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Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys (delandistrogene moxeparvovec) as a gene therapy for treating Duchenne muscular dystrophy (DMD).
Roche entered into a worldwide partnership with Sarepta Therapeutics in 2019 to commercialise the gene therapy outside of the US.
Subjects in the trial received one dose of Elevidys or a placebo with a crossover component, allowing for a comparative analysis of the treatment's impact.
Two years post-treatment with the therapy, subjects exhibited statistically significant and clinically meaningful enhancements in Time to Rise (TTR), 10m walk/run (10MWR), and the North Star Ambulatory Assessment (NSAA), compared to a pre-specified, propensity-weighted, untreated external control arm.
The functional gap between the treated individuals and the external control group increased from the first to the second year after dosing.
Roche global product development head and chief medical officer Levi Garraway said: “After two years of treatment with Elevidys, we are seeing multiple sustained benefits in the day-to-day lives of these young boys, all of which are indicators of its disease-modifying potential in Duchenne.
"These results, which include improvements in standing, walking, and running, represent meaningful progress and we plan to share them with health authorities as quickly as possible.”
Subjects from the first part of the trial maintained significant improvements in NSAA scores two years after the therapy’s administration.
Those who initially received a placebo in part one and switched to being treated with Elevidys during the second part of the trial also displayed ‘favourable’ outcomes at 52 weeks post-treatment, aligning with the results from the first cohort despite being a year older.
Muscle biopsies from a subject subset 64 weeks post-dosing in the first part of the trial confirmed ‘consistent’ and ‘sustained’ micro-dystrophin expression.
MRI assessments of muscle pathology showed minimal progression, matching the functional benefits observed. In addition, no new safety concerns were identified, supporting the consistent safety profile of the therapy.
The therapy is currently approved for individuals aged four years and above in Qatar, UAE, Kuwait, Bahrain, Oman, and the US, without restrictions on ambulatory status.
It is also authorised for ambulatory individuals aged four to seven years in Israel and Brazil.
In September 2024, Roche reported positive topline data from the Phase III CENTERSTONE trial of Xofluza (baloxavir marboxil) for influenza infection.