Aldeyra Therapeutics announced on June 26, 2025, the receipt of a Special Protocol Assessment (SPA) Agreement Letter from the U.S. Food and Drug Administration (FDA) for ADX-2191. This investigational drug candidate is being developed for the treatment of primary vitreoretinal lymphoma (PVRL), a rare and potentially fatal cancer that currently lacks FDA-approved therapy. The SPA provides a clear regulatory pathway for the drug's development.
The SPA outlines a single clinical trial designed to compare cancer cell clearance after 30 days of therapy in up to 20 patients. Participants will be randomized 1:1 to receive either a single intraocular injection or eight intraocular injections of ADX-2191. This agreement follows a June 2023 Complete Response Letter from the FDA, which had requested adequate and well-controlled trials for approval, and now confirms that a single trial, in addition to literature references, will be sufficient for NDA resubmission.
The clinical trial is expected to commence in the second half of 2025 and conclude in 2026. ADX-2191 is a novel, vitreous-compatible formulation of methotrexate, specifically designed for intraocular injection, potentially allowing for a reduced injection volume compared to compounded methotrexate. This development is a significant step forward for patients suffering from PVRL, a condition with a median survival of less than five years for newly diagnosed patients.
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