Edgewise Therapeutics Inc. CEO Kevin Koch speculated that “perhaps a different environment at the FDA” from four months ago led to reviewers’ caution on sevasemten, his firm’s fast skeletal myelin inhibitor for Becker muscular dystrophy and Duchenne muscular dystrophies.
Top-line data from the second and third parts of Edgewise Therapeutics Inc.’s four-part phase II Cirrus-HCM study in hypertrophic cardiomyopathy (HCM) has produced more positive data. There was however less enthusiasm for the results than there had been in September for the first batch of data.
Researchers from Edgewise Therapeutics Inc. have previously developed and characterized a novel bmx mouse model of Becker muscular dystrophy (BMD), which harbors a deletion of murine Dystrophin exons 45-47 on a C57/BL6J background.
Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiovascular disorder with around 85% of people with HCM remaining undiagnosed. There are no treatments approved for nonobstructive HCM (nHCM) to date.
Positive data from two studies boosted Edgewise Therapeutics Inc.’s market share and elevated analyst enthusiasm for the company and its treatment of obstructive hypertrophic cardiomyopathy. Top-line data from the phase I and phase II studies of EDG-7500 in treating the genetic disease that results in thickened heart muscles showed the therapy was well-tolerated in healthy volunteers and produced meaningful improvements in those with the disease.
Researchers from Edgewise Therapeutics Inc. presented preclinical data for the cardiac sarcomere modulator, EDG-7500, which is being developed for the treatment of hypertrophic cardiomyopathy (HCM) and other diseases of diastolic dysfunction.
With no drugs approved by the U.S. FDA for treating Becker muscular dystrophy, Edgewise Therapeutics Inc. reported positive two-year, phase Ib data looking at patients’ ability to physically function, plus biomarker data.
Edgewise Therapeutics Inc. priced an underwritten offering of 21.8 million shares at $11 each as it looks for about $240 million in gross proceeds to develop its Duchenne and Becker muscular dystrophies treatment. The offering propelled the company’s stock (NASDAQ:EWTX) Jan. 19 to close 34.5% higher at $13.04 each, their highest valuation in the past 12 months.
Edgewise Therapeutics Inc. has divulged pyridazinone compounds acting as myosin-2 (MYH2) (ATPase activity) inhibitors reported to be useful for the treatment of Duchenne muscular dystrophy, amyotrophic lateral sclerosis, encephalitis, spinal cord injury, phenylketonuria, cerebral palsy, Alzheimer’s disease and Parkinson’s disease, among others.
