruiting5 · 7%
Active, not recruiting3 · 4%
Not yet recruiting1 · 1%

Clinical Trials

Trial Activity

72 trials

Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease
Recruiting·SSkyhawk Therapeutics, Inc.·Phase 2/3·NCT06873334·120 enrolled
A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
Recruiting·Alnylam Pharmaceuticals logoAlnylam Pharmaceuticals·Phase 1·NCT06585449·66 enrolled
Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease
Recruiting·FFundación Pública Andaluza para la gestión de la Investigación en Sevilla·Phase 2·NCT04478734·24 enrolled
A Phase I, 2-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-ascending Doses of Intrathecally Administered RG6496 in a Randomized, Placebo-controlled, Investigator/Participant-blind Study With an Open-label Extension in Huntington's Disease Gene Expansion Carriers
Recruiting·Roche logoRoche·Phase 1·NCT07246941·40 enrolled
PREVAIL: Valbenazine in Chorea of Huntington's
Recruiting·Neurocrine Biosciences logoNeurocrine Biosciences·Phase 3·NCT05200010·300 enrolled
GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease
Active, not recruiting·Roche logoRoche·Phase 2·NCT05686551·301 enrolled
The Safety and Tolerability Study With ER2001 Intravenous Injection Repetitive Treatment in Adults With Early Manifest Huntington's Disease.
Active, not recruiting·EExoRNA Bioscience·Phase 1·NCT07344519·9 enrolled
A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.
Active, not recruiting·EExoRNA Bioscience·Phase 1/2·NCT07339514·27 enrolled
Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease
Not yet recruiting·—·Phase 1/2·NCT07451613·21 enrolled
A Study to Evaluate Sigma-1 and Dopamine-2 Receptor Occupancy by Pridopidine in the Human Brain of Healthy Volunteers and in Patients With Huntington's Disease
Completed·PPrilenia·Phase 1·NCT03019289·23 enrolled
An Open Label Study of ANX005 in Subjects With, or at Risk for, Manifest Huntington's Disease
Completed·AAnnexon, Inc.·Phase 2·NCT04514367·28 enrolled
Safety and Tolerability Study of Phenylbutyrate in Huntington's Disease (PHEND-HD)
Completed·UUniversity of Rochester·Phase 2·NCT00212316·60 enrolled
Dose-response Evaluation of the Cellavita HD Product in Patients With Huntington's Disease
Completed·—·Phase 2·NCT03252535·49 enrolled
Creatine Safety & Tolerability in Huntington's Disease
Completed·MMassachusetts General Hospital·Phase 2·NCT01412151·10 enrolled
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B
Completed·Supernus Pharmaceuticals, Inc. logoSupernus Pharmaceuticals, Inc.·Phase 1·NCT03787758·6 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)