A Phase 1, Double-Blind, Placebo-Controlled Trial of Sevasemten (EDG-5506), a Selective Modulator of Fast Skeletal Muscle Contraction, in Healthy Volunteers and Adults With Becker Muscular Dystrophy.

ABSTRACT:

Introduction/aims

Sevasemten (EDG-5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fiber contraction by inhibiting fast myosin ATPase. This study assessed the safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics (PD) of sevasemten in healthy adult volunteers (HVs) and adults with Becker muscular dystrophy (BMD).

Methods

This randomized, double-blind, placebo-controlled phase 1 study was conducted at a single site. Eligible participants were 18-55 years of age with a body mass index < 30 kg/m²; adults with BMD were required to have a confirmed diagnosis based on documentation of pathogenic variant(s) in the dystrophin gene and a BMD phenotype. Participants were randomized 3:1 to receive sevasemten or

SUBMITTER: Donovan J 

PROVIDER: S-EPMC12338012 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature