Shares of Klotho Neurosciences, Inc. (NASDAQ:KLTO) surged 22% following news that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s gene therapy candidate, KLTO-202, aimed at treating Amyotrophic Lateral Sclerosis (ALS).
The newly designated treatment—a secreted-Klotho gene therapy utilizing a muscle-specific delivery system—is designed to target the neuromuscular junction, a key site of degeneration in ALS patients. The gene therapy uses a “desmin” promoter to activate expression of the s-KL gene, delivering therapeutic protein precisely where it’s needed.
The orphan status unlocks a range of regulatory incentives for Klotho, including tax credits for clinical trials, waived FDA fees, and seven years of U.S. market exclusivity if the drug is ultimately approved. The FDA grants such status to treatments for conditions that affect fewer than 200,000 people in the U.S.
“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, CEO of Klotho Neurosciences.
KLTO-202 is the company’s flagship therapeutic candidate and has shown positive results in preclinical proof-of-concept studies using two separate animal models of human ALS. Klotho has already begun manufacturing activities for the therapy and plans to consult both the FDA and the European Medicines Agency (EMA) to chart a development path toward clinical trials.
ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder marked by the loss of motor neurons. Fewer than 200,000 Americans live with the disease, and around 5,000 new cases are diagnosed each year. The condition is universally fatal, with no known cure.
Klotho Neurosciences stock price
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