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Aldeyra Therapeutics starts Phase 2 clinical trial of ADX-2191 for treatment of retinitis pigmentosa


The 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.

Aldeyra Therapeutics Inc. today announced 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 to 110,000 individuals in the United States, and approximately 1 in 4,000 people worldwide.

Todd C. Brady, MD, PhD, president and CEO of Aldeyra, pointed out in a statement that ADX-2191 represents a novel approach for the treatment of patients with retinitis pigmentosa, an incurable, sight-threatening condition.

"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," he said.

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.

About ADX-2191
ADX-2191, the intravitreal formulation of methotrexate, is a compound that inhibits cellular replication and activation. In vivo research has identified the activity of methotrexate in inducing misfolded rhodopsin clearance, suggesting the potential to treat forms of retinitis pigmentosa that are characterized by misfolded rhodopsin.1

ADX-2191 has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of retinitis pigmentosa. Orphan drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials; exemption from user fees; and market exclusivity after approval, if received.


1 Xi Y, Chen Y. Pharmacological strategies for treating misfolded rhodopsin-associated autosomal dominant retinitis pigmentosa. Neural Regen Res. 2022 Jan;17(1):110-112. doi: 10.4103/1673-5374.314306. PMID: 34100444; PMCID: PMC8451548.

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