CTD Holdings Presents Compassionate Use Data on Trappsol(R) Cyclo(TM) to Treat Niemann-Pick Disease Type C and Provides Update on Progress of U.S. and EU Clinical Trials at 13th Annual WORLDSymposium

CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, presented updated compassionate use data regarding the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin, Trappsol® Cyclo™, for the treatment of Niemann-Pick Disease Type C (NPC). The data were presented in a poster entitled, “Intravenous Cyclodextrin Trials and Compassionate Use in Niemann-Pick Type C Disease”, at the 13^th Annual WORLDSymposium, the premier scientific conference focused on lysosomal storage diseases. The presentation was made by Dr. Sharon Hrynkow, CTD’s Senior Vice President for Medical Affairs, and Dr. Caroline Hastings, UCSF Benioff Children’s Hospital Oakland, who serves as the Principal Investigator for the Company’s U.S. clinical trial, and is the first physician in the U.S. to treat NPC patients with Trappsol® Cyclo™ on a compassionate use basis. CTD also provided an update on the Company’s planned U.S. (Phase I) and EU (Phase I/II) clinical trials using Trappsol® Cyclo™ intravenously to treat NPC.
The presentation included a physicians’ report on the compassionate use of Trappsol® Cyclo™ administered intravenously for more than 7 years in 11 NPC patients. Trappsol® Cyclo™ was reported to be safe and well-tolerated in these patients. Individual patients in the compassionate use program exhibited responses systemically and/or in the central nervous system, including reduction in liver size and improvement in liver transaminases; resolution of interstitial lung disease; improvement in fine and gross motor skills; and improvement in quality of life.
“We continue to be encouraged by the data being generated from Trappsol® Cyclo™ compassionate use programs,” said Dr. Hrynkow. “The opportunity to present this data at the WORLDSymposium in front of the world’s leading physicians and researchers in the area of lysosomal storage diseases will help build greater global visibility for our Trappsol® Cyclo™ development program.”
Drug regulators in the UK (Medicines and Healthcare Products Regulatory Agency), Sweden (Medical Products Agency) and U.S. (U.S. Food and Drug Administration) have accepted clinical trial applications for IV administration of Trappsol® Cyclo™ for NPC, with a fourth application pending in Italy. Patient enrollment in the U.S. and EU studies is expected to begin in early 2017.
“The compassionate use data generated to date further strengthens our confidence in the potential of Trappsol® Cyclo™ as a safe and effective treatment for NPC,” said N. Scott Fine, CTD Chairman and CEO. “We look forward to enrolling the initial patients into our U.S. Phase I and EU Phase I/II studies shortly.”
Niemann-Pick Disease Type C is a rare and fatal genetic disease that impacts primarily children but is increasingly diagnosed in older patients who may live with disability for many years. The disease impacts the brain and major organs through abnormal accumulation of cholesterol in cells. Trappsol® Cyclo™ previously received Orphan Drug Designation in both the U.S. and EU.
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
About the WORLDSymposium™:
The goal of the WORLDSymposia is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. Each year, WORLDSymposia hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases. Additional information is available at the WorldSymposium website: www.worldsymposia.org
Safe Harbor Statement:
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