Researchers plot a course into PhIII after another Alzheimer’s drug flunks a key test

Paul Aisen

Another big Alzheimer’s drug study has foundered in the clinic, but it’s being positioned for Phase III in any case.

This time the failure belongs to Toyama Chemical, a subsidiary of Fujifilm, which wound up recruiting close to 500 patients with a mild-to-moderate form of the disease for the trial.

According to the company, the drug failed the primary endpoint and the secondaries, proving unable to do any better than a placebo in improving cognition or function. But, researchers say they tracked a benefit in a key biomarker for tau on the high dose while spotting an improvement among earlier-stage patients in a post hoc analysis of the data.

Backers of this drug believe that it can influence levels of tau as well as amyloid beta, a one-two therapy targeting two big culprits behind the disease. And Toyama wants to take it into Phase III to find out how a better designed trial can work.

That has not proven to be a winning formula in the past, with 15 years of pivotal failures to prove it. But Toyama, like others in the field, believe that what they have learned from their failures can pave the way to success.

The study was launched by Paul Aisen back in 2014, well before his group split away from UC San Diego and defected to USC. Aisen has played a leading role in some of the biggest flops in the business, including his outspoken support for Eli Lilly’s solanezumab, which failed a slate of late-stage studies. According to Fujifilm, the original preclinical work on T-817MA was done by Rudy Tanzi, a Harvard professor who claimed in 2014 to have figured out a new approach to researching Alzheimer’s drugs that would deliver a winner in the clinic.

Tanzi himself is the scientific founder of Prana Biotechnology $PRAN, a tiny company with a market cap of only $22 million that claimed to see great success in a failed Phase II study of a novel Alzheimer’s drug inspired by Tanzi’s work on the role of zinc and copper balances.

Tanzi responded to our story in a comment:

For the record, I never in any forum publicly or privately “claimed to see great success on a failed Phase II” Alzheimer’s trial with PBT2. Perhaps you’re referring to the Huntington’s disease trial where I was impressed by the effects of PBT2 on executive function, while acknowledging that the overall trial failed. I will also say regarding Alzheimer’s disease that I was encouraged enough by the results for PBT2 in lowering senile plaque counts (in the treatment group) to continue believing, as I do today, that this drug may potentially be useful for preventing and treating Alzheimer’s. It is currently on partial clinical hold by the FDA.

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