SOURCE: CTD Holdings, Inc.

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August 01, 2016 08:31 ET

CTD Holdings Files Clinical Trial Application With U.K. Regulatory Agency to Advance Its Drug Development Program

Application Describes Clinical Plans and Protocol to Fight the Rare and Fatal Disease NPC

ALACHUA, FL--(Marketwired - August 01, 2016) - CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced its filing of a Clinical Trial Application (CTA) with the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA).

"This is a milestone step for our company as we prepare to begin our clinical trial of Trappsol® Cyclo™ for the treatment of Niemann-Pick Type C disease," said N. Scott Fine, Chairman and CEO of CTD. "Our submission of this application is a major step forward as we work to address the critical needs of patients with Niemann-Pick Type C, and as we build the company's cyclodextrin-based platform to treat a range of diseases."

Niemann-Pick Type C (NPC) is a rare and fatal genetic disease. It impacts primarily children but is increasingly diagnosed in older patients who may live with this disability for many years. NPC impacts the brain and major organs through abnormal accumulation of cholesterol in cells. CTD is working to develop its orphan drug designated Trappsol® Cyclo™, the company's proprietary formulation of hydroxypropyl-beta-cyclodextrin, for the treatment of NPC.

The CTA describes CTD's phase I/II clinical plans to administer Trappsol® Cyclo™ intravenously to patients in the UK as young as two years old and into adulthood. The product has Orphan Drug Designation in both the EU and the US.

Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs, said, "The application describes plans for a randomized, parallel group study, to evaluate multiple doses of Trappsol® Cyclo™. We plan to enroll 12 NPC patients in the UK and other countries over the coming months. Our study will evaluate safety and tolerability of Trappsol® Cyclo™ in this first formalized trial, as well as the clinical benefits of Trappsol® Cyclo™ relating to gross and fine motor skills, lung and liver function, behavioral and cognitive skills, and quality of life."

The UK sites named in the application are Salford Royal Hospital NHS Foundation Trust, Department of Adult Inherited Metabolic Diseases and University College London, Institute of Neurology. The Principal Investigators named in the application are Dr. Reena Sharma, Mark Holland Metabolic Unit, Salford Royal NHS Foundation Trust and Dr. Robin Lachmann, Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, respectively, who both treat NPC patients from across the UK.

Professor Alan Boyd, CEO of Boyd Consultants, a key advisor to CTD and a leader in pharmaceutical medicine in the UK, said, "We are pleased to have had a positive advice meeting with the MHRA late last year in which we described CTD's experience providing Trappsol® Cyclo™ to NPC patients on a compassionate use basis, in some cases for more than five years. We hope that this submission will move the CTD program forward quickly for the future benefit of NPC patients."

About the Company:
CTD Holdings, Inc. is a biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company's Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Type C, a rare and fatal genetic disease. Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the company's website: www.ctd-holdings.com.

Safe Harbor Statement:
This press release contains "forward-looking statements" about the company's current expectations about future results, performance, prospects and opportunities. 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.

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