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SRK-015 · 1 trial · 9 indications
The Revised Hammersmith Scale (RHS) is a 36 item clinical assessment of physical abilities in patients with Type 2 SMA and ambulatory or nonambulatory patients with Type 3 SMA. For the ambulatory Type 3 patients 5-21 years of age in Cohort 1 (N=23), the primary endpoint was the change from baseline in RHS total score at month 12. The RHS has a minimum achievable score of 0 and a maximum achievable score of 69. The RHS includes 33 items that are graded on a scale of 0, 1, 2, where 0 denotes the lowest level and 2 denotes the highest level of ability/function. The remaining 3 items are scored 0 or 1, where 0 denotes an inability and 1 denotes an ability to achieve. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
Cohort 2: For the nonambulatory Type 2 and Type 3 patients 5-21 years of age in Cohort 2 (N=15), the primary efficacy endpoint was the change from baseline in HFMSE total score at month 12. Cohort 3: For the nonambulatory Type 2 patients ≥2 years of age in Cohort 3 (N=20), the primary efficacy endpoint was the change from baseline in HFMSE total score at month 12. The Hammersmith Functional Motor Scale Expanded (HFMSE) assesses the physical abilities of patients with Type 2 and Type 3 SMA comprises 33 items graded on a scale of 0, 1, or 2, where 0 denotes unable, 1 denotes performed with modification or adaptation, and 2 denotes performed without modification or adaptation.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Ambulatory Type 3 SMA |
| Cohort 2 | EXPERIMENTAL | Type 2 SMA / Non-Ambulatory Type 3 SMA |
| Cohort 3 | EXPERIMENTAL | Type 2 SMA |
| Name | Type | Description |
|---|---|---|
| SRK-015 | BIOLOGICAL | SRK-015 is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity. SRK-015 will be administered every 4 weeks by intravenous infusion. |
Inclusion Criteria: * Age 5 through 21 years old at the time of screening for Cohorts 1 and 2; Age ≥2 years old at the time of screening for Cohort 3. * Documented diagnosis of 5q SMA. * Diagnosed as later-onset (e.g., Type 2 or Type 3) SMA prior to receiving any treatment with therapy approved for...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 13 | PHASE3 | Nusinersen |
| Novartis AG Sponsored ADR | NVS | 5 | PHASE3 | onasemnogene abeparvovec |
| Biohaven Ltd. | BHVN | 1 | PHASE3 | taldefgrobep alfa |
| Scholar Rock Holding Corp. | SRRK | 2 | PHASE3 | Apitegromab |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
SRK-015 is an investigational monoclonal antibody being developed for the treatment of Spinal Muscular Atrophy (SMA), including Type 2 and Type 3 SMA. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
SRK-015 is being developed by Scholar Rock Holding Corporation, a biopharmaceutical company traded on the NASDAQ under the ticker symbol SRRK. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for Spinal Muscular Atrophy.
SRK-015 is in Phase 2 clinical development. A Phase 2 clinical trial has been completed, and the drug remains investigational. It has not been approved by the FDA or any other regulatory agency, and its safety and efficacy have not been fully established.
SRK-015 has been studied in a completed Phase 2 clinical trial with the identifier NCT03921528. This trial enrolled 58 participants with Type 2 or Type 3 Spinal Muscular Atrophy and was conducted in the United States, Italy, Netherlands, and Spain.
SRK-015 is the investigational name for the drug developed by Scholar Rock. No alternative names have been reported for this compound. It is a monoclonal antibody being studied specifically for the treatment of Spinal Muscular Atrophy.