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Alzheon Publication Defines New Path for 2nd Generation Anti-Amyloid Drugs for Alzheimer’s Disease, Supporting Launch of Phase 3 Study with ALZ-801 Oral Tablet

November 6, 2019

FRAMINGHAM, Mass.--(BUSINESS WIRE)--Nov 6, 2019--

Alzheon, Inc., a clinical-stage biopharmaceutical company focused on developing new medicines for patients suffering from Alzheimer’s disease and other neurodegenerative disorders, today announced the publication of a peer-reviewed research paper “The Path Forward in Alzheimer’s Disease Therapeutics: Reevaluating the Amyloid Cascade Hypothesis,” in Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association, available through open access at this link: https://doi.org/10.1016/j.jalz.2019.09.075

The publication, authored by Alzheon experts, offers six key insights about therapeutic strategies for Alzheimer’s disease (AD), at a time when few drugs remain in advanced clinical trials. The key properties of 2 nd generation anti-amyloid drugs that address the limitations seen with treatments to date are: simple oral dosing; high brain penetration; selectivity for soluble amyloid oligomers, the most toxic form of amyloid; and favorable safety, avoiding the risk of brain edema and microhemorrhage associated with plaque-clearing treatments. This improved pharmacological profile will enable anti-amyloid drugs to achieve robust and meaningful clinical efficacy, and to realize the full potential of this most promising class of Alzheimer’s medicines. These insights have also guided Alzheon strategy in developing ALZ-801, which is the only remaining Phase 3 candidate with the attributes required for the next generation of anti-amyloid therapies.

The review builds upon learnings from genetic, biomarker, and clinical studies, as well as from new discoveries made by Alzheon scientists elucidating the novel mechanism of targeting toxic soluble amyloid oligomers, and provides the following insights:

The publication also highlights a series of discoveries that the Alzheon team has made and published:

“We have a historic opportunity to bring to patients and their families the first safe, oral treatment, with the potential to slow, or even stop, disease progression in Alzheimer’s. ALZ-801 augments the body’s natural mechanism for blocking the formation of toxic amyloid oligomers that can lead to memory loss, and drive the onset and progression of Alzheimer’s symptoms,” said Martin Tolar, MD, PhD, Founder, President and Chief Executive Officer of Alzheon. “Based on our insights, we are poised to initiate a pivotal Phase 3 trial with ALZ-801 – the only oral anti-amyloid drug remaining in the late-stage trials for Alzheimer’s. Our ultimate goal with ALZ-801 is to prevent the onset of clinical symptoms in the aging population, and to preserve their independence and dignity.”

“This review offers an important blueprint for Alzheimer’s drug developers, because it builds on the key learnings of the research community and capitalizes on the convergence of new science around synaptotoxic amyloid oligomers as triggers of disease progression” said Marwan Sabbagh, MD, Director of Cleveland Clinic Lou Ruvo Center for Brain Health, scientific advisor to Alzheon and co-author of the publication. “Recent validation of amyloid targeting by aducanumab brought up the necessity to prevent the formation of amyloid aggregates and the onset of the clinical symptoms of Alzheimer’s – and this is what we hope the new therapeutic approach will accomplish.”

About ALZ-801

Alzheon’s lead product candidate, ALZ-801, is an oral anti-amyloid drug candidate that is an optimized prodrug of tramiprosate, which has shown promising results in analyses of clinical data 1,2 and a novel therapeutic mechanism of action. 3 ALZ-801 received Fast Track designation from the U.S. Food and Drug Administration. The clinical data for ALZ-801 4 and its active agent, tramiprosate, suggest long-term clinical efficacy in AD patients with the apolipoprotein ε4 (APOE4) genotype and a favorable safety profile. 1,2 ALZ-801 acts through a novel molecular mechanism of action blocking the formation of toxic amyloid oligomers 3 associated with the development and progression of AD. The cognitive improvements observed in AD patients in the tramiprosate Phase 3 studies may be attributed, in part, to the therapeutic effects of 3-sulfopropanoic acid (3-SPA), an endogenous substance in the human brain, discovered by Alzheon scientists, that inhibits the formation of neurotoxic beta amyloid oligomers. 5 3-SPA is the primary metabolite of ALZ-801 in humans and its discovery elucidates the beneficial pharmaceutical attributes of ALZ-801, including a favorable safety profile, selectivity against beta amyloid oligomers, and excellent brain penetration. ALZ-801 increases levels of 3-SPA in the brain and augments the body’s natural mechanism for blocking the formation of toxic amyloid oligomers. 5 The initial Phase 3 program for ALZ-801 will focus on patients with the homozygous APOE4/4 genotype at the Mild stage of AD, with the potential for future expansion to additional Alzheimer’s populations.

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 a Phase 3-ready, first-in-class, small molecule oral inhibitor of beta amyloid aggregation and neurotoxicity – hallmarks of Alzheimer’s disease. ALZ-801 is a novel prodrug that builds on the safety and efficacy profile of the 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.

Alzheon Publications

1Abushakra et al. Journal of Prevention of Alzheimer’s Disease, 2016
2Abushakra et al. Journal of Prevention of Alzheimer’s Disease, 2017
3Kocis et al. CNS Drugs, 2017
4Hey et al. Clinical Pharmacokinetics, 2018
5Hey et al. CNS Drugs, 2018

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CONTACT: Kathryn Morris

The Yates Network

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kathryn@theyatesnetwork.com

KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS

INDUSTRY KEYWORD: BIOTECHNOLOGY PHARMACEUTICAL HEALTH CLINICAL TRIALS

SOURCE: Alzheon, Inc.

Copyright Business Wire 2019.

PUB: 11/06/2019 06:00 AM/DISC: 11/06/2019 06:01 AM

http://www.businesswire.com/news/home/20191106005355/en