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REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202
Robust microdystrophin expression observed in new data from pivotal dosePatients aged 5.8 and 8.5 years at dosing had expression levels at 77.2% and 46.5% of
About this update from Regenxbio Inc.
[{"type":"text","content":"Robust microdystrophin expression observed in new data from pivotal dosePatients aged 5.8 and 8.5 years at dosing had expression levels at 77.2% and 46.5% of control, respectivelyConsistent high expression of microdystrophin across treated patients in all age groups continues to support plans for accelerated approval Expects to initiate pivotal trial in Q4 2024 ROCKVILLE, Md., Aug. 1, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) ages 1 to 11 years old.\nRGX-202 is an investigational one-time AAV Therapeutic designed to deliver a novel microdystrophin gene that includes key regions of the naturally occurring dystrophin gene. RGX-202 is the only gene therapy approved or in development for Duchenne that encodes for the C-Terminal (CT) domain to produce a microdystrophin that is closer to naturally occurring dystrophin. In preclinical studies, the CT domain has been shown to protect the muscle from contraction-induced stress and improve its ability to repair itself.\n\"With today's new, positive data, we are seeing a clear dose response and consistent, robust microdystrophin expression levels across all treated patients in the AFFINITY DUCHENNE trial, and, notably, among the highest levels of microdystrophin expression reported in older ambulatory patients,\" said Curran M. Simpson, President and CEO, REGENXBIO. \"This data continues to build on the totality of evidence supporting the potential for RGX-202 to be a differentiated, best-in-class treatment for Duchenne. RGX-202 is well positioned to be the next potential gene therapy approved for Duchenne, and, with our commercial-ready, suspension-based manufacturing platform process, we believe we have the capacity to serve the entire market.\"\n\"I remain encouraged by the biomarker data from the AFFINITY DUCHENNE trial of RGX-202 and am eagerly anticipating the initial functional data from this program,\" said Aravindhan Veerapandiyan, M.D., Arkansas Children's Hospital. \"This update is also encouraging for the Duchenne community, which is exploring various treatment options that could influence disease progression.\"\nData UpdateIn patients aged 5.8 and 8.5 who received RGX-202 at dose level 2, RGX-202 mi...