- Framingham, MA-based Alzheon will advance its lead candidate for treatment of Alzheimer's disease into Phase 3 trials after two Phase 1b studies showed favorable tolerability and pharmacokinetic properties, the company said Monday.
- Alzheon said it would target a subset of patients who are homozygous for the APOE 4/4 gene in the planned trials, which are expected to begin registration in early 2017.
- Drug development in Alzheimer's has proved exceedingly difficult, with dozens of failed drugs in past years. A number of companies, including Alzheon and Axovant Sciences, have picked up these discarded drugs for further study in specific patient populations.
Alzheon's candidate, known as ALZ-801, is a prodrug version of tramiprosate, which was originally developed by a Canadian firm formerly called Neurochem.
While tramiprosate failed to improve cognitive function in a Phase 3 trial, Alzheon hopes to prove its benefit among patients who have a specific form of gene called apolipoprotein E (APOE). This APOE gene has been linked to an increased risk of developing Alzheimer's disease.
Two Phase 1b studies of ALZ-801 demonstrated "favorable" pharmacokinetic properties, such as gastrointestinal tolerability and sustained plasma levels. This allowed Alzheon to determine a clinical dose which would be bioequivalent to the tramiprosate dose used in the failed Phase 3 trial, bridging between Alzheon's development work and the previously demonstrated safety profile.
Like many others, Alzheon is betting on the "amyloid hypothesis," aiming to prevent excessive clumping of the beta-amyloid protein. But Alzheon has a hard road ahead. Phase 3 has proved a high hurdle for drug developers in the space.
All eyes will be on Singapore-based TauRx Pharmaceuticals, which plans to release data on a Phase 3 study of its drug, LMTX, at the Alzheimer’s Association International Conference this week. LMTX targets a different protein, known as tau, which represents a potential new avenue to fight Alzheimer's.
A number of big pharma companies, including Biogen, are working with or looking at tau-targeting compounds after years of developing amyloid drugs.