CYTH - Cyclo Therapeutics completes rare genetic disease early-stage trial

Cyclo Therapeutics (CYTH) announces that the last patient completed their last visit in the Phase 1/2 study evaluating Trappsol Cyclo, a proprietary formulation of hydroxypropyl beta cyclodextrin, administered intravenously to patients with Niemann-Pick Disease Type C1 ((NPC1)), a rare genetic disorder affecting 1 in 100,000 live births globally.The company expects to report topline data for the study in March 2021.The randomized, double-blind Phase 1/2 study of Trappsol Cyclo enrolled 12 patients aged 2 and above, and the treatment was evaluated in 3 dose groups (1500, 2000, 2500 mg/kg body weight) administered IV in NPC patients over 48 weeks.The company confirmed that a pivotal Phase 3 study for the treatment may begin enrollment in Q2 2021.

For further details see:

Cyclo Therapeutics completes rare genetic disease early-stage trial