Cyclo Therapeutics Selected to Present Overview of Pivotal Phase 3 Study for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2022

GAINESVILLE, Fla.--(BUSINESS WIRE)--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 poster presentation of its ongoing pivotal Phase 3 study, TransportNPC™ at the 18^th Annual WORLDSymposium 2022, a leading medical and scientific conference for professionals working to advance understanding and treatments for lysosomal storage diseases, including Niemann-Pick Disease Type C (“NPC”), being held virtually and in San Diego, CA, February 7–11, 2022.

The ePoster titled, “TransportNPC: a Phase 3 global trial of Trappsol^® Cyclo™ administered intravenously to patients with Niemann-Pick Disease Type C1 (NPC1),” will be presented by Lise Lund Kjems, MD, PhD, Chief Medical Officer of Cyclo Therapeutics in the Clinical Applications Abstracts session on Wednesday, February 9, 2022 from 3:00 – 5:00 PM PST.

“Our pivotal Phase 3 TransportNPC™ clinical trial was designed in close collaboration with regulatory agencies, Key Opinion Leaders, patient advocacy groups and patient communities in order to best enable a potentially positive outcome. With the severity of this disease and the limited treatment options currently available to patients, there is a significant unmet need for a product that can treat the systemic and neurologic manifestations of NPC. We continue to be encouraged by the potential of Trappsol^® Cyclo™ and are leveraging this critical product, its mechanism of action and route of administration with the hope of providing a much-needed clinical benefit to the NPC patient population. We are dedicated to progressing this trial and look forward to the new insights it will provide,” commented Dr. Lise Lund Kjems.

The Company’s ongoing pivotal Phase 3 study, TransportNPC™, is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol^® Cyclo™ administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1. The Phase 3 study intends to enroll at least 93 pediatric (age 3 years and older) and adult patients with NPC1 in at least 23 study centers in 9 countries. Eligible patients will be randomized 2:1 to receive either Trappsol^® Cyclo™ or a 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.

This study has dual primary objectives. For the U.S. and countries following FDA guidance, the primary objective is to evaluate the efficacy of Trappsol^® Cyclo™ versus placebo using a 4-Domain Niemann-Pick Disease Type C Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech and Swallow]) composite score at Weeks 48 and 96. For the EU and countries following EMA guidance, the primary objective is to evaluate the efficacy of 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) at Weeks 48 and 96.

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 patients from 3 to less than 18 years of age with NPC Type C1, and in addition, to include a single-arm sub-study of patients from birth to less than 3 years of age with NPC Type C1 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 only be conducted in the EU and countries following EMA guidelines.

Cyclo Therapeutics recently formed a Global Steering Committee (GSC) comprised of Key Opinion Leaders and experts on lysosomal storage diseases to guide the pivotal Phase 3 global clinical development program of Trappsol^® Cyclo™ for the treatment of NPC.

Cyclo Therapeutics received Orphan Drug Designation for Trappsol^® Cyclo™ to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S. The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the U.S. upon marketing authorization.

For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.

About WORLDSymposium™

WORLDSymposium™ is an annual research conference dedicated to lysosomal diseases. WORLD is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium has grown to an international research conference that attracts over 2000 participants from more than 50 countries around the globe. For more information, please visit: worldsymposia.org.

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 four 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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