Cyclo Therapeutics' Trappsol Shows Early Safety, Efficacy In Genetic Disorder

Cyclo Therapeutics Inc CYTH has announced new data from its Phase 1 extension study evaluating Trappsol Cyclo Niemann-Pick Disease Type C1 (NPC), a genetic disorder characterized by the abnormal accumulation of cholesterol in cells.
Efficacy as measured NPC Severity Scale showed a trend of stability for patients, with the mean length of exposure of 25 months.
Using a sub-set of NPC Severity Scale, assessing the quality of life (Speech, Swallow, Fine Motor skills, Ambulation and Cognition), a trend in disease stabilization was observed.
An average worsening of 0.4 pts per year was observed for all patients with all available data. It is significant as a worsening of 1.4 pts per year would be expected for NPC patients based on published calculations.
The patient who improved overall in the 5-Domain NPC Severity Score received the 1500 mg/kg dose, while the patient who worsened overall also received the 1500 mg/kg dose.
Of the 3 patients who were stable overall, 2 received the 1500 mg/kg dose, and 2 received the 2500 mg/kg dose.
Trappsol Cyclo is a proprietary formulation of hydroxypropyl beta-cyclodextrin, which has an affinity for cholesterol. The candidate is currently in a pivotal Phase 3 study.
Price Action: CYTH stock is down 0.5% at $8.31 during the market session on the last check Friday.