A new treatment for spinal muscular atrophy (SMA) in children being developed jointly by Biogen Inc and Ionis Pharmaceuticals Inc is set for launch this year after a successful phase III study.
Spinraza (nusinersen) met the primary endpoint of a significant improvement in motor function of the children treated compared to the placebo group.
“These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients,” said Michael Ehlers, executive vice president, head of Research and Development at Biogen (NASDAQ:BIIB).
CHERISH was a fifteen-month study investigating Spinraza in 126 children with later-onset SMA.
No patients discontinued the study and after the strength of these results CHERISH will be stopped and the patients switched to an open-label extension study to receive Spinraza.
“These data further validate the potential of Spinraza as a treatment for patients with SMA,” said Lynne Parshall, chief operating officer of Ionis (NASDAQ:IONS).
Shares in Ionis rocketed 21% to US$33, adding US$1bn to its market value, while the much larger Biogen jumped 6% to US$293.
Story by ProactiveInvestors