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 hydroxyalkyl derivative that normalizes the transportation and absorption of cholesterol in cells.
The company will share data from the trial at the National Niemann-Pick Disease Foundation Family Support & Medical Conference, held between July 29 and August 1. It will also present at the International Niemann Pick Disease Foundation conference scheduled for August 1.
The company is also testing the use of Trappsol Cyclo as an Alzheimer’s disease therapy targeting the reduction of amyloid beta and tau. It has released positive data from an Alzheimer’s patient treated under the Compassionate Use Program for 18 months.