Klotho Neurosciences Inc. Granted FDA Orphan Drug Designation
NEW YORK, July 10, 2025 — Klotho Neurosciences Inc. (NASDAQ: KLTO), a pioneering gene and cell therapy company, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its innovative product KLTO-202 (s-KL-AAV.myo) aimed at treating Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig's Disease.
Understanding Orphan Drug Designation
The FDA's Orphan Drug Designation is a significant milestone for drugs intended for rare diseases, which affect fewer than 200,000 individuals in the U.S. This designation not only provides incentives like tax credits for clinical trial costs but also ensures a seven-year U.S. market exclusivity independent of the company’s intellectual property protection.
Comments from Klotho's CEO
Dr. Joseph Sinkule, CEO of Klotho, expressed the importance of this designation, stating, "Receiving the Orphan Drug Designation for s-KL-AAV.myo underscores our commitment to providing new treatment options for patients suffering from this devastating and rare condition. We aim to pioneer the first gene replacement therapy that targets the neurological damage caused by ALS, enhancing motor neuron protection through therapeutic concentrations of the s-KL protein."
The Impact of ALS
Amyotrophic Lateral Sclerosis, often referred to as Lou Gehrig's disease, has affected thousands of lives, with approximately 5,000 new cases each year. The disease is characterized by progressive degeneration of motor neurons, leading to severe physical impairment.
Next Steps for Klotho Neurosciences
Klotho Neurosciences has successfully completed proof-of-concept studies in two animal models and is now moving forward with the manufacturing of KLTO-202. The company plans to engage with both the FDA and the European Medicines Agency (EMA) to align on the future development path for this promising candidate.
KLTO-202 utilizes a muscle-specific promoter, "desmin," to drive the expression of the s-KL gene and protein, specifically targeting delivery to the neuromuscular junction.
As of now, KLTO-202 is not yet approved for human use by any regulatory authority. For more details about Klotho Neurosciences and its innovative approaches in treating neurodegenerative diseases, visit www.klothoneuro.com or check out Inside Ticker for the latest updates.