ALACHUA, Fla.—CTD Holdings, Inc., a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, has posted its presentation to be made at the annual Family Support and Medical Conference organized by the National Niemann-Pick Disease Foundation (NNPDF). The company’s presentation to families will be made on August 16, followed by a Q&A session.
“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,” said N. Scott Fine, CTD chairman and CEO. “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 intravenously-administered proprietary cyclodextrin drug, Trappsol Cyclo, in the U.S., the UK, 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.
“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,” noted Sharon Hrynkow, Ph.D., CTD’s chief scientific officer and senior vice president for Medical Affairs.
“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 preclinical 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, M.D., co-principal investigator for the U.S. Phase 1 clinical trial and senior clinical advisor for the Phase 1/2 clinical trial, will make the presentation on behalf of the company. Hastings is Pediatric Hematologist and Oncologist at UCSF Benioff Children’s Hospital in Oakland, CA. She is also the first physician in the U.S. 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, Hrynkow and 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.