Aldeyra Therapeutics announced Aug. 28 that the European Medicines Agency (EMA) had granted orphan designation for ADX-2191 (methotrexate intravitreal injection) for certain cancers, including primary vitreoretinal lymphoma (PVRL).

There is currently no approved treatment for PVRL, a rare, aggressive, cancer. The candidate also gained orphan status from the US FDA.

Aldeyra, of Lexington, Massachusetts, says its methotrexate formulation was designed to be vitreous-compatible, while reducing the risk of infection and lowering injection volume compared with compounded versions.

The FDA in June 2023 rejected Aldeyra’s literature-based new drug application for methotrexate for PVRL. Regulators cited a lack of clinical evidence. Based on prior discussions with the FDA, Aldeyra did not conduct any clinical trials of ADX-2191 in PVRL.

Aldeyra said a proposed clinical trial, which received special protocol assessment agreement from the FDA, will compare cancer cell clearance after 30 days in up to 20 patients following 1:1 randomization to receive either a single intraocular injection or eight intraocular injections of ADX-2191.

The frequency of methotrexate injections has been linked to cancer cell clearance in PVRL patients, Aldeyra said.

The trial is expected to begin in the second half of 2025 and conclude in 2026, Aldeyra said.

In July, ADX-2191 gained EMA orphan status for inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa (RP).

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