Applying precision medicine to find a better solution

Alzheon’s valiltramiprosate/ALZ-801 is currently the only oral anti-amyloid treatment in Phase 3 testing for Alzheimer’s disease. We believe that valiltramiprosate has the potential to significantly slow or stop disease progression, and even prevent the onset of clinical symptoms.

Development of valiltramiprosate, a well-tolerated new treatment, is based on ten years of Alzheon research into the biology of Alzheimer’s. Safety profile in valiltramiprosate studies remains favorable & consistent with prior data in over 3,000 AD patients, with no increased risk of vasogenic brain edema.

Using our pioneering precision medicine approach, we are initially focusing our development on the highest risk patients with two copies of the APOE4 gene (APOE4/4 homozygotes), who are predicted to benefit most from the valiltramiprosate treatment. Future trials will expand treatment to patients with only one copy of the APOE4 gene, and ultimately to prevention of onset of Alzheimer’s.

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Wall Street analyst Robyn Karnauskas of Truist Securities discussing with Alzheon management team new data and therapeutic potential of valiltramiprosate.

Valiltramiprosate/ALZ-801 acts upstream of anti-amyloid drugs in AD

Alzheon publications: Tolar (2024) Int J Mol Sci; Tolar (2021) Int J Mol Sci; Tolar (2020) Alzheimers Res Ther; Tolar (2019) Alzheimers Dement

Validated precision medicine approach supported by experts

  • Received $51M in grants from National Institute on Aging to support APOLLOE4 Phase 3 study
  • Prominent global Scientific Advisory Board
  • Alzheon published 10 peer reviewed scientific publications on key discoveries in Alzheimer’s:
    1. Identification of neurotoxic amyloid oligomers that initiate and drive Alzheimer’s pathogenesis
    2. Biological diagnosis of Alzheimer’s using fluid biomarkers and imaging
    3. Use of biomarkers to assess disease progression and efficacy of treatments
    4. Role of APOE4 genotype in biology of Alzheimer’s
Phase 2 trial Biomarker Steering Committee: the top 3 experts
Kaj Blennow, MD, PhD
Developed p-tau181 biomarker for AD
(on Aduhelm’s FDA label); Professor of Clinical Neurochemistry, University of Gothenburg, Sweden
Eric Reiman, MD
Developed p-tau217 biomarker for AD; Executive Director of Banner Alzheimer’s Institute, Chief Executive Officer of Banner Research, Phoenix
Philip Scheltens, MD, PhD
Developed imaging & fluid AD biomarkers; Managing Partner of LSP Dementia Fund; Director of the Alzheimer Center, VU University, Amsterdam, Netherlands

Reflects current best internal estimates, subject to change; NDA – New Drug Application; sNDA – Supplemental New Drug Application; Phase 2 and Phase 3 studies continuing with Longterm Extension Studies (LTEs)

Valiltramiprosate development strategy