Buck Institute's First Clinical Trial Underway in Australia

First patient receives exploratory new therapeutic for treatment of Mild Cognitive Impairment

November 10, 2014/Novato, CA: The Buck Institute’s first clinical trial, which involves a treatment for amnestic mild cognitive impairment (aMCI), often an early indicator of Alzheimer’s disease, is under way in Australia.

The drug being tested, F03, is a therapeutic that binds three different receptors involved in cognitive function. The on-going Phase 1b/2a trial is a randomized, double-blind, placebo-controlled study assessing the safety, tolerability, pharmacokinetics and preliminary efficacy of two dose levels of F03 in 36 aMCI patients. If results, currently anticipated by the end of summer of 2015, are positive, F03 will advance into more advanced clinical studies involving significant numbers of patients in the U.S. as well as Australia. F03 has been approved in Australia (and 48 other countries) for use in a different indication; it is very well tolerated and has a very strong safety profile and history.

“We are excited to commence the first clinical trial of F03 for the treatment of aMCI,” says Stelios Tzannis, PhD, the Buck’s Director of Clinical Sciences, “F03’s novel, multi-targeted mechanism of action gives us increased hope that it may be a highly valuable therapeutic candidate with potential disease-modifying capacity.”

Emanating from groundbreaking research by Dale Bredesen, MD, and his lab at the Buck, F03 addresses a key process in the brain that is distorted earlyon in the development of Alzheimer’s disease: the processing of amyloid precursor protein (APP). A number of supporting preclinical studies have demonstrated that F03 significantly improves cognition and reverses memory loss in mouse models of Alzheimer’s, providing early evidence for a potentially disease-modifying effect.

“It is extremely rewarding to have this work move into human trials—we are optimistic about this important first trial,” says Bredesen, whose Buck lab continues to work on Alzheimer’s therapeutics, while he also takes the helm as the Director of the Mary S. Easton Center for Alzheimer’s Research at the University of California, Los Angeles.

The F03 clinical trial was enabled by federal grants as well as support from the Ellen and Douglas Rosenberg Foundation, Bechtel Foundation, Alzheimer’s Association, and philanthropic support from Hussam Abu Issa. “We are grateful for the varied sources of support that led to this groundbreaking work.” Tzannis says. “The Buck Institute continues to build and expand its position in the field of Alzheimer’s disease and other neurodegenerative disorders, and has multiple new funding opportunities. We are poised to move our groundbreaking science towards development of novel, safe and effective drugs to combat these devastating diseases.”

About the Buck Institute for Research on Aging

The Buck Institute is the U.S.’s first independent research organization devoted to Geroscience – focused on the connection between normal aging and chronic disease. Based in Novato, CA, The Buck is dedicated to extending “Healthspan”, the healthy years of human life and does so utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and those focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimer’s and Parkinson’s, cancer, cardiovascular disease, macular degeneration, osteoporosis, diabetes and stroke.  In their collaborative research, they are supported by the most recent developments in genomics,proteomics, bioinformatics and stem cell technologies. For more information:   www.thebuck.org

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