ASRS 2026: Durable Retina Therapies Take Center Stage as Gene Therapy, Implant and Oral Data Mature
核心洞察
At ASRS 2026 in Montreal, durable treatment strategies for wet AMD dominated research, with gene therapy, intravitreal implant and optogenetic data presented across four days.
Ocular Therapeutix reported phase 3 SOL-1 results showing a single AXPAXLI injection maintained BCVA in 74.1% of patients at Week 36 versus 55.8% with monthly aflibercept.
Nanoscope Therapeutics (搜索) presented 3-year REMAIN data showing durable BCVA gains of about 3 ETDRS lines with MCO-010 in retinitis pigmentosa (搜索), alongside a rolling BLA submission.
Durable treatment strategies for wet age-related macular degeneration (搜索) (AMD) emerged as the dominant research theme at the 2026 annual meeting of the American Society of Retina Specialists (ASRS), held in Montreal, Canada, where investigators presented late-stage data on gene therapies, an intravitreal implant and an oral agent aimed at reducing treatment burden beyond standard anti-VEGF injections.
The four-day meeting's research agenda centered on whether newer approaches can maintain outcomes comparable to anti-VEGF injections with fewer treatments. Three programs anchored that discussion: 4D Molecular Therapeutics (4DMT) with 2-year phase 2b data from the PRISM trial of 4D-150; Ocular Therapeutix with phase 3 safety and efficacy data from the SOL-1 trial of AXPAXLI; and RegenxBio, in partnership with AbbVie, with 2.5-year follow-up phase 2 data from the ALTITUDE trial of ABBV-RGX-314.
SOL-1: Single Implant Versus Monthly Aflibercept
Ocular Therapeutix presented the latest efficacy and safety results from SOL-1, a phase 3 trial evaluating OTX-TKI (AXPAXLI), an investigational bioresorbable hydrogel intravitreal implant formulated with anti-angiogenic properties, as a single injection versus aflibercept in newly diagnosed wet AMD patients.
Presented by Dilsher S. Dhoot, MD, of California Retina Consultants, the trial was positioned as the first to demonstrate meaningful durability beyond 9 months from a single AXPAXLI injection, with three key findings emphasized.
On the primary endpoint, a single OTX-TKI injection significantly outperformed monthly aflibercept, with 74.1% versus 55.8% of subjects maintaining BCVA at Week 36 (P = 0.0006). The gap widened by Week 52 (65.9% vs. 44.2%, P < 0.0001).
Time to rescue treatment was also meaningfully delayed: median time to first rescue injection differed by 24 weeks between arms (P = 0.0005), and 74.7% of OTX-TKI patients remained rescue-free through Week 36 versus 56.4% on aflibercept. According to the investigators, this directly supports reduced injection burden versus standard monthly dosing.
The implant showed a clean inflammatory and vasculitis safety profile. No cases of endophthalmitis or occlusive/non-occlusive retinal vasculitis were reported, and intraocular inflammation occurred in only 7 subjects (9 events), all mild-to-moderate and resolved. The most common adverse event, vitreous floaters (12.4% vs. 1.2%), was tied to the hydrogel's drug-elution process rather than an inflammatory reaction.
The company previously released results in February and April, including positive superiority data and an additional readout that Ocular said would serve as the basis of a future planned NDA submission.
Optogenetic Therapy Shows Durable Gains in RP
Nanoscope Therapeutics (搜索) presented two updates at the meeting. The first was data from the first direct-to-phase 2 randomized, controlled trial evaluating an optogenetic therapy, sonpiretigene isteparvovec (son-vec; MCO-010), in a nonhuman primate model for geographic atrophy (搜索) (GA). The therapy is under development as a one-time, in-office intravitreal injection intended to offer durable restoration of vision, and is also under evaluation for Stargardt disease (搜索) and retinitis pigmentosa (搜索) (RP).
That study was conducted in parallel with the phase 2 STARLIGHT study, which evaluated son-vec for Stargardt, and was presented by Jordi Mones, MD, PhD, of the Institut de la Macula and Barcelona Macula Foundation during the "Beyond Atrophy" AMD non-neovascular symposium. Investigators measured safety, expression and functional efficacy of son-vec, created using optical coherence tomography (OCT)-guided scanning green laser micro-irradiation.
Son-vec demonstrated clinically meaningful restoration of vision from previously irreversible photoreceptor loss in inherited macular degeneration, according to the findings. Combined data from STARLIGHT and the phase 2 trial informed the design of two trials: STARGAZE (phase 3) for Stargardt and GENESIS (direct-to-phase 2) for central GA secondary to AMD. Per the abstract, "Collectively, these data establish MCO optogenetics as a disease-agnostic therapeutic platform for severe vision loss from advanced macular degeneration, regardless of underlying pathophysiology."
