REGENXBIO Announces Phase I/II Trial of RGX-202, a Novel Gene Therapy Candidate for Duchenne Muscular Dystrophy

REGENXBIO today announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202 for the treatment of Duchenne muscular dystrophy (Duchenne/ DMD) is now active and recruiting patients. RGX-202 is designed to deliver a transgene for a novel microdystrophin protein that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. RGX-202 uses REGENXBIO’s proprietary NAV® AAV8 vector.

AFFINITY DUCHENNE is a multicenter, open-label dose evaluation and dose expansion clinical trial to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in patients with Duchenne. Additionally, REGENXBIO is recruiting patients in the AFFINITY BEYOND™ trial, an observational screening study. The primary objective is to evaluate the prevalence of AAV8 antibodies in patients with Duchenne up to 12 years of age. Information collected in this study may be used to identify potential participants for the AFFINITY DUCHENNE trial and potential future trials of RGX-202.

“I am pleased that we are now able to initiate the trial for RGX-202 and also begin enrollment activities in our AAV8 antibody screening study,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “The RGX-202 program is a key piece of our ‘5x’25’ strategy to have five AAV Therapeutics either on the market or in late-stage development by 2025. We look forward to continuing to work closely with the Duchenne community as we advance a highly differentiated product candidate developed with the potential to improve muscle strength and motor function in boys with Duchenne.”

“Duchenne muscular dystrophy is a devastating disease and there are still unmet therapeutic needs,” said Aravindhan Veerapandiyan, M.D., a principal investigator in the study and Director of the Comprehensive Neuromuscular Program, PPMD Certified Duchenne Care Center, and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children’s Hospital. “Gene therapies, like RGX-202, have the potential to impact the progressive nature of Duchenne.”

REGENXBIO has manufactured additional clinical supply of RGX-202 in its in-house Manufacturing Innovation Center using the NAVXpress™ process platform. Located in REGENXBIO’s 132,000 square foot headquarters in Rockville, MD, the Manufacturing Innovation Center is designed to meet global clinical and commercial regulatory standards, and includes two independent bulk drug substance production suites, a final drug product suite and integrated quality control labs. REGENXBIO is one of only a few gene therapy companies worldwide with a cGMP facility capable of production at scales up to 2,000 liters.