Alzheon raises $10m to advance oral Alzheimer's candidate


Armed with preliminary clinical data, US start-up Alzheon has raised $10 million to help take its oral Alzheimer's therapy ALZ-801 into a phase II/III trial.

The financing means that Alzheon can now press on with developing a once-daily formulation of ALZ-801 and preparing for the efficacy study, which it hopes to get underway before the end of the year, according to chief executive Martin Tolar.

Like many Alzheimer's candidates ALZ-801 targets beta amyloid, which forms the characteristic plaques that are seen in the brains of people who develop the disease. While the list of drugs that target amyloid has been a catalogue of failures to date, Alzheon has capitalised on recent data from Biogen which restored faith in the approach.

In a phase Ib trial, Biogen's beta amyloid monoclonal antibody aducanumab was shown to reduce amyloid plaques in patients in the very early stages of Alzheimer's whilst also slowing cognitive impairment.

The finding lent weight to the view that earlier trials of amyloid-targeting therapies failed because they were given too late in the disease process, and the company is now planning phase III trials later this year.

Alzheon's ALZ-801 candidate differs from most others in development because it is an oral therapy. It is a prodrug of tramiprosate, a drug originally developed by Bellus Health, which has already tried, and failed, to show an effect in two large phase III trials in Alzheimer's disease.

Tolar maintains the team at Alzheon knows why that trial failed, and has modified the tramiprosate molecule to improve its tolerability, stability and penetration into the central nervous system, as well as its duration of action in the body.

In addition, the company believes the earlier trials were flawed by a less-than-expected deterioration in the placebo group which made it hard to show a significant benefit.

It intends to test the drug in a narrower population than before – enrolling only patients with a specific gene mutation called ApoE4 that accounts for around 50 per cent of Alzheimer's patients – and believes this will improve its chances of showing a benefit.

In its phase Ib trial reported last month, ALZ-801 showed a reduced tendency to cause side effects such as nausea and vomiting compared to tramiprosate, and its clinical profile suggests once-daily dosing is feasible.

The $10 million fundraising will be enough to take the company through to the start of its pivotal trials programme, but Tolar predicts it will need to undertake another investment round later this year.

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Linda Banks