NASDAQ: CYTH cyclotherapeutics.com

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Forward-Looking Statements

Some of the information in this presentation relates to future events or future business and financial performance. Such statements constitute forward-looking information within the meaning of the Private Securities Litigation Act of 1995. Such statements can be only predictions and the actual events or results may differ from those discussed due to, among other things, the risks described in the public filings and other publications of Cyclo Therapeutics, Inc. Forward-looking statements are identified by words such as "anticipates", "projects", "expects", "plans", "intends", "believes", "estimates", "target", and other similar expressions that indicate trends and future events.

The market data and certain other statistical information used throughout this presentation are based on independent industry publications, governmental publications, reports by market research firms or other independent sources. Some data are also based on the Company's good faith estimates. In addition, this presentation includes summaries of scientific activities and outcomes that have been condensed to aid the reader in gaining general understanding.

The information about Cyclo Therapeutics, Inc. and its subsidiaries is solely for information purposes and is not to be construed as an offer to sell or the solicitation of an offer to buy any security in any state.

Factors that could cause the Company's results to differ materially from those expressed in forward looking statements include, without limitation, the Company's need for additional capital; the Company's reliance on its Trappsol® Cyclo product, which may never receive regulatory approval; the Company's ability to commercialize any of its proposed drug products if it receives regulatory approval; the outcome of the Company's clinical trials, which may not support the Company's product claims or may result in adverse side effects; the cost and timing of the Company's clinical trials; the Company's reliance on third parties to conduct clinical trials and to produce its products; and other risks associated with being a clinical stage biotechnology company.

This presentation is not to be copied, transmitted, displayed, distributed (for compensation or otherwise), or altered in any way without the prior written consent of Cyclo Therapeutics, Inc.

NASDAQ: CYTH cyclotherapeutics.com

Company Snapshot

Who:In 1990, the company was formed as Specialty Fine Chemical business specializing in cyclodextrins. In 2014, the business was expanded into a biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with challenging diseases.

What: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.

Why:Because cholesterol is so important to the normal function of our cells, its synthesis and degradation is tightly controlled by an array of cellular processes. When there is an imbalance in cholesterol synthesis or metabolism, cells and organs may not function properly, leading to disease or death.

How: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.

NASDAQ: CYTH cyclotherapeutics.com

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Currently Targeting 2 Serious Diseases with Unmet Medical Need

Niemann-Pick Disease Type C

Fatal and progressive genetic disorder Orphan indication affecting >9,000 in 80 countries (~400 in U.S. / 320 EU5) 1

Alzheimer's Disease

6th leading cause of death affecting

5 million people in the U.S.2

Platform technology has potential to fuel pipeline expansion opportunities

  1. April 2021, Tessellon Inc. (former Kantar Health experts with 25+ years of epidemiology and forecasting experience), (www.Tessellon.com); Exhaustive literature search with a broad range of MESH terms in United States + 79 other countries.
  2. https://www.alz.org/alzheimers-dementia/facts-figures

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Platform Technology Pipeline:

Trappsol® Cyclo allows for a multiple shots on goal model

Program

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Milestones

Trappsol® Cyclo

Niemann-Pick

48-week comparative

Disease Type C

interim analysis planned for 1Q 2025

Alzheimer's

First patient enrolled and

Trappsol® Cyclo

Disease

dosed in Q1 2023 for Phase 2b study

Ongoing Collaboration with University of the Witwatersrand, Johannesburg to

Advance Trappsol® Cyclo Platform and Explore Pipeline Expansion Opportunities

Orphan Drug Designation in U.S. | Fast Track Status in U.S. | Potential for Priority Review Voucher (PRV) in U.S

Orphan Designation in EU | EMA Pediatric Investigational Plan Adopted

NASDAQ: CYTH cyclotherapeutics.com

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Leadership Team with Proven Experience

N. Scott Fine

Joshua M. Fine

Chief Executive Officer & Director

Chief Financial Officer

Karen Mullen, FFPM

Michael Lisjak

Interim Chief Medical Officer

Chief Regulatory Officer

Jeffrey L. Tate, Ph.D.

Lori Gorski

Chief Operating Officer, Chief Quality Officer & Director

Global Head of Patient Advocacy

NASDAQ: CYTH cyclotherapeutics.com

Scientific Advisory Board

Rita Colwell, Ph.D.

Co-Chair

Internationally recognized scientist, microbiologist and founder of CosmosID, a privately held bioinformatics firm.

Distinguished University Professor at U. Maryland and Johns Hopkins University. Former Director, National Science Foundation (1998 - 2006). National Medal of Science awardee. Member, US National Academy of Sciences.

Benny Liu, M.D.

Key Opinion Leader in Niemann-Pick Disease Type C

Gastroenterologist at Alameda Healthy System, CA and Highland Hospital. Globally recognized expert in lipid metabolism.

First to discover that cyclodextrins release cholesterol from cells using an animal model. Assistant Clinical Professor, UCSF.

Gerald F. Cox, M.D., Ph.D.

Internationally Renowned for Clinical Drug Development

Seasoned biotechnology executive with 20-year successful track record of drug development for rare genetic diseases and extensive worldwide regulatory experience

NASDAQ: CYTH cyclotherapeutics.com

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Sharon H. Hrynkow, Ph.D.

Co-Chair

Neuroscientist with more than 25 years' experience in global health arena, public and private sectors.

