International Niemann–Pick Disease Alliance
CTD Chairman and CEO N. Scott Fine said, “We look forward to presenting data on our US and EU/Israel trials as part of our continuing effort to keep the patient community informed and involved as we advance our drug development program to bring Trappsol® Cyclo™ to market registration for Niemann-Pick Disease Type C. We are grateful to NNPDF for its stewardship of this conference and for the important work that it does to provide evidence-based information on FDA-approved clinical trials to those struggling with NPC.”
Niemann-Pick type C Disease (NPC) is a rare, genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to the defect in the NPC protein which is responsible for cholesterol processing in the cell. Because of the NPC defect in this disease, cholesterol accumulates abnormally in every cell in the body, causing symptoms in the brain, liver, spleen, lung and other organs. NPC is ultimately a fatal disease. CTD supports three clinical trial programs using its proprietary cyclodextrin drug, Trappsol® Cyclo™, administered intravenously, in the United States, the United Kingdom, Sweden, Israel and Italy for the treatment of NPC. Both main trials are nearing completion of enrollment, and one extension study is active with a second extension study soon to be active.
CTD’s Chief Scientific Officer and Senior Vice President for Medical Affairs, Sharon Hrynkow, PhD said, “Our data suggest that Trappsol® Cyclo™ is safe when administered intravenously at the doses we are using in our clinical trials, and our data show encouraging effects in addressing NPC symptoms, including ataxia, behavioral manifestations of the disease, and liver dysfunction. Our newest data, which have not been presented until now, suggest that the drug clears cholesterol from liver cells of NPC patients based on histological results: this is the first time that we have been able to visualize clearance of cholesterol in liver cells of NPC patients following intravenous administration of our drug. While the study remains blinded in terms of dose in our ongoing clinical trials, the new findings on visualization of the clearance of liver cholesterol in NPC patients are encouraging since all patients in our trials receive the study drug. The findings are in keeping with our expectations based on pre-clinical work and from our compassionate use programs in patients globally.”
In keeping with NNPDF communication policies, CTD’s presentation to families must be given by a non-employee. Dr. Caroline Hastings, MD, Co-Principal Investigator for the US Phase I clinical trial and Senior Clinical Advisor for the Phase I/II clinical trial will make the presentation on behalf of the company. Dr. Hastings is Pediatric Hematologist and Oncologist at UCSF Benioff Children’s Hospital, Oakland, CA, and is also the first physician in the US to administer hydroxypropyl beta cyclodextrins to NPC patients: this was done on a compassionate use basis with CTD’s product given intravenously. Following the presentation, Dr. Hrynkow and Dr. Hastings will participate on a panel with other industry and academic representatives to address audience questions on FDA-approved trials for NPC in the United States.
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, on a compassionate use basis as well as in three ongoing formal clinical trials (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the company’s website: www.ctd-holdings.com
The National Niemann-Pick Disease Foundation (NNPDF) is a non-profit organization dedicated to supporting and empowering patients and families affected by Niemann Pick Disease through education, collaboration, and research.
Safe Harbor Statement:
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