International Niemann–Pick Disease Alliance

Cyclo Therapeutics Appoints Gerald F. Cox, MD, PhD as Acting Chief Medical Officer

– Seasoned biotechnology executive with 20-year successful track record of drug development for rare genetic diseases and extensive worldwide regulatory experience
– Internationally renowned for clinical development of innovative drugs with broad experience in small molecules, enzyme replacement therapies, antibody therapeutics, gene therapy, and CRISPR-based genome editing

Gainesville, FL – (Businesswire) – March 1, 2021 – Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company developing cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C and Alzheimer’s Disease, today announced the appointment of Gerald F. Cox, MD, PhD as Acting Chief Medical Officer.
Dr. Cox is an internationally renowned biotechnology executive with over 20 years of experience in drug development for rare diseases. Over the course of his career, he has made major contributions to more than 15 Investigational New Drug applications (INDs) and 6 orphan drug marketing authorizations for serious and life-threatening diseases that have generated over $5.0 billion in revenue. He brings with him extensive worldwide regulatory expertise, invaluable clinical acumen, and deep scientific insights.
“As we continue to advance our Trappsol® Cyclo™ clinical development programs, we are incredibly pleased to have secured the interest of Dr. Cox. The expertise and guidance he can provide as we execute on our clinical and regulatory strategies to treat systemic and neurologic manifestations of Niemann-Pick Type C and Alzheimer’s Disease will be invaluable. We look forward leveraging Gerry’s breadth of knowledge and expertise to drive forward these programs as effectively and expeditiously as possible,” commented, N. Scott Fine, CEO of Cyclo Therapeutics.

Dr. Cox is the founder of Gerald Cox Rare Care Consulting, LLC, where since 2018 he has been providing expert advice to small companies in all phases of clinical development for investigational rare disease drugs. From 2016-2018, Dr. Cox was the Chief Medical Officer of Editas Medicine, where he led the clinical development of CRISPR-based genome editing medicines to treat human diseases, including the first approved IND for a CRISPR-based medicine to be delivered in vivo that is designed to treat a genetic form of blindness called Leber congenital amaurosis type 10. Prior to Editas Medicine, Dr. Cox held increasingly senior roles at Genzyme (now Sanofi Genzyme) for over 15 years, advancing to Vice President of Rare Disease Clinical Development. While at Genzyme, he played an instrumental role in the global development and approval of treatments for several lysosomal storage disorders, including the enzyme replacement therapies Aldurazyme® (laronidase) for Mucopolysaccharidosis type I in 2003, Elaprase® (idursulfase) for Mucopolysaccharidosis type II in Japan and the Asia Pacific region in 2007, and Cerezyme® (imiglucerase) for a label expansion in Gaucher disease type 3 in Australia and China in 2016, as well as the substrate reduction therapy Cerdelga® (eliglustat) for Gaucher disease type 1 in 2014. He also led the early clinical development of the enzyme replacement therapies Myozyme® (alglucosidase alfa) for infantile Pompe disease, which was approved in 2006, and olipudase alfa for Niemann-Pick disease type B, which recently completed a successful Phase 3 study. Dr. Cox has been affiliated with Boston Children’s Hospital during his entire career, where he is a Part-time Staff Physician in Genetics. He is also an Instructor in Pediatrics at Harvard Medical School.
Dr. Cox added, “Niemann-Pick C is a devastating childhood disease for which there is no satisfactory treatment. With Cyclo Therapeutics’ pivotal Phase 3 study in NPC commencing next quarter and topline results from the NPC Phase 1/2 study expected in the near future, this is an exciting time for the Company. I am thrilled to be joining the Cyclo Therapeutics management team and believe they have great potential to provide potentially life-changing medicines for patients with rare diseases where there remains significant unmet need.”
Dr. Cox received his MD and PhD from the University of California at San Diego and his B.A. from Harvard College. He completed an internship and residency in pediatrics followed by clinical and post-doctoral research fellowships in genetics at Boston Children’s Hospital and was Director of the Medical Genomics Mapping Facility. Dr. Cox is board-certified by the American College of Medical Genetics and Genomics in Clinical, Biochemical, and Molecular Genetics, and he was board-certified by the American Academy of Pediatrics in the past. He serves on the Board of Directors for the National Tay-Sachs and Allied Diseases organization.

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 suffering from disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is the subject of three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, ( NCT02939547, NCT02912793 and NCT02912793). The company is planning an early phase clinical trial using Trappsol® Cyclo™ intravenously in Alzheimer’s Disease based on encouraging data from an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the company’s website:

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.

Investor Contact:
Jenene Thomas
(833) 475-8247

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