Our Pipeline

Our lead oral product, ALZ-801, is ready for Phase 3 in Alzheimer’s

Focused on mild Alzheimer’s patients
Targeted patient selection based on APOE4 genotype
Granted Fast Track Designation by FDA for development in Alzheimer’s disease

ALZ-801 is an improved prodrug of tramiprosate which showed

Promising clinical signals in a subset of Alzheimer’s patients
Well tolerated, long-term safety evaluated in 2,000 patients at effective clinical dose

Our next clinical candidate is ALZ-507

Rapid path to clinic with IND planned for early 2020
In development for additional amyloid-related disorders

Our next generation candidate is ALZ-1903

New chemical entity
More potent inhibitor of amyloid misfolding

Discovery Platform

Rich and diverse compound libraries enabled by the novel mechanism of action
Designed to inhibit the misfolding of amyloids and other proteins associated with neurodegenerative diseases

Scientific insights pioneered by Alzheon

will be applied to our ALZ-801 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

Consistent efficacy across biomarker & clinical studies

Alzheon Pipeline - Efficacy Across Biomarker & Clinical Studies

ALZ-801 fully inhibits formation of toxic oligomers


ALZ-801 differentiation from amyloid antibodies

ALZ-801 differentiation from amyloid antibodies

Source: Binding profiles of BAN2401, Gantenerumab, Aducanumab, Crenezumab and Solanezumab are based on peer reviewed publications and information disclosed by competitors