RegenxBio completes dosing in RGX-202 confirmatory Duchenne study

RegenxBio (NASDAQ: RGNX) announced it has completed dosing in the confirmatory study of RGX-202, a gene therapy candidate for Duchenne muscular dystrophy, according to a press release dated June 24, 2026.

The company said the confirmatory study finished ahead of schedule. RegenxBio plans to initiate a Biologics License Application (BLA) submission under the FDA's accelerated approval pathway in the third quarter of 2026, targeting a potential FDA approval in the second half of 2027.

The planned BLA will draw on safety data from 63 participants across the pivotal and confirmatory portions of the AFFINITY DUCHENNE study, along with efficacy data from 30 pivotal study participants. The submission is expected to include 12-month functional data for at least half of the pivotal study participants.

In the pivotal dataset, RGX-202 met its primary endpoint, with more than 93% of patients achieving 10% microdystrophin expression at week 12. Among nine patients who reached 12-month assessments, functional data showed improvement across multiple timed function tests and the North Star Ambulatory Assessment. The company also reported a correlation between microdystrophin expression at week 12 and functional improvement at one year.

"Completing the enrollment of our confirmatory study ahead of schedule is a significant milestone that underscores the urgent unmet need in Duchenne," said Curran Simpson, President and Chief Executive Officer of RegenxBio.

RegenxBio said it is preparing for a potential commercial launch using in-house manufacturing at its facility in Rockville, Md., where production intended for commercial supply began last year.

Duchenne muscular dystrophy is a degenerative muscle disease affecting an estimated 1 in 3,500 to 5,000 boys born each year worldwide.

You May Also Be Interested In

• Ashland said to explore sale amid takeover interest - Bloomberg
• FDA approves Replimune melanoma therapy in combo with nivolumab
• IM Cannabis closes $250,000 convertible note deal with institutional investor