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

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) 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.”

About ADX-2191

ADX-2191 is a novel intravitreal formulation of methotrexate, which in preclinical models of retinitis pigmentosa facilitates the clearance of misfolded rhodopsin, a critical visual cycle protein susceptible to genetic mutation.¹ The prevalence of retinitis pigmentosa is more than one million people worldwide, and genetic mutations leading to rhodopsin misfolding account for approximately one-third of cases. ADX-2191 has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of retinitis pigmentosa.

About Aldeyra

Aldeyra Therapeutics is a biotechnology company devoted to discovering innovative therapies designed to treat immune-mediated diseases. Our approach is to develop pharmaceuticals that modulate immunological systems, instead of directly inhibiting or activating single protein targets, with the goal of optimizing multiple pathways at once while minimizing toxicity. Our product candidates include RASP (reactive aldehyde species) modulators ADX‑629, ADX‑246, ADX‑248, and chemically related molecules for the potential treatment of systemic and retinal immune-mediated diseases. Our pre-commercial product candidates are reproxalap, a RASP modulator for the potential treatment of dry eye disease (under U.S. Food and Drug Administration New Drug Application review) and allergic conjunctivitis, and ADX-2191, a novel formulation of intravitreal methotrexate for the potential treatment of primary vitreoretinal lymphoma (under U.S. Food and Drug Administration New Drug Application review), proliferative vitreoretinopathy, and other rare sight-threatening retinal diseases. For more information, visit https://www.aldeyra.com/ and follow us on LinkedIn, Facebook, and Twitter.