A Phase III study examining a treatment for the rare genetic disorder Niemann-Pick disease has seen 87% of its participants, all younger than three years old, show signs of stabilisation or improvement.
US-based Cyclo Therapeutics has unveiled preliminary data from its TransportNPC trial (NCT04860960) of a hydroxypropyl beta cyclodextrin formulation used in eight patients under the brand name Trappsol Cyclo.
The ongoing Phase III double-blind, placebo-controlled, multi-centre study is designed to evaluate the safety and tolerability of the treatment across 104 patients.
Results from a sub-study arm within the trial saw 87% who had reached the 24-week mark display signs of clinical improvement as measured by the Clinical Global Impression–Change (CGI-C) Scale. Additionally, six out of seven patients who continued onto week 48 showed continued improvement.
Niemann-Pick disease refers to a group of rare genetic conditions that affect a person’s ability to break down fats such as lipids. The TransportNPC trial specifically examined patients living with the type C version of the disease. The condition is typically caused by mutations in the NPC1 or NPC2 genes.
Dr Ronen Spiegel, trial investigator and director for the Center of Rare Disease at Emek Medical Center, said: “The preliminary findings from the TransportNPC sub-study are encouraging and further underscore the potential of Trappsol Cyclo to address the significant unmet medical need in the treatment of NPC.
“Slowing down disease progression through cholesterol mobilisation is an important consideration for patients with established disease, who can expect to experience neurodegeneration without treatment.
“The growing body of data from our TransportNPC study and sub-study continue to demonstrate the potential of Trappsol Cyclo to address a devastating disease with significant unmet need.”
Cyclo Therapeutics has now said it is looking to use its sub-group arm success to file for marketing applications before the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). At the same time, the company is currently examining Trappsol Cyclo in a Phase IIb study for Alzheimer’s disease.
Elsewhere in the treatment of Niemann-Pick disease, Azafaros reported positive topline findings last year from the Phase II RAINBOW clinical trial of nizubaglustat.
