Keros Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its investigational therapy, ker-065, aimed at treating Duchenne muscular dystrophy (DMD). This designation signifies a significant milestone for Keros as they advance ker-065 into a phase 2 clinical trial for DMD patients.
DMD is a rare medical condition affecting muscle degeneration and premature death, caused by the lack of functional dystrophin protein due to a gene mutation. The absence of dystrophin makes muscle cells more susceptible to damage and leads to progressive muscle degeneration.
Ker-065, a novel ligand trap, is designed to inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors. Its aim is to increase skeletal muscle regeneration, muscle size and strength, reduce body fat, fibrosis of the skeletal muscle, and increase bone strength, effectively targeting neuromuscular diseases, with an initial focus on DMD.
The orphan drug designation for ker-065 provides potential benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees, and seven years of market exclusivity if approved.
Keros Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat disorders linked to dysfunctional signaling of the TGF-ß family of proteins. Their lead product candidate, ker-065, demonstrates potential disease-modifying benefits for patients with neuromuscular diseases, particularly those with DMD.
Keros' most advanced product candidate, elritercept, is being developed for the treatment of cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and myelofibrosis. As a result of these announcements, the company's shares have moved -2.07% on the market, and are now trading at a price of $14.19. If you want to know more, read the company's complete 8-K report here.
