Biogen (BIIB) and Ionis Pharmaceuticals' (IONS) approach to Lou Gehrig's disease flopped over the weekend, leading IONS stock to collapse to a five-year low Monday.
The duo tested their drug, tofersen, in patients with amyotrophic lateral sclerosis, also known as ALS. Patients with this disease lose muscle control due to dysfunction in the nerve cells of the brain and spinal cord.
Tofersen-treated patients didn't improve enough to deem the study a success, and IONS stock tumbled. SVB Leerink analyst Mani Foroohar says the data release marks "another misstep" for one of Ionis' partnered programs in central nervous system disorders. Previously, Roche (RHHBY) discontinued a study of Ionis' Huntington's disease treatment.
IONS Stock Dives On Misstep
Patients showed improvement in function — as measured by a scale for ALS assessment — but the results weren't strong enough to be statistically significant.
Biomarker data also suggested the drug was, in fact, engaging the mutated gene responsible for patients' disease. Further, patients who received tofersen showed greater declines in a marker of neurodegeneration, known as neurofilament, than those who received the placebo.
But the drug also carried some side effects. Serious adverse events occurred in almost 6% of patients who received tofersen vs. none in the placebo group, Needham analyst Joseph Stringer said in a report. Those included inflammation in the spinal cord, an infection called meningitis, a disease of the lumbar spinal nerve root and other nervous system disorders.
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