KER-065 / Keros Therap - LARVOL DELTA

Keros Announces U.S. FDA Orphan Drug Designation Granted to KER-065 for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire)

Orphan drug • Duchenne Muscular Dystrophy

Phase 1 Trial in Healthy Participants of KER-065, Modified Activin Receptor Ligand Trap, Support Development in Duchenne Muscular Dystrophy (DMD) and Bone Disorders (ASBMR 2025) - No abstract available

Clinical • P1 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Orthopedics

Keros Announces Return of $375 Million in Excess Capital to Stockholders (Keros Therap Press Release) - "To that end, we intend to return a significant amount of excess capital to stockholders while continuing to pursue development of our lead product candidate, KER-065, for the treatment of neuromuscular diseases, with an initial focus on Duchenne muscular dystrophy."

Commercial • Duchenne Muscular Dystrophy

A Phase 1 study to Assess Safety and Tolerability of KER-065 in Healthy Adult Volunteers (ANZCTR) - P1 | N=38 | Completed | Sponsor: Keros Therapeutics, Inc. | Recruiting ➔ Completed

Trial completion • Genetic Disorders • Musculoskeletal Diseases • Obesity

Keros Therapeutics Announces Initial Topline Results from the Phase 1 Clinical Trial of KER-065 in Healthy Volunteers (GlobeNewswire) - P1 | N=NA | "Trial achieved key objectives for safety, tolerability, pharmacokinetics and pharmacodynamics...Keros Therapeutics...today 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 ('MAD') treatment period (Day 85)....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....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."

P1 data • P2 data • Regulatory • Duchenne Muscular Dystrophy

Keros Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "'We are excited to report initial data from the ongoing Phase 1 clinical trial of KER-065 in healthy volunteers in the first quarter of 2025, which we believe can inform our advancement into a Phase 2 clinical trial in neuromuscular disease, with our initial focus on Duchenne muscular dystrophy."

P1 data • Duchenne Muscular Dystrophy

RKER-065, a novel ActRII ligand trap, counteracted the adverse musculoskeletal effects of glucocorticoid treatment in a murine model of Duchenne muscular dystrophy (New Directions 2024) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases • ACVR2A

RKER-065 enhanced the therapeutic effect of phosphorodiamidate morpholino oligomer in a Duchenne muscular dystrophy mouse model. (New Directions 2024) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

RKER-065 enhanced the therapeutic effect of phosphorodiamidate morpholino oligomer in a Duchenne muscular dystrophy mouse model. (New Directions 2024) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

KER-065, a Novel ActRII-Fc Ligand Trap, Functions as a Potent Muscle Anabolic in Mice (ENDO 2024) - "Abstract is embargoed at this time."

Preclinical • Genetic Disorders • Obesity • ACVR2A

A Phase 1 study to Assess Safety and Tolerability of KER-065 in Healthy Adult Male Volunteers (ANZCTR) - P1 | N=44 | Recruiting | Sponsor: Keros Therapeutics, Inc. | Not yet recruiting ➔ Recruiting | Initiation date: Dec 2023

Enrollment open • Trial initiation date • Musculoskeletal Diseases • Orthopedics

RKER-065, a novel ActRII ligand trap, counteracted the deleterious musculoskeletal effects of chronic glucocorticoid treatment in young mice (MDA 2024) - "Our data demonstrate that RKER-065 can attenuate the negative musculoskeletal effects of chronic glucocorticoid use in young mice. Therefore, KER-065 has the potential to improve muscle mass, muscle function, and bone health in DMD patients receiving chronic glucocorticoid treatment."

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Atrophy • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • ACVR2A • INHBB • TGFB1

RKER-065 enhanced the therapeutic effect of phosphorodiamidate morpholino oligomer in a Duchenne muscular dystrophy mouse model (MDA 2024) - "Our study demonstrated RKER-065’s effect in ameliorating muscle and bone loss in a DMD mouse model. The observation that RKER-065 treatment augmented PMO-induced dystrophin expression highlighted the potential synergistic benefit of using KER-065 and PMO combination therapy for DMD."

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Osteoporosis • INHBB

RKER-065 enhanced muscle and bone in a progressive murine model of Duchenne muscular dystrophy (MDA 2024) - "These data demonstrated RKER-065 reversed muscle atrophy, improved muscle function and ameliorated bone loss in a DMD model. This further supports clinical development of KER-065 as a potential treatment for dystrophic patients and supports that targeting this pathway warrants investigation."

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Atrophy • Muscular Dystrophy • Musculoskeletal Diseases • Orthopedics • Osteoporosis • ACVR2A • TGFB1