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.
Market News and Data brought to you by Benzinga APIs
Posted In: BiotechNewsHealth CareGeneralBriefsGenetic Disorder
Benzinga simplifies the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!