Senior executive at NIH.

First president of non-profit Global Virus Network.

Former Member of President's Council of Advisors on Science and Technology.

5 years at Cyclo Therapeutics leading clinical and scientific programs.

Caroline Hastings, M.D.

Key Opinion Leader in Niemann-Pick Disease Type C

Pediatric hematologist oncologist, Director of NeuroOncology, and Professor of Pediatrics, UCSF Benioff Children's Hospital Oakland.

First physician in US to use cyclodextrins for treatment in NPC, compassionate use.

Advisor to US and Australian NPC Advocacy organizations and to physicians globally on NPC.

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Niemann-Pick

Disease Type C

Ongoing Pivotal

Phase 3 Study

NASDAQ: CYTH cyclotherapeutics.com

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NPC: A Debilitating Disease with Fatal Outcomes

  • Rare, fatal and progressive genetic disorder affecting the brain, liver, spleen and lungs
  • Characterized by a defect in the NPC1 protein
  • Cholesterol and lipids accumulate in cells of major organs and tissues
  • Leading to cell and tissue dysfunction

0

U.S. Approved NPC Therapies

1

EU Approved Therapy with No

Systemic Effects

Market Opportunity1

United States: $300 Million | Worldwide: $600 Million

Incidences

1/100,000 (~35 per year in U.S.)

Of Diagnosis

  • 3% are age 3 and below
  • 97% are age 3 and above
  • 60% age 16 and above

Median Survival

Early Infantile (2m-2): 4.6y

Late Infantile (3-6): 9.4y

Juvenile (7-15): 15.4y

Adolescent/Adult (16+): 12.2y

1: Data on file Cyclo Therapeutics

NASDAQ: CYTH

*Scope: United States + 79 other countries; *Commissioned Tessellon Inc - former Kantar Health experts with 25+ years of

epidemiology and forecasting experience, (www.Tessellon.com); *Exhaustive literature search with a broad range of MESH terms.

cyclotherapeutics.com

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Trappsol® Cyclo as a potential therapy for NPC Suggestive evidence of safety and systemic and neurological benefit

Mechanism of Action: Trappsol® Cyclo , with its cyclic structure, facilitates the transport of cholesterol between cells and subcellular structures thus circumventing blockades and enabling the normal function of cells and tissues.

  • Pre-clinicaldata: Demonstrates the amount of HPβCD getting into the brain is at a safe and efficient concentration in order to enhance

transfer of cholesterol between (intra)cellular membranes and therefore supportive of Trappsol® Cyclo having a potential safe and effective benefit for the neurological symptoms of NPC.

Phase 1 Study (CTD-TCNPC-101): Data suggests that Trappsol® Cyclo has an acceptable well-tolerated safety profile and demonstrates clearance of trapped liver cholesterol, indicates that cholesterol turnover is normalized and cholesterol synthesis rate adjusted for the whole body, shows reduced changes in metabolite level is indicative of reduced cholesterol storage and indicates normalization of cholesterol storage in brain neurons. These data are suggestive of a potential benefit for both the systemic and neurological symptoms of NPC.

Phase 2 Study (CTD-TCNPC-201): Data demonstrates that Trappsol® Cyclo has an acceptable well-tolerated safety profile and that a potential therapeutic benefit may be observed at or before 48 weeks of treatment compared to disease progression. Although studied in a small patient sample size, provides compelling evidence of disease stabilization or improvement for the 5D-NPC-SS,CGI-I and SARA scales.

  • Ongoing Extension Study (CTD-TCNPC-102): Provides long-term safety data demonstrating the product is well-tolerated along with data suggestive of durability of effect.

Conclusion

The data obtained from preclinical studies and Cyclo Therapeutics completed and ongoing clinical studies suggest Trappsol® Cyclo is well-tolerated and has the potential to treat both the systemic and neurological symptoms of NPC. The data from our early phase studies and OLE are currently being confirmed in our ongoing Phase 3 Study (CTD-TCNPC-301).

NASDAQ: CYTH cyclotherapeutics.com

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Trappsol® Cyclo

Enables the Effective Transport of Cholesterol Out of Cells

Under normal conditions, NPC1, located on the late endosomal/lysosomal (LE/L) compartments, regulates cholesterol efflux (Maetzel et al, 2014) (see left panel below). In NPC, mutations in the NPC1 gene on both alleles lead to accumulation of cholesterol in the LE/L compartments by inhibiting its efflux, and to a block in autophagic flux, which causes accumulation of autophagosomes and autophagy substrate due to impaired formation of amphisomes (see middle panel below). Trappsol® Cyclo mediated cholesterol release independent of the function of both NPC1 and NPC2 proteins, circumventing blockades in cholesterol transport, and therefore has the potential to bring significant benefit to patients with NPC (see right panel below).

Cholesterol as measured by Filipin staining at Baseline and after 7 doses over 14 weeks

Baseline

14 Weeks

Baseline

14 Weeks

Baseline

14 Weeks

1500

2500

2500

Mg/kg

Mg/kg

Mg/kg

(Mild)

(Marked)

(Marked)

Reduction

Reduction

Reduction

The lack of light blue represents the clearing of cholesterol from cells

NASDAQ: CYTH

Maetzel et al., 2014

cyclotherapeutics.com

Source : Study 101

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Cyclo Therapeutics Inc. published this content on 18 March 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 18 March 2024 13:53:08 UTC.