Edgewise Therapeutics announced positive topline results for its potential new Duchenne muscular dystrophy (DMD) treatment, EDG-5506. The results showed that EDG-5506 was generally well tolerated and demonstrated robust engagement of targeted myosin.

Joanne Donovan, MD, PhD, chief medical officer of Edgewise, stated, “We are excited by the potential that EDG-5506 may serve as an oral treatment to decrease muscle damage, the root cause of DMD and [Becker muscular dystrophy (BMD)], and we eagerly look forward to better understanding its full potential in future clinical studies.”

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The most common adverse reactions experienced by study participants were transient and self-resolving dizziness and somnolence. The incidence of adverse reactions decreased as exposure levels increased, indicating the potential development of tolerance to EDG-5506. No adverse events were observed in electrocardiograms, laboratory values, or other recorded vital signs.

Pharmacokinetic (PK) data collected during the trial showed that EDG-5506 had a high affinity for binding to myosin in fast skeletal muscle fibers.

The pharmacodynamics of EDG-5506 were tested through the inhibition of involuntary muscle activation evoked in the leg using transmagnetic stimulation. The drug successfully inhibited muscle activity in the leg without affecting voluntary grip, shoulder, or hip strength.

Other forms of myosin in other parts of the body, such as the heart, did not appear to be affected by EDG-5506.

Forty healthy volunteers received multiple ascending oral doses (ranging from 5 mg to 40 mg) of EDG-5506 or placebo. Doses above 10 mg reached pharmacologically active levels, which were determined by multiple DMD preclinical models.

Based on the success of this clinical trial, a phase 2 trial of EDG-5506 in patients with DMD and BMD is being planned for 2022. The new trial will investigate the drug’s safety, PKs, and changes in biomarkers such as fast troponin I and creatine kinase. In the meantime, a phase 1b open-label study of EDG-5506 in patients with BMD has recruited its first patient.

EDG-5506 is an oral, small molecule myosin inhibitor that is designed to help protect fast skeletal muscles from injury in patients with dystrophinopathies.


Edgewise Therapeutics announces positive topline results from the EDG-5506 phase 1 multiple ascending dose (MAD) study in healthy volunteers (HVs) and doses first Becker muscular dystrophy (BMD) patients. News release. Edgewise Therapeutics; October 28, 2021.