Describes Proposed Clinical Trials for Intravenous Administration of Trappsol(R) Cyclo(TM) for the Treatment of Niemann-Pick Type C Disease
ALACHUA, FL -- (Marketwired) -- 08/16/16 -- CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of disease, recently presented to two groups of parent advocates and physicians on its clinical trial plans for intravenous (IV) administration of its hydroxyl-propyl-beta cyclodextrin product, Trappsol® Cyclo™, in Niemann-Pick Type C patients. In both meetings, CTD summarized data from the use of Trappsol® Cyclo™ in compassionate use programs and provided overviews of its planned complementary EU and US clinical trials. "We have provided information on our clinical programs to NPC parent advocates as well as physicians and caregivers. We will continue to meet with and talk to those most affected by NPC, as well as our broader community of stakeholders and supporters," said N. Scott Fine, Chairman and CEO of CTD Holdings.
On August 11, CTD convened its 'Family and Physician Listening Circle' via webinar. The Listening Circle is a network for learning and sharing on topics of interest to its members, including updates on CTD's clinical trials, information sharing among parents on practical issues addressing management of the disease, and clinician insights on trends and challenges in the field. It is co-chaired by CTD, an NPC treating physician and a parent advocate; currently Dr. Sharon Hrynkow, also Senior Vice President for Medical Affairs and Co-Chair of the Scientific Advisory Board; Dr. Caroline Hastings, UCSF-Benioff Children's Hospital Oakland and Senior Clinical Advisor to CTD, also member of the Scientific Advisory Board; and Ms. Sue French of the UK. Listening Circle members asked CTD to present on its clinical program progress at its inaugural meeting which was attended by members from the UK, Spain, Norway and the US.
Presenters at the Listening Circle meeting were N. Scott Fine; Dr. Sharon Hrynkow; Professor Alan Boyd, CEO of Boyd Consultants, a key advisor to CTD and a leader in pharmaceutical medicine in the UK; and Dr. Caroline Hastings, who pioneered the use of cyclodextrins to treat NPC. Dr. Hastings is also the Principal Investigator for the US clinical trial. The presentation summarized data from the compassionate use program on the safety and tolerability of Trappsol® Cyclo™ given intravenously in 11 patients for up to six years, and described the clinical benefits observed by parents and physicians which allowed for continued administration. Among the benefits of IV Trappsol Cyclo observed by physicians were improvements in fine motor skills, behavior and cognition; improvements in lung function, and decreased size of the liver. The Listening Circle presentation can be viewed on the company's website and here. "Our clinical program for IV administration is built on solid data resulting from several years of experience in many patients. Families who are struggling with NPC should have information on all clinical programs and treatment options so that their personal caregiving decisions can be as informed as possible," said Dr. Hrynkow.
On August 12, CTD presented to a group of NPC-affected families in response to a request by Shannon Reedy, mother of an NPC affected child, and founder of the non-profit organization Chase the Cure. Ms. Reedy is also a member of the CTD Family and Physicians Listening Circle. The meeting, which was attended in-person by a dozen families and one physician, was held in Danvers, MA. CTD provided an overview of compassionate use data for intravenous use of Trappsol® Cyclo™, and described the proposed US and EU trials. Mr. Fine, Drs. Hrynkow and Hastings, and Professor Boyd all participated in the presentation and the question and answer period immediately following. In his concluding comments, Mr. Fine thanked Ms. Reedy for hosting the meeting, noting that her efforts and those of other NPC-affected parents have been instrumental in moving the whole field forward, for the benefit of NPC children and families everywhere. Ms. Reedy added, "Forums such as the Listening Circle and our Chase the Cure event are key for NPC parents since they provide us with opportunities to hear from experts, ask questions, and learn from others who are facing similar challenges."
Niemann-Pick Type C (NPC) is a rare and fatal genetic disease. It impacts primarily children but is also 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. CTD is working to obtain regulatory approval of its orphan drug designated Trappsol® Cyclo™ for the treatment of NPC.
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.