Mesoblast Limited announced that the U.S. Food and Drug Administration granted Investigational New Drug clearance to conduct a registrational clinical trial of its allogeneic mesenchymal stromal cell therapy, Ryoncil, in Duchenne muscular dystrophy (DMD). The trial will enroll 76 patients aged 5 to 9 years and randomize them to receive seven infusions of 2 × 10^6 cells per kilogram over nine months or placebo, in addition to standard of care. The primary endpoint is time‑to‑stand at nine months, a validated FDA endpoint for approval.
Ryoncil, also known as remestemcel‑L‑rknd, is currently approved for pediatric patients 2 months and older with steroid‑refractory acute graft‑versus‑host disease. In the first half of fiscal 2026, the product generated $49 million in sales, up from $30.3 million in the March quarter, underscoring strong commercial traction in its existing indication. Mesoblast reported total revenue of $51.3 million for the same period, a decline from the prior year’s $61.8 million, but a net loss narrowed to $40.2 million from $55.6 million, reflecting improved cost control and the impact of the Ryoncil launch.
The DMD trial represents a strategic expansion into a disease affecting roughly 15,000 children in the United States. By leveraging Ryoncil’s anti‑inflammatory mechanism, Mesoblast aims to address the inflammatory component of DMD, potentially opening a substantial new market. The partnership with Parent Project Muscular Dystrophy and the Duchenne Registry will facilitate patient identification and trial awareness, positioning the company to accelerate enrollment and gather robust data.
CEO Silviu Itescu said the IND clearance was a positive step following preclinical and safety data, highlighting the company’s confidence in the platform’s versatility. Principal investigator Dr. Aravindhan Veerapandiyan noted that Ryoncil’s anti‑inflammatory properties could mitigate the muscle degeneration seen in DMD, underscoring the scientific rationale for the study.
Mesoblast also secured a $125 million non‑dilutive credit facility and reported $130 million in cash at the end of December 2025, providing financial flexibility to support the expanded pipeline and ongoing clinical development.
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