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SRP-1005 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1: SRP-1005 Dose 1 | EXPERIMENTAL | Participants will receive SRP-1005 or placebo. |
| Cohort 2: SRP-1005 Dose 2 | EXPERIMENTAL | Participants will receive SRP-1005 or placebo. |
| Cohort 3: SRP-1005 Dose 3 | EXPERIMENTAL | Participants will receive SRP-1005 or placebo. |
| Cohort 4: SRP-1005 Dose 4 | EXPERIMENTAL | Participants will receive SRP-1005 or placebo. |
| Name | Type | Description |
|---|---|---|
| SRP-1005 | DRUG | Subcutaneous injection |
| Placebo | DRUG | Subcutaneous injection |
Key Inclusion Criteria: * Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥40. * Participant has HD Integrated Staging System (HD-ISS) Stage 2 or Mild Stage 3. At screening, the participant must be classified with the Enroll-HD HD-ISS Modified Calculator a...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| Hoffmann-La Roche | RHHBY | 3 | PHASE2 | Tominersen, RG6496, SPK-10001 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | AMT-130 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
SRP-1005 is an investigational small molecule being developed for the treatment of Huntington's Disease. It is currently in Phase 1 clinical development, meaning it has not yet been approved by regulatory authorities. The drug is being studied in a first-in-human trial to evaluate its effects in participants with Huntington's Disease.
SRP-1005 targets the HTT gene, which is associated with Huntington's Disease. By targeting HTT, the drug aims to address the underlying genetic cause of the condition. This mechanism is being evaluated in early-stage clinical trials to determine its safety and potential efficacy in patients.
SRP-1005 is being developed by Sarepta Therapeutics, Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol SRPT. Sarepta is conducting the clinical development of this investigational drug for Huntington's Disease.
SRP-1005 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is recruiting participants to assess the drug's safety, tolerability, and effects in people with Huntington's Disease.
SRP-1005 is being studied in a single Phase 1 clinical trial registered as NCT07536061. This first-in-human study is recruiting 32 participants with Huntington's Disease across Australia, New Zealand, Switzerland, and the United Kingdom. The trial is randomized, double-blind, and controlled, with participants aged 21 years and older.
SRP-1005 is the sole name provided for this investigational drug; no alternative names have been identified. It is a distinct small molecule being developed by Sarepta Therapeutics for Huntington's Disease and is not known to be identical to any other marketed or investigational product.