Cyclo Therapeutics Provides Business Update and Reports Full Year 2021 Financial Results

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 reported its financial results for the full year 2021 and provided a business update.

“Over the course of the past year, we have made significant progress across our clinical programs. We are pleased by the continued advancement of our ongoing pivotal Phase 3 study evaluating Trappsol^® Cyclo™ as a potential treatment for NPC, TransportNPC™. With the added expertise in our Global Steering Committee, to help guide this important study, we are encouraged and remain focused on driving it to completion with the potential to provide the first U.S. FDA approved therapy for NPC,” commented N. Scott Fine, CEO of Cyclo Therapeutics. “Additionally, with the clearance of our IND from the FDA, we are working towards launching our Phase 2 study for our Alzheimer’s Disease asset. 2022 is poised to be an exciting year as we execute on the milestones ahead and continue to build momentum.”

Recent Highlights

• Announced the formation of Global Steering Committee (GSC) comprised of leading experts to advise on the global Phase 3 clinical development program for Trappsol^® Cyclo™ in NPC;
• Appointed Caroline Hastings, M.D. as Global Principal Investigator for ongoing TransportNPC™ study evaluating Trappsol^® Cyclo™ for the treatment of NPC;
• Received clearance from the U.S. Food and Drug Administration (FDA) of its investigational new drug (IND) application to advance its Phase 2 Study of Trappsol^® Cyclo™ for the treatment of Alzheimer’s Disease;
• Named preeminent neuroscientist and world-renowned researcher, Cynthia A. Lemere, PhD, as Senior Advisor for advancement of Alzheimer’s Disease asset.
• Closed a $11.7 million underwritten public offering.

Trappsol^® Cyclo™ Clinical Program Update

Trappsol^® Cyclo™ is a proprietary formulation of hydroxypropyl beta cyclodextrin, used intravenously (IV) and currently in development for the treatment of NPC, a rare genetic disorder causing cholesterol accumulation in lysosomes of cells, organ dysfunction and premature death.

Niemann-Pick Disease Type C1 Development Program

In June 2021, the Company commenced the TransportNPC™ study evaluating Trappsol^® Cyclo™ for the treatment of NPC1, a rare, progressive and fatal genetic disorder. Initial sites are in the U.S.

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. Data seen to-date provide additional support for the capacity of Trappsol^® Cyclo™ to stabilize disease progression with home-based intravenous infusions as well as for a favorable safety profile of more than two years in NPC.

The Company recently announced the formation of a Global Steering Committee (GSC) to guide the pivotal Phase 3 global clinical development program of Trappsol^® Cyclo™ for the treatment of NPC. As the Global Principal Investigator for the TransportNPC™ study, Caroline Hastings, MD serves as the senior scientific and clinical expert for the trial and will also chair the GSC. Other members of the Companies GSC include leading experts and renowned Key Opinion Leaders.

Additionally, Cyclo Therapeutics 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 in the randomized study, and in addition, to include a single-arm open-label 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 sub-study in patients from birth to less than 3 years of age will only be conducted in the EU and countries following EMA guidelines.

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

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.

Alzheimer’s Disease Asset

Cyclo Therapeutics is also planning to evaluate Trappsol^® Cyclo™ for the treatment of Alzheimer’s Disease, targeting the reduction of amyloid beta and tau. In December 2021, the Company received IND clearance from the U.S. FDA to advance its Phase 2 study of intravenous Trappsol^® Cyclo™ for the treatment of early Alzheimer’s Disease. The Company expects to commence this Phase 2 study and enroll patients in 2022.

Many of the known risk factors for Alzheimer’s disease are associated with cholesterol metabolism. Cholesterol imbalance in Alzheimer’s patients is well known, and significant research exists, suggesting these imbalances are responsible for amyloid beta (Aβ) and tau accumulation. Furthermore, neurons, because of their high metabolic demands, experience an increased level of oxidative stress. Oxidative stress has also been linked to abnormal cholesterol accumulation and processing.

Summary of Financial Results for Full Year 2021

Net loss for the year ended December 31, 2021 was approximately $14.3 million. Research and development expenses increased 50% to $9.2 million for the year ended December 31, 2021, from $6.1 million for the year ended December 31, 2020. The increase in research and development expense is due to increased activity in the Company’s international clinical program and U.S. clinical trials. The Company expects research and development costs to further increase in 2022 as we continue to seek regulatory approval for the use of Trappsol^® Cyclo™ in the treatment of NPC and Alzheimer’s disease.

The Company ended the year with approximately $16.6 million of cash.

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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