Switzerland-headquartered pharmaceutical giant Roche (SWX: ROG) has announced that a Phase III trial for its gene transfer therapy Elevidys (delandistrogene moxeparvovec) did not meet its primary endpoint in Duchenne muscular dystrophy (DMD) ambulatory patients aged 4 to 7 years. The trial aimed to measure the change from baseline in motor function at week 52, with the experimental arm showing only a 2.6-point improvement in the north star ambulatory assessment (NSAA) score compared to a 1.9-point increase in the placebo group.
Secondary Endpoints and Future Analysis
Despite not achieving the primary endpoint, the study did meet all secondary endpoints on muscle function across age groups. These results have encouraged Roche to continue its analysis and engage in discussions with health authorities regarding the potential path forward for Elevidys.
Collaboration with Sarepta Therapeutics
The single-dose intravenous infusion of Elevidys was created and is manufactured by Sarepta Therapeutics (NASDAQ: SRPT), which granted Roche an ex-US license for the therapy. Elevidys currently holds an accelerated approval in the US market for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.
Conclusion
The mixed results from the Phase III trial present both challenges and opportunities for Roche and Sarepta Therapeutics. While the primary endpoint was not met, the positive outcomes in secondary endpoints provide a foundation for further investigation and potential regulatory discussions. The future of Elevidys in the treatment landscape for DMD will depend on the insights gained from ongoing analyses and interactions with regulatory bodies.-Fineline Info & Tech
