Cyclo Therapeutics Submits Investigational New Drug Application to U.S. FDA to Advance Trappsol® Cyclo™ in Phase 2 Study for Treatment of Alzheimer’s Disease

Rhea-AI Summary

Cyclo Therapeutics (NASDAQ: CYTH) has submitted an initial investigational new drug (IND) application to the FDA for Trappsol® Cyclo™, targeting early Alzheimer’s Disease. The Phase 2 study design is based on clinical data from Niemann-Pick Disease Type C and 18 months' experience with a late-onset Alzheimer’s patient. The drug aims to address cholesterol accumulation and oxidative stress, conditions shared in both diseases. The company highlights significant unmet needs in Alzheimer’s treatment, positioning Trappsol® Cyclo™ as a potential solution.

Positive

• IND application for Trappsol® Cyclo™ submitted to FDA.
• Phase 2 study designed based on extensive clinical data.
• Trappsol® Cyclo™ shows promise in managing cholesterol transport related to Alzheimer’s Disease.

Negative

• No established treatments or cures for Alzheimer’s Disease currently available.
• Regulatory and clinical trial risks may affect drug approval timelines.

– Biologic similarities demonstrated between Niemann-Pick Disease Type C and Alzheimer’s Disease, including cholesterol accumulation in regions of the brain, elevated levels of Tau in CSF, and amyloid plaques in the brain, bolsters rationale for studying Trappsol^® Cyclo™ for the treatment of early Alzheimer’s Disease

– Phase 2 protocol was developed based clinical data from completed and ongoing trials in Niemann-Pick Disease Type C and on 18 months of data from a single late-onset Alzheimer’s patient being administered monthly intravenous doses of Trappsol^® Cyclo™ under compassionate use

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 submitted its initial investigational new drug (“IND”) application with the U.S. Food and Drug Administration (“FDA”) for a Phase 2 study of Trappsol^® Cyclo™ for the treatment of early Alzheimer’s Disease (AD).

“We are excited to have submitted our Initial IND to evaluate Trappsol^® Cyclo™ for the treatment of Alzheimer’s Disease. The feedback and recommendations received from our Type B Meeting with the FDA have provided valuable input that we believe positions us for success in advancing this asset. We have taken another step towards commencing our Phase 2 study and, importantly, bringing a much-needed treatment option to patients and families,” commented Michael Lisjak, Chief Regulatory Officer, Senior Vice President for Business Development of Cyclo Therapeutics.

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 AD are associated with cholesterol metabolism. Cholesterol imbalance in AD patients is well known, and significant research exists suggesting these imbalances are responsible for 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. AD shares features with NPC-1, 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 (CSF). Cyclo Therapeutics is currently testing the same investigational Trappsol^® Cyclo™ drug in clinical trials for the treatment of Niemann-Pick Disease Type C1 (NPC-1). Taking the place of the defective NPC-1 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.

“There remain significant unmet needs within the Alzheimer’s Disease community, which we believe Trappsol^® Cyclo™ is well-positioned to address. We are committed to advancing this important program as quickly and efficiently as possible following feedback from FDA in the coming weeks,” added N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics.

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211116005289/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 Phase 2 study for CYTH?

The Phase 2 study aims to evaluate the efficacy of Trappsol® Cyclo™ in treating early Alzheimer’s Disease.

What are the key findings supporting CYTH's application for Alzheimer’s treatment?

Biologic similarities between Niemann-Pick Disease Type C and Alzheimer’s, including cholesterol accumulation and increased Tau levels.

What is the expected impact of Trappsol® Cyclo™ on Alzheimer’s Disease?

Trappsol® Cyclo™ may help address cholesterol imbalances and oxidative stress associated with Alzheimer’s Disease.

When was the IND application for CYTH submitted?

The IND application for Trappsol® Cyclo™ was submitted on November 16, 2021.

What are the next steps for CYTH following the IND application?

Cyclo Therapeutics anticipates receiving FDA feedback to advance the Phase 2 study of Trappsol® Cyclo™.