Biogen‘s made headlines of late for committing $2.5 billion on a Alzheimer’s drug development gamble – and, notably, preparing for a return to the ALS market. Making such broad commitments can stir a lot of hope up among a desperate patient base – something CEO George Scangos has learned a lesson or two about by now.
Two years ago, Biogen famously halted Phase 3 trials of its drug for amyotrophic lateral sclerosis. The biggest disappointment didn’t come from the huge financial hit. It was the emotional toll that such a failure took on the patients and families – and the employees that worked on the drug for years, Scangos said at this week’s World Medical Innovation Forum in Boston.
“Not only did it not meet it its endpoint, it was the single-most negative trial I’ve ever seen,” he said.
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The trial had generated an immense amount of hope among the ALS community – so the failure was particularly devastating, Scangos said. He’s been very transparent about the trial’s failure, having actually written a Bloomberg editorial at the time on what the failure meant to Biogen, as well as ALS patients and their families. But he cut to the heart of it this week.
“We knew that if the trial were positive, that we would be under tremendous pressure to make that drug available to patients even before approval,” he said. “So we spent some number of millions of dollars, just so we’d have it ready.”
A Piper Jaffray consultant told the Wall Street Journal that the costs of merely halting the Phase 3 portion of the ALS drug at that time was between $75 million and $100 million. When it comes to these laborious Phase 3s that cost hundreds of millions of dollars, workers of course break out the champagne when results are positive, Scangos said.
“In the case of that ALS trial, there were tears. People broke down in that room. I guarantee nobody was thinking about how much less money we’d make,” he said. Just the dashed hopes of the patients, he said.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Biogen’s current approach is to dive into the genetics of neurodegenerative disease. It’s starting with Alzheimers, aiming for approval in five years, then building on multiple sclerosis and, bravely, circling back to ALS.
But that old trial was not based on any genetics of the disease, he said – rather, it was based on a drug – dexpramipexole – whose mechanism was unknown, only hypothesized about. But it had interesting data in a small Phase 2 trial.
“It was an old-fashioned trial,” Scangos said. “That was the last trial of that kind we’d ever do.”
[Picture courtesy of Getty Images]
