Atsena Reports LIGHTHOUSE data at ARVO - Summary - MDSpire

Atsena Reports LIGHTHOUSE data at ARVO

Atsena Therapeutics announced positive clinical data results from Part A of the LIGHTHOUSE study, a phase 1/2 clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). The data were presented at the 2025 Association for Research in Vision and Ophthalmology meeting in Salt Lake City, Utah.

  • May 1, 2025

  • 3 min

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Objective:

To evaluate the safety and efficacy of ATSN-201 for the treatment of X-linked retinoschisis (XLRS) in a phase 1/2 clinical trial.

Key Findings:
  • ATSN-201 was well tolerated with no serious adverse events related to treatment.
  • Adverse events were primarily related to the surgical procedure and were of Grade 1-2 severity.
  • No dose-limiting toxicities were observed, and no subjects discontinued from the study.
Interpretation:

The preliminary results indicate that ATSN-201 is a promising candidate for treating XLRS, demonstrating good safety and tolerability.

Limitations:
  • The study is still ongoing, and results are preliminary.
  • Part A involved a small sample size of only 9 adult patients.
Conclusion:

Enrollment is underway for Part B of the study, which will further assess safety and efficacy in a larger cohort, including pediatric patients.

Original Source(s)

Atsena Reports LIGHTHOUSE data at ARVO

  • May 1, 2025

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