Ionis Pharmaceuticals (IONS) Achieves Major Milestone

Ionis shares saw a big pop in pre market trading on Monday, rising 37% to $40.10 after being down 53% year to date.

The big move came after news revealed that an experimental treatment for a rare children’s spine disorder that was developed by Ionis (Nusinersen), has achieved the primary endpoint of a late-stage clinical trial. This result allows Biogen (BIIB) to exercise an option to take over development of the drug and seek marketing approval.

Spinal muscular atrophy is a rare, genetic disease that causes muscle atrophy and weakness in infants and young children. Severe forms of the disease can lead to paralysis and even death.

Ionis’ Nusinersen is the lead drug in their antisense drug development pipeline.

Both companies announced that Nusinersen’s safety profile in the study was “acceptable.”

Ionis will receive $75 million from Biogen for the exercise of the partnership option. If nusinersen is eventually approved, Ionis is eligible for another $150 million in milestone payments plus a mid-teens percentage royalty on sales.

Disclaimer: We have no position in Ionis Pharmaceuticals Inc. (NASDAQ: IONS) nor Biogen Inc. (NASDAQ: BIIB) and have not been compensated for this article.