sevasemten (EDG-5506) / Servier - LARVOL DELTA

EDG-5506-201: Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) (clinicaltrials.gov) - P2 | N=244 | Completed | Sponsor: Edgewise Therapeutics, Inc. | Active, not recruiting ➔ Completed

Biomarker • Trial completion • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy • MB

Human Disposition, Metabolism, and Excretion of Sevasemten (EDG-5506), a Selective Modulator of Fast Myosin in Healthy Volunteers. (PubMed, J Clin Pharmacol) - P1 | "Sevasemten was well tolerated, with no serious adverse events or adverse events leading to study discontinuation. Sevasemten has favorable PK properties for once daily administration and continues to be investigated for the treatment of Becker and Duchenne muscular dystrophies."

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Effects of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults with Becker muscular dystrophy: results of a phase 1b, open-label study. (PubMed, EBioMedicine) - P1 | "Sevasemten treatment for up to 24 months in adults with BMD was well tolerated and associated with durable reductions in muscle injury biomarkers, consistent with preclinical studies and near maximal with the 10 mg dose, as well as functional stabilisation. Further clinical development is ongoing."

Biomarker • Journal • P1 data • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Fibrosis • Immunology • Inflammation • Muscular Dystrophy

MESA: Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy (clinicaltrials.gov) - P2 | N=260 | Enrolling by invitation | Sponsor: Edgewise Therapeutics, Inc. | Trial completion date: Aug 2029 ➔ Feb 2031 | Trial primary completion date: Aug 2029 ➔ Feb 2031

Biomarker • Trial completion date • Trial primary completion date • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

EDG-5506-203: Open-Label Extension of EDG-5506 in Participants with Becker Muscular Dystrophy (clinicaltrialsregister.eu) - P1/2 | N=82 | Recruiting | Sponsor: Edgewise Therapeutics Inc.

Biomarker • New P1/2 trial • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) (clinicaltrials.gov) - P2 | N=244 | Active, not recruiting | Sponsor: Edgewise Therapeutics, Inc. | N=175 ➔ 244

Biomarker • Enrollment change • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) (clinicaltrials.gov) - P2 | N=76 | Active, not recruiting | Sponsor: Edgewise Therapeutics, Inc. | Trial completion date: Feb 2026 ➔ Jan 2027 | Trial primary completion date: Feb 2026 ➔ Jan 2027

Biomarker • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (clinicaltrials.gov) - P2 | N=43 | Active, not recruiting | Sponsor: Edgewise Therapeutics, Inc. | Trial completion date: Oct 2025 ➔ Mar 2027 | Trial primary completion date: Oct 2025 ➔ Mar 2027

Biomarker • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

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. (PubMed, Muscle Nerve) - P1 | "Sevasemten was generally well tolerated. Preliminary observations of decreases in biomarkers of muscle damage in adults with BMD support further clinical development."

Clinical • Journal • P1 data • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy • MB

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX) (clinicaltrials.gov) - P2 | N=43 | Active, not recruiting | Sponsor: Edgewise Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Biomarker • Enrollment closed • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Edgewise Therapeutics to Present on Sevasemten for the Treatment of Becker Muscular Dystrophy at the 2025 MDA Clinical and Scientific Conference (Businesswire) - "Edgewise Therapeutics...announced that the company will present data on sevasemten, an investigational orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. The conference will take place at the Hilton Anatole, Dallas, TX from March 16-19, 2025....A companion poster presentation will be available for Dr. McDonald’s presentation (poster #O78)."

P2 data • Preclinical • Becker Muscular Dystrophy

MESA: Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy (clinicaltrials.gov) - P2 | N=260 | Enrolling by invitation | Sponsor: Edgewise Therapeutics, Inc. | N=200 ➔ 260 | Trial completion date: Mar 2028 ➔ Aug 2029 | Trial primary completion date: Mar 2028 ➔ Aug 2029

Biomarker • Enrollment change • Trial completion date • Trial primary completion date • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

Edgewise Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights (Businesswire) - "The Company plans to engage the U.S. FDA in the first half of 2025 to discuss marketing authorization filing strategies for sevasemten in Becker...The Company expects to report data from LYNX and FOX as well as its future clinical trial plans in the first half of 2025...The Company expects to report topline CIRRUS-HCM 28-day data in the first quarter of 2025. Further, the Company expects to report data from the 12-week CIRRUS-HCM trial in individuals with obstructive HCM and non-obstructive HCM in the second half of 2025...During 2025, the Company expects to file an investigational new drug application for a novel candidate for the treatment of heart failure, and select a proprietary cardiometabolic drug candidate based on preclinical proof-of-concept data."

