We had a drug called dexpramipexole—dex, for short—that was in development for ALS, or Lou Gehrig’s disease. It’s a devastating disease that often strikes young people. From diagnosis to death takes about three years. They stop being able to walk, then stop being able to move, and eventually they can’t breathe. All this time, their cognitive functions are completely unimpaired. There’s a person trapped inside this body that doesn’t work.
There’s only one drug on the market to treat ALS right now, and it provides only moderate benefits, so there’s a great need for something else. Biogen (BIIB) has great expertise in neurodegenerative diseases and has been interested in them for some time.
We licensed dex from another company named Knopp Biosciences that had been developing it. In Knopp’s phase II study of the drug, the results were encouraging. The decision we had to make was, should we or should we not invest in this? Research and development is risky. It’s expensive. We have to be very thoughtful and rigorous about how we spend the company’s money. We looked at the data and thought, is this a bet we wanted to take? We decided it was.
We conducted a phase III trial with 943 patients who were treated with dex for 12 to 18 months. We did a rigorous trial and got a definitive answer: The drug didn’t work. We sat around the table during the presentation of the results, and when we learned about the data, there were tears. It’s incredibly disappointing.
You read about a lot of drugs that fail in trial, and the company says something like, “We’re exploring it further.” When something like this fails, you lose money. Sometimes you don’t want to admit it. But I think we have a responsibility to patients. You shouldn’t be taking this drug.
I’ve thought a lot about whether we made the right decision to invest in dex. I think we did. If you avoid the risk, you’re never going to develop anything that’s innovative. We’ll continue to have programs on the disease, and I’m hopeful we’ll come up with something else. In the past year and a half we’ve had four drugs in phase III trials. The other three were positive, and this one was negative. I’ll take that 75 percent batting average any day. — As told to Claire Suddath