REGENXBIO Eyes Q2 RGX-202 Data and $100M AbbVie Milestone
RGX-202 showed a 7.4-point mean functional improvement versus CTAP at 18 months with no serious adverse events, and pivotal AFFINITY trial top-line data are expected early Q2 2026 ahead of an accelerated BLA. The AbbVie partnership’s NAVIGATE DR dosing will trigger a $100 million milestone, extending cash runway into early 2027.
1. Phase I/II RGX-202 Functional Data
Phase I/II data for RGX-202 in Duchenne muscular dystrophy showed an average 7.4-point functional improvement versus a CTAP model at 18 months, with no serious adverse events reported in patients aged eight and older.
2. AFFINITY Trial Timeline and BLA Pathway
Dosing in the pivotal AFFINITY DUCHENNE trial completed in October 2025, with top-line data expected in early Q2 2026; management plans mid-year FDA engagement to pursue an accelerated BLA submission based on external control comparisons and functional outcomes.
3. AbbVie Partnership and Milestones
Under the AbbVie partnership, REGENXBIO anticipates first patient dosing in the NAVIGATE diabetic retinopathy trial next quarter, which will trigger a $100 million milestone payment, and expects wet AMD pivotal results in Q4 2026.
4. Financial Position and Regulatory Outlook
The company reported $241 million in cash at year-end 2025, supporting operations into early 2027, and is preparing a response to a complete response letter and clinical holds for its MPS gene therapy programs while scheduling FDA Type A meetings to de-risk submissions.