Dr. Caroline Hastings, a pediatric hematologist/oncologist at UCSF Benioff Children’s Hospital Oakland, discusses her role in treating the first Niemann-Pick Disease Type C (NPC) patients in the U.S. with Trappsol® Cyclo™ and how this grew to a global compassionate-use program for NPC patients, then formal clinical trials at Cyclo Therapeutics. Dr. Hastings is the Principal Investigator on Cyclo Therapeutics’ phase I clinical trial, Senior Clinical Advisor on CTD’s phase I/II clinical trial in Europe, and she co-chairs Cyclo Therapeutics’ Family and Physicians Listening Circle.
Trappsol® Cyclo™ is an experimental treatment for people with Niemann-Pick Disease Type C. It is a proprietary formulation of a ring of sugar molecules known as cyclodextrins. Cyclo Therapeutics uses hydroxypropyl beta cyclodextrin in its formulation of Trappsol® Cyclo™. Laboratory experiments have shown that Trappsol® Cyclo™ can remove the harmful cholesterol build up from animal cells, and it has been used by NPC patients on a compassionate basis for up to 7 years.
Dr. Benny Liu, a gastroenterologist at Highland Hospital and UCSF Benioff Children’s Hospital, Oakland, California, discusses his groundbreaking research on cyclodextrins as a treatment of Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease that impacts the brain, lung, liver, spleen, and other organs. Dr. Liu is a member of Cyclo Therapeutics’ Scientific Advisory Board and a co-Investigator on Cyclo Therapeutics’ phase I clinical trial using Trappsol® Cyclo™, CTD’s proprietary orphan drug for treatment of NPC.
N. Scott Fine, Chairman and CEO of Cyclo Therapeutics, discusses Cyclo Therapeutics’ evolution from a fine chemicals company to a clinical-stage pharmaceutical company that develops cyclodextrins to treat human disease, particularly the orphan drug Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C, a rare and fatal genetic disease that impacts the brain, lung, liver, spleen, and other organs.
July 17, 2020
Dear NPC Families and Community, Joslyn and Justin,
Cyclo Therapeutics, Inc. has supported a role for all companies in the NPC space, recognizing that patients will ultimately benefit from multiple treatment options. Cyclo Therapeutics Inc. remains committed to move forward with our planned Phase III pivotal trial with our cyclodextrin formulation, Trappsol® Cyclo™, through Intravenous administration in patients with Niemann-Pick Disease Type C1. And, our support for the NPC community continues to be unwavering.
Our decision to develop Trappsol® Cyclo ™ through an intravenous route of administration was based on knowledge gained through our Expanded Access programs dating back to 2009: safety of the drug when provided intravenously; patient and caregiver acceptance of intravenous administration; higher safety profile for intravenous over other routes of administration; convenience to patients and families and ability to provide administration of the drugat home (as is happening now in a Cyclo Therapeutics’ extension protocol of the US Phase I trial); and benefit as measured by efficacy tools both in Expanded Access programs and now in the formal clinical trials.
Cyclo Therapeutics recently closed its Phase I safety trial of intravenous Trappsol® Cyclo™ cyclodextrin and reported top line results. The safety profile, as expected, was highly favorable, and it validated 10 years’ of safety data from Expanded Access programs.In addition, Cyclo Therapeuticsrecently reported the Interim Analysis of our Phase I/II trial, showing signals of efficacy in all dose groups studied. While the Phase I/II trial will not be completed until early 2021, there are compelling individual examples of drug benefit showing improvements in walking, speaking, word finding ability, and fine motor skills underpinning activities of daily living.
We look forward to continuing to collaborate with patients, families, physicians and health agencies with the intention of launching ourPhase III intravenous cyclodextrin trial in 4Q2020.
Dr. Sharon H. Hrynkow
Chief Scientific Officer and
Senior Vice President for Medical Affairs
Cyclo Therapeutics, Inc.