Keros Therapeutics reports results from Phase 1 clinical trial of KER-065

Keros Therapeutics (KROS) announced initial topline results from the Phase 1 clinical trial of KER-065 in healthy volunteers. Topline results from this ongoing trial are through the multiple ascending dose treatment period. “We are pleased to report topline results that met the key objectives of the Phase 1 clinical trial and provided important insights to inform the development of KER-065 for patients with DMD,” said Jasbir S. Seehra, Ph.D., Chair and CEO. “Considering the limitations of currently available therapies, the need for additional treatments in DMD remains high, and KER-065 has the potential to address multiple aspects of the disease, including across important tissues and underlying genetic deficiencies. Our strong financial foundation enables us to continue advancing our promising pipeline of novel therapeutics, and we look forward to engaging with regulators towards the aim of moving KER-065 to a Phase 2 clinical trial in the first quarter of 2026.” Key findings of this trial as of a February 6, 2025 data cut-off date include the following: KER-065 was generally well-tolerated, with no major safety signals observed to date. No serious adverse events or dose-limiting toxicities reported. Evidence for activin inhibition across tissues of interest, as KER-065 elicited: Increases in bone specific alkaline phosphate, a biomarker of bone formation, and decreases in C-Terminal Telopeptide, a biomarker of bone resorption. Increases in adiponectin, a biomarker of fat mobilization, and decreases in leptin, a biomarker of fat mass Changes in body composition, as demonstrated by increases in bone mineral density and muscle mass and decreases in fat mass, which in totality were consistent with activin inhibition. “We observed evidence of activin inhibition based on multiple biomarkers and body composition data. These data, coupled with preclinical and mechanistic insights on the pivotal role of the activin pathway in neuromuscular pathobiology, demonstrate the exciting therapeutic potential of KER-065 in DMD and other neuromuscular disorders,” said Yung H. Chyung, M.D., Chief Medical Officer. Keros plans on engaging with regulatory authorities, starting in the third quarter of 2025. Subject to the outcome of these regulatory interactions, Keros expects to initiate a Phase 2 clinical trial of KER-065 in patients with DMD in the first quarter of 2026.