Keros Therapeutics, Inc. ("Keros") presented additional clinical data from its phase 1 clinical trial of ker-065 in healthy male volunteers at the American Society of Bone and Mineral Research 2025 annual meeting on Saturday, September 6, 2025. The trial was a randomized, double-blind, placebo-controlled, two-part dose escalation trial with the primary objectives of assessing safety, tolerability, and pharmacokinetics of ker-065. As of the final data cut-off date of April 29, 2025, treatment with ker-065 was generally well-tolerated at all dose levels tested, with no dose-limiting toxicities or serious adverse events reported.
The additional results from the trial demonstrated significant changes in bone biomarkers, including increased bone formation and reduced bone resorption consistent with tissue level changes, as indicated by observed increases in bone mineral density (BMD). Whole body BMD improvements at day 85 were sustained through day 141, suggesting a balance between osteoblast and osteoclast activity. Furthermore, increased lumbar spine BMD following three doses was sustained through day 141.
Ker-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin a, two ligands that signal through activin receptors, to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle, and increase bone strength. The company is developing ker-065 for the treatment of neuromuscular diseases, with an initial focus on Duchenne muscular dystrophy (DMD).
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. The absence of dystrophin in muscle cells leads to significant cell damage, muscle cell death, and the replacement of muscle with fibrotic and fatty tissue, causing progressive loss of muscle strength and function, leading to immobility and respiratory and cardiac complications.
Keros Therapeutics, Inc. is a clinical-stage biopharmaceutical company 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. The company's 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, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and in patients with myelofibrosis. The market has reacted to these announcements by moving the company's shares -0.1% to a price of $15.575. For the full picture, make sure to review Keros Therapeutics's 8-K report.
