10 Jan 2017

Keystone Nano, Inc., a biopharmaceutical company focused on improving cancer treatments through the application of nanotechnology, announced today that the U.S. Food and Drug Administration has approved the company's Investigational New Drug (IND) Application to assess Ceramide NanoLiposome in the treatment of solid tumors. The therapy will be tested in a Phase I clinical trial at three sites: the University of Maryland, the University of Virginia and the Medical University of South Carolina. The clinical trial will enable the Company to establish a safe dose level, and begin gathering information about the efficacy of the product as a cancer therapy.

"This approval represents the conclusion of one phase of developing Ceramide, a truly novel anti-cancer agent, and the beginning of the very important human testing phase," noted Jeff Davidson, Keystone Nano's CEO. "We are excited by the efficacy and lack of toxicity Ceramide NanoLiposome has shown in preclinical testing, and we look forward to continuing to evaluate Ceramide NanoLiposome for human use. This is a significant step forward in the development of Ceramide NanoLiposome for the treatment of cancer."
Ceramide is a bioactive lipid that has shown efficacy in multiple models of liver cancer, as well as in breast cancer, leukemia and pancreatic cancer. Evidence collected in a large number of research tests (in vitro and in vivo) has clearly demonstrated that Ceramide NanoLiposome kills cancer cells while leaving normal cells alone, providing treatment without undue toxicity.

"The anti-cancer activity of Ceramide has long been known in the laboratory," said Dr. Mylisa Parette, Keystone Nano's Vice President of Research, "Until now, however, no-one has had an effective method for delivery of Ceramide, which is insoluble and has an extremely short half-life in the body. The proprietary formulation we are evaluating uses ceramide integrated into the membrane of a nanoliposome to overcome these barriers and allow us to exploit the novel properties of Ceramide as an anti-cancer therapy."
The Phase I portion of the trial will recruit patients with solid tumors to establish dosing and safety. The Phase II component of the trial is expected to focus on Liver Cancer, which kills approximately 27,000 people in the United States and 700,000 worldwide each year. There is currently no effective therapy for this disease. Keystone Nano was recently awarded orphan drug status for the treatment of liver cancer with ceramides in the United States. Keystone Nano previously announced that it was awarded a $2 million Phase II Small Business Innovation Research grant from the National Cancer Institute of the National Institutes of Health, which is funding part of this trial.