Global clinical protocol agreed upon by US Food and Drug Administration (FDA) and European Medicines Agency (EMA)
Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today announced the commencement of its pivotal Phase 3 study (“TransportNPC”) evaluating Trappsol® Cyclo™, a proprietary formulation of hydroxypropyl beta cyclodextrin, delivered intravenously, for the treatment of Niemann-Pick Disease Type C1 (NPC1). The TransportNPC study has the regulatory and IRB approval required to commence patient enrollment, and site activation is underway.
NPC is a rare genetic disease affecting 1 in 100,000 live births globally. Approximately 95% of individuals with NPC have mutations in the NPC1 gene and 5% have mutations in the NPC2 gene. NPC affects nearly every cell in the body due to a deficiency in either the NPC1 or NPC2 protein, which are required for the transport and processing of cholesterol within the cell. As cholesterol accumulates within cells, NPC causes symptoms that affect the brain, liver, spleen, lung and other organs and often leads to premature death.
“The start of patient enrollment in our pivotal Phase 3 study is an important milestone for both the NPC community and Cyclo Therapeutics. We are grateful for the hard work on the part of so many in getting us to the point of enrollment. We are excited to move forward with speed, and remain deeply committed to advancing our NPC clinical program forward with the goal of bringing a safe and effective treatment to NPC patients, families and physicians,” said N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics.
The TransportNPC study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2000 mg/kg of Trappsol® Cyclo™ administered intravenously and standard of care (SOC) compared to placebo administered intravenously and SOC in patients with NPC1. The TransportNPC study intends to enroll at least 93 pediatric (age 3 to less than 18 years) and adult patients with NPC1 in at least 23 study centers in 9 countries. Eligible patients will be randomized 2:1 to receive Trappsol® Cyclo™ 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. The Company expects to report topline results from the interim analysis in the first half (H1) of 2023.
This study has dual primary objectives based on regulatory preferences. For the US and countries following FDA guidance, the primary objective is to evaluate the mean change from baseline to 48 weeks or 96 weeks as measured by improvement in Trappsol® Cyclo™ versus placebo using a 4-Domain modified Niemann-Pick Disease Type C Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech and Swallow]) composite score. For the EU and countries following EMA guidance, the primary objective is to evaluate the mean change from baseline to 48 weeks or 96 weeks as measured by improvement in Trappsol® Cyclo™ versus placebo using the 5-Domain Niemann-Pick Disease Type C Severity Scale (5D-NPC-SS) composite score (Ambulation, Fine Motor, Speech, Swallow, and Cognition).
“The initiation of this clinical trial is an important advance in research and a new opportunity for hope for the NPC community, building upon data that has been accumulated over many years supporting the development of this promising therapy to treat NPC,” added Caroline Hastings, MD, UCSF Benioff Children’s Hospital Oakland, California, and Principal Investigator. “We applaud all the families and researchers who have made this opportunity possible, and we look forward to the new insights that will be available as this Phase 3 trial advances in the months ahead.”
“The launch of the Phase 3 pivotal program for Trappsol® Cyclo™ is a significant milestone for the NPC community,” commented Joslyn Crowe, Executive Director of the National Niemann-Pick Disease Foundation. “With the heterogeneity of the disease, we hope that patients will one day have multiple options for treatment regimens that will be effective to meet individual patient needs.”
As previously announced, the Company received a positive opinion from the Paediatric Committee (PDCO) of the EMA and agreement on its Paediatric Investigation Plan (PIP) for Trappsol® Cyclo™. The PIP opinion from PDCO endorsed the clinical program to evaluate the safety, tolerability and efficacy of Trappsol® Cyclo™ in pediatric patients of all ages. The Phase 3 study will consist of a main study cohort that includes pediatric patients with NPC1 from age 3 to less than 18 years, and in addition, a single-arm sub-study of patients from birth to less than 3 years of age with NPC1 irrespective of symptoms to evaluate safety and to obtain descriptive data on global disease severity and the response to Trappsol® Cyclo™. The substudy in patients from birth to less than 3 years of age will be conducted in the EU, and countries following EMA guidelines, and others as approved by local regulatory authorities.
“We are incredibly pleased with the strides the NPC program continues to make, and we remain optimistic as Trappsol® Cyclo™ advances into the final phase of clinical development. Having met all of the primary endpoints of our Phase 1 and Phase 1/2 studies showing favorable safety and promising trends in efficacy with Trappsol® Cyclo™, we believe that Cyclo Therapeutics is well-positioned to address the systemic and neurologic manifestations of NPC, and ultimately, provide hope to people living with NPC and their families,” added Gerry Cox, MD, PhD, Acting Chief Medical Officer of the Company.
Cyclo Therapeutics has received Orphan Drug Designation for Trappsol® Cyclo™ to treat NPC in both the US and EU, and additionally, Fast Track and Rare Pediatric Disease Designations in the US. The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the US upon marketing authorization.
For more information about the pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.
About Cyclo Therapeutics
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families suffering from disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is the subject of three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (www.ClinicalTrials.gov NCT02939547, NCT02912793, NCT03893071 and NCT04860960). The Company is planning an early phase clinical trial using Trappsol® Cyclo™ intravenously in Alzheimer’s Disease based on encouraging data from an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the Company’s website: www.cyclotherapeutics.com.
Safe Harbor Statement
This press release contains “forward-looking statements” about the company’s current expectations about future results, performance, prospects and opportunities, including, without limitation, statements regarding the satisfaction of closing conditions relating to the offering and the anticipated use of proceeds from the offering. Statements that are not historical facts, such as “anticipates,” “believes” and “expects” or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company’s future performance include the company’s ability to obtain additional capital to expand operations as planned, success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company’s biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.
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