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

« back to Press Releases • 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. ### Media Contact: Hilary Lefebvre [email protected] (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.

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