AgeneBio Publishes Phase 2 Results for Delaying Onset of Alzheimer’s

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, has published complete Phase 2 clinical trial results in NeuroImage: Clinical for its lead product candidate. According to the company, 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.

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 company says 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.”