- Biogen Inc will end its efforts to test its multiple sclerosis drug Tysabri in acute ischemic stroke, pulling the plug on the program after the drug failed to meet both primary and secondary endpoints in a study known as ACTION 2.
- The Phase 2b dose-ranging trial in people with acute ischemic stroke showed no improvements in clinical outcomes compared with placebo, as gauged by clinical measures of independence and activities of daily living. Further details of the results will be released at a future scientific forum.
- Biogen still retains a foothold in stroke, with a mid-2018 Phase 3 trial planned for a candidate called BIIB093, for prevention and treatment of edema in a severe form of stroke known as large hemispheric infarction.
Correction: A previous version of this article incorrectly stated the number of Alzheimer's drugs in Biogen's pipeline and their phases.
Despite not hitting the mark in the Phase 2a ACTION study, Biogen continued to move Tysabri forward into Phase 2b, where the focus centered on improving clinical outcomes like independence and daily activity.
Now that ACTION 2 has failed as well, Biogen has finally put to bed the idea of developing Tysabri in stroke. One plus point, though — there were no new safety signals, and no impact on the benefit-risk profile of Tysabri in its approved indications.
"While we are disappointed with the ACTION 2 study results, we have furthered our knowledge of the disease and will continue to pursue innovative approaches in this area, including BIIB093 [intravenous glibenclamide]," said Michael Ehlers, executive vice president, Research & Development at Biogen.
The failure of the ACTION 2 study skews Biogen's clinical pipeline even further towards Alzheimer's disease. In its 17-product clinical pipeline, Biogen has six drugs in clinical trials in Alzheimer's, including two (aducanumab and E2609) in Phase 3 trials.
This fact concerns Jefferies equity analyst Michael Yee: "We remain long-term positive and believe the Phase 3 Alzheimer's trial can work but the company needs more balanced programs in between as it is levered too much towards Alzheimer's," Yee wrote in a note to investors.
"We viewed the Phase 2b Tysabri stroke trial as high risk/high reward… [and believe] that the company has perhaps too many high risk/high reward programs and needs perhaps more balanced programs that are lower risk but still offer promising revenue opportunity."
Last May, Biogen acquired Remedy Pharmaceuticals' Cirara (intravenous glyburide) for $120 million in cash. Cirara is expected to complete a Phase 3 trial in cerebral edema in large hemispheric infarction (LHI), a form of stroke, in May 2019. The drug, however, does not appear on Biogen's pipeline, and recruitment in the study does not appear to have started.