Edgewise Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of individuals with Becker. EDG-5506, a selective, small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in DMD and BMD, is advancing in a Phase 1 clinical trial.

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need.

Read the Full Update from Edgewise

Read the announcement from Edgewise:

Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Individuals with Becker Muscular Dystrophy (BMD)

August, 16, 2021

–EDG-5506, a drug candidate designed to arrest muscle fiber breakdown in BMD and Duchenne muscular dystrophy (DMD), continues to advance in Phase 1 –

Boulder, Colo., (August 16, 2021) – Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of individuals with BMD. EDG-5506, a selective, small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in DMD and BMD, is advancing in a Phase 1 clinical trial.

“The FDA’s decision to grant EDG-5506 Fast Track designation underscores the urgency to address a significant unmet medical need for individuals with Becker muscular dystrophy,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “Becker is a serious, progressive disease that leads to severe disability, including loss of ambulation and heart disease that has substantial impact on day-to-day function in many individuals. We look forward to working closely with the FDA towards establishing EDG-5506 as the potential first muscle-directed therapy for individuals with Becker.”

“I am pleased to see that the FDA recognizes Becker muscular dystrophy as a serious disease and that the team at Edgewise is acknowledging the important unmet need in Becker,” said Craig McDonald, M.D., Professor and Chair, Department of Physical Medicine & Rehabilitation, University of California Davis.

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need. The Fast Track Program supports important new therapeutics to reach patients earlier. The designation is granted to therapeutics that offer potential to meaningfully impact survival, day-to-day functioning, or if left untreated, progression of the condition. Therapeutics that receive this designation receive a number of benefits that include more frequent meetings with the FDA to discuss development of the drug candidate and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.

The decision to give EDG-5506 Fast Track designation was supported by a robust preclinical data package and an ongoing Phase 1 clinical trial (NCT04585464). The Company expects to have Phase 1 topline Multiple Ascending Dose (MAD) data in healthy volunteers and in individuals with BMD later in 2021.

About EDG-5506 for DMD and BMD

EDG-5506 is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit injurious hypercontraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development.

The Phase 1 study of EDG-5506 is designed to evaluate safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). EDG-5506 is advancing in the Multiple Ascending Dose (MAD) portion of the study. The Company expects to have topline MAD data in healthy volunteers and in individuals with BMD later in 2021. Go to clinicaltrials.gov to learn more about this study (NCT04585464).