Application Describes Clinical Plans and Protocol to Fight the Rare and Fatal Disease NPC
ALACHUA, FL -- (Marketwired) -- 09/12/16 -- 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 Sweden's Medical Products Agency.
"This is another milestone step for our company as we continue to build our international clinical trial program of Trappsol® Cyclo™ for the treatment of Niemann-Pick Type C disease," said N. Scott Fine, Chairman and CEO of CTD. "Our submission in Sweden complements the progress we are making in the US, where the FDA recently approved our Investigational New Drug application, and in the UK, where our Phase I/II Clinical Trial Application is currently under review."
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 is a systemic disease, impacting the brain, liver and other 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 product has Orphan Drug Designation in both the EU and the US.
The CTA describes CTD's phase I/II clinical plans to administer Trappsol® Cyclo™ intravenously to patients in Sweden as young as two years old and into adulthood. The study will be led by Dr. Martin Paucar, Karolinska University Hospital, Department of Clinical Neuroscience. Dr. Paucar has extensive experience in treating movement disorders, and inherited neurologic diseases. The study is a randomized double blind parallel group study to examine the safety and tolerability of three doses of Trappsol® Cyclo™, and effects of Trappsol® Cyclo™ on the body as well as how the body metabolizes the drug. Three doses will be examined: 1500 mg/kgBW; 2000 mg/kgBW; and 2500 mg/kgBW. Outcome measures will include markers of cholesterol synthesis and cholesterol degradation, and clinical assessments on swallowing, lung function, cognitive ability, and fine motor skills.
Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs, said, "The trial in Sweden coupled with expected trials in both the United Kingdom and Italy will provide us with formal clinical data on Trappsol® Cyclo™ in NPC. These data will complement what we know about our product's impact already in NPC, based on its compassionate use globally, and phase I data from the FDA approved trial commencing soon in the United States."
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.
Source: CTD Holdings, Inc.