Klotho Neurosciences Receives FDA Orphan Drug Designation for KLTO-202 in ALS

NEW YORK – July 10, 2025 – Klotho Neurosciences, Inc. (Nasdaq: KLTO), a gene and cell therapy company focused on neurodegenerative and aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KLTO-202 (s-KL-AAV.myo). This designation is for the company’s novel secreted-Klotho (s-KL) promoter, gene, and delivery system for the treatment of Amyotrophic Lateral Sclerosis (ALS).

ALS, also known as Lou Gehrig’s disease, is a rare, universally fatal neurodegenerative disorder affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides significant incentives, including tax credits for clinical trials, waiver of GDUFA User Fees, and seven years of U.S. market exclusivity upon approval.

Dr. Joseph Sinkule, Klotho’s Chief Executive Officer, stated, “Receiving the Orphan Drug Designation for s-KL-AAV.myo for the early treatment of ALS underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease… we believe this ODD designation provides strong validation of our science and our approach to treat this disease.”

KLTO-202, Klotho Neurosciences’ lead product candidate, is a gene replacement therapy composed of a muscle-specific “desmin” promoter driving the expression of the s-KL gene transcript and s-KL protein. It is designed for targeted delivery to the neuromuscular junction, aiming to address motor neuron damage and provide neurologic protection.