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The US Food and Drug Administration (FDA) has granted Priority Review for Aldeyra’s New Drug Application (NDA) for ADX-2191, an investigational drug candidate for the treatment of primary vitreoretinal lymphoma (PVRL) and proliferative vitreoretinopathy.

ADX-2191 is a preservative-free, non-compounded intravitreal formulation of methotrexate for the potential prevention or treatment of specific rare retinal diseases. “The FDA’s decision underscores the significant need for an FDA-approved treatment of primary vitreoretinal lymphoma, a rare but potentially fatal cancer,” said Dr Todd Brady, Aldeyra’s president and CEO. “We are working closely with the FDA during the review process to bring ADX‑2191 to patients as quickly as possible, and plan to launch ADX-2191 in the United States in the second half of this year, pending approval.”

Alongside three decades of published literature on the safety and efficacy of methotrexate for primary vitreoretinal lymphoma treatment, Aldeyra said its NDA application was supported by safety data from its recently completed phase 3 Guard trial of ADX-2191 in patients with proliferative vitreoretinopathy. Guard trial researchers said no safety signals were observed and ADX-2191 was well tolerated. The most common adverse event associated with treatment was punctate keratitis, which was most commonly mild in severity.

Aldeyra’s GA treatment progresses

In separate news Aldeyra Therapeutics also announced it had advanced two investigational new drug candidates to clinical testing, including ADX-‑248 for the treatment of geographic atrophy (GA).

ADX-246 and ADX-248 are novel reactive aldehyde species (RASP) modulators designed to decrease immune responses that lead to disease. Pending completion of the FDA Investigational New Drug requirements, a phase 1/2 clinical trial of intravitreally injected ADX‑-248 and a phase 1 clinical trial of orally administered ADX‑-246 for the treatment of systemic immune-mediated diseases, are expected to begin in the second half of 2023 or early 2024, said Aldeyra.

Pro-inflammatory RASP are associated with inflammation in GA and potentially contribute to visual impairment in low-light settings early in the course of the disease. They also lead to the formation of macromolecular aggregates that comprise, in part, retinal inclusions characteristic of GA and related diseases, including Stargardt disease and dry age-related macular degeneration, said the company.

In February 2023, Apellis’ Syfovre (pegcetacoplan for intravitreal injection) become the first US Food and Drug Administration (FDA)-approved treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).