Arialys Therapeutics Announces U.S. FDA Fast Track Designation for ART5803, the First Precision Therapeutic Candidate in Development for the Treatment of Anti-NMDA Receptor Encephalitis

LA JOLLA, Calif.--(BUSINESS WIRE)--Arialys Therapeutics, a clinical-stage biotechnology company pioneering precision medicines for autoimmune neuropsychiatric diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ART5803 for the treatment of anti-NMDA receptor encephalitis (ANRE). ART5803 is a novel therapeutic candidate designed using structural biology to specifically inhibit the pathogenic effects of autoantibodies that target the NMDA receptor.

“Fast Track designation is an important step as we expedite the development of ART5803 as a potential treatment option for patients suffering from ANRE, a serious disease with no currently approved therapeutics,” said Peter Flynn, Ph.D.

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“Fast Track designation is an important step as we expedite the development of ART5803 as a potential treatment option for patients suffering from ANRE, a serious disease with no currently approved therapeutics,” said Peter Flynn, Ph.D., President and CEO of Arialys Therapeutics. “We appreciate the FDA’s recognition of the unmet medical need for ANRE patients and ART5803’s therapeutic potential. Our team is efficiently executing our development plan, with enrollment ongoing in an open-label Phase 2 clinical study and a randomized Phase 2 study planned to initiate in the U.S. later this year.”

About Fast Track Designation

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of investigational therapies intended to treat serious or life-threatening conditions and that have the potential to address unmet medical needs. A drug that receives Fast Track designation is eligible for increased communication with the FDA throughout the development process, rolling review of a marketing application, as well as the potential for Accelerated Approval and Priority Review.

About ANRE

Anti-NMDA receptor encephalitis (ANRE) is a rare, life-threatening, and often misdiagnosed neurological disease. ANRE is caused by pathogenic autoantibodies that bind to and crosslink NMDA receptors in the brain, leading to receptor internalization and synaptic dysfunction. The disease can cause debilitating neuropsychiatric symptoms, including psychiatric and behavioral alterations, cognitive decline, seizures, coma, and autonomic dysfunction. A significant percentage of ANRE patients are pediatric, and NMDA receptor-specific autoantibodies can also contribute to neurodevelopmental deficits. There are no approved therapies for ANRE, and current treatments rely on broad immunosuppressive agents, which are associated with delayed efficacy and significant side effects.

About ART5803

Arialys scientists used crystallographic structures and pharmacological assessments to develop ART5803, the first precision therapeutic designed to directly inhibit the pathological effects of autoantibodies on the NMDA receptor. ART5803 is a humanized, monovalent monoclonal antibody. Arialys has initiated an open-label Phase 2a signal seeking study of ART5803 in ANRE patients. In addition, the FDA has cleared an investigational new drug (IND) application for a separate randomized Phase 2 study. Arialys has completed Phase 1 SAD and MAD clinical assessments in healthy volunteers. ART5803 demonstrated safety, pharmacokinetic, and CNS penetration profiles supportive of further clinical assessment. In preclinical models, ART5803 rapidly reversed behavioral symptoms caused by NMDAR autoantibody pathogenicity. ART5803 has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the U.S. FDA and Orphan Drug designation from South Korea’s Ministry of Food and Drug Safety (MFDS).

About Arialys Therapeutics

Arialys is a clinical-stage biotechnology company pioneering precision medicines for autoimmune neuropsychiatric diseases. Using a combination of highly sensitive autoantibody detection, patient sampling, and receptor structural biology, Arialys has developed a first-in-class precision therapy to specifically block pathogenic autoantibodies in the brain. Arialys is supported by Avalon Bioventures, Catalys Pacific, MPM BioImpact and JJDC, and is headquartered in La Jolla, California. For more information, visit www.arialysrx.com.