Favorable risk/benefit analysis outlined in report to FDA
ALACHUA, FL / ACCESSWIRE / June 10, 2019 / CTD Holdings, Inc. (CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that the annual report on "Expanded Access to Trappsol® Cyclo™ for an Individual Patient with Late-Onset Alzheimer's Disease" has been received by FDA according to investigator-sponsor Diana R. Kerwin, MD, President, Kerwin Research Center, allowing the company to continue to support the program. The program tests CTD's drug, Trappsol® Cyclo™, its proprietary formulation of hydroxypropyl beta cyclodextrin, as a potential disease modifying treatment in a single patient with dementia of the Alzheimer's type. The drug is administered once a month by intravenous injection.
CTD's Chairman and CEO N. Scott Fine said, "We are grateful to FDA for allowing CTD to provide Trappsol® Cyclo™ to the patient, and we look forward to continuing to advance our understanding of the drug's effects in this first geriatric patient to use our drug. We expect that the results from this Expanded Access program will be critical in our design of formal clinical studies for the Alzheimer's population."
CTD collaborates with Diana R. Kerwin, MD, President, Kerwin Research Center, as the investigator-sponsor, in the conduct of this expanded access program. Over the course of the first year of the program, safety was assessed by MRIs, audiology, ECGs and standard laboratory tests. Cognitive and behavioral assessments included the Mini-Mental State Examination and standard neurologic testing (see ClinicalTrials.gov NCT03624842 for protocol details).
The one-year report describes a favorable safety profile as well as encouraging initial observations on potential benefit. "The report suggests cognitive and neurologic stability, indicating possible benefit," said Sharon Hrynkow PhD, CTD's Chief Scientific Officer and Senior Vice President for Medical Affairs. This is a positive outcome given that persons with Alzheimer's Disease dementia are generally expected to decline during a one-year timeframe. "The data to date suggest that the patient continue with monthly dosings," said Dr. Hrynkow. Biomarker data for cholesterol synthesis and metabolism and imaging are now underway.
Adverse events observed during the first year of dosings were mild in most instances and moderate in one. Mild events included diarrhea, phlebitis and blood pressure increase, all of which were found to be clinically insignificant. One moderate event was observed, a vasovagal episode: it was unclear if the event was related to the drug or to underlying medical conditions.
With the support of the treating physician, the patient and the patient's family, the second year of monthly dosing is expected to commence this month.
As part of the partnership with the Kerwin Research Center on the expanded access program, CTD holds rights to all data for the purposes of commercialization.
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.
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SOURCE: CTD Holdings, Inc.
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