Biogen barrels toward Alzheimer's Phase III after a promising early signal

Biogen Idec ($BIIB) is heralding hope in one of the industry's toughest fields, plotting a late-stage trial for an Alzheimer's disease treatment after charting some surprising positive results in an early study.

Doug Williams

The treatment, BIIB037, is an antibody targeting beta amyloid proteins, which build up in the brain and, it is widely believed, contribute to Alzheimer's neurodegenerative effects. In a Phase Ib study designed to measure the drug's safety, Biogen noted a dose- and time-dependent reduction in amyloid levels at 52 weeks, R&D chief Doug Williams said at the Deutsche Bank BioFEST conference, and BIIB037 also had a positive effect on patients' cognition.

Now Biogen is "planning very aggressively" to get the antibody into Phase III, Williams said, as quoted by CNBC, a pronouncement that sent the Big Biotech's shares up nearly 8% on Tuesday morning. The company hasn't disclosed a particular timeline for its late-stage program.

Biogen hopes BIIB037 can succeed where so many once-promising programs have failed. Many Alzheimer's treatments--including Eli Lilly's ($LLY) solanezumab and Pfizer ($PFE) and Johnson & Johnson's ($JNJ) bapineuzumab--have demonstrated great potential in early trials only to fail miserably in Phase III, contributing to a daunting 99.6% clinical trial failure rate for such drugs, according to Cleveland Clinic's Jeffrey Cummings.

And the same fate could well befall Biogen's drug, but the rampant spread of Alzheimer's and potential windfall in store for a successful treatment are enough to make any measurable clinical activity a big deal to investors and investigators.

BIIB037 is one facet of Biogen's all-hands approach to Alzheimer's. Alongside partner Eisai, the company is working on a treatment that targets beta secretase, which key to the production of amyloid, and one that homes in on the tau protein, also found in Alzheimer's sufferers. Per a deal signed in March, Eisai has the option of jointly developing BIIB037 under undisclosed terms.

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