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Axovant’s promising Alzheimer’s therapy fizzles

NATIONAL INSTITUTE ON AGING

Yet another once-promising treatment for Alzheimer’s disease has come up short in the final stage of development, this time adding Wall Street drama to the familiar disappointment that has plagued the drug industry for decades.

A pill called intepirdine, developed by the biotech startup Axovant Sciences, failed to blunt the symptoms of Alzheimer’s in a large trial. The drug was never considered a cure, but the company had hoped it would delay the worst symptoms of the disease, giving patients a few more months of health before needing around-the-clock care.

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Axovant, a well-funded and hotly debated company, lost about 70 percent of its value in early-morning trading Tuesday. The New York biotech was worth more than $2.6 billion before the news of intepirdine’s failure.

The company rose to that multibillion-dollar valuation on the promise that intepirdine, which had failed in four previous trials, could slow cognitive decline when paired with a generic memory-booster called Aricept. In a trial involving more than 1,300 patients with mild to moderate Alzheimer’s, the combination of intepirdine and Aricept failed to outpace the old drug alone, missing its key goals of improving memory and physical function.

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The drug industry has spent billions over the past decade-plus developing pills and injections that might reverse the course of the disease or at least arrest its progress. All showed early promise; all eventually failed, leaving the more than 5 million Americans who suffer from Alzheimer’s with few options.

The drug’s negative results did not surprise many in the industry, given that it had failed in four previous clinical trials. Other drugs that work the same way have also fallen short.

The data are devastating for Axovant, a three-year-old startup that has been hailed as one of biotech’s most ambitious endeavors.

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Axovant’s parent company, a privately held firm called Roivant, has built an inchoate empire on the idea of finding undervalued drugs languishing in other companies’ pipelines, betting it can succeed where Big Pharma inertia has failed.

Roivant paid GlaxoSmithKline just $5 million upfront for intepirdine and built Axovant around it, taking the company to Wall Street in 2015 in what was then biotech’s biggest initial public offering. Roivant’s young founder, former hedge fund manager Vivek Ramaswamy, then spun off other startups devoted to women’s health, dermatology, and rare diseases. He’s attracted major investment and media attention alike for this strategy.

But for Roivant to live up to its billing, at least some of the drugs have to work. Intepirdine’s failure, while unrelated to the fates of the firm’s other drugs, may lead investors to question the broader idea behind Ramaswamy’s empire.

Damian Garde can be reached at damian.garde@statnews.com. Follow him on Twitter @damiangarde. Follow Stat on Twitter @statnews.
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