LEXINGTON, Mass. — Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191 (methotrexate intravitreal injection, USP) for the treatment of primary large B-Cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma. There is currently no approved treatment for patients with primary vitreoretinal lymphoma, a rare, aggressive, high-grade cancer that affects approximately 100 to 200 people per year in the European Union.
“Primary vitreoretinal lymphoma is a potentially fatal cancer that today is treated with off-label compounded formulations of methotrexate that are injected into the eye,” stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “The receipt of Orphan Designation from the EMA, in conjunction with the previously announced Orphan Drug Designation from the U.S. Food and Drug Administration, is another important step forward for our retinal disease program, complementing our ongoing clinical development of ADX-2191, a novel, vitreous-compatible formulation of methotrexate that is specifically designed for intraocular injection.”
A proposed clinical trial, which received Special Protocol Assessment agreement from the U.S. Food and Drug Administration, is designed to compare cancer cell clearance after 30 days of therapy in up to 20 patients following 1:1 randomization to receive either a single intraocular injection or eight intraocular injections of ADX-2191. The frequency of methotrexate injections has been linked to cancer cell clearance in patients with primary vitreoretinal lymphoma,1 and approximately five injections on average has been observed to achieve cancer cell clearance.2 The clinical trial is expected to begin in the second half of 2025 and conclude in 2026.
The EMA grants orphan designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare, life-threatening, or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union.
About ADX-2191
ADX-2191 (methotrexate intravitreal injection, USP) is a sterile, non-compounded intravitreal formulation of methotrexate for the potential treatment of specific rare retinal diseases, including primary vitreoretinal lymphoma and retinitis pigmentosa. The ADX-2191 intravitreal formulation is preservative-free, is designed to be vitreous-compatible, and is optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. ADX-2191 received FDA Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa, and EMA Orphan Designation for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa, and the treatment of primary large B-cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma.
About Primary Vitreoretinal Lymphoma
Primary vitreoretinal lymphoma is a rare, aggressive, and potentially fatal retinal cancer that is diagnosed in approximately 200 to 600 patients in the United States and 100 to 200 patients in the European Union per year. The median survival for newly diagnosed patients is less than five years. No approved treatments are currently available, though intravitreal injection of compounded methotrexate represents the current standard of care.
About Aldeyra
Aldeyra Therapeutics is a biotechnology company devoted to discovering innovative therapies designed to treat immune-mediated and metabolic diseases. Our approach is to develop pharmaceuticals that modulate protein systems, instead of directly inhibiting or activating single protein targets, with the goal of optimizing multiple pathways at once while minimizing toxicity. Our product candidates include RASP (reactive aldehyde species) modulators ADX-629, ADX‑248, ADX-743, ADX-631, ADX-246, and chemically related molecules for the potential treatment of systemic and retinal immune-mediated and metabolic diseases. Our late-stage product candidates are reproxalap, a RASP modulator for the potential treatment of dry eye disease and allergic conjunctivitis, and ADX-2191, a novel formulation of intravitreal methotrexate for the potential treatment of primary vitreoretinal lymphoma and retinitis pigmentosa.
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References 1 Am J Ophthalmol 251: 189–196, 2023 2 Br J Haematol, 194, 92–100, 2021; Cancer Sci. 107:1458-1464, 2016 |
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