NIH Funds Novel Small Molecule Approach to Interrupt Alzheimer’s Dementia

The grant fuels a novel drug discovery program targeting hippocampal overactivity.

Neurotherapeutic drug developer AgeneBio recently disclosed the company’s novel small-molecule discovery program has been awarded a grant for as much as $10 million from the National Institutes of Health’s (NIH) National Institute on Aging (NIA). The funds are intended to support exploration of GABAA a5, the company’s Positive Allosteric Modulator (PAM) program which targets hippocampal overactivity identified with mild cognitive impairment (MCI), a precursor to the onset of Alzheimer’s dementia.

AgeneBio said the grant is funded as part of the Blueprint Neurotherapeutics Network (BPN), an initiative launched by the NIH Blueprint for Neuroscience Research, a collaboration among affiliated, relevant NIH Institutes and centers. The grant is designed to also foster collaboration between the NIH and private industry. Under the alliance, AgeneBio will team with the BPN network, academic institutions and contract research organizations to forward the GABAA a5 discovery process and advance it to Phase I clinical trials.

AgeneBio explained GABA functions as an inhibitory neurotransmitter able to limit overactive neurons. Because of the high density of GABAA a5 receptors in the hippocampus, said AgenBio, “compounds that act as GABAA a5 PAMs are well positioned to attenuate and control hippocampal overactivity.” The program said the company, is designed to enhance the activity of GABA at the GABAA a5 receptor, which improves memory function and demonstrated in preclinical testing.

According to AgeneBio, grant funds will accrue to $10 million if the program reaches agreed upon milestones. AgeneBio's Vice President of Research and Development Sharon Rosenzweig-Lipson, noted "We are grateful to the NIH for this grant and the significant Blueprint resources to help advance AgeneBio's GABAA discovery program. "This support for our GABAA discovery program recognizes the scientific potential to delay the onset of Alzheimer's dementia by targeting the marked hippocampal overactivity that is present during MCI due to AD. We look forward to furthering our program with this tremendous support."

Speaking for the NIA, Program Director Lorenzo M. Refolo, explained that the project continues the NIA’s commitment to develop effective therapies by supporting a pipeline of innovation to support potential new therapies against novel drug targets including addressing MCI. “Research that advances understanding of the underlying causes of MCI is vital to the field of neuroscience," said Charles Cywin, National Institute of Neurological Disorders and Stroke Program Director, who manages the network.


Nigel Walker

Mr. Walker is the founder and managing director of That’s Nice LLC, a research-driven marketing agency with 20 years dedicated to life sciences. Nigel harnesses the strategic capabilities of Nice Insight, the research arm of That’s Nice, to help companies communicate science-based visions to grow their businesses. Mr. Walker earned a bachelor’s degree in graphic design with honors from London College of Communication, University of the Arts London, England.