Keros Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Ker-065, a novel therapy designed to treat Duchenne Muscular Dystrophy (DMD). This designation is significant for Keros as it highlights the unmet medical need for patients with DMD and provides potential benefits such as tax credits for qualified clinical testing, waiver or partial payment of FDA application fees, and seven years of market exclusivity if approved.
Ker-065 is a ligand trap designed to inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors. It is aimed at increasing skeletal muscle regeneration, muscle size and strength, reducing body fat, fibrosis of the skeletal muscle, and increasing bone strength. Keros is initially focusing on developing Ker-065 for the treatment of neuromuscular diseases, with DMD as the primary target.
DMD is the most common form of muscular dystrophy, resulting in muscle degeneration and premature death due to the lack of functional dystrophin protein caused by a gene mutation. This absence leads to significant cell damage, muscle cell death, and the replacement of muscle with fibrotic and fatty tissue, ultimately resulting in the progressive loss of muscle strength and function, immobility, and respiratory and cardiac complications. Keros' aim is to address this critical medical need with the development of Ker-065.
Keros Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat patients with disorders related to dysfunctional signaling of the TGF-ß family of proteins. Their lead product candidate, Ker-065, holds promise for providing meaningful and potentially disease-modifying benefits to patients with neuromuscular diseases, particularly DMD.
The granting of orphan drug designation for Ker-065 marks a significant milestone for Keros as they advance into a phase 2 clinical trial in patients with DMD, further emphasizing their commitment to addressing the unmet medical needs of this patient population. The market has reacted to these announcements by moving the company's shares -2.07% to a price of $14.19. Check out the company's full 8-K submission here.
