CEO of Cyclo Therapeutics Discusses Niemann-Pick Disease Type C1 Clinical Trial

Scott Fine, CEO of Cyclo Therapeutics, discusses the phase 3 trial evaluating hydroxypropyl betacyclodextrin (Trappsol® Cyclo™) for the treatment of Niemann-Pick disease type C1 (NPC1).

NPC is a disabling neurogenetic disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood. This rare disorder is marked by progressive motor dysfunction and a highly variable symptom profile and onset of symptoms. It can result in the patient’s death soon after birth or manifest as a chronic disorder with symptoms worsening slowly over time. The community is in need of a new therapeutic approach as to this disease. Earlier this year, the FDA denied approval of arimoclomol as a treatment for NPC.

One possible treatment option is hydroxypropyl betacyclodextrin. As Mr. Fine explains, the drug is being evaluated in the TransportNPC study that is designed to evaluate the safety, tolerability, and efficacy of hydroxypropyl betacyclodextrin administered intravenously (2000 mg/kg) plus standard of care (SOC) compared to placebo plus SOC in patients with NPC1. The study intends to enroll at least 93 pediatric and adult patients with NPC1 in at least 23 study centers in 9 countries. Eligible patients will be randomized 2:1 to receive hydroxypropyl betacyclodextrin or placebo. Randomization will not be constrained based on patient age, nor will patient enrollment be gated by patient age. The study duration is 96 weeks and includes an interim analysis at 48 weeks. Cyclo Therapeutics expects to report topline results from the interim analysis in the first half of 2023.

For more information about the study and enrollment, visit https://clinicaltrials.gov/ct2/show/NCT04860960

To learn more about NPC and other lysosomal storage diseases, visit checkrare.com/diseases/lysosomal-storage-disorders/