Whalatane
3 months ago
CANYON Phase 2 placebo-controlled trial in adults with Becker : CANYON, the largest interventional Becker trial to date, includes 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. The primary endpoint of CANYON is change in creatine kinase (CK) over the treatment period with additional measures collected, including North Star Ambulatory Assessment (NSAA), 100-meter timed test, biomarkers of muscle damage and MRI. The Company expects to report CANYON data in the fourth quarter of 2024.
Kiwi
Whalatane
3 months ago
From IBD ....The biotech company tested its drug, EDG-7500, in healthy volunteers and patients with obstructive hypertrophic cardiomyopathy. In this disease, a genetic mutation causes the heart ventricles to thicken. This limits cardiac function and exercise capacity.
After a single dose, patients showed improved blood flow from the left ventricle. But sometimes drugs can do their job too well, suppressing cardiac function — a measure known as reduced left ventricular ejection fraction, or LVEF. But patients didn't show meaningful reductions in LVEF.. my emphasis
Kiwi
Whalatane
10 months ago
BOULDER, Colo., February 13, 2024--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 for the treatment of Duchenne.
EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker muscular dystrophy (Becker).
The FDA previously granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne and Becker, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designation for the treatment of Becker.
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Kiwi
Whalatane
11 months ago
Jonathan C. Fox, M.D., Ph.D., FACC
DIRECTOR
Jonathan C. Fox, M.D., Ph.D., FACC, serves as a member of our board of directors and is the president and chief medical officer for cardiovascular and renal diseases at BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical stage biotechnology company developing novel, genetically targeted therapies. Prior to that, Dr. Fox served as the chief medical officer of MyoKardia, Inc. Additionally, he worked as a consultant at Nigel-Montgomery, LLC, and held various senior R&D positions successively at SmithKline Beecham, Merck Research Laboratories and AstraZeneca. He was also on the faculty of the University of Pennsylvania School of Medicine. Dr. Fox serves on the board at ML BioSolutions and is a trustee of the Lankenau Institute for Medical Research. He is currently an adjunct Professor of Medicine at the Stanford University Cardiovascular Institute. He received his A.B. in biology, his Ph.D. in experimental pathology and his M.D. from the University of Chicago, and completed his training in Internal Medicine and Cardiology at Duke University. Dr. Fox is ABIM Certified in Cardiovascular Diseases and is a Fellow of the American College of Cardiology.
MyoKardia was bt out by BMS
Kiwi
crudeoil24
4 years ago
Edgewise Therapeutics, Inc. is a clinical-stage biopharmaceutical company that is developing orally bioavailable, small molecule therapies for musculoskeletal diseases. The Company is principally focused on discovering, developing and commercializing treatments for rare muscle disorders. Its platform utilizes custom-built 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. The Company's lead candidate, EDG-5506, is an orally administered allosteric, selective, fast myofiber (type II) myosin small molecule inhibitor designed to address the root cause of dystrophinopathies, including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The Company's research programs include EDG-6289, EDG-002 and EDG-003.