Alzheon Pipeline 2017-10-13T17:55:51+00:00



Amyloid Oligomers: The Missing Piece in AD Puzzle

Beta amyloid proteins are produced and cleared from the brain as a part of a normal biological process. With advanced age, the clearance of beta amyloid is impaired, and beta amyloid proteins aggregate into small soluble amyloid aggregates, called oligomers, as well as longer amyloid fibrils and plaques, the hallmark pathology of Alzheimer’s disease (AD). Oligomers have been identified as the toxic agents that lead to injury of brain cells and neurodegeneration, progressive cognitive and behavioral impairment, and ultimately, death.

Aging is the main risk factor for the development of AD, resulting in the growing prevalence of the disease as people live longer. Alzheon is building a pipeline of oral, beta amyloid-targeting drugs to block this pathological process and to treat a wide range of diseases caused or worsened by beta amyloid deposits.

How-ALZ-801-works_Alzheon_Alzheimers-Disease-treatmentALZ-801 – An Optimized Prodrug

ALZ-801, our lead drug candidate, is an optimized, oral prodrug of the active molecule tramiprosate. Tramiprosate has been studied in more than 2,000 Alzheimer’s patients with up to 2.5 years of treatment, and has a favorable safety profile. We have leveraged this significant dataset in Alzheimer’s patients and uncovered new insights into the efficacy of tramiprosate in genetically-defined subpopulations allowing us to accelerate ALZ-801 into a pivotal clinical program. After confirmation of clinical efficacy and initial approval, ALZ-801 can be developed for all patients with Alzheimer’s disease.  Learn about the Underlying Clinical Data of ALZ-801.

ALZ-1903 – Next Generation Clinical Lead

Improved capacity to block formation of neurotoxic oligomers compared to tramiprosate

New chemical entity with favorable profile:

  • Robust activity in multi-ligand binding assay, directly translating to MOA
  • High aqueous solubility and excellent predicted bioavailability supporting oral dosing
  • Alzheon-owned composition of matter IP expires in 2036




First Indication: Mild AD patients with homozygous APOE4/4 genotype


Second Indication: Mild AD patients with heterozygous APOE4 genotype


Down syndrome


Familial AD and cerebral amyloid angiopathy and traumatic brain injury



Other beta amyloid indications – glaucoma, age-related macular degeneration, inclusion body myositis