The second dataset comprised durable 3-year REMAIN follow-up data from the RESTORE phase 2b/3 trial of the optogenetic therapy for RP. A rolling Biologics License Application submission was initiated in July 2025 and is currently underway for this RP indication. Investigators sought to evaluate long-term efficacy and safety of MCO-010 among RP patients in what they described as "the first-ever RCT of optogenetic therapy."
Presented by Benjamin Bakall, MD, PhD, of Associated Retina Consultants, the 3-year outcomes determined that MCO-010 demonstrated "durable, clinically meaningful" BCVA improvements of approximately 3 Early Treatment Diabetic Retinopathy Study (ETDRS) lines with "favorable safety, supporting durable efficacy."
Oral Tinlarebant Meets Primary Endpoint in Stargardt Disease
Belite Bio (搜索) delivered its latest update from the phase 3 DRAGON trial evaluating oral tinlarebant for Stargardt disease (搜索). The once-daily tablet is being developed as a potential first FDA-approved treatment for Stargardt disease type 1 (STGD1), and the company completed its rolling new drug application submission to the FDA last month.
Results were presented by Paul Bernstein, MD, of the Moran Eye Center, University of Utah, during the Hereditary Retinal Disease & Genetics panel. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful 36% reduction in retinal lesion growth versus placebo. A statistically significant treatment effect was also observed in the fellow eye, with a 33.6% lesion growth reduction (p = 0.041).
Real-World Data for Approved GA Therapies
Post-market outcomes featured prominently as well. Astellas presented new real-world data on its FDA-approved and commercially available IZERVAY (avacincaptad pegol), showing similarities in safety and dosing intervals among patients undergoing concomitant anti-VEGF and IZERVAY treatment.
Apellis Pharmaceuticals' SYFOVRE (pegcetacoplan injection), an FDA-approved therapy for geographic atrophy (搜索) recently acquired by Biogen, was the subject of 18-month real-world results as well as new data extending GA benefit to patients with concurrent wet AMD. The meeting marked Biogen's first ASRS with SYFOVRE under its ownership, following its $5.6 billion purchase of Apellis earlier this year.
Surgical Debates and Meeting Themes
Beyond pharmacotherapy, the meeting opened with a programming agenda emphasizing one key message: look beyond the retina and examine the whole patient. Sessions included "Retina Surgery Around the World," in which international retina specialists debated uveitic and traumatic detachments, autologous retinal transplants and pediatric trauma from the standpoint of adapting techniques to surgical restraints, and "Circus of Complications," a new session featuring the younger generation of surgeons discussing clinical practice complications, including cutter incarceration, suprachoroidal cannula displacement and anesthesia-block globe perforation.
Additional research themes included an ongoing macular hole "arms race," in which advanced techniques carry broadly similar success rates, leaving the choice to surgeons' comfort and resource access rather than clear superiority; debate over tamponade positioning, with silicone oil migration viewed as a shared enemy and ongoing discussion over how long to leave perfluorocarbon in place and how to direct perfluorocarbon liquid-to-silicone exchange to avoid air embolism; and a pneumatic retinopexy renaissance, favored for its superior injection capabilities and near-90% success rates, with subretinal gas migration considered a major, prognosis-worsening complication. Investigators also emphasized that inflammation must be controlled prior to operation to improve outcomes in uveitic and traumatic detachments, and that anti-VEGF is considered a hazard in tractional disease.
The Foundation of the ASRS hosted a private reception ahead of the meeting to introduce a new strategic program focused on advancing retina health initiatives with global impact, featuring speakers from the FOCUS Foundation, Small World Vision and the Global Retina Institute discussing the role of humanitarianism and philanthropy in advancing global health outcomes.
The Exhibit Hall featured surgical and diagnostic innovations, including Bausch + Lomb (搜索)'s Stellaris Elite Vision Enhancement System and its Bi-Blade dual-port vitrectomy cutter, which received FDA 510(k) clearance in April 2026; Alcon's UNITY Vitreoretinal Cataract System, cleared in June 2024 and commercially launched in April 2025, and the Valeda Light Delivery System for intermediate dry AMD, authorized by the FDA in November 2024 and acquired by Alcon in September 2025; and ZEISS's ARTEVO 850 3D heads-up ophthalmic microscope. Canadian-based MacoMira, in partnership with Keeler, showcased a non-invasive dry AMD therapy delivering low-level microcurrents through the eyelids in 30-to-40-minute in-office sessions; the device is cleared in Canada and Australia, with potential European Union clearance planned by Q4 2026 and U.S. clinical trial evaluations planned to begin by the end of 2026.