AgeneBio Announces Completion of Patient Enrollment in Phase 2B Clinical Trial to Evaluate AGB101 to Treat Amnestic Mild Cognitive Impairment Due to Alzheimer's Disease

BALTIMORE, April 21, 2021 /PRNewswire/ -- AgeneBio announced today that it has completed enrollment in a Phase 2B clinical trial evaluating the efficacy of AGB101, a once-a-day investigational medication to treat amnestic Mild Cognitive Impairment due to Alzheimer's Disease (MCI due to AD). The clinical trial, known as "HOPE4MCI" (NCT03486938), is a 78-week study, randomized, double-blind, placebo controlled study that has enrolled 164 patients across 23 sites in the United States and Canada.

Accumulated evidence has demonstrated that neural overactivity drives both amyloid and tau pathology in brain. The HOPE4MCI study builds on an earlier trial¹ that demonstrated the efficacy of AGB101 to reduce hippocampal overactivity and improve episodic memory. Cognitive and functional efficacy in the HOPE4MCI study will be measured by the Clinical Dementia Ratings Scale – Sum of Boxes (CDR-SB) (primary endpoint), Mini Mental State Exam and Functional Activities Questionnaire (MMSE, FAQ). The CDR-SB has been recognized by the FDA as an appropriate endpoint for potential registration trials.

The HOPE4MCI trial also includes a sub-study of brain PET imaging to track measures of neurodegeneration (structural MRI, Tau PET). The use of [18F]MK-6240 scanning for tau, developed by Cerveau Technologies, together with a structural brain analysis developed in collaboration with the Johns Hopkins Center for Imaging Science and Bioclinica, will trace the spread of pathology over the length of the clinical protocol and its potential modification by therapeutic treatment.

"The scale of interest that we've seen in this trial, even as patients and their doctors navigate unprecedented challenges posed by the global pandemic, underlines the urgency of unmet needs in Alzheimer's research," said Sharon Rosenzweig-Lipson, Vice President of Research and Development for AgeneBio.

"As the population continues to age, the need for approved treatment options for Alzheimer's Disease will become even more critical," said Richard Mohs, Vice President of Clinical Development for AgeneBio and Principal Investigator for HOPE4MCI. "We are thrilled with the progress of the trial and are looking forward to sharing our results at the end of next year."

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 MCI due to AD, the symptomatic pre-dementia stage of Alzheimer's Disease. AgeneBio's Phase 3 candidate AGB101 is the first and only therapeutic being investigated to target hippocampal overactivity to slow progression to and delay the onset of Alzheimer's dementia. The HOPE4MCI trial is partially supported under a public/private partnership by grant funding from NIA and awards from the Alzheimer's Drug Discovery Foundation. The HOPE4 MCI trial is registered in clinicaltrials.gov as NCT03486938. AgeneBio also has a novel GABA-A alpha5 small molecule program in late discovery stage being investigated for a spectrum of untreated conditions including MCI due to AD, autism and schizophrenia. Learn more at www.agenebio.com and follow us on Twitter @AgeneBio.

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Statements herein relating to the future of AgeneBio, Inc. and the ongoing development of its products are forward-looking statements. AgeneBio, Inc. cautions that these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Disclosure: Research reported in this publication was supported by the National Institute On Aging of the National Institutes of Health under Award Number R01AG061091. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

¹ ClinicalTrials.gov. John Hopkins University. (2017, August 21). Levetiracetam and Memory Function in Amnestic Mild Cognitive Impairment (MCI).  Identifier NCT01044758. Retrieved from: https://clinicaltrials.gov/ct2/show/study/NCT01044758?term=levetiracetam%2C+alzheimer%27s&rank=5.

SOURCE AgeneBio