REGENXBIO, a leading clinical-stage biotechnology company focused on advancing transformative gene therapies, announced in late November that the pivotal phase of the AFFINITY DUCHENNE® study of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has commenced.
The AFFINITY DUCHENNE® Initial trial sites are currently located in the U.S., with additional sites in Canada and Europe expected to follow in the future.
Key Highlights:
- Pivotal Phase Enrolment: The pivotal phase of the AFFINITY DUCHENNE study is now enrolling patients, with the first patient already having been dosed.
- Positive Phase I/II Data: The Phase I/II portion of the study yielded positive safety and efficacy data, including functional improvements in patients based on the North Star Ambulatory Assessment (NSAA) and timed function tests.
- Strong Biomarker Data: New biomarker data confirm high expression and transduction of RGX-202 microdystrophin in muscle tissue, indicating the therapy is reaching its target.
- Adverse Events (AE): As of November 1st, RGX-202 was well tolerated with no serious adverse events (SAEs) or AEs of special interest (AESIs) reported. Common drug-related AEs included nausea, vomiting and fatigue. All resolved and are typically anticipated with gene therapy administration.
- Regulatory Alignment: REGENXBIO has reached alignment with the U.S. Federal Drugs Agency (FDA) on the pivotal program and expects to file a Biologics License Application (BLA) in 2026.
About RGX-202: RGX-202 is a novel gene, one-time gene therapy for the treatment of Duchenne that includes an optimised transgene for a novel microdystrophin. The therapy is designed to deliver a functional microdystrophin protein to muscle cells, potentially improving muscle function and quality of life for patients with Duchenne muscular dystrophy.
AFFINITY DUCHENNE® is a multi-centre, open label research study of RGX-202. The trial is currently in its pivotal phase, with the goal of demonstrating the effectiveness of RGX-202 in treating Duchenne muscular. The pivotal phase of the AFFINITY DUCHENNE® trial is a critical step in the development of RGX-202. This phase will provide the necessary data to support the FDA approval of RGX-202, potentially through the accelerated approval pathway in 2026.
