Huntington Disease

Neurology
18
Pipeline Programs
11
Companies
20
Clinical Trials
6 recruiting
6
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
2
4
3
2
6
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
150%
Small Molecule
150%
+ 14 programs with unclassified modality

On Market (6)

Approved therapies currently available

Teva
AUSTEDOApproved
deutetrabenazine
Teva
oral2017
Teva
AUSTEDO XRApproved
deutetrabenazine
Teva
oral2023
U
DEUTETRABENAZINEApproved
deutetrabenazine
Unknown Company
oral2024
U
EXSERVANApproved
riluzole
Unknown Company
oral2019
U
RILUTEKApproved
riluzole
Unknown Company
oral1995
U
RILUZOLEApproved
riluzole
Unknown Company
oral2024

Competitive Landscape

11 companies ranked by most advanced pipeline stage

Teva
TevaIsrael - Petach Tikva
3 programs
1
1
1
DeutetrabenazinePhase 2/32 trials
ACR16Phase 22 trials
Protocol Intervention GroupN/A1 trial
Active Trials
NCT03417583Completed100Est. Oct 2023
NCT00724048Completed227Est. Jul 2010
NCT00665223Completed437Est. Jun 2010
+2 more trials
Sanofi
SanofiPARIS, France
1 program
1
RiluzolePhase 3Small Molecule2 trials
Active Trials
NCT00542412Completed414Est. Dec 2004
NCT00277602Completed537Est. Jul 2004
Novartis
NovartisBASEL, Switzerland
1 program
1
VotoplamPhase 32 trials
Active Trials
NCT07326709Recruiting770Est. Apr 2030
NCT06254482Active Not Recruiting144Est. Jul 2029
Skyhawk Therapeutics
2 programs
2
SKY-0515Phase 2/31 trial
SKY-0515Phase 2/31 trial
Active Trials
NCT07378644RecruitingEst. Aug 2029
NCT06873334RecruitingEst. Dec 2027
Annexon Biosciences
Annexon BiosciencesCA - Brisbane
1 program
1
ANX005Phase 21 trial
Active Trials
NCT04514367CompletedEst. Jan 2022
PTC Therapeutics
1 program
1
PTC518Phase 21 trial
Active Trials
NCT05358717Completed159Est. Jul 2025
Ultragenyx Pharmaceutical
1 program
1
Triheptanoin oilPhase 21 trial
Active Trials
NCT02453061Completed100Est. Dec 2019
Roche
RocheSTAVANGER NORWAY, Norway
3 programs
1
SPK-10001Phase 1/21 trial
Biospecimen CollectionN/A1 trial
Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion CarriersN/A1 trial
Active Trials
NCT05773196Recruiting5Est. Feb 2025
NCT06667414Recruiting600Est. Sep 2028
NCT06826612Recruiting53Est. Jan 2035
AskBio
AskBioNC - Durham
1 program
1
AB-1001 Gene TherapyPhase 1/2Gene Therapy1 trial
Active Trials
NCT05541627Active Not Recruiting5Est. Apr 2028
Voyager Therapeutics
1 program
1
Intraparenchymal rAAV1 -Phase 11 trial
Active Trials
NCT04885114WithdrawnEst. Dec 2024
T-Therapeutics
T-TherapeuticsUK - Cambridge
1 program
FuRST 2.0N/A1 trial
Active Trials
NCT03709173CompletedEst. Oct 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
SanofiRiluzole
NovartisVotoplam
TevaACR16
SanofiRiluzole
Skyhawk TherapeuticsSKY-0515
Skyhawk TherapeuticsSKY-0515
TevaDeutetrabenazine
NovartisVotoplam
PTC TherapeuticsPTC518
Annexon BiosciencesANX005
Ultragenyx PharmaceuticalTriheptanoin oil
TevaACR16
RocheSPK-10001
AskBioAB-1001 Gene Therapy
TevaDeutetrabenazine

Showing 15 of 20 trials with date data

Clinical Trials (20)

Total enrollment: 3,596 patients across 20 trials

NCT00542412SanofiRiluzole

CARE Canadian ALS Riluzole Evaluation

Start: Jan 2001Est. completion: Dec 2004414 patients
Phase 4Completed
NCT07326709NovartisVotoplam

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

Start: Jun 2026Est. completion: Apr 2030770 patients
Phase 3Recruiting
NCT00665223TevaACR16

A Study of Treatment With Pridopidine (ACR16) in Participants With Huntington's Disease

Start: Apr 2008Est. completion: Jun 2010437 patients
Phase 3Completed
NCT00277602SanofiRiluzole

Riluzole in Huntington's Disease

Start: Nov 1999Est. completion: Jul 2004537 patients
Phase 3Completed
NCT07378644Skyhawk TherapeuticsSKY-0515

Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease

Start: Jan 2026Est. completion: Aug 2029
Phase 2/3Recruiting
NCT06873334Skyhawk TherapeuticsSKY-0515

Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease

Start: May 2025Est. completion: Dec 2027
Phase 2/3Recruiting
NCT04713982TevaDeutetrabenazine

Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease

Start: Nov 2021Est. completion: Oct 202630 patients
Phase 2/3Active Not Recruiting
NCT06254482NovartisVotoplam

An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)

Start: Aug 2023Est. completion: Jul 2029144 patients
Phase 2Active Not Recruiting
NCT05358717PTC TherapeuticsPTC518

A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

Start: Jun 2022Est. completion: Jul 2025159 patients
Phase 2Completed
NCT04514367Annexon BiosciencesANX005

An Open Label Study of ANX005 in Subjects With, or at Risk for, Manifest Huntington's Disease

Start: Aug 2020Est. completion: Jan 2022
Phase 2Completed
NCT02453061Ultragenyx PharmaceuticalTriheptanoin oil

A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease

Start: Jun 2015Est. completion: Dec 2019100 patients
Phase 2Completed
NCT00724048TevaACR16

A Study of Pridopidine (ACR16) for the Treatment of Participants With Huntington's Disease

Start: Oct 2008Est. completion: Jul 2010227 patients
Phase 2Completed
NCT06826612RocheSPK-10001

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

Start: Feb 2025Est. completion: Jan 203553 patients
Phase 1/2Recruiting
NCT05541627AskBioAB-1001 Gene Therapy

A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease

Start: Oct 2022Est. completion: Apr 20285 patients
Phase 1/2Active Not Recruiting
NCT04173260TevaDeutetrabenazine

An Open-label Study to Define the Safety, Tolerability and Clinical Activity of Deutetrabenazine (AUstedo) in Adult Study Subjects With DYsTonia

Start: Apr 2021Est. completion: Aug 202415 patients
Phase 1/2Completed
NCT04885114Voyager TherapeuticsIntraparenchymal rAAV1 -

Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease

Start: Jul 2021Est. completion: Dec 2024
Phase 1Withdrawn
NCT06667414RocheFrequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers

Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers

Start: Sep 2024Est. completion: Sep 2028600 patients
N/ARecruiting
NCT05773196RocheBiospecimen Collection

Prospective Single-Timepoint Huntington's Disease Biospecimen Collection Study

Start: Mar 2023Est. completion: Feb 20255 patients
N/ARecruiting
NCT03709173T-TherapeuticsFuRST 2.0

FuRST 2.0 Cognitive Pre-testing - Round 2

Start: Aug 2019Est. completion: Oct 2019
N/ACompleted
NCT03417583TevaProtocol Intervention Group

Assessing Efficacy of Neuropsychiatric Assessment and Treatment Protocols in Huntington's Disease Patients

Start: Nov 2018Est. completion: Oct 2023100 patients
N/ACompleted

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
6 actively recruiting trials targeting 3,596 patients
11 companies competing in this space

Related Indications

Multiple Sclerosis(274)Schizophrenia(255)Epilepsy(242)Parkinson Disease(171)Alzheimer's Disease(165)Parkinson's Disease(160)Alzheimer Disease(117)Stroke(102)Relapsing-Remitting Multiple Sclerosis(83)Depression(82)