Clinical TrialMay 14, 2026, 07:21 AM
REGENXBIO RGX-202 Phase III Duchenne trial meets primary endpoint
AI Summary
REGENXBIO announced positive topline results from its pivotal Phase III AFFINITY DUCHENNE® trial of RGX-202, a gene therapy for Duchenne Muscular Dystrophy, achieving its primary endpoint with high statistical significance and demonstrating a correlation between microdystrophin expression and functional improvement. The company is preparing for potential accelerated approval in 2027. For Q1 2026, REGENXBIO reported $6.4 million in revenue and a net loss of $90.1 million, primarily due to a decrease in license revenue and ZOLGENSMA® royalties compared to the prior year. The company also provided updates on surabgene lomparvovec and announced the lifting of the clinical hold for RGX-121.
Key Highlights
- RGX-202 Phase III trial achieved primary endpoint; 93% of patients had microdystrophin expression >10% (p<0.0001).
- RGX-202 microdystrophin expression averaged 71.1% across participants.
- Statistically significant correlation between RGX-202 expression and functional improvement (NSAA).
- Q1 2026 revenue was $6.4 million, down from $89.0 million in Q1 2025.
- Q1 2026 net loss was $90.1 million, compared to net income of $6.1 million in Q1 2025.
- Cash, cash equivalents, and marketable securities totaled $150.5 million as of March 31, 2026.
- R&D expenses increased to $57.3 million in Q1 2026 from $53.1 million in Q1 2025.
- Expected $100 million milestone payment from AbbVie upon first patient dosed in NAAVIGATE study (Q2 2026).