To evaluate the efficacy of ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS), in a Phase 3 pivotal trial.
Approach:
Trial Design: The LIGHTHOUSE trial is enrolling 76 adults and children aged six years and older, randomized 1:1 to receive ATSN-201 or enter a control arm for 12 months.
Endpoints: The primary endpoint is the change in microperimetry at 52 weeks.
Key Findings:
Enrollment began in May and has reached 10% of the planned study population.
Topline results are anticipated in the first half of 2028.
A Biologics License Application filing is targeted for the second half of 2028.
Interpretation:
XLRS is a rare inherited retinal disease with no approved treatments, and ATSN-201 represents a potential therapeutic option.
Limitations:
The study is still ongoing, and results are not yet available.
Conclusion:
Dosing the first patient marks a significant milestone for Atsena and the XLRS community.
