we are
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 Therapeutic
Technology
Platform technology has demonstrated to be safe and effective with over 10 years of patient exposure
Completion of enrollment in Pivotal Phase 3 study expected by year end 2023
Significant market opportunity with no approved therapy to treat both systemic and neurological manifestations of NPC
FDA: Orphan Drug Designation (ODD), Fast-Track, Rare Pediatric Disease Designation, potential PRV; EMA: ODD and adopted PIP
Alzheimer's Disease
Patient enrollment and dosing in Phase 2b study underway
Pipeline expansion into Alzheimer's Disease (AD), patent filed globally and is currently being executed
Cash Balance1 | Market Cap3 | Shares Outstanding2 | Average Volume3 |
$5.2M | ~$9M | 10.5M | ~91K |
1: Pro Forma as of December 31, 2022 post the financing completed in January with single institutional investor; 2: As of March 15, 2023; 3: As of March 31, 2023 with a closing price of $0.84
Our Mission
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 it can be further processed and excreted out of cells. With the biologic similarities demonstrated between Niemann-Pick disease Type C1 and Alzheimer's disease, including cholesterol accumulation in regions of the brain, elevated levels of Tau in cerebrospinal fluid ("CSF"), and amyloid plaques in the brain, the Company believes Trappsol® Cyclo™ has significant potential to be an effective treatment option for Alzheimer's disease.
Our Team
N. Scott Fine | Joshua M. Fine | Karen Mullen, FFPM | ||
Chief Financial Officer | Interim Chief Medical Officer | |||
Chief Executive Officer | ||||
& Director | ||||
Michael Lisjak | Lori McKenna Gorski | |
Jeffrey L. Tate, PhD | Global Head of Patient | |
Chief Regulatory Officer | Advocacy |
Chief Operating Officer,
Chief Quality Officer
& Director
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)
Completion of enrollment expected by year end 2023
Patient enrollment and dosing underway
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, 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
1/100,000 Instances of NPC (~35 per year in U.S.)
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, cyth@jtcir.com
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Cyclo Therapeutics Inc. published this content on 06 April 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 06 April 2023 20:26:07 UTC.