Atsena Therapeutics announced in a press release that the first patient has been dosed in the phase 3 pivotal cohort of the LIGHTHOUSE trial evaluating ATSN-201 for the treatment of X-linked retinoschisis (XLRS).

Since beginning enrollment in May, Atsena said it has enrolled 10% of the study and expects rapid continued enrollment as additional planned sites are activated across North America and Europe. The company also said it expects to complete enrollment by the end of the first quarter of 2027, with topline results anticipated in the first half of 2028 and a biologics license application filing targeted for the second half of 2028.

According to the company, the phase 3 pivotal portion of the LIGHTHOUSE trial is enrolling 76 patients with XLRS, including both adults and children as young as age 6. Patients are randomized approximately 1:1 between a treatment arm, which receives ATSN-201, and a control arm, which is observed for 12 months and then offered the option to receive treatment. The primary endpoint is change in microperimetry at 52 weeks, as aligned with both the US Food and Drug Administration (FDA) and the European Medicines Agency. Key secondary endpoints include visual acuity and optical coherence tomography-based measures of retinal structure. RP