Cyclo Therapeutics Receives IND Clearance from the U.S. FDA to Advance its Phase 2 Study of Trappsol® Cyclo™ for the Treatment of Alzheimer’s Disease

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 it has received notification that its study may proceed from the U.S. Food and Drug Administration (“FDA”) for its initial investigational new drug (“IND”) application for a Phase 2 study of Trappsol^® Cyclo™ for the treatment of early Alzheimer’s disease.

“We are incredibly pleased with the interaction we’ve had with the FDA throughout this initial IND process, culminating in their decision for our proposed Phase 2 program to proceed. We look forward to commencing the study and the potential to address the need for an effective treatment option for all those affected by this devastating disease,” commented Michael Lisjak, Chief Regulatory Officer, Senior Vice President for Business Development of Cyclo Therapeutics.

"Alzheimer’s disease shares characteristics with Niemann-Pick disease Type C1 a neurovisceral, genetic disease in which cholesterol accumulates in lysosomes, including progressive decline in cognitive ability, amyloid beta plaques in the CNS, and increased levels of tau in the cerebrospinal fluid. With the data we’ve amassed to-date in both our Niemann-Pick disease Type C1 clinical program and the 18 months of data under compassionate use for the treatment of Alzheimer’s disease, we believe we are well-positioned for success in advancing Trappsol^® Cyclo™ through the clinic in order to address the unmet medical need that physicians, patients and families are facing,” added Lise Kjems M.D. PhD. Chief Medical Officer.

Trappsol^® Cyclo™ is a proprietary formulation of hydroxypropyl beta cyclodextrin, and in multiple clinical studies, has shown encouraging results to effectively manage the transportation of cholesterol. 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.

“This milestone further demonstrates our vision of utilizing Trappsol^® Cyclo™ as a platform technology and is a strong representation of our team’s commitment to the patient communities and our stakeholders. While our current focus continues to be on the development of novel therapies for two neurodegenerative diseases, Niemann-Pick disease Type C1 and Alzheimer’s disease, future activities will include assessing other viable applications of Trappsol^® Cyclo™ technology in order to expand our development pipeline,” commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics.

Cyclo Therapeutics is currently testing the same investigational Trappsol^® Cyclo™ drug in a Phase 3 clinical trial and a long-term extension study for the treatment of Niemann-Pick disease Type C1, a rare, fatal and progressive genetic disorder. 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. With the biologic similarities demonstrated between Niemann-Pick disease Type C1 and Alzheimer’s Disease, including cholesterol accumulation in regions of the brain, elevated levels of Tau in cerebrospinal fluid (“CSF”), and amyloid plaques in the brain, the Company believes Trappsol^® Cyclo™ has significant potential to be an effective treatment option for Alzheimer’s disease.

About Alzheimer’s Disease

Alzheimer’s disease is a progressive neurologic disorder that causes the brain to shrink (atrophy) and brain cells to die. Estimates vary, but experts suggest that more than 5.5 million Americans, most of them age 65 or older, may have dementia caused by Alzheimer’s. Most people with Alzheimer’s have the late-onset form of the disease, in which symptoms become apparent in their mid-60s. Early-onset Alzheimer’s disease occurs between a person’s 30s and mid-60s and represents less than 10 percent of all people with Alzheimer’s. The early signs of the disease include forgetting recent events or conversations. As the disease progresses, a person with Alzheimer’s disease will develop severe memory and thinking skills impairment, then lose ability to learn, reason, make judgments, communicate and carry out daily activities. Medications may temporarily improve or slow progression of symptoms, however there is currently no treatment that cures Alzheimer’s disease or alters the disease process in the brain.

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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