Atsena Therapeutics Reports Positive 18-Month Results for ATSN-201 in X-Linked Retinoschisis Trial

DURHAM, N.C., Sept. 30, 2026 /CourierPR/ -- Atsena Therapeutics, a clinical-stage gene therapy company focused on reversing or preventing blindness from inherited retinal diseases, has announced positive 18-month results from Part A of the Phase 1/2/3 LIGHTHOUSE Trial evaluating ATSN-201 in patients with X-linked retinoschisis (XLRS). The findings, presented at the American Academy of Optometry 2026 Annual Meeting, held from September 30 to October 3 in Anaheim, California, reinforce the favorable safety profile and durable structural and functional benefits of ATSN-201.

The LIGHTHOUSE Trial, which consists of three parts (A, B, and C), aims to evaluate the efficacy and safety of ATSN-201 in patients with XLRS. Part A, completed in 2025, involved nine adult patients, with each receiving a subretinal injection of ATSN-201 at one of three different dose concentrations. The results showed that ATSN-201 maintained foveal schisis closure in seven of the nine treated eyes, a significant improvement not observed in untreated eyes. Additionally, the treated eyes demonstrated statistically significant improvements in central retinal thickness, microperimetry, best-corrected visual acuity (BCVA), and low-luminance visual acuity (LLVA). Six of the nine treated eyes achieved a 7 dB or greater improvement in microperimetry, and seven of the nine treated eyes showed visual acuity improvements of 10 letters or more at 18 months, compared to only one of the nine untreated eyes.

Enrollment is ongoing for Part C of the LIGHTHOUSE Trial, the pivotal Phase 3 portion, which is expected to complete by the end of the first quarter of 2027. Topline results are anticipated in the first half of 2028, and a Biologics License Application (BLA) filing is targeted for the second half of 2028. The pivotal cohort began dosing in June 2026 and is enrolling ahead of expectations at sites in the U.S. and the United Kingdom, with additional sites in Europe and Canada expected to open later this year. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).

X-linked retinoschisis (XLRS) is a monogenic X-linked disease caused by mutations in the RS1 gene, which encodes retinoschisin (RS1). XLRS is characterized by schisis, or abnormal splitting of retinal layers, leading to impaired visual acuity that is not correctable with glasses and often resulting in progressive vision loss and blindness. Approximately 30,000 males in North America and Europe have XLRS, for which there are currently no approved, disease-specific treatments.

Atsena is also developing ATSN-101, an investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. ATSN-101 has completed a Phase 1/2 trial, and Atsena expects to initiate a global pivotal Phase 3 clinical trial evaluating ATSN-101 in the coming months.