REGENXBIO Inc. (RGNX) on Thursday announced positive topline and interim functional data from the pivotal Phase III AFFINITY DUCHENNE trial of RGX-202, the company's gene therapy candidate for Duchenne Muscular Dystrophy.
The trial evaluated RGX-202 in 31 ambulatory boys aged 1 year of age and older. Key topline interim results include safety, biomarker, and functional data.
The trial met its primary endpoint, with 93% of patients achieving at least 10% microdystrophin expression at Week 12. The company also said RGX-202 showed a statistically significant correlation between microdystrophin expression and functional improvement, supporting validity of surrogate endpoint.
RGX-202 was generally well tolerated, with no new safety concerns identified.
Interim results from 9 participants aged about 5 to 12 years showed improvements in functional measures, including the North Star Ambulatory Assessment and timed function tests, one year after treatment.
Following the positive data, REGENXBIO Inc. said it plans to seek accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027.
The company added that it is finalizing the design of an ex-U.S. study to support global regulatory submissions for RGX-202.
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