Pipeline

KER-065 is an investigational selective activin receptor ligand trap being developed for the treatment of neuromuscular disorders, with an initial focus on Duchenne muscular dystrophy (DMD). KER-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, which are negative regulators of muscle and bone mass and strength, to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength. In March 2025, we announced initial topline results from the Phase 1 clinical trial of KER-065 in healthy volunteers. KER-065 was generally well-tolerated, with no major safety signals observed as of the February 6, 2025 data cut-off date. No serious adverse events or dose-limiting toxicities were reported. See the relevant Keros press release here.

Elritercept (KER-050) is an investigational activin receptor type IIA (ActRIIA) fusion protein designed to inhibit select TGF-β ligands, including activin A. Based on data from our completed Phase 1 clinical trial, multiple preclinical studies and our ongoing Phase 2 clinical trials, we believe elritercept has the potential to increase red blood cell and platelet production by acting across the spectrum of cellular differentiation and maturation in hematopoiesis while also improving bone health.

Elritercept is being developed for the treatment of ineffective hematopoiesis in myelodysplastic syndromes (MDS) and myelofibrosis (MF), where the impact of the disease in the bone marrow microenvironment can contribute to anemia, thrombocytopenia, neutropenia and an increased risk of acute myeloid leukemia. In December 2024, we entered into an exclusive license agreement with Takeda Pharmaceuticals U.S.A., Inc. to further develop, manufacture and commercialize elritercept worldwide outside of mainland China, Hong Kong and Macau, which became effective in January 2025.