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CTD Holdings Presents Compassionate Use Data on Trappsol(R) Cyclo(TM) to Treat Niemann-Pick Disease Type C and Provides Update on Progress of U.S. and EU Clinical Trials at 13th Annual WORLDSymposium

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Company's Phase I U.S. and Phase I/II EU Clinical Trials Expected to Begin Enrolling Initial Patients Shortly

ALACHUA, FL -- (Marketwired) -- 02/16/17 -- CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, presented updated compassionate use data regarding the Company's proprietary formulation of hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, for the treatment of Niemann-Pick Disease Type C (NPC). The data were presented in a poster entitled, "Intravenous Cyclodextrin Trials and Compassionate Use in Niemann-Pick Type C Disease", at the 13th Annual WORLDSymposium, the premier scientific conference focused on lysosomal storage diseases. The presentation was made by Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs, and Dr. Caroline Hastings, UCSF Benioff Children's Hospital Oakland, who serves as the Principal Investigator for the Company's U.S. clinical trial, and is the first physician in the U.S. to treat NPC patients with Trappsol® Cyclo™ on a compassionate use basis. CTD also provided an update on the Company's planned U.S. (Phase I) and EU (Phase I/II) clinical trials using Trappsol® Cyclo™ intravenously to treat NPC.

The presentation included a physicians' report on the compassionate use of Trappsol® Cyclo™ administered intravenously for more than 7 years in 11 NPC patients. Trappsol® Cyclo™ was reported to be safe and well-tolerated in these patients. Individual patients in the compassionate use program exhibited responses systemically and/or in the central nervous system, including reduction in liver size and improvement in liver transaminases; resolution of interstitial lung disease; improvement in fine and gross motor skills; and improvement in quality of life.

"We continue to be encouraged by the data being generated from Trappsol® Cyclo™ compassionate use programs," said Dr. Hrynkow. "The opportunity to present this data at the WORLDSymposium in front of the world's leading physicians and researchers in the area of lysosomal storage diseases will help build greater global visibility for our Trappsol® Cyclo™ development program."

Drug regulators in the UK (Medicines and Healthcare Products Regulatory Agency), Sweden (Medical Products Agency) and U.S. (U.S. Food and Drug Administration) have accepted clinical trial applications for IV administration of Trappsol® Cyclo™ for NPC, with a fourth application pending in Italy. Patient enrollment in the U.S. and EU studies is expected to begin in early 2017.

"The compassionate use data generated to date further strengthens our confidence in the potential of Trappsol® Cyclo™ as a safe and effective treatment for NPC," said N. Scott Fine, CTD Chairman and CEO. "We look forward to enrolling the initial patients into our U.S. Phase I and EU Phase I/II studies shortly."

Niemann-Pick Disease Type C is a rare and fatal genetic disease that impacts primarily children but is increasingly diagnosed in older patients who may live with disability for many years. The disease impacts the brain and major organs through abnormal accumulation of cholesterol in cells. Trappsol® Cyclo™ previously received Orphan Drug Designation in both the U.S. and EU.

About CTD Holdings:
CTD Holdings, Inc. is a clinical-stage 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 Disease 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

About the WORLDSymposium™:
The goal of the WORLDSymposia is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. Each year, WORLDSymposia hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases. Additional information is available at the WorldSymposium website: www.worldsymposia.org

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.

Contacts:

For CTD Holdings, Inc.:

Hans Vitzthum
LifeSci Advisors, LLC
212-915-2568
Hans@lifesciadvisors.com

For WORLDSymposia:

WORLDSymposia
PO Box 14806
Minneapolis, MN 55414
info@worldsymposia.org

Source: CTD Holdings, Inc.