REGENXBIO reported third-quarter 2026 results alongside updates on its gene-therapy pipeline. The company remains on track to seek U.S. approval next year for its Duchenne muscular dystrophy candidate, while pivotal results for its wet age-related macular degeneration program are expected before year-end.
The quarter’s financial update also reflects a $100 million milestone payment from AbbVie and proceeds from a stock offering. Separately, REGENXBIO said the FDA’s clinical hold on its Hunter syndrome program remains under review as it gathers more patient data.
Key details
- Duchenne program: REGENXBIO expects to meet with the FDA before submitting an application for RGX-202 in the fourth quarter of 2026. It remains on track to submit in the first quarter of 2027, with potential accelerated approval in the second half of 2027. The company is making intended commercial supply at its own manufacturing center.
- The company plans to start an ex-U.S. study, AFFINITY RISE, in the first half of 2027 to support global regulatory submissions. RGX-202 is being studied in the AFFINITY DUCHENNE trial, including a confirmatory study in ambulatory patients aged 1 and older.
- Wet AMD: REGENXBIO and AbbVie expect topline results in the fourth quarter of 2026 from the pivotal ATMOSPHERE and ASCENT studies of sura-vec, delivered under the retina. Global regulatory submissions are expected in 2027.
- Diabetic retinopathy: The Phase IIb/III NAAVIGATE trial of sura-vec, delivered through the suprachoroidal space, is enrolling patients with non-proliferative diabetic retinopathy. The company cited three-year follow-up from an earlier study as showing durable safety and efficacy at the dose being tested in NAAVIGATE.
- Hunter syndrome: The FDA placed RGX-121 on clinical hold in August after asymptomatic findings appeared on spine MRIs in five participants in the CAMPSIITE study. REGENXBIO is continuing to collect and assess imaging and long-term follow-up data.
Financial position
- Cash, cash equivalents and marketable securities totaled $251 million at Sept. 30, up from $240.9 million at year-end 2025. The quarter-end figure does not include a $39 million payment received in October under a settlement with Sarepta Therapeutics.
- The increase from year-end primarily reflected a $100 million AbbVie milestone payment in July, triggered by the first patient dosing in NAAVIGATE, and $107.8 million in net proceeds from a July public offering of common stock and pre-funded warrants. Cash used for operations partly offset those inflows.
- Third-quarter revenue was $9.2 million, compared with $29.7 million a year earlier. The decline was driven mainly by a $21.2 million drop in ZOLGENSMA royalty revenue, largely following the expiration of U.S. licensed patents in January 2026. ITVISMA royalties contributed $2.8 million in the quarter.
The near-term development milestones center on the expected wet AMD trial results and the planned RGX-202 application. In the meantime, the Hunter syndrome program remains on clinical hold, and the company’s reported quarter-end cash balance excludes the later Sarepta settlement payment.