Edgewise Therapeutics has announced the FDA has authorized the initiation of the LYNX Phase 2 clinical trial of EDG-5506 in participants with Duchenne. EDG-5506 is a small molecule, taken orally, that is intended to help protect muscle by reducing injury of fast-twitch skeletal muscle.
The placebo-controlled LYNX Phase 2 study will assess the effects of three doses of EDG-5506, over 12 weeks on safety, pharmacokinetics, and biomarkers of muscle damage. All participants will continue in an open-label extension for a total of 12 months of study. Approximately 27 children with Duchenne, ages 4 and up to their 10th birthday on stable corticosteroids, are expected to enroll at up to 12 sites across the US. Edgewise expects to begin dosing participants in the 4thquarter of 2022.
Read the Press Release from Edgewise Therapeutics:
Edgewise Therapeutics Announces FDA Authorization for Phase 2 Clinical Trial of EDG-5506 for the Treatment of Duchenne Muscular Dystrophy
BOULDER, Colo.–(BUSINESS WIRE)– Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today announced that the U.S. Food and Drug Administration (FDA) has authorized a clinical trial of EDG-5506 in children with DMD. The Company expects to begin dosing participants in the LYNX Phase 2 trial in the fourth quarter of 2022. EDG-5506 is an investigational orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as DMD and Becker muscular dystrophy (BMD).
“This is an important achievement for our team as we seek to expand our studies with EDG-5506 into individuals with DMD. Our team has worked with key opinion leaders and patient advocacy groups to thoughtfully design our LYNX Phase 2 clinical trial,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “The promising tolerability and changes in biomarkers of muscle damage that we have seen in studies with EDG-5506 in individuals with BMD supports our next step of expanding enrollment to individuals with DMD.”
The LYNX Phase 2 trial is a placebo-controlled trial to assess the effect of three doses of EDG-5506 over 12 weeks on safety, pharmacokinetics (PK) and biomarkers of muscle damage. Approximately 27 children with DMD aged 4 to 9 years on stable corticosteroids are expected to be enrolled at up to 12 sites across the United States. Participants will then continue in an open-label extension part of the trial for a total of 12 months to gain further insights into safety and functional measures. Importantly, this trial is designed to identify the doses of EDG-5506 that have the potential to reduce biomarkers of muscle damage and provide functional benefit to patients in a Phase 3 trial.
“As observed in many neuromuscular disorders, we need to have complementary approaches to address the underlying cause of the condition,” said Pat Furlong, Founding President and Chief Executive Officer, Parent Project Muscular Dystrophy. “By Edgewise expanding from Becker to Duchenne, this adds hope for those affected by Duchenne.”