REGENXBIO — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenues were $9.2 million for the three months ended September 30, 2026, a decrease from $29.7 million in the same period of 2025.
• License and royalty revenue decreased to $5.2 million from $23.6 million year-over-year, primarily due to a $21.2 million decline in ZOLGENSMA® royalties following the expiration of U.S. licensed patents in January 2026.
• ITVISMA® royalty revenues contributed $2.8 million in the third quarter of 2026, offsetting some of the ZOLGENSMA® decline.
• Research and development expenses increased to $61.0 million from $56.1 million year-over-year, driven by clinical trial expenses for RGX-202.
• General and administrative expenses were $20.6 million, consistent with $20.3 million in the prior year period.
• Net loss was $40.7 million ($0.62 per share) for the quarter, compared to a net loss of $61.9 million ($1.20 per share) in the prior year period.
• The company recorded a $38.0 million gain on a litigation settlement with Sarepta Therapeutics in the third quarter of 2026.
• Cash, cash equivalents, and marketable securities totaled $251.0 million as of September 30, 2026, up from $240.9 million as of December 31, 2025.
• The increase in cash position was driven by a $100.0 million milestone payment from AbbVie and $107.8 million in net proceeds from a public offering completed in July 2026.
• Deferred revenue decreased to $22.2 million ($5.9 million current, $16.2 million non-current) from $29.4 million ($10.5 million current, $18.9 million non-current) as of December 31, 2025.
• Royalty monetization liabilities increased to $185.7 million ($12.1 million current, $173.6 million non-current) from $187.0 million ($39.6 million current, $147.4 million non-current) as of December 31, 2025.

Guidance and Future Outlook:

• The company expects its current cash balance of $251.0 million, plus $39.0 million in settlement proceeds received in October 2026, to fund operations into late Q4 2027.
• This cash runway guidance excludes potential future milestone payments from partners or licensees and excludes additional funding opportunities.
• Topline data from the pivotal ATMOSPHERE® and ASCENT® studies for sura-vec in wet AMD is expected in Q4 2026.
• A BLA submission for RGX-202 is on track for Q1 2027, with potential accelerated approval in the second half of 2027.
• Global regulatory submissions for sura-vec are expected in 2027.
• The AFFINITY® RISE study for RGX-202 is expected to initiate in the first half of 2027.

Business Segments and Product Lines:

• Neuromuscular Disease: RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy, is being evaluated in the Phase I/II/III AFFINITY DUCHENNE® trial. The company is manufacturing intended commercial supply at its in-house Manufacturing Innovation Center.
• Retinal Disease: Sura-vec (ABBV-RGX-314), developed with AbbVie, is a potential first-in-class gene therapy for wet AMD and diabetic retinopathy. Long-term follow-up data from the ALTITUDE® trial showed durable safety and efficacy through 3 years. The Phase IIb/III NAAVIGATE trial for diabetic retinopathy is ongoing.
• Neurodegenerative Disease: RGX-121 (clemidsogene lanparvovec) for MPS II is under development with Nippon Shinyaku. The FDA placed a clinical hold on RGX-121 in August 2026 following asymptomatic spine MRI findings in five CAMPSIITE® study participants; the company is evaluating additional imaging and follow-up data.
• ITVISMA has launched U.S. sales in Q1 2026 and is now approved in the EU, Japan, and multiple Gulf countries.

Market and Competitive Landscape:

• The company positions RGX-202 as a potential best-in-class therapy and sura-vec as a potential first-in-class treatment for wet AMD.
• The expiration of ZOLGENSMA® patents in the U.S. in January 2026 significantly impacted royalty revenue streams.
• ITVISMA is now approved in multiple jurisdictions beyond the U.S., with patents extending until 2037.

Risks and Challenges:

• A clinical hold remains in place for RGX-121 pending evaluation of MRI findings and long-term follow-up data.
• Future cash runway excludes the impact of potential milestone payments or commercialization revenues from partners.
• Forward-looking statements regarding clinical trial success, regulatory approval timing, and market acceptance are subject to significant risks and uncertainties.

Management Commentary and Tone:

• CEO Curran Simpson stated the company is "continuing to build momentum" to launch potential first- or best-in-class gene therapies in 2027 and 2028.
• Management expressed confidence that sura-vec has the potential to "transform the treatment paradigm" for wet AMD patients.
• The company emphasized its focus on executing near-term milestones and leveraging fully integrated capabilities.

Other Key Points:

• REGENXBIO received a $100.0 million milestone payment from AbbVie in July 2026 for dosing the first patient in the Phase IIb/III NAAVIGATE study.
• The company completed an underwritten public offering of common stock and pre-funded warrants in July 2026, generating $107.8 million in net proceeds.
• A $39.0 million settlement payment from Sarepta Therapeutics was received in October 2026, resolving all patent litigation matters between the parties.
• The company issued 66,119 shares of common stock as of September 30, 2026, compared to 50,892 shares as of December 31, 2025.
• A conference call and webcast were scheduled for November 5, 2026, at 8:00 a.m. ET.