Mesoblast has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) to proceed directly to a registrational clinical trial of Ryoncil (remestemcel-L-rknd) in Duchenne muscular dystrophy.
The randomized, placebo-controlled study will enroll 76 patients aged 5 to 9 years and deliver seven infusions of 2 x 10^6 cells per kg over nine months on top of standard of care, with the primary endpoint set as time-to-stand at nine months, a validated FDA endpoint for approval.
The company says the trial builds on Ryoncil’s pediatric safety data in steroid-refractory acute graft-versus-host disease and preclinical efficacy in DMD models, and Mesoblast aims to leverage the product’s anti-inflammatory mechanism to preserve muscle function and slow disease progression.
Mesoblast will work with Parent Project Muscular Dystrophy to support patient identification and trial awareness. The company notes approximately 15,000 children in the U.S. are affected by DMD. Timelines for trial initiation and completion were not provided by the company.
