Management's first flush of excitement two years ago in creating new therapies and a new company led also to the creation of Edgewise Therapeutics Inc.'s name.

"We were all talking and no one could get a word in edgewise," Kevin Koch, Edgewise's president and CEO, told BioWorld. "It just made sense."

Boulder, Colo.-based Edgewise just closed a $50 million series B financing to advance its lead candidate's preclinical research into Duchenne and Becker muscular dystrophy. Edgewise also has candidates for treating adult neuromuscular disorders and metabolic disorders. The funding is designed to get its lead candidate, EDG-001, for both Duchenne and Becker, into proof-of-concept clinical testing and file an IND application with the FDA in the second half of 2020, Koch said.

The round was co-led by Novo Holdings A/S and U.S. Venture Partners with participation from Deerfield Management, New Leaf Venture Partners, Cure Duchenne Ventures and a founding investor, Orbimed Advisors, one of the largest health care investment companies, with more than $14 billion in management assets. Orbimed led Edgewise's $15.5 million series A fundraising round in 2017.

Edgewise's goal is to arrest muscle damage in muscular dystrophy patients, reducing the inflammatory and fibrotic response while preserving muscle integrity and physical function.

"We've had major discussions on the mechanism and how it stabilizes muscle and by doing that you decrease injury of the muscle. That's the hallmark of the disease," Koch said. "We have quite a bit of preclinical data on it."

Alan Russell, Edgewise's co-founder and chief science officer, told BioWorld that EDG-001 is potentially combinable with currently approved therapies. Russell has been vice president and head of Glaxosmithkline plc's Muscle Metabolism Discovery Performance Unit, which focused on muscle-compromised patients. He also spent time at Cytokinetics Inc. Focusing on Duchenne has been one of his professional goals.

"Of all patient advocacy communities, Duchenne is one of the strongest ones," Russell said.

Duchenne is a rare but fatal X-linked degenerative neuromuscular disorder. It affects an estimated one in every 3,500 to 5,000 males born worldwide.

Edgewise is not relying on dystrophin replacement, making candidates applicable for patients with any dystrophin mutation and for use in combination with all approved and experimental therapies for Duchenne, an inherited muscle disease caused by mutations in the dystrophin gene. Dystrophin is a protein that connects muscle contraction to the supporting membrane around muscle fibers.

Edgewise's research includes enzymology and high-throughput biochemistry, skeletal and cardiac muscle physiology and in vivo pharmacology. The research is being conducted in the University of Colorado Boulder's Biofrontiers Institute, home to both academic and industrial research.

"Some of our ideas come from observations from the '80s and '90s in Duchenne patients," Koch said. "No one recognized this particular target class. The endpoints are highly measurable in the clinic. It makes for a neat, crisp story in you could make this class of drugs."

Because the company has identified multiple modulators for muscle, there could be licensing opportunities in the future as offshoot agreements, Koch said.

The series B funds will also allow the company to grow itself as it's seeking to build out its clinical staff. There are currently 10 full-time employees and a number of key consultants. Edgewise is looking for a chief medical officer and a director of clinical operations.

Also in the pipeline is EDG-002, which is in preclinical proof-of-concept research for adult neuromuscular disorders, and EDG-003, which is being researched for metabolic disorders.

"EDG-003 is still in research mode and we're not quite ready to talk about it yet," Koch said.

Koch has been a venture partner with Orbimed as well as having worked in life sciences for more than 20 years. Most recently he was the senior vice president of drug discovery: chemical and molecular therapeutics at Biogen Inc., where he managed global drug discovery and biomarker development as well as the immunology and hematology teams. From 1998 to 2013, he was president, chief scientific officer, board member and co-founder of Array Biopharma Inc. While there, he built a fully integrated R&D team that oversaw the invention of more than 20 clinical development candidates in multiple therapeutic areas. Prior to Array, he held senior positions at Amgen Inc. and Pfizer Central Research.

Joining Edgewise's board are Kenneth Harrison, a principal at Novo Ventures, and Jonathan Root, a general partner at U.S. Venture Partners. Robert Jackson, a partner and chief science officer at Deerfield Management, joins as a board observer.

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