For families interested in knowing more, contact:
Dr. Caroline Hastings at Children's Hospital Oakland: firstname.lastname@example.org
Dr. Caroline Hastings, a pediatric hematologist/oncologist at UCSF Benioff Children’s Hospital Oakland, discusses her role in treating the first Niemann-Pick Disease Type C (NPC) patients in the U.S. with Trappsol® Cyclo™ and how this grew to a global compassionate-use program for NPC patients, then formal clinical trials at CTD. Dr. Hastings is the Principal Investigator on CTD’s phase I clinical trial, Senior Clinical Advisor on CTD’s phase I/II clinical trial in Europe, and she co-chairs CTD’s Family and Physicians Listening Circle.
Trappsol® Cyclo™ is an experimental treatment for people with Niemann-Pick Disease Type C. It is a proprietary formulation of a ring of sugar molecules known as cyclodextrins. CTD uses hydroxypropyl beta cyclodextrin in its formulation of Trappsol® Cyclo™. Laboratory experiments have shown that Trappsol® Cyclo™ can remove the harmful cholesterol build up from animal cells, and it has been used by NPC patients on a compassionate basis for up to 7 years.
Dr. Benny Liu, a gastroenterologist at Highland Hospital and UCSF Benioff Children’s Hospital, Oakland, California, discusses his groundbreaking research on cyclodextrins as a treatment of Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease that impacts the brain, lung, liver, spleen, and other organs. Dr. Liu is a member of CTD’s Scientific Advisory Board and a co-Investigator on CTD’s phase I clinical trial using Trappsol® Cyclo™, CTD’s proprietary orphan drug for treatment of NPC.
N. Scott Fine, Chairman and CEO of CTD Holdings, discusses CTD’s evolution from a fine chemicals company to a clinical-stage pharmaceutical company that develops cyclodextrins to treat human disease, particularly the orphan drug Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C, a rare and fatal genetic disease that impacts the brain, lung, liver, spleen, and other organs.
Company Expects Final Data from Trial by End of 2018
ALACHUA, FL--(Marketwired - June 21, 2017) - CTD Holdings, Inc. (
The Phase I/II clinical trial, which will include additional sites in the UK and Sweden and is also expected to be expanded to Italy, will require 12 patients to be fully enrolled. The trial will evaluate the safety and efficacy of Trappsol® Cyclo™ in NPC patients ages two and older, with the primary objective of determining the optimum dose for further study. Patients will be randomized into three dose groups of 1500 mg/kg, 2000 mg/kg and 2500 mg/kg of Trappsol® Cyclo™, which will be administered via bi-weekly intravenous injections over a period of 48 weeks.
"Enrollment of the first patient at the Salford clinical site is a significant milestone for the Company in the development of this treatment for a devastating disease," said CTD Chairman and CEO, N. Scott Fine. "We expect final data from this important clinical trial by the end of 2018. In addition, we anticipate enrolling the first patient in our U.S. Phase I clinical trial of Trappsol® Cyclo™ in the near future. CTD is grateful for the continued support from the many patient families, researchers and clinicians who have worked with us to get to this point."
Trappsol® Cyclo™ is a parenteral grade of hydroxypropyl beta cyclodextrin, a donut-shaped molecule comprised of seven glucopyranose units. To date, intravenous Trappsol® Cyclo™ has been administered to 21 NPC patients worldwide, some for more than six years, via Compassionate Use Programs. Data from treating physicians have demonstrated that multiple patients have shown marked improvements in neurological symptoms, lung function or liver morphology, or had stabilization of disease progression, with no significant safety concerns.
"I am pleased to be participating in this promising clinical trial," said Dr. Sharma. "Based on the data generated from the ongoing Compassionate Use Programs, Trappsol® Cyclo™ administered intravenously has significant potential to be a safe and effective treatment for NPC, a catastrophic condition with a substantial unmet need. I look forward to further evaluating this important drug candidate in a clinical setting."
"NPC is a systemic disease, which is why we are administering Trappsol® Cyclo™ intravenously in this study," said Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs. "We look forward to working with our colleagues to enroll additional patients in Salford, and other sites, as we move this promising drug candidate forward in this Phase I/II study."
In addition to the European study, CTD Holdings has initiated a Phase I clinical trial in the U.S. evaluating intravenous administration of Trappsol® Cyclo™ in NPC patients. CTD previously received Fast Track Designation and Orphan Drug Designation for the use of Trappsol® Cyclo™ in the treatment of NPC from the U.S. Food and Drug Administration and the European Medicines Agency.
About CTD Holdings:
CTD Holdings, Inc. is a clinical-stage 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 Disease 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.
For families interested in learning more about the EU trial or the US trial, please contact CTD's Patient Liaisons:
For additional information, please visit:
Phase I/II: https://clinicaltrials.gov/ct2/show/NCT02912793
LifeSci Advisors, LLC
Source: CTD Holdings, Inc.
Released June 21, 2017