Aldeyra Thinks It May Have Broadly Applicable Retinitis Pigmentosa Therapy

Data from a small Phase II study show that Aldeyra’s methotrexate formulation may address the underlying cause of 20%-30% of RP cases. The drug recently was denied US FDA approval in a rare ocular cancer.

Genetic eye disease
Aldeyra's drug may address the underlying cause of retinitis pigmentosa • Source: Shutterstock

Potentially competing with gene therapy in the unmet medical need of retinitis pigmentosa (RP), Aldeyra Therapeutics, Inc. reported statistical significance across multiple measures in a small Phase II study of its intravitreal injection formulation of methotrexate on 29 June, data the biotech said it hopes will lead to a pivotal Phase II/III study for ADX-2191 in RP.

The data came roughly a week after the US Food and Drug Administration issued a complete response letter for the drug candidate in a rare cancer, primary vitreoretinal lymphoma. In that case, Aldeyra had filed a “paper NDA” without data from new clinical studies in the sought indication, basing its application instead on “more than three decades of published literature” about the use of methotrexate in primary vitreoretinal lymphoma

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