Klotho Neurosciences Receives Orphan Drug Designation for ALS Treatment Candidate

Klotho Neurosciences has received US FDA Orphan Drug Designation for its ALS drug candidate. The biopharmaceutical company is developing disease-modifying cell and gene therapies using the anti-aging Klotho gene to treat neurodegenerative disorders. Its portfolio includes KLTO-202, KLTO-101, and KLTO-301 gene therapy programs, as well as clinical-stage programs for antibody biologics targeting cancer and autoimmune diseases.

Klotho Neurosciences, Inc. (NASDAQ: KLTO), a biotechnology company specializing in the development of innovative cell and gene therapies, has received a significant regulatory milestone from the U.S. Food and Drug Administration (FDA). The FDA has granted Orphan Drug Designation (ODD) to KLTO-202, the company's gene therapy candidate for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease.

The FDA's Orphan Drug Designation provides KLTO-202 with substantial benefits, including tax credits for clinical trial costs, GDUFA user fee waivers, and seven years of market exclusivity upon approval. This designation underscores the potential of KLTO-202 to address a critical unmet medical need in ALS, a rare and universally fatal neurodegenerative disorder affecting fewer than 200,000 people in the U.S. with approximately 5,000 new diagnoses annually.

KLTO-202 utilizes a novel approach combining a muscle-specific promoter called "desmin" with gene therapy to deliver the secreted-Klotho (s-KL) gene and protein to the neuromuscular junction. The therapy aims to address both the neurological damage and offer neuroprotection through therapeutic concentrations of the s-KL protein in blood, brain, and muscle tissues. The company has completed proof-of-concept studies in two animal models and is initiating manufacturing of the candidate, with upcoming regulatory meetings scheduled with both the FDA and the European Medicines Agency (EMA).

"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," said Dr. Joseph Sinkule, Klotho's Chief Executive Officer. "We aim to deliver the first gene replacement therapy addressing the neurologic insult resulting in motor neuron damage and the potential neurologic protection induced by providing therapeutic blood, brain, and muscle concentrations of the s-KL protein."

Klotho Neurosciences is also developing other gene therapy programs, including KLTO-101 and KLTO-301, and clinical-stage programs for antibody biologics targeting cancer and autoimmune diseases. The company's focus on innovative therapies using the anti-aging Klotho gene positions it as a leader in the development of disease-modifying treatments for neurodegenerative and age-related disorders.

References:
[1] https://www.stocktitan.net/news/KLTO/klotho-neurosciences-inc-granted-fda-orphan-drug-designation-for-orcinwaydbve.html
[2] https://www.morningstar.com/news/pr-newswire/20250710la27685/klotho-neurosciences-inc-granted-fda-orphan-drug-designation-for-klto-202-for-treatment-of-amyotrophic-lateral-sclerosis-als-or-lou-gehrigs-disease
[3] https://www.bioprocessintl.com/facilities-capacity/klotho-neurosciences-advances-production-of-gene-therapy-for-als