Mesoblast (ASX:MSB) announced that the US Food and Drug Administration has granted investigational new drug clearance to commence a registrational clinical trial for Ryoncil (remestemcel-L).
The study will evaluate the efficacy of the mesenchymal stromal cell therapy in treating Duchenne muscular dystrophy, a debilitating X-linked genetic disorder affecting approximately 15,000 children in the United States.
Characterised by progressive muscle degeneration and chronic inflammation, DMD typically leads to a loss of ambulation and fatal respiratory or cardiac failure by a patient’s third decade.
While current gene therapies focus on replacing dystrophin, Mesoblast intends to target the "inflammatory cascade" that drives disease progression.
By leveraging Ryoncil’s established anti-inflammatory mechanism—already proven safe in paediatric treatments for graft-versus-host disease—researchers aim to preserve muscle tissue and alter the disease's trajectory.
The upcoming trial will enrol 76 children aged 5 to 9, randomising participants to receive either a series of seven Ryoncil infusions or a placebo over nine months.
The primary endpoint is the "time-to-stand" metric, a validated FDA measure of functional mobility.
Mesoblast is partnering with Parent Project Muscular Dystrophy to accelerate patient recruitment.
CEO Silviu Itescu expressed optimism, noting that the trial builds upon robust preclinical data and the company’s extensive experience in paediatric cellular medicines to address the critical unmet needs of those living with this devastating condition.
At the time of reporting, Mesoblast’s share price was $2.01.