Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Sevasemten
Sevasemten Dose 1 · 2 trials · 1 indication
All participants
All participants
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 2 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 3 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 4 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 5 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 2NS | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Name | Type | Description |
|---|---|---|
| Sevasemten Dose 1 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten Dose 2 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten Dose 3 | DRUG | Sevasemten is administered orally once per day |
| Placebo | DRUG | Placebo is administered orally once per day |
| Sevasemten Dose 4 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten Dose 5 | DRUG | Sevasemten is administered orally once per day |
Key Inclusion Criteria: * Aged 6 to 17 with a documented mutation on the DMD gene and phenotype consistent with DMD. * Prior receipt of an AAV-based gene therapy (≥ 2 years after documented receipt of gene therapy administration or ≥ 3 years after randomization in a randomized study). * Able to com...
Sevasemten is an investigational small molecule being developed for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. It is also studied in healthy subjects for drug interaction and food effect evaluations. Sevasemten is in Phase 2 clinical development for these rare neuromuscular diseases.
Sevasemten is a small molecule designed to modulate the fast skeletal muscle troponin complex. By targeting this complex, it aims to stabilize the muscle contractile apparatus and reduce muscle damage associated with muscular dystrophies. This mechanism is being evaluated in Duchenne and Becker Muscular Dystrophy.
Sevasemten is being developed by Edgewise Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker EWTX. The company is conducting Phase 2 clinical trials for Sevasemten in Duchenne and Becker Muscular Dystrophy.
Sevasemten is in Phase 2 clinical development. It is not FDA approved and remains investigational. The drug has received FDA designations including Orphan Drug, Rare Pediatric Disease, and Fast Track for its muscular dystrophy indications.
Sevasemten is being studied in three active Phase 2 trials: NCT05291091 in Becker Muscular Dystrophy, NCT05540860 in children with Duchenne Muscular Dystrophy, and NCT06100887 in children with Duchenne previously treated with gene therapy. A Phase 1 trial in healthy subjects has been completed.
Sevasemten is also known as Sevasemten Dose 1 and Sevasemten 10 mg. The clinical trials listed for Sevasemten reference the compound EDG-5506, indicating these names refer to the same investigational drug being developed by Edgewise Therapeutics.