DMD Program Advances with Q2 2026 Phase II Trial
Keros Therapeutics confirmed on March 17, 2026, that it is advancing its drug candidate, rinvatercept, for the treatment of Duchenne muscular dystrophy (DMD). The company announced it has secured orphan drug status for the indication and released fresh Phase I clinical data. This progress paves the way for a planned Phase II trial, which is scheduled to begin in the second quarter of 2026, marking a critical execution milestone for the company's clinical pipeline.
Dual-Disease Strategy Expands into ALS
The therapeutic potential of rinvatercept extends beyond DMD, as Keros is also developing the drug for amyotrophic lateral sclerosis (ALS). Rinvatercept is designed to block myostatin and activin A, proteins that inhibit muscle growth, with the goal of preserving muscle strength and function. To accelerate its ALS program, Keros is collaborating with Massachusetts General Hospital's MyMatch initiative, a biomarker-driven program designed to match patients to experimental therapies most likely to benefit them. This strategic partnership aims to optimize the design for larger Phase 2/3 clinical trials.
Early Data Shows Muscle Mass Gains, Supports Outlook
Early clinical results support the scientific rationale behind rinvatercept. Data from a Phase 1 trial in healthy volunteers showed the therapy was generally well-tolerated and demonstrated an ability to increase muscle mass. Furthermore, preclinical studies in a mouse model of ALS showed rinvatercept preserved muscle strength and protected the neuromuscular junction where nerves communicate with muscles. The combination of a clear mechanism, a multi-disease strategy, and positive early data reinforces the potential of rinvatercept as a key value driver for Keros.
rinvatercept represents a potentially novel approach to treat patients with ALS.
— Jasbir S. Seehra, PhD, President and CEO of Keros.