Cyclo Therapeutics Announces Abstract Accepted for Poster Presentation at the 14th International Congress of Inborn Errors of Metabolism (ICIEM)

Rhea-AI Summary

Cyclo Therapeutics, Inc. (NASDAQ: CYTH) announced that its abstract has been chosen for poster presentation at the 14th International Congress of Inborn Errors of Metabolism in Sydney, Australia, from November 21-24, 2021. The poster will highlight the TransportNPC Phase 3 global trial of Trappsol® Cyclo™ for patients with Niemann-Pick disease type C1 (NPC1). This trial aims to evaluate safety and efficacy in a multicenter study involving at least 93 patients across 9 countries. The study duration is 96 weeks, including an interim analysis at 48 weeks.

Positive

• Abstract selected for presentation at a significant international congress, potentially enhancing Cyclo Therapeutics' visibility.
• Phase 3 trial of Trappsol® Cyclo™ aims to address a critical unmet medical need for patients with NPC1.
• Multicenter study involves a diverse patient population across 9 countries, which may provide robust data.

Negative

• None.

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 its abstract has been selected for poster presentation at the upcoming 14^th International Congress of Inborn Errors of Metabolism (ICIEM) being held November 21-24, 2021 in Sydney, Australia and virtually.

Summarized below is the poster title selected for presentation:

Title: TransportNPC: A Phase 3 global trial of Trappsol^® Cyclo™ administered intravenously to patients with Niemann-Pick disease type C1 (NPC1)
Poster Number: #445
Authors: Gerald Cox, MD, PhD, Member of the Cyclo Therapeutics Scientific Advisory Board and former Acting Chief Medical Officer; Sharon H. Hrynkow, PhD, Chief Scientific Officer, Senior Vice President of Medical Affairs of Cyclo Therapeutics; and Michael Murphy, MD, PhD, Co-Founder, Chief Medical and Scientific Officer of Worldwide Clinical Trials
Presenter: Dr. Gerald Cox

Trappsol^® Cyclo™ is Cyclo Therapeutics’ proprietary formulation of hydroxypropyl beta cyclodextrin and in multiple clinical studies has shown encouraging results in removal of accumulated cholesterol in cells of NPC patients. Taking the place of the defective NPC1 protein, Trappsol^® Cyclo™, with its cyclic structure, facilitates the transport of accumulated cholesterol out of cellular lysosomes so it can be further processed and excreted out of cells.

Trappsol^® Cyclo™ is currently being evaluated in the pivotal, Phase 3 study, TransportNPC™, for the treatment of NPC1, a rare, progressive and fatal genetic disorder. Initial sites are in the U.S. 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 every 2 weeks along with 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.

About the 2021 ICIEM

ICIEM provides an exciting opportunity for all members of the metabolic team to share knowledge, interact, connect and innovate. ICIEM 2021 is taking that to the next level. The 2021 Congress will be a compelling mix of innovative basic science and cutting-edge translational research tempered by pragmatic medical and allied health practitioners in the field. The Congress welcomes all members of the metabolic team including doctors, nurses, dietitians, scientists, counsellors and industry representatives to convene and share the latest knowledge in our field and network.

For more information about the Congress, please visit iciem2021.com.

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211018005178/en/

Investor Contact:

JTC Team, LLC

Jenene Thomas

(833) 475-8247

CYTH@jtcir.com

Source: Cyclo Therapeutics, Inc.

FAQ

What is the purpose of the TransportNPC Phase 3 trial for CYTH?

The TransportNPC Phase 3 trial aims to evaluate the safety and efficacy of Trappsol® Cyclo™ in patients with Niemann-Pick disease type C1.

When and where will the CYTH abstract be presented?

The CYTH abstract will be presented at the 14th International Congress of Inborn Errors of Metabolism in Sydney, Australia, from November 21-24, 2021.

How many patients are targeted for the TransportNPC study by CYTH?

The TransportNPC study aims to enroll at least 93 pediatric and adult patients.

What is Trappsol® Cyclo™ used for in the context of CYTH's research?

Trappsol® Cyclo™ is evaluated for its effectiveness in treating Niemann-Pick disease type C1.

What type of trial design is the TransportNPC study?

The TransportNPC study is a randomized, double-blind, placebo-controlled, multicenter trial.