Aldeyra Therapeutics Inc. (NASDAQ:ALDX) saw its stock rise by 8% after announcing a key agreement with the U.S. Food and Drug Administration (FDA) concerning its planned clinical trial for ADX-2191, a treatment candidate for primary vitreoretinal lymphoma (PVRL), a rare and aggressive eye cancer.
The company received a Special Protocol Assessment (SPA) Agreement Letter from the FDA, confirming alignment on the trial’s design. The upcoming study will evaluate the effectiveness of ADX-2191 in eliminating cancer cells through two different treatment regimens—one involving a single intraocular injection, and the other using a series of eight injections. The trial is scheduled to begin in the latter half of 2025, with results anticipated in 2026.
ADX-2191 is a proprietary, vitreous-compatible formulation of methotrexate tailored specifically for use inside the eye. Its unique composition is designed to improve upon conventional compounding approaches, potentially allowing for smaller injection volumes and more consistent delivery.
“Current treatment for primary vitreoretinal lymphoma relies on off-label use of compounded methotrexate,” said Aldeyra CEO Dr. Todd Brady. “We believe ADX-2191 could offer a standardized, more reliable option for patients battling this life-threatening disease.”
Previously, Aldeyra submitted a literature-based New Drug Application (NDA) for ADX-2191, which was granted Priority Review by the FDA in early 2023. However, the agency later issued a Complete Response Letter in June 2023, citing the need for direct clinical trial data to demonstrate the drug’s efficacy.
Despite the earlier setback, investor sentiment has turned optimistic with the FDA’s formal agreement on the new trial framework. JonesTrading analyst Catherine Novack maintained a “Buy” rating and a $6.00 price target, noting that a successful approval for PVRL could eventually lead to broader use of ADX-2191 in more common conditions, including retinitis pigmentosa.
Currently, no FDA-approved therapies exist for PVRL, making Aldeyra’s investigational approach a potentially significant advancement in ocular oncology.