MESO · Mesoblast Limited

Mesoblast Limited announced a significant regulatory advancement with the FDA granting Investigational New Drug (IND) clearance to directly proceed to a registrational clinical trial for Ryoncil® in Duchenne Muscular Dystrophy (DMD). This clearance allows the company to bypass earlier-phase studies, leveraging existing preclinical data in DMD animal models and the established safety profile of Ryoncil® in children with steroid-refractory acute graft-versus-host disease (SR-aGvHD). Ryoncil® is noted as the first mesenchymal stromal cell (MSC) product approved by the FDA.

The registrational trial is designed to randomize 76 patients aged 5 to 9 years to receive either Ryoncil® or placebo on top of standard of care. The dosing regimen consists of 7 infusions of 2 x 10^6 cells/kg over 9 months. The trial's primary endpoint is time-to-stand at nine months, which is described as a validated FDA endpoint for approval. The therapeutic approach aims to leverage Ryoncil's® anti-inflammatory mechanism to address the chronic inflammation characteristic of DMD, potentially preserving muscle tissue and altering disease trajectory.

To support trial execution, Mesoblast is collaborating with Parent Project Muscular Dystrophy (PPMD) to facilitate patient identification and awareness. DMD affects approximately 15,000 individuals in the United States. Silviu Itescu, Chief Executive of Mesoblast, highlighted the potential for a unique approach to treating this devastating disease based on the company's experience with Ryoncil®.