AgeneBio to Receive up to $10 Million in NIH Funding to Advance Program for Mild Cognitive Impairment Due to Alzheimer’s Disease

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September 13, 2017

AgeneBio to Receive up to $10 Million in NIH Funding to Advance Program for Mild Cognitive Impairment Due to Alzheimer’s Disease

Grant to support novel GABAA program targeting hippocampal overactivity that leads to neurodegeneration and cognitive decline in Alzheimer’s disease patients

Baltimore, MD, September 13, 2017 — AgeneBio, a pharmaceutical company developing innovative therapeutics for unserved patients battling neurodegeneration, today announced it has been awarded a grant for up to $10 million from the National Institute on Aging (NIA) of the National Institutes of Health (NIH) to support its novel GABAA discovery program to treat mild cognitive impairment due to Alzheimer’s disease (MCI due to AD) and delay the onset of Alzheimer’s dementia. MCI due to AD is an intermediate stage between normal cognition and Alzheimer’s dementia in which memory and cognitive abilities are markedly worse than expected for a person’s age.

The grant is funded as part of the Blueprint Neurotherapeutics Network (BPN) of the NIH Blueprint for Neuroscience Research, a collaboration of NIH Institutes and Centers that supports research on the nervous system with one goal of developing new neurotherapeutic drugs. As part of the grant, AgeneBio will team with the BPN network, capitalizing on the scientific expertise of the company while expanding access to contract research organizations and academic institutions to advance the GABAA discovery program to the first Phase 1 clinical trial. Under terms of the grant, AgeneBio can receive up to $10 million if the program continues to advance and reach milestones.

“We are grateful to the NIH for this grant and the significant Blueprint resources to help advance AgeneBio’s GABAA discovery program,” said Sharon Rosenzweig-Lipson, PhD, AgeneBio’s Vice President of Research and Development and the Principal Investigator on studies supported by this grant. “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.”

“In funding this project, the NIA continues in its commitment to feed a pipeline of innovative potential new therapies against novel drug targets,” said Lorenzo M. Refolo, PhD, Program Director, Alzheimer’s Drug Development Program, NIA.

“The Blueprint Neurotherapeutics Network is excited to provide its drug discovery and development expertise to advance this GABAA a5 Positive Allosteric Modulators program directed at MCI due to AD into the clinic. Research that advances understanding of the underlying causes of MCI is vital to the field of neuroscience,” said Charles Cywin, PhD, the National Institute of Neurological Disorders and Stroke Program Director who manages the network.

“We believe our GABAA discovery program could one day positively impact many of the millions of patients and their families affected by heavy burden of MCI due to AD,” said AgeneBio President and CEO Jerry McLaughlin. “We thank the NIA and are honored to collaborate with the Blueprint Neurotherapeutics Network on this important neuroscience research.”

AgeneBio’s pipeline of development programs is based on the research of its founder and Chief Scientific Officer, Michela Gallagher, PhD, Krieger-Eisenhower Professor of Psychological and Brain Sciences at Johns Hopkins University.

About GABAA Drug Discovery Program
AgeneBio’s novel GABAA a5 small molecule program is in discovery stage with potential to address unmet needs for several diseases of the central nervous system including MCI due to AD, autism and schizophrenia. The GABAA a5 Positive Allosteric Modulator (PAM) program targets hippocampal overactivity, which prior research suggests is a major contributor to cognitive decline and MCI due to AD. GABA functions as an inhibitory neurotransmitter thus limiting overactivity of neurons. With a high density of GABAA a5 receptors in the hippocampus, compounds that act as GABAA a5 PAMs are well positioned to attenuate and control hippocampal overactivity. This discovery program is designed to enhance the activity of GABA at the GABAA a5 receptor, which improves memory function in preclinical testing under conditions of hippocampal overactivity and aging. Proof of biology studies demonstrate that GABAA a5 PAMs from multiple structural classes occupy GABAA a5 receptors in the hippocampus and improve memory impairment in aged animals.

About MCI due to AD, the Pre-Dementia Stage of Alzheimer’s Disease
MCI due to AD is a clinical condition between normal aging and early Alzheimer’s dementia characterized by impaired memory. There is no treatment for MCI due to AD, sometimes referred to as the pre-dementia stage of Alzheimer’s disease, and most patients progress to Alzheimer’s dementia within seven to 10 years. Today 5.6 million Americans and 25 million people globally suffer from MCI due to AD, and this population will double by 2030. By age 85, one of every three people will have Alzheimer’s disease. Alzheimer’s disease currently costs Medicare and Medicaid $150 billion in direct medical costs annually, which are expected to exceed $1 trillion by 2050. Data from the Alzheimer’s Association suggests that a five-year delay in the onset of Alzheimer’s dementia could reduce its prevalence by more than 40%.

About the NIH Blueprint for Neuroscience Research
The NIH Blueprint for Neuroscience Research is a collaborative framework that includes the NIH Office of the Director and 15 NIH Institutes and Centers that support research on the nervous system. By pooling resources and expertise, Blueprint identifies cross-cutting areas of research, and confronts challenges too large for any single Institute or Center. One of Blueprint’s Grand Challenges, launched in 2011 to catalyze research with the potential to transform basic understanding of the brain and approaches to treating brain disorders, is the Blueprint Neurotherapeutics Network. The Network provides funding to researchers and biopharmaceutical companies for small molecule drug discovery and development. For more information, please visit 

About AgeneBio
AgeneBio, Inc., is a development-stage CNS biopharmaceutical company developing innovative therapeutics aimed at preserving and restoring brain function for unserved patients afflicted with neurological and psychiatric diseases. AgeneBio’s novel pipeline of therapies is based on decades of research at Johns Hopkins University and leading research centers worldwide showing that overactivity in the hippocampus contributes to cognitive impairment and drives neurodegeneration if not controlled. This overactivity is a characteristic feature of mild cognitive impairment due to Alzheimer’s disease (MCI due to AD), the symptomatic pre-dementia stage of Alzheimer’s disease. If approved, AgeneBio’s Phase 3-ready lead candidate AGB101 will be the first and only therapeutic targeting hippocampal overactivity and potentially the first therapeutic to slow progression to and delay the onset of Alzheimer’s dementia. AgeneBio also has a novel GABAA a5 small molecule program in discovery stage with therapeutic potential for a spectrum of untreated conditions including MCI due to AD, autism and schizophrenia. Learn more at and follow us on Twitter @AgeneBio.

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