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October 17, 2024

AgeneBio’s Phase 2B Trial Shows AGB101 Slows Progression of Brain Atrophy in Patients with Mild Cognitive Impairment Due to Alzheimer’s Disease Who Are Non-Carriers of the APoE-4 Allele

• The Phase 2B trial showed 40% less clinical decline and significantly reduced atrophy of the

entorhinal cortex, a key marker of disease progression, compared to placebo in ApoE-4 non-

carriers with MCI due to AD

• Patients were given a once-daily dose of AGB-101, a proprietary low-dose extended-release

formulation of levetiracetam or placebo in a randomized controlled trial.

 

(Baltimore, Maryland) October 11, 2024 – Today, AgeneBio, Inc. published new findings from the

HOPE4MCI Phase 2B study (NCT03486938) sharing promising results for Alzheimer’s patients who

do not carry an ApoE-4 allele. The trial evaluated 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). Following the publication of the clinical-cognitive results showing a 40% reduction

in progression over 18 months on the Clinical Dementia Rating Scale (CDR) published earlier this

year, AgeneBio has now published the results of AGB101 treatment on biomarkers

of AD in Alzheimer’s & Dementia: Translational Research & Clinical Interventions.

 

Reported findings show that in ApoE-4 non-carriers, AGB101 not only matched the efficacy of FDA-

approved biologics in clinical-cognitive progression in prodromal AD but surpassed any currently

published data for AD therapeutics by significantly reducing atrophy of the entorhinal cortex, a key

marker of disease progression in MCI due to AD. In addition, the reduction of brain tissue loss in

AGB101 treated participants was correlated with a reduction of blood-based biomarkers of

neurodegeneration, further supporting a slowing of neurodegeneration in treated participants

compared to controls.

 

Results of the HOPE4MCI trial to date provide strong support for further testing of AGB101 in

patients with MCI due to AD who are non-carriers of ApoE-4. AgeneBio is currently completing an

extension of this study in the United States (NCT05986721).

 

AGB-101 is being investigated to target hippocampal overactivity to slow progression and delay the

onset of AD dementia. This therapeutic approach was motivated by extensive preclinical data

indicating that hippocampal overactivity is a driver of neurodegeneration and the spread of tau

pathology, along with clinical data showing that patients with MCI clinical symptoms and amyloid

deposits in brain have levels of hippocampal overactivity that exceed those associated with normal

aging. The HOPE4MCI study builds on earlier clinical research that demonstrated the efficacy of

AGB101 to reduce hippocampal overactivity and improve episodic memory.i

 

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.

 

Cautionary Note Regarding Forward-Looking Statements

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.

 

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Media Contact:

Hilary Lefebvre

hilary@foxcroftstrategy.com

(212) 300-4929

 

i 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.