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.