BALTIMORE, MD — AgeneBio, a pharmaceutical company developing innovative therapeutics for unserved patients battling amnestic mild cognitive impairment (aMCI), the pre-dementia stage of Alzheimer's disease, and other neurological and psychiatric diseases, announced today the publication of complete Phase 2 clinical trial results in NeuroImage: Clinical for its lead product candidate. The results show that AGB101 restored brain network function and significantly improved memory in elderly patients with aMCI. The Phase 2 trial was conducted by Johns Hopkins University and supported by a grant from the National Institutes of Health.
AgeneBio's AGB101 is a proprietary once-a-day low-dose formulation of levetiracetam, an anti-epileptic commercialized for more than a decade with a well-characterized safety profile. AGB101 will be given to patients at approximately one-fifteenth of the dose most commonly prescribed for epilepsy. AgeneBio expects to initiate the AGB101 Phase 3 clinical program, the primary endpoint for which is aligned with recent US Food and Drug Administration guidance for aMCI trials, in the second half of 2015. If approved, AGB101 will be the first and only therapeutic that reduces hippocampus overactivity and could be the first therapeutic to slow progression to, and delay the onset of, Alzheimer's dementia.
Phase 2 Trial
Hippocampal overactivity in aMCI patients is well documented by leading research centers around the world as the signature characteristic in the aMCI stage of Alzheimer's disease and the best predictor of subsequent cognitive decline and progression to Alzheimer's dementia. The Phase 2 trial is the first and only clinical trial to target hippocampal overactivity during aMCI. The randomized double-blind study with subject crossover employed fMRI BOLD activation to detect effects of drug treatment across a range of doses on brain function together with memory performance. The study included 69 patients, with 54 aMCI and 17 control patients completing. Results showed that AGB101 dosed at 125 or 250 mg daily reduced excess hippocampal activity to a normal range and significantly improved memory performance in aMCI patients.
AgeneBio's drug discovery work is based on the research of its founder, Michela Gallagher, PhD, Krieger-Eisenhower Professor of Psychological and Brain Sciences and Principal Investigator of the Neurogenetics and Behavior Center at Johns Hopkins University. Dr. Gallagher noted, "Based on our research to date, AGB101 maps across all preclinical and clinical studies, consistently restoring normal brain function and preserving memory. Given the consistent translation of our research to date, we expect our upcoming AGB101 Phase 3 trial to demonstrate efficacy in preserving cognition and memory in aMCI patients while delaying progression to the clinical stage of Alzheimer's dementia."
"The AGB101 Phase 2 results show that intervening during aMCI, the symptomatic pre-dementia stage of Alzheimer's, offers an excellent prospect to delay disease progression," said AgeneBio CEO Jerry McLaughlin. "More than five million Americans battle aMCI, and the pathway to Alzheimer's dementia is almost assured with more than seven of every 10 aMCI patients progressing in less than seven years. If approved, we believe this breakthrough approach to delaying the onset of Alzheimer's dementia offers both a significant commercial opportunity and momentous social value to patients and families battling this debilitating disease."
About aMCI, the Pre-Dementia Stage of Alzheimer's Disease
Today 5.6 million Americans and 25 million people globally suffer from amnestic mild cognitive impairment (aMCI), the symptomatic, pre-dementia stage of Alzheimer's disease characterized by significant loss of memory, 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 with the cost expected to exceed $1 trillion by 2050. Data from the National Institute on Aging show that modest delays have massive benefits to patients, their families, and society as a whole. If the onset of Alzheimer's dementia can be delayed, there is an approximately 10% reduction in the prevalence of this disease for every one-year delay in the onset of Alzheimer's dementia.
AgeneBio, Inc., is an emerging pharmaceutical company dedicated to developing innovative therapeutics that prevent neurodegeneration and preserve and restore cognitive function for unserved patients battling amnestic mild cognitive impairment (aMCI), the symptomatic pre-dementia stage of Alzheimer's disease, and other 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 aMCI. 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 GABA-A alpha5 small molecule program in late discovery stage with therapeutic potential for a spectrum of untreated conditions including aMCI, autism and schizophrenia. Learn more at www.agenebio.com.