Biogen Idec had some encouraging word for the treatment of Parkinson’s disease this morning. The Cambridge, MA-based biotech company (NASDAQ: BIIB) said its experimental drug for the disease reached its goal of demonstrating effectiveness in a pair of mid-stage clinical trials, although it didn’t go into detail about how good the data really was.
The drug, BIIB-014, reached its goal in a study of 40 patients who took the treatment alone for early stage Parkinson’s, and was also effective in about 70 patients with more severe forms of the disease who took it in combination with standard L-dopa, Biogen said. The news came from Cecil Pickett, Biogen’s president of research and development, in a presentation today to investors at the JP Morgan Healthcare Conference in San Francisco.
Biogen isn’t saying yet what its next steps will be in developing the drug, said spokeswoman Naomi Aoki. But the company clearly has high hopes for this program, as we described last week. Parkinson’s 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 ailment, according to the National Parkinson Foundation. The last truly big innovation for patients with Parkinson’s came in the 1960s, with L-dopa, a drug used to help replenish the brain’s diminishing supply of a neurotransmitter called dopamine. But the drug’s effect diminishes after a few years, and patients develop secondary symptoms over time like cognitive decline, depression, anxiety, and involuntary movements as the drug’s effect wears off.
Researchers have tried lots of different approaches to treating Parkinson’s, like gene therapies, cell-replacement therapies, and deep-brain stimulation, with little to show for it. 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, the company says. Kenilworth, NJ-based Schering-Plough, and Swiss drug giant Roche are also pursuing drugs designed to hit the same target on cells.
Pickett’s R&D update also had some newsy nuggets for treatment of multiple sclerosis and cancer. The company is planning to go ahead with a final-stage clinical trial program of a longer-lasting form of interferon beta therapy (Avonex) made through pegylation chemistry. This treatment would allow patients to take injections every other week or once a month, instead of the much more frequent injections currently required by standard treatments. The final stage trial will enroll 1,260 patients with a goal of reducing the number of MS flare-ups over a year, he said. “We think this will increase patient compliance, and increase convenience,” Pickett said.
The company is also forging ahead with a drug designed to block heat shock protein 90, a so-called chaperone protein that’s plays a role in tumor growth. An interim analysis of a mid-stage clinical trial of the drug among patients with gastrointestinal stromal tumors produced positive data, and the company now plans to initiate other mid-stage studies against different tumor types, Pickett says. This program would put Biogen in competition with Cambridge, MA-based Infinity Pharmaceuticals among others aiming to block this protein. “We think ours may be best in class,” Pickett said.