AgeneBio, a leading biopharmaceutical company specialising in central nervous system (CNS) disorders, has announced the enrolment of the first patient in its pivotal phase 3 clinical trial for AGB101, a promising treatment for amnestic Mild Cognitive Impairment (MCI) caused by Alzheimer’s Disease (AD). The trial, named HOPE4MCI, aims to assess the efficacy and safety of AGB101 in slowing the progression of MCI due to AD, with the goal of preventing or delaying the onset of Alzheimer’s dementia.

A pivotal step towards new Alzheimer’s treatment

The HOPE4MCI trial, a late-stage clinical study, will enrol 830 patients, aged between 55 and 85 years, across the US, Canada, and Europe. The primary objective of this trial is to evaluate whether AGB101 can halt the progression of symptoms associated with MCI due to AD. The patients will be randomly assigned to receive either a placebo or 220mg of AGB101, administered once daily for 78 weeks.

Currently, no approved treatments are available for MCI due to AD, making this trial a critical step forward for Alzheimer’s research. AgeneBio’s efforts could potentially offer a new therapeutic option for millions of patients grappling with this early stage of cognitive decline.

Building on promising phase 2 results

This phase 3 trial builds on the success of AgeneBio’s previous phase 2 clinical trial, which demonstrated that AGB101 could effectively address hippocampal overactivity, a hallmark feature of MCI due to AD. The earlier trial also showed positive outcomes in enhancing episodic memory, which is often the first cognitive function to be impaired in patients with MCI.

As part of the HOPE4MCI trial, AgeneBio will also conduct a sub-study featuring specialized brain imaging techniques. These advanced imaging methods will track changes in the brain over the course of the trial, providing valuable insights into the progression of neurodegeneration. Secondary imaging endpoints will include measures of structural changes in the brain such as MRI scans and Tau PET imaging.

Expert insights on a breakthrough approach to Alzheimer’s treatment

Sharon Rosenzweig-Lipson, AgeneBio’s Vice President of Research and Development, expressed her enthusiasm about the trial, highlighting the innovative approach of targeting neuronal overactivity. She noted that the trial marks a shift from traditional amyloid-focused treatments, as it explores the potential to alleviate cognitive impairment by addressing the underlying neural activity that drives Alzheimer’s pathology.

“We are pleased to be at the forefront of a new direction in Alzheimer’s Disease research, moving beyond amyloid-directed therapeutics to target the neuronal overactivity that drives downstream pathology and memory impairment,” Rosenzweig-Lipson said. “Based on our Phase 2 findings, we are excited about the potential for this novel therapy to provide hope for millions of people who suffer from amnestic mild cognitive impairment due to Alzheimer’s Disease, a condition with no approved treatment options.”

AGB101: a unique therapeutic candidate

AGB101 is a novel, once-daily extended-release formulation of levetiracetam, an FDA-approved antiepileptic drug. AgeneBio’s formulation delivers the drug at a fraction of the typical dose used to treat epilepsy, specifically targeting brain network imbalances that lead to memory loss and brain atrophy. This innovative approach distinguishes AGB101 as the first and only product candidate designed to address these underlying neurological issues.

With the onset of Alzheimer’s dementia being an unmet clinical need, the results of the HOPE4MCI trial could potentially lead to a breakthrough treatment for millions of patients suffering from MCI due to Alzheimer’s Disease.

  AGB101, AgeneBio, Alzheimer’s Disease, Brain Atrophy, HOPE4MCI, Levetiracetam, Memory loss, Mild Cognitive Impairment, phase 3 clinical trial