FDA event • IND • P2 data • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Heart Failure • Hypertrophic Cardiomyopathy

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) (clinicaltrials.gov) - P2 | N=170 | Active, not recruiting | Sponsor: Edgewise Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Biomarker • Enrollment closed • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) (clinicaltrials.gov) - P2 | N=76 | Active, not recruiting | Sponsor: Edgewise Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Biomarker • Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Phase 2 Study of EDG-5506 in Children and Adolescents with Duchenne Muscular Dystrophy Previously Treated with Gene Therapy (FOX) (clinicaltrials.gov) - P2 | N=48 | Recruiting | Sponsor: Edgewise Therapeutics, Inc. | N=24 ➔ 48 | Trial completion date: Jun 2025 ➔ Oct 2025 | Trial primary completion date: Jun 2025 ➔ Oct 2025

Biomarker • Enrollment change • Gene therapy • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Study of EDG-5506 in Children with Duchenne Muscular Dystrophy (LYNX) (clinicaltrials.gov) - P2 | N=72 | Recruiting | Sponsor: Edgewise Therapeutics, Inc. | N=54 ➔ 72

Biomarker • Enrollment change • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Edgewise Receives European Medicines Agency (EMA) Orphan Drug Designations for Sevasemten (EDG-5506) for the Treatment of Becker and Duchenne Muscular Dystrophies (Businesswire) - "Edgewise Therapeutics, Inc...announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne). Sevasemten is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage. Sevasemten is currently in late-stage clinical trials for individuals with Becker and is also being studied in Duchenne."

European regulatory • Orphan drug • Duchenne Muscular Dystrophy • Muscular Dystrophy

ARCH: A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy (clinicaltrials.gov) - P1 | N=12 | Completed | Sponsor: Edgewise Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy

Effects of EDG-5506, a Fast Myosin Modulator, on Function and Biomarkers of Muscle Damage in Adults with Becker Muscular Dystrophy (AAN 2024) - P2 | "EDG-5506 was well tolerated with rapid and sustained reductions in biomarkers of muscle damage. Trends toward improvements in function compared to expected natural history trajectories were observed. Phase 2 trials in BMD and DMD are ongoing (NCT05291091 and NCT05540860) with a cohort designed to be registrational enrolling in BMD."

Biomarker • Clinical • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Muscular Dystrophy • Novel Coronavirus Disease • MB

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX) (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Edgewise Therapeutics, Inc. | Not yet recruiting ➔ Recruiting | Initiation date: Dec 2023 ➔ Mar 2024

Biomarker • Enrollment open • Gene therapy • Trial initiation date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Comparison of short- and long-term proteomic response to the fast skeletal myosin inhibitor, EDG-5506, in Becker muscular dystrophy (BMD) (MDA 2024) - P1 | "Conclusions We validated the use of SOMAscan aptamers to measure CK and TNNI2 and demonstrated that reductions in muscle injury after short-term EDG-5506 therapy are maintained through long-term treatment. Further, we show that long-term treatment also results in changes to inflammatory signatures towards those of healthy individuals."

Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Genetic Disorders • Inflammation • Muscular Dystrophy

Functional and Muscle Damage Biomarker Changes Following Treatment with EDG-5506, a Fast Myosin Modulator, in Adults with Becker Muscular Dystrophy (MDA 2024) - P2 | "Phase 2 trials in BMD and DMD are ongoing (NCT05291091 and NCT05540860) with a pivotal cohort enrolling BMD adults. Additional data will be available at the time of presentation."

Biomarker • Clinical • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Muscular Dystrophy • Novel Coronavirus Disease • MB

North Star (NSAA and NSAD) Functional Assessments in Individuals with Becker Muscular Dystrophy (MDA 2024) - P1 | "Objectives To understand reproducibility of NSAA and NSAD in Becker using repeated pre-treatment assessments in clinical trials of EDG-5506, an orally administered fast skeletal muscle myosin inhibitor designed to prevent contraction-induced muscle damage (NCT05160415/05291091), and to examine patterns of compensation/loss of individual functions over a range of baseline NSAA/NSAD scores...Certain measures appear to be affected earlier (stand from chair), i.e., at higher NSAA scores, or later (lift head) than has been observed in Duchenne. This information is helpful in interpreting the clinical meaningfulness of changes in these clinical outcome measures in clinical trials in Becker, a serious disease without approved therapies."

Clinical • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Fibrosis • Immunology • Muscular Dystrophy

Functional and Muscle Damage Biomarker Changes Following Treatment with EDG-5506, a Fast Myosin Modulator, in Adults with Becker Muscular Dystrophy (MDA 2024) - No abstract available

Biomarker • Clinical • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy • Muscular Dystrophy