FILE - In this Aug. 14, 2018 file photo, Dr. William Burke goes over a PET brain scan at Banner Alzheimers Institute in Phoenix. The drug company Biogen Inc. says it will seek federal approval for a medicine to treat early Alzheimer's disease, a landmark step toward finding a treatment that can alter the course of the most common form of dementia. The announcement Tuesday, Oct. 22, 2019, is a surprise because the company earlier this year stopped two studies of the drug, called aducanumab, after partial results suggested it was not working. (AP Photo/Matt York, File)
Biogen completed two, virtually identical, trials on aducanumab, with diverging results © AP

The revival of a potential drug to fight Alzheimer’s, whose clinical trials had been declared futile just months ago, proved the value of sticking with the search for treatments for the disease when other pharmaceuticals companies are “running away”, according to the chief executive of its developer, Biogen.

By announcing on Tuesday that it would seek regulatory approval after all for its drug aducanumab, the US biotech company gave new hope to millions of patients and caregivers — and its shareholders.

Michel Vounatsos, Biogen chief executive, told the Financial Times that the new data analysed since the company halted a patient trial in March marked a “turning point in the fight against Alzheimer’s disease”. 

But among Alzheimer’s researchers and campaigners, who have lived through decades of dashed hopes, the unmistakable excitement was tempered by a battle-hardened caution. Biogen shares reflected that: having opened almost 40 per cent higher, they closed on Tuesday with a somewhat more modest 26 per cent gain.

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While the opportunity is huge, so are the hurdles. The theory that Alzheimer’s patients suffer from a build-up of amyloid plaques — hard clumps of protein between nerve cells in the brain — may have found statistical support in a large-scale human trial for the first time, but the full data will not be published until December.

Biogen licensed aducanumab in 2007 from Neurimmune, a Swiss company which had found evidence some people benefited from antibodies that cleared away the plaques, and has been working to turn these antibodies into a potential drug ever since.

In March a so-called futility analysis of its two late-stage trials for the drug found the benefits did not outweigh the risks. The company abandoned the trials, seemingly adding aducanumab to the list of once-promising Alzheimer’s drugs that have failed in the late stages of testing, leaving the industry with little to show for the billions of dollars spent.

Mr Vounatsos said Biogen remained focused on finding treatments for the degenerative condition when other “companies are running away” and called the reversal “a testament to our persistence and pioneering role”.

Higher doses proved key to trial’s success

The Cambridge, Massachusetts-based company said studying a larger data set had shown aducanumab did work if it was taken at higher doses, improving patient cognition and their ability to perform daily tasks such as cleaning and shopping and slowing their clinical decline by 23 per cent compared to a placebo. Crucially, it announced that the US drug regulator was open to it filing for an approval. 

One of the two trials met its goal. On the study that failed, a subset of patients saw success: those who had taken more of the drug also had their decline slowed. Biogen had at first been cautious about giving higher doses because of a side effect that swelled the brain — but the majority of patients did not experience any symptoms. 

Paola Barbarino, chief executive of Alzheimer’s Disease International, which represents 100 Alzheimer’s associations and federations around the world, described the announcement as “the most positive piece of news we have had in decades” but warned that it was “quite early stages”. 

Analysts questioned whether the data really would be enough to convince the US Food and Drug Adminstration to approve the drug. Evan Seigerman, an analyst at Credit Suisse, warned investors to beware of “hype” around the prospective Alzheimer’s drug while it remains unclear which patients it works best for or how to decide on the right dose while avoiding side effects. 

Others were relieved they had not been wrong to be shocked in March. “After months of apologising for getting this story so wrong, we are relieved and not entirely surprised,” said Thomas Shrader, an analyst at BTIG. “Our detailed molecular thesis for aducanumab was that removing ‘junk’ from a patient’s brain should help.”

Biogen must have received a “very strong signal” from the FDA to announce that it will seek approval, just a day after its second meeting with the agency, said Geoffrey Porges, an analyst at SVB Leerink. 

“I think the FDA of five to 10 years ago would have thrown this out and said, ‘Go and do another study.’ But the current FDA is fairly patient — and industry-friendly and seems inclined to find a way to approve the drug,” he said.

The prize, and the price, of an Alzheimer’s drug

Even if it does win approval, Biogen will have to be careful how it prices the treatment, given insurers and healthcare systems may struggle to pay for it for millions of patients. Mr Vounatsos would not discuss pricing at this stage, but said the disease created half a trillion dollars in direct and indirect costs, indicating how the company will seek to justify its price tag.

James Kupiec, chief medical officer of PromisNeurosciences and the former head of Pfizer’s neuroscience unit, said Biogen’s reversal will “revitalise” the work pursuing the hypothesis that amyloid plaque causes the cognitive decline seen in Alzheimer’s patients, which many scientists had recently “poo-pooed”.

Ms Barbarino agreed it could catalyse investment in the field. “If one company is successful . . . that will create more interest and more pharmacological innovation,” she said. It would also help draw new young researchers, with fresh ideas, to the work. “If all you hear is failure, people are less likely to join the fray,” she said. 

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Few companies had given up entirely on Alzheimer’s research, Ms Barbarino suggested, since many big-name pharma businesses were at least continuing to explore data sets. Shares in smaller, Alzheimer’s-focused biotechs Denali Therapeutics and Alector rose more than 5 per cent on Tuesday.

Alison Evans, head of policy at Alzheimer’s Research UK, said that scientists should also continue to look beyond the amyloid plaque. “It is really important we explore all avenues,” she said. “We have so much unmet need, we need as many shots on goal as possible.”

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