A precision medicine approach to Alzheimer’s disease

Alzheon is developing ALZ-801, a Phase 3-ready tablet, one of the few oral drug candidates in advanced stages of clinical testing in Alzheimer’s disease (AD). ALZ-801 is well-tolerated and has the potential to slow or stop the course of the disease in patients with Alzheimer’s.

ALZ-801, an improved prodrug formulation, builds upon a large dataset of approximately 2,000 Alzheimer’s patients treated with the active molecule, tramiprosate. Using a precision medicine approach, we are focusing our initial development on high risk APOE4/4 homozygous patients with early to mild Alzheimer’s disease.

About ALZ-801

Scientific Insights Pioneered by Alzheon

Will be Applied to our Phase 3 Program

  • Effective inhibition of brain toxins causing Alzheimer’s – addressed by ALZ-8011
    • ALZ-801 fully inhibits formation of soluble amyloid oligomers = neurotoxic form of beta amyloid & avoids interaction with insoluble fibrillar plaque
    • Robust brain penetration & target engagement using small molecule approach drives clinical benefits
    • ALZ-801 enhances body’s natural defense against formation of toxic amyloid oligomers
    • In contrast, brain levels of anti-amyloid antibodies too low to remove amyloid oligomers
  • Safe oral tablet designed to slow or stop disease progression – addressed by ALZ-8012
    • Inhibits brain shrinkage associated with disease onset & progression
    • Favorable safety without brain edema or microbleeds caused by some antibodies
  • Accurate patient selection by application of precision medicine – addressed by ALZ-8013
    • Genetic testing identified patients at high risk for AD & highly responsive to our medication
    • Avoids inaccurate diagnosis: almost third of clinically diagnosed patients had no brain amyloid
1 Alzheon publications:  Kocis (2017) CNS Drugs; Hey (2018) CNS Drugs; Hey (2018) Clin Pharmacokinet
2 Alzheon publications:  Hey (2018) Clin Pharmacokinet; Tolar (2019) Alzheimers Dement
3 Alzheon publications:  Abushakra (2016) J Prev Alz Dis; Abushakra (2017) J Prev Alz Dis; Tolar (2019) Alzheimers Dement

APOE4/4 AD Patients Optimal for Initial Approval

APOE4-4 Alzheimer's Disease

Alzheimer’s Program Summary

ALZ-801 Alzheimers

Projected Readout of Alzheimer’s Late-Stage Trials

ALZ-801 Potentially One of the First Oral Approved Disease Modifying Drugs