The company's presentation to families was made on August 16, followed by a Q and A session. CTD announced its participation at the conference and support for the conference via an unrestricted grant in a release July 31, 2019.
Niemann-Pick type C Disease 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.
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.
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.
The National Niemann-Pick Disease Foundation (NNPDF) is a non-profit organisation dedicated to supporting and empowering patients and families affected by Niemann Pick Disease through education, collaboration, and research.
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