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Also known as SRP-1001 for Injection
SRP-1001 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| SRP-1001 | EXPERIMENTAL | SRP-1001 for injection |
| Placebo | PLACEBO_COMPARATOR | Sodium chloride (0.9%) |
| Name | Type | Description |
|---|---|---|
| SRP-1001 for Injection | DRUG | Single or multiple doses of SRP-1001 by intravenous (IV) infusion |
| Placebo | DRUG | Calculated volume to match active treatment by IV infusion |
Key Inclusion Criteria: * Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record * Clinical severity score between 3 and 8 (scale, 0 to 10) * Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fr...
SRP-1001 is an investigational therapy being developed for facioscapulohumeral muscular dystrophy, a form of muscular dystrophy. It is in Phase 1 clinical development and is not approved for any indication. The program is led by Arrowhead Pharmaceuticals, Inc. (ticker: ARWR).
SRP-1001 targets DUX4, the gene whose abnormal expression drives facioscapulohumeral muscular dystrophy. By targeting DUX4, the therapy aims to address the underlying cause of the disease. SRP-1001 is a small molecule in Phase 1 development for this condition.
SRP-1001 is being developed by Arrowhead Pharmaceuticals, Inc., which trades under the ticker ARWR. The company is running a Phase 1 trial of SRP-1001 in facioscapulohumeral muscular dystrophy. No other developers are listed for this asset.
SRP-1001 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA or any other regulatory agency. The Phase 1 trial is currently enrolling by invitation and has an estimated enrollment of 60 participants.
SRP-1001 is being studied in one active Phase 1 trial, NCT06131983, titled Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1. The trial is enrolling by invitation at sites in Australia, Canada, Germany, Italy, the Netherlands, New Zealand, and Spain.
Yes, SRP-1001 for Injection is an alternative name for SRP-1001. Both refer to the same investigational small molecule developed by Arrowhead Pharmaceuticals for facioscapulohumeral muscular dystrophy. Searches for either name point to the same Phase 1 program.