EMBARK Trial Results - Positive topline results from year two of the EMBARK trial show statistically significant and clinically meaningful improvements in motor function measures (NSAA, TTR, 10MWR) for individuals treated with Elevidys compared to an external control group [1] - Functional differences between Elevidys-treated individuals and the external control group increased between one and two years after treatment, demonstrating sustained benefits [1][6] - Detailed year two results will be shared at an upcoming medical meeting and discussed with health authorities [2] Functional Improvements - Individuals treated in part one of EMBARK (n=63) showed sustained improvements in NSAA (+2.88 points, P<0.0001), TTR (-2.06 seconds, P<0.0033), and 10MWR (-1.36 seconds, P<0.0028) two years after treatment [4] - The crossover treated group (n=59) experienced similar improvements 52 weeks after treatment, despite being one year older than those treated in part one [4] - Muscle biopsies from a subset of patients showed consistent and sustained expression of micro-dystrophin 64 weeks after dosing, with minimal progression in underlying muscle pathology [7] Safety and Regulatory Status - No new safety signals were observed, reinforcing the consistent and manageable safety profile of Elevidys [6][8] - Elevidys is approved for individuals aged four years and older in the US, UAE, Qatar, Kuwait, Bahrain, and Oman, and for ambulatory individuals aged four through seven years in Brazil and Israel [9] - Regulatory filings have been submitted to the EMA and authorities in Japan, Switzerland, Singapore, Hong Kong, and Saudi Arabia [9] Clinical Development Program - EMBARK is a multinational, phase III, randomised, double-blind, two-part crossover, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged four to seven years [12] - The clinical development program includes multiple studies evaluating the safety, efficacy, and long-term effects of Elevidys in various age groups and disease stages [13] About Elevidys and Duchenne Muscular Dystrophy - Elevidys is the first approved disease-modifying gene therapy for Duchenne, designed to address the underlying cause through targeted skeletal, respiratory, and cardiac muscle expression of shortened dystrophin [16] - Duchenne is a rare, genetic, muscle-wasting disease affecting approximately 1 in 5,000 boys worldwide, leading to loss of mobility, respiratory and cardiac function, and a mean life expectancy of 28 years [17][18] Roche's Neuroscience Focus - Neuroscience is a major focus of Roche's research and development, with a goal to develop new treatments for chronic and potentially devastating diseases [19] - Roche is investigating more than a dozen medicines for neurological disorders, including Duchenne muscular dystrophy, multiple sclerosis, and Alzheimer's disease [20]
Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD)