CureDuchenne is pleased to share the news that Edgewise Therapeutics is starting a Phase 1 clinical trial to assess the safety, tolerability, and appropriate dose of their experimental therapeutic aimed at minimizing muscle fiber damage in Duchenne and Becker muscular dystrophies. CureDuchenne Ventures is a funder of Edgewise and this program, and we are thrilled to see our investment reach the clinical stage.
An important and early contributor to muscle pathology in Duchenne and Becker muscular dystrophies is the damage that muscles undergo when contracting. While this wear-and-tear can be repaired in normal muscles, in Duchenne and Becker it can lead to inflammation, fibrosis, and eventual loss of function. Edgewise’s EDG-5506 was designed to minimize this contraction-induced muscle fiber break- down, and thus also the subsequent pathological steps in the disease processes.
Edgewise’s approach is a novel strategy for treating these diseases, has the potential to benefit all individuals with Duchenne or Becker (no matter what DMD mutation they carry), and likely could prove to be additive with other therapies aimed at restoring dystrophin. For all of these reasons, as well as our great respect for the team at Edgewise, CureDuchenne Ventures was excited to provide funding to Edgewise in September 2019.
Edgewise’s Phase 1 clinical trial of EDG-5506 will begin by testing the safety, tolerability, and drug distribution and metabolism properties in adult healthy volunteers, to help find the optimal drug dose. Adults with Becker muscular dystrophy will be enrolled after the results in healthy volunteers. This trial represents an important opportunity for the Becker community, who don’t have many treatment or clinical trial opportunities. The results of this Phase 1 trial will inform the pivotal trials Edgewise is planning to conduct in both Duchenne and Becker muscular dystrophy.