GlobeNewswire·Apr 10, 2026, 4:09 PM ET

Mesoblast Receives IND Clearance From FDA to Directly Proceed to Registrational Trial for Approval of Ryoncil® in Duchenne Muscular Dystrophy - CORRECTION

FDA granted Investigational New Drug (IND) clearance to Mesoblast Limited to proceed directly to a registrational clinical trial for Ryoncil® (remestemcel-L-rknd) in Duchenne muscular dystrophy (DMD).
Approximately 15,000 children in the U.S. are living with DMD.
Ryoncil® is the first FDA-approved mesenchymal stromal cell (MSC) product, currently authorized for children under age 12 with steroid-refractory acute graft-versus-host disease (SR-aGvHD).
The registrational trial will randomize 76 patients aged 5 to 9 years to receive either Ryoncil® (7 infusions of 2 x 10^6 cells/kg over 9 months) or a placebo, in addition to standard of care.
The trial's primary endpoint is time-to-stand at nine months, a validated FDA endpoint for approval.
Mesoblast contracted Parent Project Muscular Dystrophy (PPMD) & the Duchenne Registry to support patient identification & trial awareness efforts.
Mesoblast aims to utilize Ryoncil®'s anti-inflammatory mechanism to reduce the inflammatory cascade in DMD, preserve muscle function, & slow disease progression.
This press release is a correction to a version dated April 7, 2026, clarifying that the announcement does not imply an endorsement by PPMD.