Regenxbio Inc

REGENXBIO Inc. announced positive three-year long-term follow-up data from the Phase II ALTITUDE study of investigational surabgene lomparvovec, also known as sura-vec or ABBV-RGX-314, in non-proliferative diabetic retinopathy using suprachoroidal delivery, presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles. In data as of August 17, 2026, 60% of all Dose Level 3 participants with three-year visits, or 6 of 10, achieved a greater than 2-step improvement on the Diabetic Retinopathy Severity Scale without additional treatment for diabetic retinopathy, and these participants experienced no vision-threatening events. Additionally, the majority of participants, 3 of 4, who achieved a 1-step DRSS improvement at one year without supplemental anti-VEGF injections went on to achieve a greater than 2-step DRSS improvement by three years without additional treatment. No new sura-vec-related safety signals and no intraocular inflammation were observed through three years in 17 participants receiving short-course prophylactic topical steroids. Dose Level 3 is being evaluated in the Phase IIb/III NAAVIGATE trial of sura-vec in NPDR, and REGENXBIO is developing sura-vec in collaboration with AbbVie.

Regenxbio ended the second quarter of 2026 with cash, equivalents, and marketable securities of $106 million, and on a pro forma basis more than $310 million after receiving a $100 million milestone payment from AbbVie and approximately $108 million in net proceeds from a follow-on public offering, extending its cash runway into the fourth quarter of 2027. The company completed enrollment in the confirmatory study for RGX-202 ahead of schedule, with over 60 patients in pivotal and confirmatory trials, and the FDA confirmed that available data for RGX-121 is sufficient for review under accelerated approval with no additional studies required for BLA resubmission. Long-term data for sura-vec in wet AMD and diabetic retinopathy showed durable efficacy and safety, and the Phase 2B/3 NAVIGATE study for diabetic retinopathy was initiated. Top-line data for sura-vec in wet AMD is expected in the fourth quarter of 2026, while the BLA submission for RGX-202 is a multi-module process with the clinical module not expected to be complete until the first quarter of 2027. The US patent on Zolgensma has expired, reducing future royalty revenue from that product in the US, though coverage remains in about 20 countries outside the US and on Invisma worldwide.

REGENXBIO announced positive long-term data for its investigational gene therapy surabgene lomparvovec in wet age-related macular degeneration and diabetic retinopathy at the American Society of Retina Specialists annual meeting. In a Phase I/IIa trial, wet AMD patients who had required frequent anti-VEGF injections prior to a single subretinal dose of sura-vec showed stable to improved vision through five years, with no drug-related intraocular inflammation observed in a setting of no prophylactic steroids. In a separate Phase II ALTITUDE trial, a single suprachoroidal administration of sura-vec at the dose being evaluated in the ongoing Phase IIb/III NAAVIGATE trial prevented vision-threatening events without any additional treatment in the majority of non-proliferative diabetic retinopathy participants at 2.5 years, and over half achieved a greater than two-step improvement on the Diabetic Retinopathy Severity Scale; no intraocular inflammation was observed with short-course prophylactic topical steroids. Topline data from the pivotal ATMOSPHERE and ASCENT trials in wet AMD are expected in the fourth quarter of 2026.

Regenxbio shares rose 16.3% after the company announced alignment with the FDA on the next regulatory steps for the potential accelerated approval of Navsunli, a one-time investigational gene therapy for Mucopolysaccharidosis II, also known as Hunter syndrome. The FDA acknowledged that existing clinical data are sufficient to support accelerated approval and asked the company to request a Type A meeting to review longer-term biomarker and clinical data. Regenxbio plans to resubmit the biologics license application in the third quarter of 2026, with the Type A meeting expected in July. The development follows a complete response letter from the FDA in February 2026 and the lifting of a clinical hold last month. Year to date, Regenxbio shares have declined 37%, compared with a 1.1% decrease for the industry.

Several biotech stocks posted sharp gains Monday, led by Tenon Medical after an S-1 filing, Nuvectis Pharma on a licensing deal with Haisco, and REGENXBIO on a regulatory reversal for its Hunter syndrome gene therapy. Tenon filed to offer up to 1.81 million shares and 6.98 million warrants at $0.6021 per unit, with shares closing up 77.57% at $0.62. Nuvectis announced exclusive ex-China rights to two clinical-stage compounds, NXP100 and NXP200, from Haisco Pharmaceutical Group, sending its stock up 19.61% to $16.53. REGENXBIO gained 16.28% to $9.07 after the FDA agreed that existing data for NAVSUNLI is sufficient for an accelerated approval resubmission, with a Type A meeting set for next month and BLA resubmission expected in the third quarter of 2026. Other notable movers included Azitra, up 33.95% after a shareholder letter outlined a 2026 roadmap for its cosmetic ingredient program ATR-COSF, and Apollomics and Faeth Therapeutics, both rising over 30% on no specific news.

Apogee Therapeutics shares are soaring on news that AbbVie is closing in on a nearly $11 billion deal to buy the inflammatory disease drug developer, according to a Financial Times report. Separately, Regenxbio shares are moving after the company aligned with the FDA on next steps for a potential accelerated approval of Navsunli, a gene therapy for Mucopolysaccharidosis II, with the FDA confirming no additional studies are needed. SpaceX shares are lower this morning, continuing their slide after a record IPO.

Computers and biotechnology stocks led sector gains on Wednesday. Computers shares rose about 5.6% as a group, with AstroNova surging about 70.2% and Nano Dimension Ltd up about 9.8%. Biotechnology shares advanced about 4.5%, driven by uniQure's roughly 74.1% jump and REGENXBIO's approximately 22.5% increase.