REGENXBIO STARTS PIVOTAL PHASE OF DUCHENNE TRIAL, 'POSITIVE FUNCTIONAL DATA'

 

• 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

Webcast Details
REGENXBIO will host a webcast featuring Dr. Veerapandiyan, and Michael Kelly, PhD, Chief Scientific Officer of CureDuchenne, to discuss today's developments at 8:00 a.m. EST.

The live webcast can be accessed here and in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

https://finance.yahoo.com/news/regenxbio-initiates-pivotal-phase-affinity-120500075.html