Cyclo Therapeutics Announces First Patient Dosed in Phase 2b Study of Trappsol® Cyclo™ for the Treatment of Early Alzheimer’s Disease

Rhea-AI Summary

Cyclo Therapeutics (NASDAQ: CYTH) has enrolled and dosed the first patient in its Phase 2b study of Trappsol^® Cyclo™ for early Alzheimer’s disease. The study will analyze the safety and efficacy of the drug over six months, targeting about 120 patients experiencing cognitive decline. This milestone follows promising results from previous studies highlighting Trappsol's ability to manage cholesterol transport, a critical factor in neurodegenerative diseases. The company is also testing Trappsol in a Phase 3 trial for Niemann-Pick disease Type C.

Positive

• First patient enrolled in Phase 2b study for Trappsol Cyclo™ targeting early Alzheimer's disease.
• Study evaluates safety and efficacy over 6 months with 120 patients expected.
• Trappsol Cyclo™ has shown promising results in managing cholesterol transport relevant to Alzheimer’s.

Negative

• Potential regulatory hurdles in obtaining approval for clinical trials.
• Success in demonstrating efficacy in clinical trials remains uncertain.

– 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

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 first patient has been enrolled and dosed in its Phase 2b study of Trappsol^® Cyclo™ for the treatment of early Alzheimer’s disease (AD), targeting the disrupted lipid pathways and the reduction of amyloid beta and tau.

“The start of this study is a significant milestone for our Company and the Alzheimer’s disease community. Based on the encouraging results Trappsol^® Cyclo™ has demonstrated to date we believe we are well-positioned to address the unmet medical need that patients, families and physicians are facing,” commented Lise Kjems M.D. PhD. Chief Medical Officer.

The Phase 2b study is a U.S. multicenter, randomized, placebo-controlled, double-blind, parallel group, 6-month study evaluating the safety, tolerability, and potential efficacy of monthly Trappsol^® Cyclo™ infusions in patients with early Alzheimer’s disease. The study will enroll approximately 120 patients who have shown evidence of progressive cognitive decline in the last year as determined by serial cognitive test scores, if available, or patient or informant/caregiver/study partner report as documented by the Investigator. Enrolled patients will be randomized across three study arms: 500 mg/kg or 1000 mg/kg of Trappsol^® Cyclo™ and placebo. The study will consist of a screening period, treatment period up to 24 weeks, and a safety follow-up period.

Trappsol^® Cyclo™ is the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin, and in multiple clinical studies, has shown encouraging results to effectively manage the transportation of cholesterol, a known defect in neurodegenerative diseases. Many of the known risk factors for Alzheimer’s disease are associated with cholesterol metabolism.

Cyclo Therapeutics is currently testing the same investigational Trappsol^® Cyclo™ drug in a Phase 3 clinical trial (TransportNPC™) 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. 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 living with 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 conducting a Phase 2b clinical trial using Trappsol^® Cyclo™ intravenously in early Alzheimer’s disease based on encouraging data from an Expanded Access program for 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/20230109005220/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 2b study for Trappsol Cyclo™?

The Phase 2b study aims to evaluate the safety, tolerability, and potential efficacy of Trappsol Cyclo™ in treating early Alzheimer's disease.

How many patients will be enrolled in the Alzheimer's study?

The Phase 2b study will enroll approximately 120 patients.

What are the expected results from the Phase 2b study?

The study aims to provide insights into the drug's safety and efficacy over a 6-month treatment period.

How does Trappsol Cyclo™ relate to cholesterol management in Alzheimer's?

Trappsol Cyclo™ is designed to manage cholesterol transport, addressing a key defect in neurodegenerative diseases like Alzheimer's.

What is the stock symbol for Cyclo Therapeutics?

The stock symbol for Cyclo Therapeutics is CYTH.