Cyclo Therapeutics (NSDQ:CYTH) has announced new safety and efficacy data from the its ongoing Phase 1 open-label extension study of Trappsol Cyclo to treat Niemann-Pick disease type C (NPC). NPC is a rare, progressive genetic disorder that involves abnormal cellular cholesterol accumulation.
Initial results from the study demonstrated disease stabilization with considerable clinical improvements in some patients. In addition, Trappsol Cyclo was well-tolerated, and no adverse events were linked to the treatment.
The study measured efficacy using the 17-domain NPC Severity Scale. Eight patients measured from the baseline Phase 1 study to the extension study demonstrated a trend of overall stability.
Patients in the trial had an average worsening of their NPC Severity Scale of 0.4 points per year. In general, NPC patients have an average worsening of 1.4 points.
The Trappsol Cyclo is a proprietary formulation of hydroxypropyl beta-cyclodextrin, a hy…