Biogen Idec will get an early glimpse next week into whether it has created an important new innovation for Parkinson’s disease. If the company’s drug candidate lives up to its promise, it could be one of the early movers in a new class of medicines that minimize the secondary symptoms and keep standard therapy from wearing off over time.
The drug is code-named BIIB-014, and I got an overview of it from Spyros Papapetropoulos, an associate medical director at Biogen. It was a good time to talk, since the Cambridge, MA-based biotech company (NASDAQ: BIIB), which has operations in San Diego, expects to release some results from a mid-stage clinical trial next week at the JP Morgan Healthcare Conference in San Francisco.
Parkinson’s, as many people understand thanks to actor Michael J. Fox, is a degenerative disease of the central nervous system that robs patients of their ability to control movement and speech. About 1.5 million people in the U.S. have this chronic disease, according to the National Parkinson Foundation. The last truly big innovation for this disease came in the 1960s, with L-dopa, a drug used to help replenish the brain’s diminishing supply of a neurotransmitter called dopamine. Researchers have tried lots of different approaches, like gene therapies, cell-replacement therapies, and deep-brain stimulation, with little to show for it.
“Dare I say that we’re optimistic,” Papapetropoulos says.
Here’s the problem as Biogen sees it. L-dopa-related treatments are reasonably effective at controlling the most visible symptom of the disease, the uncontrolled tremors and rigidity, but aren’t as good at controlling secondary symptoms like depression, anxiety, or cognitive decline, Papapetropoulos says. Usually, after a few years of treatment on L-dopa, the drug starts wearing off after a couple hours, instead of maintaining its effect for a full five to six hours. Plus, strangely, in an attempt to enable movement, the treatment can go overboard, stimulating too many involuntary twitches and movements through what are known as “dyskinesias,” he says.
Biogen’s goal is for its product to keep the L-dopa from wearing off, and to stop those uncontrolled movements from cropping up. The Biogen candidate, an oral pill, is designed to block a specific receptor on brain cells called A2a. Blocking this target is supposed to help restore normal brain circuitry, Papapetropoulos says.
Biogen isn’t the only company pursuing this concept. Kenilworth, NJ-based Schering-Plough (NYSE: SGP) recently released positive results from a mid-stage clinical trial that showed its candidate, preladenant, could keep the L-dopa from wearing off, Papapetropoulos says. Swiss-based pharmaceutical giant Roche also has a candidate in this class of treatment, he says.
The drug, BIIB-014, which Biogen in-licensed from U.K.-based Vernalis in 2005, is now being tested in a pair of mid-stage clinical trials. The first study involves about 40 patients with early-stage Parkinson’s taking the drug by itself in a variety of doses, compared with a placebo. The second study involves about 70 patients with later-stages forms of Parkinson’s who took the Biogen candidate in a couple different doses in tandem with L-dopa, and will compare their performance with patients who got a placebo. Preliminary results from both studies should be available in the first quarter, says Biogen spokeswoman Shannon Altimari.
Critical work on this program on the medical side is happening at Biogen’s offices in Cambridge, while important toxicology work in animals is being done in San Diego, Papapetropoulos says. This is the only Parkinson’s candidate in the Biogen clinical trial pipeline, although the company has developed a backup compound to hit this same target, he says.
Papapetropoulos didn’t try to overpromise what this compound can do, other than to say, “We will be expecting some efficacy trends to be present.” Biogen highlighted a 30-60 minute improvement in maintaining the effective control of Parkinson’s without involuntary movements, in a meeting last month with analyst Christopher James of Rodman & Renshaw. James noted that the company emphasized its R&D pipeline candidates for multiple sclerosis and heart failure, as well as the Parkinson’s program.
Papapetropoulos was recovering from a case of laryngitis when we spoke, and after politely answering all my questions, he made sure to get the last word in our conversation. “I’d really like to end on a note of optimism,” he said.