CTD supports two clinical research studies with intravenous administration of its proprietary cyclodextrin Trappsol® CycloTM for people with Niemann-Pick Disease Type C1 (NPC-1). The goal of the studies is to understand the safety of this new treatment at different doses in a Phase 1 US study, at Children’s Hospital Oakland, CA, and safety and efficacy in a Phase 1/2 study in the EU, running in the UK, Sweden and expected in Italy.
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.
ALACHUA, FL -- (Marketwired) -- 04/10/17 -- CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today issued an Open Letter to address false and misleading statements, recently made by another public company, regarding CTD's hydroxypropyl beta cyclodextrin (HPBCD) product, Trappsol® Cyclo™, and its clinical use to treat Niemann-Pick Disease Type C (NPC). The false and misleading statements were made as part of another public company's announcement of their acquisition of a hydroxypropyl beta cyclodextrin product.
CTD Holdings, Inc. issues the following Open Letter to the NPC Community and CTD Holdings Stakeholders:
Dear NPC Community and CTD Holdings Stakeholders,
Many in the patient and investor communities have contacted us to ask about statements in the public domain made by another public company with a cyclodextrin product in clinical trials for NPC, VTS-270. As a result, CTD has requested that the FDA review and take appropriate action to address the false and misleading statements made about CTD's cyclodextrin product, Trappsol® Cyclo™, also in clinical trials.
The four specific misleading and inaccurate statements and CTD's factual corrections are:
(a) a statement that CTD's product is not likely to be able to be used intrathecally. In fact, Trappsol® Cyclo™ has been and currently is in use in compassionate use programs via three routes of administration -- intrathecal (IT), intravenous (IV), and intracerebral ventricular (ICV);
(b) a statement that intravenously administered versions HPBCDs do not cross the blood-brain barrier, or do so only in limited amounts. There are no formal clinical trial data showing that HPBCD does not cross the blood-brain-barrier in NPC patients.
(c) a statement that HPBCDs may only impact neurologic aspects of NPC disease when provided directly into the cerebrospinal fluid. In fact, CTD has presented data from its compassionate use program, demonstrating that IV administration has led to neurologic benefit in some patients, and
(d) an exaggerated claim regarding the use of VTS-270 in pre-clinical studies. In fact, VTS-270 did not come into existence until early 2015, when the parent company was established. The significant proportion of pre-clinical data generated to date in mice and cat models of NPC resulted from use of CTD's Trappsol® HPBCD and others, that existed in the market when the early work was conducted, not VTS-270.
We hope that this information sheds a more accurate light on the NPC clinical trials. We know that this information is accurate with respect to CTD's Trappsol® Cyclo™ product and its use. We thank the patient community for their trust and support of our programs, with special thanks to those who have used and are still using our product in compassionate use programs. Without the support from the patient community, and physicians working alongside us all, we would not be in our current position of recruiting patients into our US and EU clinical studies.
N. Scott Fine
Chairman and CEO
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.
LifeSci Advisors, LLC
Source: CTD Holdings, Inc.
Released April 10, 2017