Mesoblast Limited (MESO), a commercial-stage biotechnology firm, announced that the United States Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance to directly proceed with a registrational clinical trial evaluating Ryoncil (Remestemcel-L-rknd) in Duchenne muscular dystrophy.
Duchenne Muscular Dystrophy (DMD) is a genetic disease in which a person's muscles gradually weaken and become damaged over time. Mesoblast estimates DMD affects approximately 15,000 children in the U.S.
Ryoncil is the first mesenchymal stromal cell (MSC) product approved by the FDA, and the only product approved for children under age 12 with steroid-refractory acute graft-versus-host disease (SR-aGvHD).
Leveraging Ryoncil's existing anti-inflammatory mechanism in SR-aGvHD, Mesoblast aims to reduce the inflammatory cascade characteristic of DMD, preserve muscle function, and slow disease progression.
The approved trial will randomise 76 patients aged 5 to 9 years to either Ryoncil with 7 infusions of 2 × 10^6 cells/kg over 9 months or placebo, in addition to standard of care. The trial's primary endpoint will be time-to-stand at nine months, a validated FDA endpoint for approval.
Mesoblast is collaborating with Parent Project Muscular Dystrophy (PPMD) to foster patient identification and trial awareness through proactive community engagement.
"We are very pleased to have received clearance to proceed directly to a registrational study for DMD", said Silviu Itescu, Chief Executive of Mesoblast.
The FDA approved Ryoncil for SR-aGvHD for pediatric patients aged 2 months and older in December 2024, and Mesoblast launched Ryoncil for purchase in the United States in late March 2025.
In the first year of launch, Ryoncil is nearing $100 million in revenue, as noted by the firm in recent reports.
MESO has traded between $9.66 and $21.50 in the last year. The stock closed Tuesday's trade at $13.69, down 7.97%.
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