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Cyclo Therapeutics Announces New Positive Safety and Efficacy Data from Ongoing Phase 1 Open-Label Extension Study of Trappsol® Cyclotm for the Treatment of Niemann-Pick Disease Type C1


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 new data from its Phase 1 extension study evaluating Trappsol® Cyclotm for the treatment of Niemann-Pick Disease Type C1 ("NPC"), a rare, progressive and fatal genetic disorder characterized by abnormal accumulation of cholesterol in cells. These data are being presented at the National Niemann Pick Disease Foundation Family Support & Medical Conference being held virtually July 29-August 1, 2021, and at the International Niemann Pick Disease Foundation biennial meeting held on August 1, 2021, also being held virtually.

Trappsol® Cyclotm is a proprietary formulation of hydroxypropyl beta cyclodextrin which has an affinity for cholesterol. In multiple clinical studies, Trappsol® Cyclotm has shown encouraging results to normalize the transportation of cholesterol in cells.

"We are incredibly pleased with the continued trends of safety and efficacy being demonstrated by Trappsol® Cyclotm in the treatment of NPC. We are grateful for the opportunity to present these data to patients and their families and hope to provide continued encouragement as we work diligently to treat the systemic and neurologic manifestations of NPC," commented Sharon Hrynkow, PhD, the Company's Chief Scientific Officer and Senior Vice President for Medical Affairs.

"We remain focused on expeditiously advancing this clinical program toward potential market approval and believe strongly that Trappsol® Cyclotm has enormous potential to meet the significant unmet needs for NPC patients and their families," stated N. Scott Fine, CEO and Executive Director of Cyclo Therapeutics.

All 8 eligible patients (U.S. residents) enrolled in the Extension Protocol, which supports IV infusions in the home setting under the care of a healthcare professional. Initial efficacy evaluation in September 2020 showed disease stabilization with notable clinical improvements in some patients.

The first patient was dosed in the Open-Label extension study in May 2019 and the last patient enrolled was in February 2020. The Company previously reported data on the Phase 1 extension study in January 2021 based on the initial efficacy results at a data cut-off of September 2020. The results summarized below have a data cut-off of July 2021.

These findings provide additional support for the capacity of Trappsol® Cyclotm to stabilize disease progression in NPC1.

Trappsol® Cyclotm is currently being evaluated in the pivotal Phase 3 study, TransportNPCtm, for the treatment of NPC1. As previously announced, Cyclo Therapeutics received Orphan Drug Designation for Trappsol® Cyclotm to treat NPC1 in both the US and EU and 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 Niemann-Pick Disease Type C1 (NPC)

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.

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® Cyclotm, 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® Cyclotm 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® Cyclotm 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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News published on 30 july 2021 at 08:10 and distributed by: