- Pivotal Phase 3 study of Trappsol^® Cyclo™ for Niemann-Pick Disease Type C (NPC) on track to commence enrollment and dosing in Q2 2021

- Company advancing Alzheimer’s Disease Phase 2 asset towards investigational new drug application (IND) filing anticipated in H2 2021

- Exercise of warrants throughout first quarter further strengthens balance sheet

- Company continues to drive towards achieving multiple clinical and regulatory milestones expected throughout 2021

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 suffering from diseases, today reported its financial results for the first quarter of 2021, and provided a business update.

“Our team continues to build momentum across multiple fronts and is focused on successfully achieving the multiple value-driving milestones we have ahead of us this year. The positive topline results from our Phase 1/2 study in NPC that we reported in March bolsters our confidence as we progress into the next stage of drug development, our pivotal Phase 3 study, which remains on track to commence enrollment and dosing imminently. Coupled with the key appointments of proven industry leaders to our executive team, we are poised to execute on the milestones ahead, achieve success, and make a positive impact on families and patient communities where there remains significant unmet need,” commented N. Scott Fine, CEO of Cyclo Therapeutics.

Recent Highlights

• Appointed Lori McKenna Gorski as Global Head of Patient Advocacy. Ms. Gorski is a leading biotechnology executive with more than 20 years of experience specializing in patient advocacy for rare disease communities;
• Announced the design of the global clinical protocol agreed with U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the pivotal Phase 3 study evaluating Trappsol^® Cyclo™ for the treatment of NPC;
• Received feedback from the FDA supporting the Company’s development strategy to submit an IND application for a Phase 2 study of Trappsol^® Cyclo™ in the treatment of early Alzheimer’s disease, following a positive Type B interaction;
• Announced topline data from its Phase 1/2 clinical trial, which demonstrated promising safety and efficacy results for Trappsol^® Cyclo™ in the treatment of NPC;
• Appointed Russ Belden as Acting Chief Commercial Officer. Mr. Belden is a well-established biotechnology commercialization leader with more than 33 years of industry experience;
• Received a positive opinion from the Paediatric Committee (PDCO) of the EMA on the agreement of a Paediatric Investigation Plan (PIP) for lead candidate, Trappsol^® Cyclo™;
• Appointed Gerald F. Cox, MD, PhD as Acting Chief Medical Officer. Dr. Cox is a seasoned executive with 20-year track record of success in drug development for rare genetic diseases and extensive worldwide regulatory experience;
• Presented favorable data from its clinical and drug development program for Trappsol^® Cyclo™ at the 17th Annual WORLDSymposium 2021; and
• Reported positive efficacy data from the extension protocol with Trappsol^® Cyclo™ in patients with NPC.

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 Type C1 Development Program

In March 2021, the Company reported topline data from its Phase 1/2 clinical trial, which demonstrated promising safety and efficacy results for Trappsol^® Cyclo™ in the treatment of NPC. Of patients who completed the trial, 100% improved or remained stable, and 89% met the efficacy outcome measure of improvement in at least 2 domains of the 17-domain NPC Severity Scale. Pharmacokinetic analysis from the trial data confirmed that Trappsol^® Cyclo™ crosses the blood-brain-barrier after intravenous infusion and further supports neurological benefit.

The Company is now preparing to commence its pivotal Phase 3 study evaluating Trappsol^® Cyclo™ and remains on track to commence enrollment and dose the first patient in the second quarter of 2021. The pivotal Phase 3 study will be 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.

As recently announced, the Company 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, and in addition, to include a single-arm 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 pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.

Expected Upcoming Milestones

• Commence enrollment and dose the first patient in pivotal Phase 3 study for NPC in Q2 2021;
• Commence commercial-scale manufacturing of Trappsol® Cyclo™ in Q3 2021; and
• NPC pivotal Phase 3 study interim analysis topline results expected in Q4 2022.

Cyclo Therapeutics has received Orphan Drug Designation for Trappsol^® Cyclo™ to treat NPC in both the US and EU, and additionally, Fast Track and Rare Pediatric Disease Designations in the US. The Rare Pediatric Disease Designation enables sponsors to receive a Priority Review Voucher (PRV) for another investigational drug upon marketing authorization of the Rare Pediatric Disease drug in the U.S.

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. Following a positive Type B interaction with the FDA, Cyclo Therapeutics received positive feedback supporting the Company’s development strategy to submit an IND application for a Phase 2 study of intravenous Trappsol^® Cyclo™ in the treatment of early Alzheimer’s disease. The Company is on track to file its IND for a Phase 2 study of Alzheimer’s disease in the second half of 2021.

As previously announced, the Company filed an international patent application titled “Methods for Treating Alzheimer’s Disease,” published as International Publication Number: WO 2020/092107 Al.

Expected Upcoming Milestones

• Target filing IND in H2 2021 for potential Phase 2 study evaluating Trappsol^® Cyclo™ for the treatment of Alzheimer’s Disease.

   

Summary of Financial Results for First Quarter 2021

Net loss for the quarter ended March 31, 2021 was approximately $4.0 million. Research and development expenses increased 58% to $3.2 million for the three months ended March 31, 2021, from $2.0 million for the three months ended March 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 2021 as we continue to seek regulatory approval for the use of Trappsol^® Cyclo™ in the treatment of NPC and Alzheimer’s disease.

In December 2020, the Company closed a public offering, which including the exercise of the underwriter’s over-allotment option, resulting in total gross proceeds of $14.4 million. Subsequent to the closing of the public offering through March 12, 2021, warrants to purchase an aggregate of 1,654,184 shares of Common Stock were exercised resulting in additional gross proceeds to the Company of $8.3 million. The Company ended the quarter with approximately $15.5 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 three ongoing 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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