Biogen's Antisense Candidate Flunks In Late-Stage Neurodegenerative Disorder Trial

Biogen's Antisense Candidate Flunks In Late-Stage Neurodegenerative Disorder Trial
  • Biogen Inc BIIB has announced topline results from its Phase 3 VALOR study of tofersen (BIIB067), an investigational antisense drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
  • The drug failed the primary endpoint on significantly improving patients' functional and neurologic decline over 28 weeks and the extension period for continued observation.
  • There were other key misses on the secondary front as well. Still, Biogen's CEO Michel Vounatsos is gain choosing to put anything supporting a positive outcome in the spotlight. 
  • Data showed trends favoring tofersen across multiple secondary and exploratory measures of biologic activity and clinical function.
  • Change from baseline in total CSF SOD1 protein, a marker of target engagement, differences were observed between the tofersen and placebo groups of 38% and 26% in the faster- and slower-progressing populations, respectively. 
  • Change from baseline in plasma neurofilament light chain, a potential marker of neuronal degeneration, differences between the tofersen and placebo groups were 67% and 48%.
  • Biogen licensed tofersen from Ionis Pharmaceuticals Inc IONS under a collaborative development and license agreement.
  • Price Action: BIIB shares traded 1.13% lower at $278, while IONS shares are down 5.13% higher at $33.30 premarket on the last check Monday.

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