GAINESVILLE, Fla. / Mar 20, 2023 / 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 2022 and provided a business update.

“We continue to be extremely active and engaged with the NPC patient and scientific communities and believe these valuable interactions underpin the significant progress we have seen with the advancement of our pivotal Phase 3 program. Importantly, this momentum gives us the line of sight necessary to confirm our goal of completing enrollment before year end, a monumental milestone for the Company. Further we continue to have encouraging strategic discussions across the industry,” commented N. Scott Fine, CEO of Cyclo Therapeutics. “I am extremely proud of our entire team. As we think about the remainder of 2023, we believe this will be a breakthrough year for our company, driving value for all stakeholders. We continue to be cognizant of the markets but believe we are well positioned strategically. We have not been affected by the recent activity in the Banking Sector, and our commitment to driving our programs forward remains unwavering.”

Recent Pipeline Highlights

• Progressed patient enrollment to 40% complete in the ongoing TransportNPC™ study completed and dosing ongoing across multiple regions;
• Initiated patient dosing in Phase 2b Study of Trappsol^® Cyclo™ for the treatment of early Alzheimer’s Disease; and
• Presented poster titled “TransportNPC: A Phase 3 Global Trial of Trappsol^® Cyclo™ Administered Intravenously to Patients with Niemann-Pick Disease Type C1 (NPC1),” at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting.

Trappsol^® Cyclo™ Clinical Program Update

Trappsol^® Cyclo™ is the Company’s 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, and early Alzheimer’s Disease (AD), where disrupted lipid pathways play a key role in the etiology and disease progression.

Niemann-Pick Disease Type C1 Development Program

The Company continues to advance enrollment in its ongoing pivotal Phase 3 study, TransportNPC™. 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.

To date, the Company remains on track for expected completion of enrollment by year end 2023.

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.

For more information about the Company’s Trappsol^® Cyclo™ clinical program for the treatment of NPC1, visit www.ClinicalTrials.gov and reference identifiers NCT02939547, NCT02912793, NCT03893071 and NCT04860960.

Alzheimer’s Disease Development Program

The Company’s ongoing 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.

For more information about the Company’s Trappsol^® Cyclo™ clinical program for the treatment of early AD and Expanded Access program, visit www.ClinicalTrials.gov and reference identifiers NCT05607615 and NCT03624842, respectively.

Summary of Financial Results for the Full Year 2022

Net loss for the year ended December 31, 2022 was approximately $15.5 million. Research and development expenses decreased 2% to $9.0 million for the year ended December 31, 2022, from $9.2 million for the year ended December 31, 2021. The changes in research and development expenses relate to the timing of startup costs in our clinical programs. We expect research and development costs to increase in 2023 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 $1.5 million of cash. Pro-Forma year end cash of $5.2 million post the financing completed on with single institutional investor in January of 2023.

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 (NCT05607615) 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.