The billion-dollar gamble: Biotech CEO takes a risky second shot at an Alzheimer’s drug

The executives packed into the room, eager to hear the good news on their blockbuster-to-be.

Medivation, a California startup, was developing a treatment for Alzheimer’s disease, and pivotal results from a major clinical trial were finally available. An earlier study, conducted in Russia, had generated what Alzheimer’s experts hailed as the best results the field had ever seen. Dr. David Hung, the CEO, believed he had a billion-dollar product.

But the results disclosed that day in 2010 were disastrous. The drug, Dimebon, failed all five of the trial’s key metrics, performing even worse than placebo on two of them. Medivation lost more than $1 billion in value in the first hour of trading as the company’s leaders struggled to process the startling failure.

By shifting focus to oncology, Hung was able to save Medivation — and, eventually, sell it to Pfizer for $14 billion — in a rebound that made him something of a legend in biotech circles, not to mention a very wealthy man. But Hung, revered by his colleagues for his almost childlike sense of scientific wonder, never shook his ambition to develop a treatment for Alzheimer’s.

So he’s doing it again — headed for another crowded room and another make-or-break clinical trial. This time it’s with Axovant Sciences. Axovant’s big trial, scheduled to read out this month, is . If the drug succeeds, Axovant’s stock price could rise