Klotho Neurosciences' ALS Therapy Receives FDA Orphan Drug Designation, Shares Soar

Klotho Neurosciences' ALS therapy, KLTO-202, received FDA Orphan Drug Designation for treatment of Amyotrophic Lateral Sclerosis (ALS), a rare neurodegenerative disease. The therapy aims to protect and preserve motor neurons by delivering neuroprotective concentrations of secreted Klotho protein. KLTO-202 has shown promise in preclinical studies, with delayed disease onset and preservation of motor neuron function. The company plans to launch Phase 1/2 clinical trials in Q3 2026, with a Biologics License Application filing potentially following in 2028.

Klotho Neurosciences (NASDAQ: KLTOW) has received a significant milestone in its quest to develop a new treatment for Amyotrophic Lateral Sclerosis (ALS). The company's novel therapy, KLTO-202, also known as s-KL-AAV.myo, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) [1].

ALS, often referred to as Lou Gehrig’s disease, is a rare, progressive neurodegenerative condition that affects the motor neurons responsible for controlling voluntary muscle movement. With approximately 5,000 new diagnoses each year in the U.S., ALS meets the FDA's criteria for rare diseases, which affects fewer than 200,000 individuals [1].

KLTO-202 is a gene therapy designed to deliver the secreted Klotho (s-KL) protein using a muscle-specific promoter, "desmin." This therapy aims to protect and preserve motor neurons by delivering neuroprotective concentrations of s-KL in the blood, brain, and muscle tissues [1].

The FDA's Orphan Drug Designation provides several benefits, including tax credits for clinical trials, a waiver for the payment of the GDUFA User Fee for market applications, and seven years of market exclusivity. Dr. Joseph Sinkule, CEO of Klotho Neurosciences, stated that this designation underscores the importance of bringing new treatment options to ALS patients and validates the company's scientific approach [1].

Preclinical studies have shown promising results for KLTO-202. The company has completed proof-of-concept studies in two animal models of ALS, demonstrating delayed disease onset and preservation of motor neuron function [1]. These studies support the initiation of manufacturing efforts and regulatory planning.

Klotho Neurosciences is now preparing for regulatory interactions with both the FDA and the European Medicines Agency (EMA) to align on clinical development pathways. The company expects to launch Phase 1/2 clinical trials in the third quarter of 2026, with a potential Biologics License Application filing as early as 2028 [1].

The company's progress in developing KLTO-202 is further bolstered by its collaboration with the Okinawa Research Centre for Longevity Science (ORCLS), which aims to study the impact of the alpha-Klotho gene on the population of Okinawa, a longevity hotspot [2].

Klotho Neurosciences is a US-based biotechnologies company focused on genomic therapies that target the alpha-Klotho gene to treat neurodegenerative disorders. The company's current portfolio includes products using both DNA and RNA treatments and diagnostic assays [2].

References:
[1] https://www.proactiveinvestors.co.uk/companies/news/1074521/klotho-neurosciences-als-therapy-awarded-fda-orphan-drug-designation-1074521.html
[2] https://www.bioprocessintl.com/facilities-capacity/klotho-neurosciences-advances-production-of-gene-therapy-for-als
[3] https://www.biospace.com/press-releases/klotho-neurosciences-inc-granted-fda-orphan-drug-designation-for-klto-202-for-treatment-of-amyotrophic-lateral-sclerosis-als-or-lou-gehrigs-disease