CYTH - Cyclo Therapeutics' lead candidate shows durable benefit in Niemann-Pick Disease

Thinly-traded nano-cap Cyclo Therapeutics (CYTH) soars 55% in premarket on initial data from its open-label study of long-term safety and efficacy of intravenous Trappsol Cyclo, its lead candidate, for the treatment of Niemann-Pick Disease Type C ((NPC)), a rare inherited progressive neurodegenerative disorder caused by the body's inability to transport cholesterol and other lipids inside cells.Eight patients were enrolled, who completed the Phase 1 trial were eligible for the extension program.All patients included in this analysis demonstrated disease stability or improvement as measured by the 17-domain NPC Severity Score scale that primarily measures neurologic features of the disease, including ambulation, fine motor skills, ability to speak and swallow, and cognition.Of the patients who received Trappsol Cyclo at the 1500 mg/kg, four patients remained stable in terms of their NPC-SS and two patients improved. Of the patients in the 2500 mg/kg dose group, both improved overall.The FDA signed off Trappsol's Phase 3 trial and the company plans to enroll the first

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Cyclo Therapeutics' lead candidate shows durable benefit in Niemann-Pick Disease