Aldeyra Therapeutics, Inc., a biotechnology company discovering and developing innovative therapies for the treatment of immune-mediated diseases, today announced that it has initiated a Phase 2 clinical trial of ADX-2191 (intravitreal methotrexate 0.8%), an investigational new drug product, for the treatment of retinitis pigmentosa (RP). RP is a clinical group of rare genetic eye diseases characterized by retinal cell death and loss of vision. There are no approved treatments for RP, which affects an estimated 82,000-110,000 individuals in the United States, and approximately 1 in 4,000 people worldwide.
“ADX-2191 represents a novel approach for the treatment of patients with retinitis pigmentosa, an incurable, sight-threatening condition,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “We are excited about the opportunity to advance our ADX-2191 retinal program across a variety of rare and underserved diseases, and look forward to reporting the top-line results of the retinitis pigmentosa Phase 2 clinical trial in the second half of this year.”
The single-center, open-label Phase 2 clinical trial will evaluate the safety and tolerability of ADX-2191 in patients diagnosed with RP due to mutations of the rhodopsin gene, including the P23H gene mutation. The trial is expected to enroll eight patients, with four patients receiving monthly and four patients receiving twice-monthly intravitreal injections of ADX-2191 over a period of three months. The trial is being conducted at Duke University Medical Center in Durham, North Carolina. Top-line results are expected in the second half of 2022.