Edgewise Will Use $95M Funding to Advance EDG-5506 for BMD, DMD

Edgewise Therapeutics has received $95 million in funding to advance the clinical development of its experimental therapy EDG-5506, designed to improve physical functioning in people with Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD).

Following the closing of the Series C financing, the proceeds also will be used to support Edgewise’s other rare disease programs and future licensing opportunities.

“We are excited about the potential of EDG-5506 for patients affected by BMD and DMD, who are currently underserved with limited treatment options,” Kevin Koch, PhD, president and CEO of Edgewise, said in a press release.

EDG-5506 is an oral small molecule therapy designed to address the root cause of progressive hereditary diseases that target the skeletal muscles. It functions by selectively limiting excessive muscle fiber contraction and the deterioration that results from a loss of the protein dystrophin, as occurs in muscular dystrophy. By easing muscle hypercontraction, EDG-5506 can potentially improve physical function in people with these diseases.

The treatment may be effective for a variety of neuromuscular disorders and can be used as a single therapy or in combination with existing or potential therapies to boost effects.

Edgewise recently announced the launch of a Phase 1 clinical trial (NCT04585464) to evaluate the safety and tolerability of EDG-5506, first in healthy volunteers, then in BMD patients. The study is recruiting up to 152 adults, ages 19 to 55, to participate in the trial, which will be conducted at a single study site in San Antonio, Texas. More information can be found here.

In the first part of the study, healthy volunteers will be randomly assigned to receive either once-daily EDG-5506 or a placebo in a single ascending dose — with or without food — or multiple ascending doses. Because the trial is double-blind, neither the study participants nor the researchers will know who received the treatment and who received the placebo. Edgewise expects the trial to conclude late next year. 

Both DMD and BMD are caused by mutations in the DMD gene that result in a deficit in functional dystrophin. DMD is the more common and severe of the two disorders. 

The financing, led by Viking Global Investors, included participation from Janus Henderson Investors, Surveyor Capital (a Citadel company), RA Capital Management, Cormorant Asset Management, Logos Capital, and Wellington Management, as well as existing investors such as CureDuchenne Ventures.

“We are delighted to partner with this distinguished group of investors as we advance our drug pipeline,” Koch added.