SOURCE: CTD Holdings, Inc.

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November 10, 2016 08:00 ET

CTD Holdings Announces Approval of Clinical Trial Application to Conduct Phase I/II Clinical Study of Trappsol® Cyclo™ in Patients with Niemann-Pick Type C Disease at Karolinska University Hospital

Karolinska Represents Third Approved Study Site in Europe; First Patient Expected to be Enrolled in Phase I/II Study in First Quarter of 2017

ALACHUA, FL--(Marketwired - November 10, 2016) - CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company developing cyclodextrin-based products for the treatment of diseases with unmet medical need, today announced that the Swedish Medical Products Agency has approved CTD Holdings' Clinical Trial Application to conduct a Phase I/II clinical study at Karolinska University Hospital in Sweden evaluating Trappsol® Cyclo™ in patients with Niemann-Pick Type C (NPC) Disease, a rare and fatal illness tied to an autosomal recessive genetic defect. Karolinska represents the third site in Europe approved to conduct this study. CTD Holdings expects that the first patient in this Phase I/II clinical trial will be dosed in the first quarter of 2017.

Trappsol® Cyclo™ is a parenteral grade of hydroxypropyl beta cyclodextrin, a donut-shaped molecule comprised of seven glucopyranose units. Its hydrophilic exterior allows it to move easily through the body, and its inner hydrophobic cavity allows it to capture and hold certain types of molecules. To date, intravenous Trappsol® Cyclo™ has been administered to 21 NPC patients worldwide, some for more than five years, via multiple Compassionate Use Programs. During this time, multiple patients have shown marked improvements in neurological symptoms, lung function or liver morphology, or had stabilization of disease progression, with no significant, unexpected, safety concerns.

"We view this third site approval at such a prestigious institution in Europe, Karolinska University Hospital, as validation of the potential of intravenous Trappsol® Cyclo™ in treating NPC," said N. Scott Fine, Chief Executive Officer of CTD Holdings. "In addition to the Phase I/II study we are conducting in Europe, we also expect to begin enrolling patients in a U.S. Phase I study to evaluate the safety and impact of intravenous Trappsol® Cyclo™ on biomarkers of NPC Disease in the first quarter of 2017. NPC is a highly under-served orphan disease with an addressable market of over $400 million, and represents a significant opportunity for CTD Holdings."

"NPC is a fatal disease that is difficult to diagnose and is consequently under-diagnosed," said Dr. Sharon Hrynkow, CTD's Senior VP for Medical Affairs. "There is currently only one approved treatment for the disease in Europe, and none in the U.S. Based on the Compassionate Use Program data garnered to date, we believe Trappsol® Cyclo™ administered intravenously has the potential to effectively treat NPC. We look forward to working with our colleagues in Sweden to further evaluate this promising drug candidate in this Phase I/II study."

CTD Holdings received Orphan Drug designation for the use of Trappsol® Cyclo™ in NPC from the U.S. Food and Drug Administration and the European Medicines Agency.

For further information on this trial and the U.S. trial, please visit www.ClinicalTrials.gov.

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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