Our Technology

TREVENTIS has a unique and revolutionary process for identifying a single small molecule that can enter the brain and neutralize both misfolded proteins in Alzheimer’s: beta-amyloid and tau. By turning off the formation of neurotoxic proteins, we have the potential to make curative agents for Alzheimer’s disease and other diseases of protein misfolding.

The Disease

There is a large and rapidly growing unmet need for disease modifying drugs for Alzheimer’s disease.  In 2015, the Alzheimer’s Association estimated that there were 5.3 million Americans with Alzheimer’s disease; by 2050 they estimate that this number will triple to 13.8 million. It is the sixth-leading cause of death in the industrialized world – and there is no cure.

Alzheimer’s disease pathology includes “plaques” of beta-amyloid and “tangles” of tau protein.  These proteins tend to abnormally “clump”, and such protein misfolding processes give rise to inflammation and neuronal death. In order to make an impact on this disease, drugs must be found that can potently and selectively stop this process.

Therapeutic Concept

Because amyloid proteins fold into various conformations and are generally disordered, it has been impossible to develop crystal structures to support rational drug design. Treventis scientists identified a common binding site based on epitope commonality between multiple misfolded amyloids. This information was used to build an experimentally validated model of the earliest stage of misfolding: a proprietary, patent-pending system known as “common conformational morphology” (CCM).

Using CCM, we can digitally screen, identify, and optimize numerous classes of potent, drug-like compounds before making and testing them experimentally – making structure-based design a reality for anti-amyloid drug discovery.

Where We Stand

In collaboration with the Wellcome Trust, we developed compounds that have potent effects on both beta-amyloid and tau protein in vitro, are brain-penetrant, and are well tolerated in oral dosing.  We have recently shown significant effects on amyloid levels in vivo with our dual-action lead compound.  Candidate selection in tauopathy is ongoing in partnership with Servier.

A Discovery platform with wide application

Our discovery platform can identify compounds that potently block the progression of amyloid toxicity.  These compounds can be designed with selectivity toward a particular amyloid or with polypharmocology, for proteins implicated in over 30 rare/orphan diseases and major indications including

  • Alzheimer’s disease
  • Parkinson’s disease
  • Huntington’s disease
  • Type II diabetes
  • Progressive Supranuclear Palsy
  • Frontotemporal Dementia
  • Creutzfeldt-Jacob
  • Transthyretin amyloidosis