Biogen, Ionis' experimental SMA drug Spinraza meets main goal of Phase III study

Biogen and Ionis Pharmaceuticals on Monday said that the experimental drug Spinraza (nusinersen) met the primary endpoint of a Phase III study in patients with later-onset spinal muscular atrophy (SMA). Results from an interim analysis of the CHERISH study showed that the therapy was associated with significant improvements in motor function versus patients who received no treatment. Shares in Ionis climbed as much as 25 percent on the news.

Last month, Biogen announced that a marketing application for Spinraza was accepted for priority review by the FDA, while a filing has been validated by the European Medicines Agency. The company noted Monday that it is preparing for the potential launch of Spinraza in the US possibly as early as the end of 2016 or the first quarter of 2017.

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In the CHERISH study, 126 non-ambulatory patients with later-onset SMA were assigned to treatment with Spinraza or no treatment for 15 months. The primary endpoint of the trial was improvement in motor function as examined using the Hammersmith Functional Motor Scale Expanded (HFMSE).

Results from the pre-specified interim analysis identified a difference of 5.9 points on the HFMSE between the groups at 15 months. Specifically, an improvement of 4.0 points versus baseline was recorded on the HFMSE at 15 months for patients who received Spinraza, while the score in the no treatment arm worsened by 1.9 points. Biogen and Ionis added that data from the other endpoints analysed were consistently in favour of children who received treatment, while Spinraza also demonstrated a "favourable" safety profile.

"These results, along with our successful trial in infantile-onset SMA, reinforce the potential of Spinraza to benefit a broad range of SMA patients," noted Biogen R&D chief Michael Ehlers, adding "we will make regulators around the globe aware of this data and will continue working closely with them to bring Spinraza to families affected by SMA as quickly as possible." For further analysis, see ViewPoints: A lot to cherish as Biogen spins into action with second set of positive SMA data.

In August, Biogen and Ionis announced that Spinraza met the primary endpoint of the ENDEAR Phase III study, with the infants receiving the drug exhibiting a significant improvement in the achievement of motor milestones compared to those who did not receive the therapy. For related analysis, read ViewPoints: Biogen, Ionis keep building case for nusinersen but competitive footsteps getting louder.

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