Aldeyra Therapeutics Announces Planned Pivotal Clinical Trial for RP Drug Candidate

Aldeyra Therapeutics hosted the Aldeyra 2024 Research & Development Day with investors and financial analysts in New York City on April 25 where it presented recent pipeline developments relating to the RASP modulation platform and ADX-2191 for the treatment of retinitis pigmentosa.

Based on recent discussions with the FDA, Aldeyra said it intends to initiate a potentially pivotal phase 2/3 clinical trial of investigational product candidate ADX-2191 (methotrexate injection, USP) in patients with retinitis pigmentosa due to rhodopsin misfolding mutations. The potential activity of ADX-2191 in retinitis pigmentosa is supported by results from a phase 2 clinical trial, announced in 2023, which demonstrated improvements from baseline in retinal sensitivity following treatment.

An overview of the unmet medical need in retinitis pigmentosa will be provided by Ramiro Maldonado, MD, principal investigator of the phase 2 clinical trial and Assistant Professor of Ophthalmology, Vitreoretinal Diseases, and Surgery at Duke University.