Klotho Neurosciences’ ALS therapy awarded FDA Orphan Drug Designation
Klotho Neurosciences announced that its novel therapy KLTO-202, also known as s-KL-AAV.myo, for the treatment of Amyotrophic Lateral Sclerosis (ALS) has received Orphan Drug Designation from the US Food and Drug Administration (FDA).
The news initially sent Klotho’s shares rocketing in early trade on Thursday, up around 44%, before settling at the open around 5.8% higher at about $1.45.
ALS is a rare, progressive neurodegenerative disease commonly known as Lou Gehrig’s disease. KLTO-202 is a novel gene therapy engineered to deliver the secreted Klotho (s-KL) protein using a muscle-specific promoter, “desmin,” designed to direct expression at the neuromuscular junction. Its goal is to protect and preserve motor neurons by delivering neuroprotective concentrations of s-KL in the blood, brain, and muscle tissues.
Dr Joseph Sinkule said that receiving the ODD underscored the importance of bringing new treatment options to ALS patients. “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,” Dr Sinkule said. “After the FDA's review of the data leading to the Orphan Drug Designation, we believe this ODD designation provides strong validation of our science and our approach to treat this disease.”
The FDA grants Orphan Drug Designation to drugs and biologics intended to treat diseases affecting fewer than 200,000 individuals in the United States. ALS fits within this category, with approximately 5,000 new diagnoses each year and a prevalence that remains below the 200,000 threshold.
KLTO-202 has shown promise in preclinical studies. The company has completed proof-of-concept studies in two animal models of ALS, demonstrating delayed disease onset and preservation of motor neuron function. These results 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 to align on clinical development pathways. The company expects to launch Phase 1/2 clinical trials in the third quarter of 2026. If the development proceeds as planned, a Biologics License Application filing could follow as early as 2028.
