3-SPA, also a Product of ALZ-801, Prevents Formation of Toxic Beta Amyloid Oligomers, the Key Driver of Alzheimer’s Disease

FRAMINGHAM, Mass., July 14, 2019 – Alzheon, Inc., a clinical-stage biopharmaceutical company focused on developing new medicines for patients suffering from Alzheimer’s disease (AD) and other neurological and psychiatric disorders, today announced the upcoming presentation at the Alzheimer’s Association International Conference (AAIC) being held in Los Angeles, California, from July 14-18, 2019.

At the AAIC, Alzheon will present nonclinical and clinical data from patients with Alzheimer’s and other neurodegenerative diseases, showing that an endogenous substance in the human brain inhibits the formation of neurotoxic beta amyloid oligomers, which are key drivers of AD pathogenesis.  The substance was identified as 3-sulfopropanoic acid (3-SPA), the primary metabolite of tramiprosate and of its prodrug ALZ-801 in humans.  The cognitive improvements observed in AD patients in the tramiprosate Phase 3 studies may be attributed, in part, to the therapeutic effects of 3-SPA in the brain.  This discovery indicates a potential protective role of 3-SPA in aging human brains and in AD and elucidates the beneficial pharmaceutical attributes of ALZ-801, including a favorable safety profile, selectivity against beta amyloid oligomers, and excellent brain penetration.

“We are excited to advance the understanding of the pathogenic and therapeutic mechanisms underlying Alzheimer’s disease.  The results from this publication point to a potential protective role of endogenous 3-SPA in normal human brain, guarding against the formation of beta amyloid oligomers that cause neurodegenerative disorders such as Alzheimer’s,” said Martin Tolar, MD, PhD, Founder, President and CEO of Alzheon.

The details for the presentation at AAIC are as follows:

Poster Presentation #P1-043

Title: Inhibition of Amyloid Oligomer Formation by Endogenous Metabolite of ALZ-801/ Tramiprosate in Human Brain: Discovery and Characterization of 3-SPA in Normal Subjects and Alzheimer’s Disease Patients
Session: Therapeutics – Clinical
Date/Time:  Sunday, July 14, 9:30am – 4:15pm
Location: South Hall GH – Los Angeles Convention Center
Presenter: John A. Hey, PhD, Chief Scientific Officer, Alzheon, Inc.
Other Authors:  Jakub Hort, MD, PhD, Motol University Hospital, Charles University, Prague, Czech Republic; Susan Abushakra, MD, Alzheon, Inc; Aidan Power, MD, Alzheon, Inc.; Martin Vyhnalek, MD, PhD, International Clinical Research Center, St. Anne’s University Hospital, Czech Republic; Petr Kocis, PhD, Alzheon, Inc.; Jeremy Yu, MD, PhD, Alzheon, Inc.; Martin Tolar, MD, PhD, Alzheon, Inc.


About ALZ-801

Alzheon’s lead product candidate, ALZ-801, is a novel, oral anti-amyloid drug candidate that is an optimized prodrug of tramiprosate, which has shown promising results in analyses of clinical data and therapeutic mechanism of action.  ALZ-801 received Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2017. The clinical data for ALZ-8011 and its active agent, tramiprosate, suggest long-term clinical efficacy in AD patients with the APOE4 genotype, along with a favorable safety profile.2,3  ALZ-801 acts through a novel molecular mechanism of action blocking the formation of toxic amyloid oligomers4 associated with the development and progression of AD.  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:

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


Kathryn Morris
The Yates Network