Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Sevasemten Dose 1, EDG-5506
Sevasemten · 8 trials · 10 indications
All participants
All participants
All participants
All participants
Adult participants
Adult participants
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The numerator of the percentage is the number of participants experiencing at least one AE after first dose of study drug up to 25 months.
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The endpoint is the cumulative total number of AEs occurring after first dose of study drug up to 25 months among participants who received at least one dose of study drug.
An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The severity of an AE is graded, according to the study protocol definitions of AE severity/intensity, as "mild", "moderate" or "severe". Participants who reported multiple AEs are counted only once at the highest severity reported.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 2 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Cohort 3 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Treatment | EXPERIMENTAL | Drug: Sevasemten |
| Adult Cohort 1 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Adult Cohort 2 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Adult Cohort 6 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Adolescent Cohort 4 | EXPERIMENTAL | Drug: Sevasemten Drug: Placebo |
| Adolescent Cohort 5 | 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 |
| Part A - AME | EXPERIMENTAL | Evaluation of absorption, metabolism, excretion and pharmacokinetics of a single oral dose of radiolabeled EDG-5506 in healthy male volunteers |
| Part B - aBA | EXPERIMENTAL | Evaluation of bioavailability of a single oral dose of EDG-5506 followed by a single intravenous dose of radiolabeled EDG-5506 in healthy male volunteers |
| Healthy Volunteer: Single Ascending Dose | EXPERIMENTAL | Single oral ascending dose in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo |
| Healthy Volunteer: Multiple Ascending Dose | EXPERIMENTAL | Multiple oral ascending doses in healthy volunteers Interventions: Drug: EDG-5506 Drug: Placebo |
| Becker Muscular Dystrophy: Multiple Ascending Dose | EXPERIMENTAL | Multiple oral ascending doses in adults with Becker muscular dystrophy Interventions: Drug: EDG-5506 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 | DRUG | Sevasemten is administered orally once per day |
| Sevasemten 10 mg | DRUG | Sevasemten is administered orally once per day |
| Sevasemten 5 mg | DRUG | Sevasemten is administered orally once per day |
| Sevasemten 12.5 mg | DRUG | Sevasemten 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 |
| EDG-5506 Tablet | DRUG | Single Oral Dose - Tablet |
| Radiolabeled EDG-5506 Suspension | DRUG | Single Oral Dose |
| Radiolabeled EDG-5506 Intravenous | DRUG | Single Intravenous Dose |
| EDG-5506 | DRUG | single oral dose |
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 Becker muscular dystrophy and Duchenne muscular dystrophy, two rare genetic muscle-wasting conditions. It is in Phase 2 clinical development and has not been approved by the FDA. Edgewise Therapeutics holds Orphan Drug and Fast Track designations for the program.
Sevasemten targets MYH4, the myosin heavy chain 4 protein. By acting on this target, the small molecule is designed to address the muscle function deficits associated with Becker and Duchenne muscular dystrophy. MYH4 is the molecular target identified for the Sevasemten development program.
Sevasemten is being developed by Edgewise Therapeutics, Inc., which trades on the Nasdaq under the ticker EWTX. The company is running the Phase 2 clinical program for the drug in Becker muscular dystrophy and Duchenne muscular dystrophy, including the GRAND CANYON and LYNX studies.
Sevasemten is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA for any indication. The program includes completed and ongoing Phase 2 trials in Becker muscular dystrophy and Duchenne muscular dystrophy, with Orphan Drug and Fast Track designations from the FDA.
Sevasemten has been studied in Phase 2 trials including NCT05291091 (GRAND CANYON) in Becker muscular dystrophy, NCT05540860 (LYNX) in Duchenne muscular dystrophy, NCT06100887 in Duchenne muscular dystrophy patients previously treated with gene therapy, and the open-label extension NCT06066580 in Becker muscular dystrophy.