Company News

CTD Holdings Files Investigational New Drug Application With US FDA to Further Advance Its Drug Development Program

Download PDF

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

ALACHUA, FL -- (Marketwired) -- 08/08/16 -- CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced its filing of an Investigational New Drug application (IND) with the US Food and Drug Administration (FDA).

"This is another milestone for our company as we move forward with our comprehensive drug development program 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 IND complements and expands our work in Europe. Working with US families is an integral part of our global clinical program, which is why this is such an exciting moment for our company, stakeholders, and mostly for the NPC families who stand to benefit."

Niemann-Pick Type C (NPC) is a rare and fatal genetic disease that 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 IND describes CTD's US Phase I clinical plans for a randomized, double blind, parallel group study enrolling six patients at a single clinical site. The objectives of this study are to investigate in detail both the pharmacokinetic parameters and the pharmacological effects of three different doses of Trappsol® Cyclo™, following its administration in both single and repeat intravenous doses to adult patients with NPC. CTD's product has Orphan Drug Designation in both the EU and the US.

"NPC is a systemic disease that impacts cholesterol accumulation in every cell in the body," said Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs. "Our IND application describes plans to evaluate safety and tolerability of Trappsol® Cyclo™ administered intravenously while examining outcome measures on cholesterol synthesis and cholesterol storage in major organs, including the liver. We expect this study to provide critical insights on links between cholesterol accumulation, symptoms and disease, all with a focus on bringing effective treatments to NPC families."

The clinical site for this study will be Children's Hospital and Research Center at Oakland, with Dr. Caroline Hastings, Pediatric Hematologist/Oncologist, UCSF Benioff Children's Hospital Oakland, as the Principal Investigator. Dr. Hastings was the first physician in the US to administer hydroxy-proply-beta cyclodextrins (HPBCD) in the form of Trappsol® Cyclo™ to NPC patients. Dr. Benny Liu, Gastroenterologist, Alameda Health System Oakland, and also affiliated with Children's Hospital and Research Center at Oakland, will be acting as a Co-Investigator. Dr. Liu made the seminal discovery showing that HPBCD is effective in treating NPC in animal models.

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 US FDA earlier this 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 benefit of NPC patients globally."

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:

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.

Sitrick and Company
Wendy Tanaka
(415) 369-8447

Thomas Mulligan
(212) 573-6100, Ext. 395

Source: CTD Holdings, Inc.