Cyclo Therapeutics, Inc., a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, announced that the Safety Review Committee for its Phase I trial in NPC has determined that Trappsol® Cyclo™ has an acceptable safety profile in patients with Niemann-Pick Disease Type C1. The Committee evaluated all safety data in a blinded fashion from the Phase I trial, “A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol® Cyclo™ (HP-ß-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease,” in making its determination. The last patient in the trial received the last dose in February 2020. Patients in this trial are age 18 years and older.
“Today’s announcement represents another major milestone for our company and all stakeholders,” said Company Chairman and CEO, N. Scott Fine. “The Safety Review Committee findings are consistent with our experience gained over many years from our named patient use of Trappsol® Cyclo™. With our first formal clinical trial coming to its conclusion, we are pleased to learn of the Safety Review Committee findings, and we look forward to sharing this information with the U.S. Food and Drug Administration (FDA) and other regulatory bodies in due course.”
“The review of individual and cumulative blinded safety data shows that Trappsol® Cyclo™ is well tolerated with no serious safety signals observed,” said Professor Alan Boyd, MD, Chairman of the Safety Review Committee. “Overall, the intravenous route of administration of the study drug resulted in only minor and expected adverse events.”
The Company is now in the process of unblinding the data from the Phase I trial. Since all patients participating in the Phase I trial received study drug, the findings of the Safety Review Committee are critical to release at this time. Cyclo Therapeutics, Inc. expects to release Top Line results from the Phase I trial, inclusive of unblinded evaluations, in May 2020.
Data from the current study combined with those of the companion Phase I/II companion study underway in Europe and Israel will be used to inform the design of the Company’s Phase III global pivotal trial. Cyclo Therapeutics, Inc. met with FDA in the February 2020 to discuss the design of the Phase III global pivotal trial and expects to meet with EMA for the same purpose in the second quarter of 2020.
“We gratefully acknowledge the many patients and families who participated in our Phase I trial, and we thank our Co-Principal Investigators, Dr. Caroline Hastings and Dr. Benny Liu, and all of our supporting partners, for the hard work that allowed us to reach this point,” said Sharon Hrynkow, PhD, Cyclo Therapeutics’ Chief Scientific Officer and Senior Vice President for Medical Affairs. “Without the concerted effort on the part of many, we would not be able to share such positive news with the community.”
About the Clinical Trial:
Complete enrollment in the study required 12 patients aged 18 and above who had NPC Type C1 disease. The study was a randomized, double-blind study using Trappsol® Cyclo™ intravenously. Study subjects received 7 doses of the drug at either 1500 mg/kg or 2500 mg/kg and were assessed for adverse events, markers for cholesterol metabolism following drug administration, and symptomatic changes using an NPC severity scoring tool, among other tests.
The company has previously reported initial data from this study suggesting that the use of Trappsol® Cyclo™ administered intravenously has a favorable safety profile; that there is a temporal link between administration of the drug and clearance of cholesterol from cells; that demonstrate the presence of the drug in the cerebrospinal fluid following IV administration; and that show a reduction in a neuron-specific biomarker, tau, associated with neuronal degeneration in NPC patients. On unblinding of the data, full interpretation will be possible.