LEXINGTON, Mass. — August 20, 2025 — Leads & Copy — Keros Therapeutics, Inc. (Nasdaq: KROS) announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (DMD). According to President and Chief Executive Officer Jasbir S. Seehra, the designation highlights the unmet medical need for patients with DMD and serves as a significant milestone as Keros advances KER-065 into a Phase 2 clinical trial in patients with DMD.
The FDA grants Orphan Drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. The designation provides potential benefits, including tax credits, waiver 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 to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle, and increase bone strength. Keros is developing KER-065 for the treatment of neuromuscular diseases, with an initial focus on DMD.
DMD is the most common form of muscular dystrophy and results in muscle degeneration and premature death. It results from the lack of functional dystrophin protein that helps promote myofiber stability, caused by a gene mutation. The National Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
Keros Therapeutics is focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders linked to dysfunctional signaling of the TGF-ß family of proteins. Keros’ lead product candidate, KER-065, is being developed for the treatment of neuromuscular diseases, with an initial focus on DMD. Keros’ most advanced product candidate, elritercept, is being developed for the treatment of cytopenias.
Investor Contact: Justin Frantz, jfrantz@kerostx.com, 617-221-6042
Media Contact: Mahmoud Siddig / Adam Pollack / Viveca Tress, Joele Frank, Wilkinson Brimmer Katcher, (212) 355-4449
Source: Keros Therapeutics, Inc.
