Biogen (Nasdaq: BIIB) announced today that in the Phase 2b dose-ranging ACTION 2 study in individuals with acute ischemic stroke (AIS), natalizumab did not demonstrate improvement in clinical outcomes compared to placebo. Both doses of natalizumab were generally well-tolerated and no new or important safety signals were observed. The results of the Phase 2b ACTION 2 study do not impact the benefit-risk profile of natalizumab in approved indications, including multiple sclerosis.
“As pioneers in neuroscience, Biogen remains committed to developing treatments for people with acute neurological conditions including stroke,” said Michael Ehlers, executive vice president, Research & Development at Biogen. “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) for prevention and treatment of edema in large hemispheric infarction, one of the most severe types of stroke.”
In the middle of 2018, Biogen plans to initiate a global Phase 3 study of BIIB093 in individuals with large hemispheric infarction, where brain swelling (cerebral edema) often leads to high morbidity and mortality.
Detailed Phase 2b ACTION 2 study findings will be made available in a future scientific forum.
About the natalizumab Phase 2 Development Program in AIS
ACTION 2 was a Phase 2b multicenter, double-blind, placebo-controlled, randomized, dose-ranging study with a 90 day follow up to evaluate the safety and efficacy of natalizumab primarily in patients with moderate severity acute ischemic stroke (AIS). The study investigated natalizumab vs placebo in approximately 270 individuals who had a clinical diagnosis of AIS with last known normal (LKN) ≤ 24 hours prior to treatment initiation. The study evaluated a 300 mg dose and a 600 mg dose versus placebo, both either within 9 hours of LKN or between 9-24 hours after LKN.
The primary objective of ACTION 2 was to assess the effects of natalizumab compared to placebo on clinical measures of independence and activities of daily living. The primary endpoint was a composite global measure of functional disability based on a score of 0 to 1 on a modified Rankin scale (mRS) and a score of ≥ 95 on the Barthel Index (BI) at Day 90. The mRS measures independence with specific tasks pre- and post-stroke. BI is a scale that consists of 10 items that measure activities of daily living and mobility.
Natalizumab was previously evaluated in AIS in the Phase 2a ACTION study. In this study, although natalizumab did not significantly decrease the primary endpoint of infarct volume at Day 5, secondary and exploratory endpoints suggested natalizumab treatment improved clinical outcomes compared with placebo which warranted further evaluation.
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases. Founded in 1978 as one of the world’s first global biotechnology companies by Charles Weissman, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, today Biogen has the leading portfolio of medicines to treat multiple sclerosis; has introduced the first and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programs in Alzheimer’s disease and dementia, multiple sclerosis and neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology. Biogen also manufactures and commercializes biosimilars of advanced biologics.
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This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, relating to potential clinical effects of natalizumab, the potential benefits, safety, and efficacy of natalizumab, risks and uncertainties associated with drug development and commercialization, the results of certain real-world data, the timing and scope of future clinical trials, including for BIIB093, the timing and status of Biogen’s current and future regulatory filings, and the potential of Biogen’s commercial business and pipeline programs, including natalizumab and/or BIIB093. These forward-looking statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “except,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will,” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.
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