REGENXBIO INITIATES PIVOTAL PHASE OF AFFINITY DUCHENNE® TRIAL OF RGX-202 GENE THERAPY AND REPORTS POSITIVE FUNCTIONAL DATA

In This Article:

  • Alignment achieved with FDA on AFFINITY DUCHENNE® pivotal program and access to accelerated approval; BLA expected in 2026

  • Pivotal trial of RGX-202 is enrolling ambulatory patients aged 1 and above with first patient dosed

  • Phase I/II data show RGX-202 recipients exceeding external natural history and established benchmarks for clinical outcomes

    • Functional improvements seen in all patients treated with dose level 1 and dose level 2 at 12 and 9 months respectively

    • New biomarker data confirms consistent robust expression of differentiated RGX-202 microdystrophin in the muscle

    • Favorable safety profile observed at both dose levels; no serious adverse events or AEs of special interest 

  • Webcast to be held at 8:00 a.m. today

ROCKVILLE, Md., Nov. 18, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that AFFINITY DUCHENNE®, the multi-center, open-label trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne), has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data.

"The initiation of our pivotal trial and newly released positive functional data are exciting milestones on our path to rapidly deliver RGX-202, the only next generation gene therapy in pivotal phase, to the Duchenne community," said Curran M. Simpson President and Chief Executive Officer of REGENXBIO. "The totality of our data demonstrates that RGX-202 provides evidence of improving outcomes for boys with Duchenne and altering the trajectory of this devastating disease, with consistent, robust expression of our novel microdystrophin translating into significant clinical benefit. Based on the strength of the Phase I/II data and our positive discussions and alignment with the FDA, we are quickly advancing RGX-202 toward a BLA filing in 2026 using the accelerated approval pathway. We continue to evaluate opportunities to expand the RGX-202 program to benefit Duchenne patients worldwide."

"There remains a critical need for new therapeutic options for patients with Duchenne muscular dystrophy", said Aravindhan Veerapandiyan M.D., Arkansas Children's Hospital. "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."

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