Cyclo Therapeutics : Factsheet

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Investment Summary

Leveraging over 3 decades of experience with cyclodextrins to advance clinically de-risked programs towards approval in diseases with unmet medical need.

Platform technology has demonstrated to be safe and effective with over 10 years of patient exposure.

FDA: Orphan Drug Designation (ODD), Fast-Track, Rare Pediatric Disease Designation, potential PRV; EMA: ODD and adopted PIP

Enrolling and dosing patients in Pivotal Phase 3 study in Niemann-Pick Disease Type C.

Significant market opportunity with no approved therapy to treat both systemic and neurological manifestations of NPC.

Pipeline expansion into Alzheimer's Disease (AD), patent filed globally and is currently being prosecuted.

Received Study May Proceed from FDA December 2021, FPI targeted H2 2022.

Cash Balance	Market Cap	Shares Outstanding	Average Volume
$11.8M	~19M*	8.4M	~42K
			*As of May 31, 2022

Our Mission

Our Team

We are dedicated to developing life-changing medicines through science and innovation for patients and families living with challenging diseases.

Our lead therapeutic asset, 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. 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 that it can be further processed and excreted out of cells.

N. Scott Fine	Joshua M. Fine	Lisa Lund Kjems, MD, PhD
CFO	CMO
CEO

	Michael Lisjak	Lori McKenna Gorski
Jeffrey L. Tate, PhD	Global Head of Patient
CRO, SVP for Business
Advocacy
Director, COO, CQO	Development

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 Disease Type C

Trappsol® Cyclo™ Alzheimer's Disease

(Pivotal Phase 3 Study)

Site activation ongoing, enrolling and dosing patients.

Open IND for Phase 2 study

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

Orphan Drug Designation in EU | EMA Pediatric Investigational Plan Adopted

Therapeutic Focus Areas

Niemann-Pick Disease Type C (NPC)

Rare, fatal and progressive genetic disorder affecting the brain, liver and spleen 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

1/100,000 instances of NPC

0 U.S. approved NPC therapies

1 E.U approved therapy with no systemic effects

Alzheimer's Disease

The most common form of

Dementia

An irreversible, progressive neurologic disorder that slowly degrades memory, thinking and social skills that affects a person's ability to function independently

5 million+ people affected in the U.S.1

6th leading cause of death in the U.S.1

500,000 new cases every year2

13.8 million cases projected by 20501

Similarities: (1) Cognitive decline, (2) Elevated levels of tau, (3) Amyloid plaques

1. https://www.alz.org/alzheimers-dementia/facts-figures | 2. https://www.brightfocus.org/alzheimers/article/alzheimers-disease-facts-figures

cyclotherapeutics.com | NASDAQ: CYTH | Investor Relations: JTC Team, [e-mail]