Biogen ALS Failure Highlights Clinical Trial Frustrations

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the drug into a Phase 3 trial because an analysis after the trial ended revealed better results when the outcomes were combined with historical data of an unspecified number of patients on placebo—a very unusual type of data analysis for clinical trials.

Perrin said he wasn’t keen about the design of the NP001 Phase 2 trial, but he still agrees with the decision to keep testing the drug: “NP001 needs another shot with a larger trial. I wouldn’t throw it out.” He is more enthusiastic about tirasemtiv, an experimental drug from Cytokinetics  (NASDAQ: CYTK) of South San Francisco, CA, that is meant to boost muscle strength by amplifying the response of muscles to nerve signals. As Xconomy reported in November, Cytokinetics started enrolling approximately 400 patients in a Phase 2b trial for the drug in October. “They are doing the right type of trial,” Perrin enthused. “This drug looks pretty exciting.”

Meanwhile, Perrin worries that the Biogen Idec failure will be a setback for the whole field of ALS research. He realizes that a larger Phase 2 trial would not likely have saved the drug, “but we would have known earlier that this wasn’t the right one. A lot of patient and financial resources were wasted.” The setback with dexpramipexole “is certainly not going to help drug development [for ALS]. If it were successful I think investment in the disease would have gone up.” Instead, he fears pharmaceutical companies will shy away from ALS because they will figure it doesn’t make sense to pour money into such a risky target.

But Biogen Idec isn’t quite ready to throw in the towel. At the JP Morgan Healthcare Conference in San Francisco on Monday, CEO George Scangos told investors that the failure of dexpramipexole “has not dampened our determination to do something about this disease. We continue to work on the biology to come forward with rationally designed compounds.” He highlighted the company’s $10 million investment in a research collaboration searching for new disease targets.

Perrin’s ALS Institute also continues to explore other approaches, and on January 8 announced a collaboration with to-BBB, a Dutch company, to develop potential treatments that can cross the blood-brain barrier, which can block drugs from reaching the brain.

Desperate patients can only hope that some of this research finally pays off.

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