REGENXBIO Unveils 2026 Pipeline Catalysts with Strong 18-Month DMD Trial Data

REGENXBIO Inc. announced new 18-month functional data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 (NCT05693142), its investigational AAV gene therapy for Duchenne muscular dystrophy (DMD), demonstrating durable treatment effects at the pivotal dose. The company also outlined a robust lineup of 2026 milestones across its late-stage pipeline, positioning it for potential commercial launches between 2026 and 2028.

Breakthrough Data from AFFINITY DUCHENNE Trial

Patients treated with the pivotal dose of RGX-202 (n=4) in the Phase I/II trial showed significant improvements on the North Star Ambulatory Assessment (NSAA) compared to the cTAP natural history disease progression model. All participants exceeded expected trajectories, with an average gain of 7.4 points at 18 months and 6.6 points at 12 months post-treatment.

This long-term durability underscores RGX-202’s potential to alter disease progression in ambulatory boys aged 4-7 with DMD. REGENXBIO plans to present comprehensive Phase I/II data including safety, biomarkers, and additional functional metrics at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March 2026.

Pivotal trial enrolment (n=30) completed in October 2025, with robust confirmatory trial recruitment ongoing; the majority of patients are expected to be dosed by mid-2026 BLA filing under accelerated approval.

 Strategic Pipeline Catalysts for 2026

REGENXBIO anticipates pivotal top-line data for RGX-202 in early Q2 2026, enabling a Biologics License Application (BLA) submission mid-year, followed by regulatory interactions with the FDA and EMA in H1 2026.

For RGX-121 (clemidsogene lanparvovec) in MPS II (Hunter syndrome), partnered with Nippon Shinyaku, the FDA PDUFA date is February 8, 2026, which could yield a Priority Review Voucher. Similarly, RGX-111 for MPS I advances under the same partnership.

In retinal diseases, ABBV-RGX-314 (sura-vec) for wet AMD and diabetic retinopathy, in collaboration with AbbVie, targets pivotal readouts from ATMOSPHERE® and ASCENT® trials in Q4 2026, with first patient dosing in diabetic retinopathy triggering a $100 million milestone in H1 2026.

Leadership Perspective and Commercial Readiness

“2026 is set to be a transformative year for REGENXBIO, as we enter commercial stage with two near-term catalysts from our three late-stage assets and a clear path to sustained growth,” stated Curran Simpson, President and CEO. He highlighted the company’s comprehensive strategy maximizing therapeutic benefits, evidenced by the new Duchenne data.

Supporting these launches, REGENXBIO leverages in-house manufacturing at its REGENXBIO Manufacturing Innovation Center in Rockville, Md., and strategic global partnerships for scalability and reliability. President and CEO Curran Simpson will present at the 44th Annual J.P. Morgan Healthcare Conference on January 14, 2026, at 10:30 a.m. PT.

References

REGENXBIO Highlights Key 2026 Catalysts and Announces Positive Long-Term Functional Outcomes in Lead Duchenne Gene Therapy Program, 11 January 2026, REGENXBIO Highlights Key 2026 Catalysts and Announces Positive Long-Term Functional Outcomes in Lead Duchenne Gene Therapy Program | Regenxbio Inc

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants with Duchenne Muscular Dystrophy (DMD), ClinicalTrials.gov ID NCT05693142, https://clinicaltrials.gov/study/NCT05693142

Long Term Follow-up for RGX-202, ClinicalTrials.gov ID NCT06491927. https://clinicaltrials.gov/study/NCT06491927