Regenxbio Delays RGX-202 BLA to First Quarter 2027

The Tip Desk · November 11, 2026 · 3 min read

The gene-therapy company booked a $38.0 million Sarepta settlement gain as ZOLGENSMA royalty revenue declined following U.S. patent expirations and cash reached $251.0 million.

REGENXBIO Inc. (RGNX) is now on track to submit a biologics license application for RGX-202 in the first quarter of 2027, after previously saying it would initiate the filing in the third quarter of 2026. The company said it expects a pre-BLA meeting with the FDA in the fourth quarter of 2026 and remains on track for potential accelerated approval in the second half of 2027.

The Rockville, Maryland, biotechnology company also reported that revenues for the three months ended September 30, 2026, fell to $9.2 million from $29.7 million a year earlier. The decline was primarily attributable to ZOLGENSMA royalty revenues, which decreased by $21.2 million after licensed patents in the United States expired in January 2026. The drop was partially offset by $2.8 million of ITVISMA royalties after Novartis launched U.S. sales of the product in the first quarter of 2026. License and royalty revenue was $5.2 million, and service revenue was $3.9 million.

Net loss narrowed to $40.7 million, or $0.62 a share, from $61.9 million, or $1.20 a share, a year earlier. The company recorded a $38.0 million gain on a litigation settlement with Sarepta Therapeutics to resolve all patent litigation matters between the parties. The gain consisted of a $39.0 million settlement payment due from Sarepta, net of proceeds payable to certain licensors. Sarepta paid that $39.0 million in October 2026.

Research and development expenses were $61.0 million, compared with $56.1 million a year earlier, which the company said was primarily attributable to clinical trial expenses for RGX-202. General and administrative expenses were $20.6 million, consistent with $20.3 million in the year-ago quarter.

Cash, cash equivalents and marketable securities were $251.0 million as of September 30, 2026, compared with $240.9 million as of December 31, 2025. The company said the increase was primarily attributable to a $100.0 million milestone payment from AbbVie in July 2026 for dosing the first patient in the Phase IIb/III NAAVIGATE study and net proceeds of $107.8 million from an underwritten public offering completed in July 2026, partially offset by cash used to fund operating activities during the nine months ended September 30, 2026. The September 30 cash balance excludes the $39.0 million Sarepta payment received in October.

REGENXBIO said that balance, together with the October Sarepta proceeds, is sufficient to fund operations into late fourth quarter 2027, based on current operational plans and excluding potential milestone payments, partner receipts, or additional funding.

On RGX-202, the company continues to manufacture intended commercial supply at its in-house Manufacturing Innovation Center. It expects to initiate AFFINITY RISE, an ex-U.S. randomized, placebo-controlled study to support global regulatory submissions, in the first half of 2027.

For surabgene lomparvovec, developed with AbbVie, the company expects to announce topline data from the ATMOSPHERE and ASCENT pivotal trials of subretinal wet AMD in the fourth quarter of 2026, with global regulatory submissions expected in 2027. Long-term ALTITUDE follow-up presented in September 2026 showed a durable safety and efficacy profile through three years at the dose being evaluated in NAAVIGATE. NAAVIGATE, a Phase IIb/III study in non-proliferative diabetic retinopathy, is enrolling.

In August 2026, the FDA placed a clinical hold on RGX-121 for MPS II after asymptomatic spine MRI findings in five participants in the CAMPSIITE study. That reversed the first-quarter lift of the hold and the second-quarter plan for a third-quarter resubmission after a July Type A meeting. The company said it continues to collect and evaluate additional patient imaging and long-term follow-up data.