Roche announces EMBARK trial in DMD did not reach primary endpoint

Roche (RHHBY) announced topline results from the global, randomized, double-blind Phase 3 EMBARK study of Elevidys in ambulatory boys with Duchenne muscular dystrophy, or DMD, aged 4-7 years. In the study, Elevidys-treated patients improved 2.6 points on their NSAA total score 52 weeks after treatment, compared to 1.9 points in placebo-treated patients. In all pre-specified, timed functional key secondary endpoints, time to rise from floor and 10 metre walk test, clinically meaningful and statistically significant improvements were observed. Both endpoints are prognostic factors for disease progression and loss of ability to walk. Additionally, a clinically meaningful and statistically significant improvement was also observed for the pre-specified secondary endpoint stride velocity 95th centile. This novel digital endpoint, qualified by the European Medicines Agency, measures speed of walking via a wearable device. The time to ascend 4-steps secondary endpoint also demonstrated consistent treatment benefit in favour of Elevidys. All data are being further analysed and will be discussed with health authorities to determine the path forward. Detailed results from the EMBARK study will be shared at an upcoming scientific congress and a medical journal publication will be pursued. “High unmet need remains in Duchenne and we are encouraged by the consistent and meaningful results seen in all key secondary functional endpoints for Elevidys, an innovative gene therapy,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. “We will work to further analyse the EMBARK results and consult with health authorities as quickly as possible. We sincerely thank all the boys, their families and the wider Duchenne community involved in this important research effort.” As part of a collaboration agreement Roche is working with Sarepta Therapeutics (SRPT) “to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function, keeping them stronger for longer. Sarepta is responsible for managing regulatory approval and the commercialisation of Elevidys in the US. Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world. Sarepta is responsible for the manufacturing of Elevidys and together, the companies are working on a comprehensive joint clinical development plan to maximise the chances of broad approval and access,” Roche stated.