Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as EDG-5506 Tablet, Sevasemten
EDG-5506 · 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...
EDG-5506 is an investigational small molecule being developed by Edgewise Therapeutics, Inc. for Becker muscular dystrophy and other muscular dystrophies. It is in Phase 1 clinical development and has been studied in healthy volunteers and adults with Becker muscular dystrophy. It is also referred to as EDG-5506 Tablet.
EDG-5506 is being developed for Becker muscular dystrophy, a rare genetic muscle disorder, and for muscular dystrophies more broadly. It is an investigational therapy in Phase 1 development and has not been approved for any indication. Clinical work to date includes healthy volunteers and adults with Becker muscular dystrophy.
EDG-5506 is developed by Edgewise Therapeutics, Inc., which trades under the ticker EWTX. The company is advancing the small molecule for Becker muscular dystrophy and related muscular dystrophies as part of its rare disease pipeline.
EDG-5506 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA for any indication. Two completed Phase 1 trials are registered, and no active trials are currently listed for the program.
EDG-5506 has been evaluated in completed Phase 1 trials, including NCT04585464, which assessed safety, tolerability, and pharmacokinetics in healthy volunteers and adults with Becker muscular dystrophy, and NCT05730842, which studied absorption, metabolism, excretion, and absolute bioavailability in healthy volunteers. A third study, NCT05492734, assessed non-invasive dried blood sampling.
Yes, EDG-5506 Tablet is an alternative name for EDG-5506, the investigational small molecule from Edgewise Therapeutics, Inc. Both names refer to the same Phase 1 asset being developed for Becker muscular dystrophy and other muscular dystrophies.