Aldeyra receives FDA special protocol assessment for lymphoma treatment

Published 26/06/2025, 14:06
Aldeyra receives FDA special protocol assessment for lymphoma treatment

Investing.com - Aldeyra Therapeutics (NASDAQ:ALDX), trading at $3.51 after gaining over 10% in the past week, has secured a special protocol assessment (SPA) from the FDA for its ALDX-2191 treatment targeting primary vitreoretinal lymphoma (PVRL), the company announced Monday.

The clinical trial for ALDX-2191, an intraocular formulation of methotrexate, is scheduled to begin in the second half of 2025 with data expected in 2026. The study will compare cancer cell clearance after 30 days in 20 patients randomized to receive either a single injection or eight intraocular injections of the treatment.

This development follows a Complete Response Letter (CRL) Aldeyra received on June 2023, when the FDA rejected the company’s literature-based New Drug Application, citing insufficient efficacy data. The regulatory agency has now agreed that a single well-controlled trial, alongside literature references, will support NDA resubmission.

PVRL is a rare condition with only 300-600 cases annually in the United States. The disease is currently treated off-label with compounded methotrexate intravitreal injections, with approximately five injections typically needed for cancer cell clearance. ALDX-2191 potentially offers reduced injection volume compared to compounded alternatives.

Aldeyra ended the first quarter of 2025 with $90.1 million in cash and cash equivalents, which the company estimates is sufficient to fund operations through 2026, covering the planned clinical trial and potential regulatory submissions. According to InvestingPro data, the company maintains a healthy current ratio of 6.49 and holds more cash than debt on its balance sheet. Analysts have set price targets ranging from $6 to $11, suggesting potential upside from current levels. For deeper insights into Aldeyra’s financial health and additional analyst coverage, explore InvestingPro, which offers 11 more exclusive tips about ALDX.

In other recent news, Aldeyra Therapeutics has resubmitted its New Drug Application to the FDA for Reproxalap, targeting dry eye disease, after a successful Phase 3 trial. This follows a Complete Response Letter from the FDA earlier this year, which highlighted methodological concerns in previous trials. Aldeyra’s new trial achieved its primary endpoint, addressing the FDA’s earlier concerns, and the company expects an FDA review within six months. Meanwhile, Aldeyra received a Special Protocol Assessment from the FDA for its lymphoma drug ADX-2191, designed to treat primary vitreoretinal lymphoma, a rare retinal cancer. The FDA has agreed that a single clinical trial, alongside literature references, will suffice for a new application.

Analyst activity around Aldeyra has been notable, with BTIG reducing its price target to $9 while maintaining a Buy rating, citing the FDA’s cautious stance on Reproxalap. Jefferies also cut its target to $6 but kept a Buy rating, noting the CRL was not due to safety concerns but rather baseline differences in trials. Laidlaw reiterated its Buy rating and a price target of $11, viewing the recent stock price drop as a buying opportunity despite the FDA setback. Aldeyra is conducting additional trials to address the FDA’s feedback, with results expected soon, which could influence future regulatory decisions.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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