Alzheon CEO Presents Overview of Oral Anti-Amyloid ALZ-801 Phase 3 Program at 9th Neurodegenerative Drug Development Summit

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FRAMINGHAM, Mass.--(BUSINESS WIRE)--Alzheon, Inc., a clinical-stage biopharmaceutical company focused on developing new medicines for patients suffering from Alzheimer's disease (AD) and other neurodegenerative disorders, today announced that Founder, President and CEO Martin Tolar, MD, PhD, will deliver a presentation and join a panel discussion at the Neurodegenerative Drug Development Summit on February 24th.

The kick-off panel discussion at 9:10 AM ET will focus on recent progress in drug development across the industry and new research into the mechanisms driving neurodegenerative pathology, among other topics.

"Using genetics-based precision medicine and building on the remarkable advances in fluid biomarkers, we are advancing our oral tablet ALZ-801 into Phase 3," said Dr. Tolar. "This Phase 3 study could support New Drug Application submission in 2024, and we are maximizing the probability of success by incorporating the latest fluid and imaging biomarkers to ensure that we have the most robust data elucidating patient benefits throughout the trial."

Dr. Tolar's presentation at 3:10 PM ET will provide an overview of Alzheon's oral Phase 3 drug candidate, ALZ-801, which prevents formation of neurotoxic soluble amyloid oligomers that drive onset and progression of AD. As an oral small molecule drug targeting amyloid toxicity, ALZ-801 is in a class of its own, and has shown potential for robust efficacy and favorable safety in the high-risk population of patients with two copies of the apolipoprotein ε4 allele (APOE4/4).

With endorsement and support from the National Institute of Aging in the form of a $47 million grant, Alzheon's Phase 3 trial of ALZ-801 will begin enrolling patients in the 2nd quarter of 2021. Alzheon's precision medicine approach will enroll the highest-risk patient group of APOE4/4 subjects, and will incorporate the latest biomarkers to track patient benefits — p-tau blood biomarkers, hippocampal volume and cortical thickness measures, as well as tau positron emission tomography imaging in a subset of enrolled subjects.

"ALZ-801 has the potential to be used as a disease modifying monotherapy or in combination with anti-amyloid antibodies," said Dr. Tolar. "There have been several developments in the last year that dramatically changed the risk-reward calculus in assessing the likelihood of approval for late-stage Alzheimer's treatments. Multiple trials now support the role of toxic forms of amyloid in driving tau pathology and disease progression; new, low-cost and non-invasive biomarkers associated with clinical benefit are being incorporated into clinical trials to help guide therapy; and there is unprecedented support to bring treatments to patients by 2025."

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Following the presentation, Dr. Tolar will participate in a live Q&A session at 4:20 PM ET.

About ALZ-801

An oral anti-amyloid drug, ALZ-801 is an optimized prodrug of tramiprosate that has shown promising results in analyses of Phase 3 clinical data,6,8 and has a novel anti-amyloid oligomer mechanism of action.4,7 ALZ-801 received Fast Track designation from the U.S. Food and Drug Administration in 2017. The clinical data for ALZ-801 and its active agent, tramiprosate, indicate long-term clinical efficacy in AD patients with the APOE4 genotype and a favorable safety profile.4,6,8 ALZ‑801 acts through a novel enveloping molecular mechanism of action to fully block formation of neurotoxic soluble amyloid oligomers6 associated with the onset of cognitive symptoms and progression of AD.2,3 The cognitive improvements observed in the tramiprosate Phase 3 studies may also be attributed in part to the therapeutic effects of 3-sulfopropanoic acid (3-SPA), an endogenous anti-oligomer substance in the human brain discovered by Alzheon scientists that, like tramiprosate, inhibits formation of toxic amyloid oligomers.4 3-SPA is the primary metabolite of ALZ-801 and its discovery helps explain the beneficial pharmaceutical attributes of ALZ-801, including favorable safety profile, high selectivity for amyloid, and excellent brain penetration. ALZ-801 treatment increases levels of 3-SPA in the brain and augments the body's natural mechanism to inhibit formation of toxic amyloid oligomers.4,5 The initial Phase 3 program for ALZ-801 will focus on Early AD patients with the APOE4/4 genotype, with future expansion to investigate ALZ‑801 for prevention of Alzheimer's onset and in patients carrying only one copy of the APOE4 gene.1,2,3

About Alzheon

Alzheon, Inc. is committed to developing innovative medicines by directly addressing the underlying pathology of devastating neurodegenerative disorders. Our lead Alzheimer's clinical candidate, ALZ-801, is an oral small molecule prodrug of tramiprosate that fully blocks formation of neurotoxic soluble amyloid oligomers in the brain. ALZ-801 is an easy-to-take tablet that builds on the safety and efficacy profile of its active compound tramiprosate, which has been evaluated in clinical trials involving over 2,000 Alzheimer's patients. Our clinical expertise and technology platform are focused on developing drug candidates using a precision medicine approach based on individual genetic and biological information to advance therapies with the greatest impact for patients.

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Alzheon Scientific Publications

1 Abushakra S, et al: APOE ε4/ε4 Homozygotes with Early Alzheimer's Disease Show Accelerated Hippocampal Atrophy and Cortical Thinning that Correlates with Cognitive Decline, Alzheimer's & Dementia 2020; 6: e12117.
2 Tolar M, et al: Aducanumab, Gantenerumab, BAN2401, and ALZ-801—the First Wave of Amyloid-Targeting Drugs for Alzheimer's Disease with Potential for Near Term Approval, Alzheimer's Research & Therapy, 2020; 12: 95.
3 Tolar M, et al: The Path Forward in Alzheimer's Disease Therapeutics: Reevaluating the Amyloid Cascade Hypothesis, Alzheimer's & Dementia, 2019; 1-8.
4 Hey JA, et al: Discovery and Identification of an Endogenous Metabolite of Tramiprosate and Its Prodrug ALZ-801 that Inhibits Beta Amyloid Oligomer Formation in the Human Brain, CNS Drugs, 2018; 32(9): 849-861.
5 Hey JA, et al: Clinical Pharmacokinetics and Safety of ALZ-801, a Novel Prodrug of Tramiprosate in Development for the Treatment of Alzheimer's Disease, Clinical Pharmacokinetics, 2018; 57(3): 315–333.
6 Abushakra S, et al: Clinical Effects of Tramiprosate in APOE4/4 Homozygous Patients with Mild Alzheimer's Disease Suggest Disease Modification Potential, Journal of Prevention of Alzheimer's Disease, 2017; 4(3): 149-156.
7 Kocis P, et al: Elucidating the Aβ42 Anti-Aggregation Mechanism of Action of Tramiprosate in Alzheimer's Disease: Integrating Molecular Analytical Methods, Pharmacokinetic and Clinical Data, CNS Drugs, 2017; 31(6): 495-509.
8 Abushakra S, et al: Clinical Benefits of Tramiprosate in Alzheimer's Disease Are Associated with Higher Number of APOE4 Alleles: The "APOE4 Gene-Dose Effect," Journal of Prevention of Alzheimer's Disease, 2016; 3(4): 219-228.stats
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