Shares of REGENXBIO Inc. (RGNX) are up over 11% at $16.27 in premarket trading on Monday, following encouraging new long-term data for its Duchenne program and key catalysts lined up for this year.
The company's investigational gene therapy for Duchenne Muscular Dystrophy, RGX-202, is under a trial, dubbed AFFINITY DUCHENNE. This study is being conducted in three sequential parts: a phase I/II study (Part 1), a phase 3 pivotal study (Part 2) and a confirmatory study (Part 3).
New, positive 18-month functional data from patients treated with the pivotal dose in the phase I/II portion of the AFFINITY DUCHENNE trial were reported yesterday. All patients exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model. cTAP is the controlled natural history disease progression model for Duchenne muscular dystrophy, and it predicts how patients' function would decline over time without treatment.
At 18 months, patients treated with RGX-202 improved an average of 7.4 points compared to cTAP. These same patients improved an average of 6.6 points compared to cTAP at 12 months post-treatment.
The company plans to share additional phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference in March 2026.
The pivotal trial part of the AFFINITY DUCHENNE study is fully enrolled with 30 patients, while enrolment is ongoing in the confirmatory trial portion.
The company expects to report pivotal topline data from the AFFINITY DUCHENNE study in early Q2 2026, with submission of a Biologics License Application (BLA) under the accelerated approval pathway planned for mid-2026.
The other investigational gene therapies in the pipeline are Clemidsogene lanparvovec (RGX-121) for MPS II, also known as Hunter syndrome, and Surabgene lomparvovec (sura-vec, ABBV-RGX-314) for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).
-- Clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II) is under FDA review, with a decision due on February 8, 2026.
Subretinal Sura-vec is under phase 3 pivotal trials for neovascular (wet) age-related macular degeneration (nAMD), dubbed ATMOSPHERE and ASCENT, with topline data anticipated in Q4 2026.
The company is scheduled to present at the J.P Morgan Healthcare Conference on Wednesday, January 14, 2026, at 10:30 a.m. PT.
RGNX has traded in a range of $5.03 to $15.80 in the last 1 year. The stock closed Friday's trading at $14.59, down 2.73%.
For comments and feedback contact: editorial@rttnews.com
Business News
June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.