NASDAQ: CYTH cyclotherapeutics.com

Forward-Looking Statements

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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.

Certain information set forth in this presentation contains "forward-looking information", including "future-oriented financial information" and "financial outlook", under applicable securities laws (collectively referred to herein as forward-looking statements). Except for statements of historical fact, the information contained herein constitutes forward-looking statements and includes, but is not limited to, the (i) projected financial performance of Cyclo Therapeutics, Inc.; (ii) completion of, and the use of proceeds from, the sale of the securities being offered hereunder; (iii) execution of the Cyclo Therapeutics, Inc. vision and growth strategy, including with respect to future M&A activity and global growth; (iv) sources and availability of third-party financing; (v) renewal of the Cyclo Therapeutics, Inc. current customer, supplier and other material agreements; and (vi) future liquidity, working capital, and capital requirements.

Forward-looking statements are provided to allow potential investors the opportunity to understand management's beliefs and opinions in respect of the future so that they may use such beliefs and opinions as a factor in evaluating the proposed investment.

These statements are not a guarantee of future performance, and undue reliance should not be placed on them. Although forward-looking statements contained in this presentation are based upon what management of Cyclo Therapeutics, Inc. believes are reasonable assumptions, such forward-looking statements necessarily involve known and unknown risks and uncertainties, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or result expressed or implied by such forward-looking statements. Cyclo Therapeutics, Inc. undertakes no obligation to update these forward-looking statements if circumstances or management's estimates or opinions should change except as required by applicable securities laws.

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

Platform Technology Pipeline:

4

Trappsol® Cyclo

allows for a multiple shots on goal model

Program

Indication

Preclinical

Phase 1

Phase 2

Phase 3

Milestones

Trappsol® Cyclo

Niemann-Pick

Disease Type C

Trappsol® Cyclo

Alzheimer's

Disease

48-week comparative interim analysis planned for 1H 2025

First patient enrolled and dosed in Q1 2023 for Phase 2b study

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

5

Niemann-Pick

Disease Type C

Ongoing Pivotal

Phase 3 Study

NASDAQ: CYTH cyclotherapeutics.com

NPC: A Debilitating Disease with Fatal Outcomes

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  • 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

NASDAQ: CYTH cyclotherapeutics.com

1: Data on file Cyclo Therapeutics

*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.

Trappsol® Cyclo as a potential therapy for NPC

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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

Trappsol® Cyclo

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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

2500

Baseline

14 Weeks

2500

Baseline

14 Weeks

1500

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

Trappsol® Cyclo Summary of completed studies and OLE

9

Study 101

Study 201

Study 102

Phase 1 study in NPC patients age

18 years and older showed Trappsol® Cyclo was well- tolerated with an acceptable safety and tolerability profile

  • After IV infusion, the drug is detectable in the cerebrospinal fluid within hours after the start of infusion
  • Cholesterol synthesis and metabolism affected, and cholesterol cleared from cells, mimicking effects from nonclinical studies (in vitro and in vivo) in NPC models
  • Publication to the Molecular Genetics and Metabolism Report: Intravenous 2-hydroxypropyl-β-cyclodextrin

(Trappsol® Cyclo ) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial (2022)

Consistent pharmacodynamic

effects and safety profile

observed in a 48-week Phase 1/2 study in NPC patients aged 2 years and older

  • 100% of patients assessed by treating physicians to be either stable or improved
  • 8 of 9 (89%) completer patients showed Improvements over disease progression in the 17D-NPC-SS
  • Based on totality of data from the Phase 1 and Phase 2 studies, the 2000 mg/kg dose was selected for the Phase 3 study
  • Publication to the Molecular Genetics and Metabolism

Report: The Long-term administration of intravenous Trappsol® Cyclo (HPβCD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial (2023)

Has the potential to treat both the systemic and neurological manifestations of NPC and is well-tolerated with an acceptable safety and tolerability profile

  • The available data from the OLE study indicate that the study medication has an acceptable tolerability profile
  • Although the number of patients is small, the data for the 5-Domain clinical severity scores (5-D-NPC-CSS)

does show a potential for stabilization and slower disease progression following administration of Trappsol® Cyclo than may be otherwise expected based on published literature on the natural course of the disease over the longer term, therefore providing evidence of durability of effect

NASDAQ: CYTH cyclotherapeutics.com

Phase 1/2 Study (CTD-TCNPC-201)

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Potential benefit may be observed at or before 48 weeks

Data Included

9 completers (2 patients in the 1500 mg/kg group; 4 patients in the 2000 mg/kg group; 3 patients in the 2500 mg/kg group)

Three patients were excluded from this analysis due to 1 only having baseline data and 2 patients discontinued prior to Week 48 for non-safety reasons

Data represented in above graph is from different studies. Solid line is from Cyclo Therapeutics CTD-TCNPC-201 Study. The dashed lines are disease progression rates identified from the following 3 sources:

  • In the most recent studies on disease progression in 2 reference patient cohorts, both with and without baseline treatment by miglustat, an average annual progression of 2.94 points (21 patients:Ory, 2017) and 2.92 points (36 patients: Mengel, 2020) on the 17D scale was found, with the 5D-NPC-CSS scale showing a progress of 1.5 units per year (Mengel, 2020).
  • The placebo group in the Orphazyme Phase 2/3 study (16 patients: Mengel, 2021) showed an annual progression on the 5D-scale of 2.15 points and on the 17D-scale (excluding hearing domains) of 2.8 points. The 17D-NPC-CSS parameter is in good agreement with the 2 natural history cohorts mentioned above, while the 5D-NPC-CSS parameter is higher than in the 2 earlier studies (Mengel, 2021).
  • The predicted disease progression of Cyclo Therapeutics Study CTD-TCNPC-201 cohort (completers) without treatment, based on 50% of baseline ASIS.

NASDAQ: CYTH cyclotherapeutics.com

Key Outcomes

  • Potential therapeutic benefit which may be observed at or before 48 weeks of treatment compared to disease progression
  • The small cohort size (9 patients) limits extensive analysis of the changes within the study
  • Importantly, transient variations cannot be separated from disease progression, but are expected to be reduced in a larger cohort (the ongoing Phase 3 CTD-TCNPC-301)

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Cyclo Therapeutics Inc. published this content on 01 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 02 May 2024 10:43:19 UTC.