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CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC W

Tuesday, September 6, 2016   (0 Comments)
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CTD Holdings Announces FDA Acceptance of Investigational New Drug Application for Treatment of NPC With Trappsol(R) Cyclo(TM)

Enrollment of Study Participants to Begin in the Coming Weeks

ALACHUA, FL -- (Marketwired) -- 09/06/16 -- CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug application (IND) for its orphan drug product Trappsol® Cyclo™ in the treatment of Niemann-Pick Type C (NPC) disease. CTD will be initiating a Phase I study to investigate safety, tolerability, pharmacokinetic parameters and pharmacological effects of the intravenous administration of Trappsol® Cyclo™, CTD's hydroxypropyl beta cyclodextrin. Trappsol® Cyclo™ has Orphan Drug Designation in both the EU and the US.

"We are pleased to see Trappsol® Cyclo™ moving into its first formal clinical trial," said N. Scott Fine, CTD Chairman and CEO. "With patients soon to be enrolled in the US, and with the Phase I/II Clinical Trial Application under review in the UK, this is an exciting time for our company and all stakeholders, and most importantly for the NPC families who are waiting for approved cyclodextrin-based treatments."

Niemann-Pick Type C 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.

The randomized, double-blinded study will be centered at one site, UCSF Benioff Children's Hospital Oakland, and is expected to enroll 12 patients over the age of 18. It will examine two dosage levels of Trappsol® Cyclo™, 1500 mg/kgBW and 2500 mg/kgBW, administered intravenously, and will examine outcome measures on cholesterol synthesis and cholesterol storage in major organs, including the liver. "We were pleased by the level of cooperation and input from FDA as we finalized our study design," said Dr. Sharon H. Hrynkow, CTD's Senior Vice President for Medical Affairs. More information on the study parameters will be posted to ClinicalTrials.gov.

Dr. Caroline Hastings, Pediatric Hematologist/Oncologist, UCSF Benioff Children's Hospital Oakland, is the Principal Investigator. Dr. Hastings was the first physician in the US to administer hydroxypropyl beta cyclodextrin in the form of Trappsol® Cyclo™ to NPC patients. Dr. Benny Liu, Gastroenterologist, Alameda Health System Oakland, and also affiliated with UCSF Benioff Children's Hospital Oakland, will be acting as a Co-Investigator. Dr. Liu made the seminal discovery showing that hydroxypropyl beta cyclodextrin is effective in treating NPC in animal models. "This approval is the culmination of years of work by many families and physicians along with CTD and other partners," said Dr. Hastings. "It is an honor to lead the formal clinical trial of IV administration of Trappsol® Cyclo™, the same cyclodextrin product used in named patient programs going back to 2009."

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.

Contact:
Sitrick and Company

Wendy Tanaka
(415) 369-8447
wtanaka@sitrick.com

Thomas Mulligan
(212) 573-6100, Ext. 395
tmulligan@sitrick.com

Source: CTD Holdings, Inc.


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