28 April 2021 - - US-based biopharmaceutical company Edgewise Therapeutics, Inc., (NASDAQ: EWTX) has appointed Joanne M. Donovan, M.D., Ph.D., to the position of chief medical officer, the company said.

Donovan will be responsible for leading clinical, medical and regulatory strategy and operations for the company's pipeline, including EDG-5506, the company's lead product candidate that is advancing in clinical development for Duchenne and Becker muscular dystrophy (DMD and BMD).

Prior to joining Edgewise, Dr. Donovan served as chief medical officer and senior vice president, Clinical Development at Catabasis, a biopharmaceutical company focused on rare diseases.

In this role, Dr. Donovan led clinical development in DMD from first-in-human through Phase 3 studies and New Drug Application preparation.

She led and built all clinical and regulatory functional areas, overseeing regulatory and quality affairs, pharmacovigilance, medical affairs and clinical operations. Since 1989, she has been a staff physician at the VA Boston Healthcare System, where she was formerly Chief of Gastroenterology.

Dr. Donovan has held an appointment at Harvard Medical School since 1990, most recently as Associate Clinical Professor of Medicine.

From 1998 to 2011, Dr. Donovan served in positions of increasing responsibility, ultimately as vice president of Clinical Development, at Genzyme, a biotechnology company focused on rare diseases, which she joined through its acquisition of GelTex Pharmaceuticals.

Dr. Donovan holds a Ph.D. in medical engineering and medical physics from the Massachusetts Institute of Technology, an M.D. from Harvard Medical School and an S.B. from the Massachusetts Institute of Technology.

She completed residency training in internal medicine and a fellowship in gastroenterology at the Brigham and Women's Hospital.

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 can 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 the safety, tolerability, pharmacokinetics and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and adults with BMD (Phase 1b).

In March 2021, EDG-5506 advanced into the Multiple Ascending Dose cohort, having successfully completed the Single Ascending Dose portion of the study.

The company expects to report topline MAD and BMD patient data, later in 2021.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need.

Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform.

Edgewise's platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases.