CTD Holdings’ Clinical Trial Design Receives Positive Responses from U.K.’s Medicines and Healthcare
Monday, December 14, 2015
CTD Holdings’ Clinical Trial Design Receives Positive Responses from U.K.’s Medicines and Healthcare Products Regulatory Agency
Responses Assist CTD in its Clinical Trial Application for Trappsol(R) Cyclo(TM) as a Treatment for Niemann Pick Type C Disease
ALACHUA, FL – (Marketwired) – 12/14/15 – CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of disease, has received positive responses from a major U.K. healthcare regulator to the clinical trial design for its Trappsol(R) Cyclo(TM) drug, which is used to treat Niemann-Pick Type C (NPC). NPC is a rare and fatal cholesterol metabolism disease found primarily in children and young adults.
The Medicines and Healthcare Products Regulatory Agency (MHRA) provided positive responses to CTD’s major clinical study design elements, including safety and toxicity, patient inclusion and exclusion criteria, and biochemical analysis and clinical outcomes. The MHRA advice holds that CTD has sufficient pre-clinical data to support its clinical trial. The MHRA’s responses follow its Scientific Advice Meeting with CTD in November.
CTD will prepare a formal clinical trial application for the MHRA at which time CTD may receive additional feedback. CTD will select sites in the U.K. for its Phase II trial and expects to begin enrolling U.K. patients in early 2016. The company also plans to enroll patients in other European countries and the U.S. at a later date.
“MHRA’s responses are a powerful signal to NPC patients, families and CTD stakeholders that our Phase II clinical study design is on target,” said CTD Chairman and CEO N. Scott Fine, “We are pleased with the positive reception and look forward to submitting our clinical trial application to the MHRA .”
CTD’s clinical study design calls for intravenous administration of Trappsol(R) Cyclo(TM) (hydroxypropyl beta cyclodextrin) in NPC patients two years of age and older. The study will examine the effect of Trappsol(R) Cyclo(TM) on biochemical markers of cholesterol metabolism and clearance post-administration, as well as the drug’s effect on neurologic, lung and liver symptoms. NPC disease causes the build-up of cholesterol in every cell of the body, leading to cognitive and movement disorders, liver dysfunction and major organ failure.
“The elements of CTD’s clinical study design were drawn largely from protocols using Trappsol(R) Cyclo(TM) in compassionate use programs around the world,” said Dr. Sharon Hrynkow, CTD’s Senior Vice President for Medical Affairs. “We are particularly grateful to the NPC families and physicians from the U.S., Brazil and Spain who shared their data and experiences with us, including those who have been administering Trappsol(R) Cyclo(TM) intravenously for more than five years.”
CTD plans to engage with additional regulatory agencies in early 2016.
About the Company:
CTD Holdings, Inc. is a biotechnology company developing cyclodextrin-based products for the treatment of disease, including Trappsol(R) Cyclo(TM), an orphan drug designated product, for the treatment of Niemann-Pick Type C, a rare and often fatal genetic disease in young children which also results in significant health impairment for affected adults. Additional indications for the active ingredient in Trappsol(R) Cyclo(TM), including peripheral artery disease, diabetic nephropathy, and acute viral infections, are also in development.
The company’s other divisions distribute and manufacture the trademarked Trappsol(R) and Aquaplex(R) cyclodextrins, cyclodextrin derivatives, and cyclodextrin complexes for biotechnology and life science companies involved in the research, pharmaceutical, medical device, cosmetics and nutrition markets. They also operate the world's only cGMP pulse drying facility for the production of UltraPure(TM) cyclodextrin derivatives and pharmaceutical grade Aquaplex(R) cyclodextrin complexes and supply cyclodextrins to biotechnology and life science researchers around the globe from the world's largest catalog of cyclodextrins. For additional information, visit the company's websites: www.ctd-holdings.com and www.cyclodex.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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Source: CTD Holdings, Inc.