In an effort to develop and commercialize central nervous system disorder therapies, such as Alzheimer’s disease and schizophrenia, Vanderbilt University’s Warren Center for Neuroscience Drug Discovery (VCNDD) entered into an exclusive worldwide licensing and collaboration deal with San Diego-based ACADIA Pharmaceuticals Inc. The deal targets a lead compound discovered by Vanderbilt researchers called VU319. Now in a Phase I clinical trial, the hope is that this new drug could help slow memory loss associated with these severe brain disorders. VCNDD showcases a model for de-risking drug discovery by blending academic basic research with state-of-the-art drug discovery resources.
TrialSite News provides a breakdown of this recent activity.
What is VU319?
VU319 was developed by a team of scientists at the Vanderbilt Center for Neuroscience Drug Discovery (VCNDD) led by center director Jeffrey P. Conn and Craig W. Lindsley, VCNDD co-director of Medicinal Chemistry. Representing a small-molecule approach back in 2017, the target was identified as “an exciting advance from current available therapies” by Paul Newhouse, director of the Center for Cognitive Medicine at Vanderbilt University. The investigational product is here today thanks to a value-added partnership between VCNDD and the William K. Warren Foundation of Tulsa, Oklahoma which has injected over $8 million into the discovery effort since 2014.
A putative cognitive enhancer, VU319 is a muscarinic M1-positive allosteric modulator current under investigation as a potential therapy for Alzheimer’s disease. Its target, the M1 muscarinic acetylcholine receptors, are located on neurons activated by the neurotransmitter acetylcholine.
By selectively tuning up the activity of the M1 receptor, Vanderbilt researchers suspect VU319 can overcomes some past failings involving previous attempts to ramp up M1: notably adverse effects, such as incidents of abnormally slow heart rate and gastrointestinal distress triggered by collateral activation of other muscarinic receptors.
Quite Possibly a First in 2017
Research on the potential drug started over ten years ago, and by 2017, the university received U.S. Food and Drug Administration (FDA) approval to initiate Phase I clinical trial. Professor Conn declared then that this was the first time he was aware that an academic drug discovery team had progressed a molecule designed to treat a chronic brain disorder from laboratory to bench to early clinical trials without a pharmaceutical company partner.
What will the primary collaboration focus on?
The Vanderbilt & ACADIA collaboration centers on intellectual property developed at the university involved with positive allosteric modulators (PAMs) of the M1 receptor, including the investigational product VU139 as well as other compounds in preclinical development as well as those in an ongoing discovery program based on early-stage research in the basic sciences. These candidates, discovered by the Warren Center, work by increasing sensitivity to a neurotransmitter called acetylcholine, which researchers suspect helps regulate learning and memory.
Why did this Partnership make sense?
Professor Conn suggests the two are a great fit: ACADIA brings “proven development and commercialization capabilities in neuropsychiatric disorders” while the Warren Center’s focus on basic science and early discovery targeting important unmet needs in central nervous system disorders. Vanderbilt Law School graduate Steve Davis, who also happens to be ACADIA’s Chief Executive Officer, suggests his company benefits from Vanderbilt’s modulation approach helping his company bring new therapies to the market, noting, “ACADIA’s collaboration with Vanderbilt’s Warren Center complements our innovative late-stage pipeline.
And hammering the point home, Warren Center co-director Craig Lindsley reports, “The ACADIA collaboration is another example of the need for focused academic drug discovery to de-risk novel mechanisms and advance beyond tool compounds to clinical candidates.”
What is the ACADIA & Vanderbilt Agreement?
Actual numerical terms weren’t disclosed. However, we do know that ACADIA paid Vanderbilt an upfront payment and the university is eligible for potential milestone payments and tiered royalties.
How else does the Warren Center secure financial support?
Research at the Warren Center is supported often by the National Institute of Mental Health, the William K. Warren Foundation, the Alzheimer’s Drug Discovery Foundation, the Harrington Discovery Institute and the Alzheimer’s Association.
Warren Center Background
Introducing a new model for drug discovery and development, the Warren Center for Neuroscience Drug Discovery (VCNDD) “de-risks” the drug discovery process by blending academic basic research with state-of-the-art drug discovery resources. VCNDD extends traditional academic pursuits in basic science to take the most exciting advances in the understanding of human disease and drug targets to a point where these breakthroughs can directly impact patient care. See the link for leadership.
Jeffrey P. Conn, PhD, Department of Pharmacology, Professor of Pharmacology, Lee E. Limbird Chair in Pharmacology, Director, Warren Center for Neuroscience Drug Discovery
Craig W. Lindsley, PhD, UCSB, University Professor of Pharmacology, Biochemistry & Chemistry
Note, drug development is a team sport and a number of researchers were instrumental in this effort. Their names can be viewed at the Vanderbilt News source.
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