ADX-2191 is currently in clinical development to treat retinitis pigmentosa and proliferative vitreoretinopathy. Credit: Kateryna Kon via Shutterstock.com.

Aldeyra Therapeutics has reported positive top-line results from the Phase II clinical trial of intravitreal ADX-2191 (methotrexate injection, USP) in retinitis pigmentosa patients.

The single-centre, open-label Phase II clinical trial was conducted in eight retinitis pigmentosa patients, who have rhodopsin misfolding mutations.

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Four patients received monthly injections and the remaining four received twice-monthly injections during a three-month treatment period with ADX-2191.

The findings showed that the treatment with ADX-2191 significantly improved retinal function across many different physiological and psychophysical assessments, from baseline.

Participants have also experienced an improvement in best-corrected and low‑light visual acuity.

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Time taken for retinal response has statistically improved significantly, as evaluated by electroretinography, and retinal sensitivity has also significantly improved, as assessed by macular and dark-adapted perimetry.

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With no safety concerns, ADX‑2191 was found to be well tolerated.

Aldeyra Therapeutics president and CEO Todd Brady said: “The improvement in retinal function relative to baseline observed in this retinitis pigmentosa clinical trial of ADX-2191 may offer hope to patients that today have no therapeutic options.

“Based on compelling proof-of-concept clinical activity that is consistent with a well-defined mechanism of action supported by preclinical evidence, we are excited to meet with regulatory authorities to discuss initiation of a potentially pivotal Phase II/III clinical trial, as we enthusiastically advance ADX-2191 to the next stage of development.”

The new intravitreal formulation of methotrexate, ADX-2191 is currently in clinical development to treat retinitis pigmentosa and proliferative vitreoretinopathy.

The US Food and Drug Administration granted orphan drug designation to the therapy to treat both indications.