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 formulation 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 lead pre-clinical 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

Consistent Efficacy Across Biomarker & Clinical Studies

Alzheon Pipeline - Efficacy Across Biomarker & Clinical Studies

ALZ-801 Pill Inhibits Formation of Amyloid 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

Critical Need for Drugs that Stop Disease Course

No New Alzheimer’s Drugs Brought to Patients in 16 years*

  • Only 1 drug out of 244 successfully completed development between 2002 & 2012*
  • Why have so many Alzheimer’s trials failed?
    • Patient selection addressed by ALZ-801
      • Inaccurate diagnosis: almost third of patients had no brain amyloid
      • Considerable disease heterogeneity even in amyloid-positive patients
      • Intervention too late in disease: need to intervene at early or mild AD stage
    • Candidate drug mechanism of action addressed by ALZ-801
      • Targeting wrong type of amyloid: solid plaque vs. small soluble oligomers
      • Doses of anti-amyloid antibodies too low to remove amyloid oligomers
    • Safety & route of administration addressed by ALZ-801
      • Most antibodies cause dose-dependent brain edema & microbleeds
      • Route of administration: infusion vs. safe oral pill
*Cummings (2018) Alzheimers Dement; Cummings (2014) Alzheimers Dement