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Investing.com -- Klotho Neurosciences, Inc. (NASDAQ:KLTO) stock surged 22% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its novel secreted-Klotho promoter, gene and delivery system (KLTO-202) for the treatment of Amyotrophic Lateral Sclerosis (ALS).
The designation provides Klotho with several benefits, including tax credits toward clinical trial costs, waiver of certain FDA user fees, and seven years of market exclusivity in the United States upon approval. This status is reserved for treatments targeting rare diseases affecting 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, Klotho’s Chief Executive Officer.
KLTO-202, the company’s lead product candidate, uses a muscle-specific promoter called "desmin" to drive expression of the s-KL gene transcript and protein. The therapy is designed with targeted delivery to the neuromuscular junction—the interface between the spinal cord and muscles.
The gene therapy company has completed "proof of concept" studies in two animal models of human ALS and is initiating manufacturing of the ALS-targeted product candidate. Klotho plans to meet with U.S. FDA and European Medicines Agency officials to determine the development path forward.
ALS, sometimes called Lou Gehrig’s disease, affects fewer than 200,000 people in the U.S., with approximately 5,000 new cases diagnosed annually. The condition is universally fatal, with patients experiencing progressive loss of motor neuron function.
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