“Today’s ‘Last-Patient-In’ announcement is a significant milestone for our company and the NPC community,” said Chairman and CEO, N. Scott Fine. “It completes another important step in our development and registration strategies for Trappsol Cyclo to treat NPC, a disease which causes much suffering for the patients and their families. We are delighted to share this news with our many supporters and all of our stakeholders.”
Niemann-Pick Disease Type C is a rare and fatal genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to a defect in the NPC protein which is responsible for cholesterol processing in the cell. NPC causes symptoms in the brain, liver, spleen, lung and other organs. There are no approved drug therapies for NPC in the United States and only one approved therapy in Europe.
“We thank all of the patients and their caregivers who have or are currently participating in this trial. We recognize the sacrifice of time and energy on the part of families as they participate in this trial,” said Sharon Hrynkow PhD, Chief Scientific Officer and Senior Vice President for Medical Affairs. “We are also deeply grateful to the study team at UCSF Benioff Children’s Hospital Oakland, especially Co-Principal Investigators Caroline Hastings MD and Benny Liu, MD, for all that they have done and continue to do to support the development of this drug.”
It is anticipated that data from the last patient enrolled in the study will be available in February 2020. Cyclo Therapeutics Inc. will then unblind the study results and analyze the data. Data from the current study combined with those of the companion Phase I/II study, nearing completion of enrollment in EU-Israel (ClinicalTrials.gov NCT02912793), are expected to be used in the company’s market registration applications.
Source: Company Press Release