Edgewise Therapeutics shows positive results from muscular dystrophy drug trials

BOULDER — Edgewise Therapeutics has seen positive results from its first-in-human Phase 1 clinical trials for its muscular dystrophy treatment drug. 

The drug, called EDG-5506, is designed to be taken orally and treat rare muscle disorders such as Duchenne muscular dystrophy and Becker muscular dystrophy. Those little-known diseases target the muscles of the hips, thighs, shoulders and upper arms, leading to difficulties in rising from the floor, climbing stairs, maintaining balance and raising arms.

This trial was the first trial involving humans with BMD. Previous trials involved healthy volunteers. The trial was used to assess safety and dosage in BMD patients. 

According to the press release announcing the findings, “treatment with EDG-5506 led to a significant and time-dependent decrease in key biomarkers of muscle damage…This reduction in muscle damage biomarkers supports the hypothesis that EDG-5506 meaningfully reduces damage from the excessive stress present in dystrophic muscle, thus potentially preserving muscle function and preventing disease progression in dystrophinopathies.”

Phase 2 clinical trials for EDG-5506 are expected to conclude in June.

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