Aldeyra Therapeutics Announces Completion of Enrollment in Phase 2 Clinical Trial of ADX‑2191 in Retinitis Pigmentosa

Aldeyra Therapeutics today announced the completion of enrollment in the Phase 2 clinical trial of ADX‑2191 (methotrexate injection, USP), an investigational drug candidate, for the treatment of retinitis pigmentosa, a group of rare genetic eye diseases characterized by retinal cell death and loss of vision, for which there is no U.S. Food and Drug Administration (FDA)-approved treatment.

The open-label, single-center Phase 2 clinical trial enrolled a total of eight retinitis pigmentosa patients with rhodopsin gene mutations, including the P23H gene mutation. Patients receive either monthly or twice-monthly intravitreal doses of ADX‑2191 for three months. The primary endpoint of the trial is safety. Secondary endpoints include change from baseline in visual acuity; retinal function, as assessed by foveal microperimetry, electroretinography, and dark adaptation; and retinal morphology, as assessed by optical coherence tomography.

“Given the lack of FDA-approved therapies for retinitis pigmentosa, a sight-threatening group of diseases that affect more than one million patients worldwide, novel therapeutic approaches are in demand,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “Aldeyra is committed to working with patients and physicians to attempt to ameliorate the unrelenting burden of retinitis pigmentosa, and we look forward to reporting top-line results of the Phase 2 clinical trial in the first half of this year.”