REGENXBIO Inc. announced positive safety and effectiveness data from the Phase I/II portion of the AFFINITY DUCHENNE® study, and that the study has moved into Phase 3, or “pivotal” phase, which is usually the last phase before submitting a product for approval. RGX-202 is a gene therapy that uses an adeno-associated viral (AAV; a virus that does not cause disease and acts like a package for the healthy gene) vector to deliver a transgene (a manufactured version of a piece of DNA) to deliver a novel microdystrophin which is also in naturally-occurring dystrophin. RGX-202 is a one-time treatment designed to specifically address underlying cause of Duchenne muscular dystrophy, allowing a person with DMD to produce microdystrophin, and prevent damage to skeletal and heart muscles.
Positive functional results from the first five participants in the Phase I/II portion of the ongoing AFFINITY DUCHENNE trial include 12-month data from three dose level 1 patients aged 4-10 years and nine-month data from two dose level 2 patients aged 8 and 12 years. In all five participants, across both dose levels, RGX-202 demonstrated improved function on the North Star Ambulatory Assessment (NSAA) and timed function tests, which are tests commonly used to see how much the person’s DMD has progressed. The higher dose will be used for the Phase 3 trial. The participants who were on the higher pivotal dose demonstrated improved performance on NSAA and timed function tests at nine months when compared to external natural history controls who were not treated.
“I am very pleased to see the advancement of the RGX-202 program to the pivotal stage, which offers promise for a broader patient population and am highly encouraged by the functional data presented today demonstrating RGX-202’s potential to alter the course of the disease. The safety, functional, and biomarker data shared today reinforce the positive feedback from families, highlighting improvements in patients’ daily activities and underscoring the potential benefits of this treatment.”
Aravindhan Veerapandiyan M.D., Arkansas Children’s Hospital
Recently, REGENXBIO announced that their AFFINITY DUCHENNE trial would be opening a Canadian centres in the first half of 2025. For any families who would like more information about REGENXBIO’s Canadian trial, please reach out to [email protected].
See the full press release and community letter below.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: