Shares of Biogen $BIIB and Ionis Pharmaceuticals $IONS both jumped early Monday morning after the two biotech partners announced another preliminary win in a late-stage study of nusinersen for spinal muscular atrophy.
The latest batch of good news — badly needed by both of these companies — centers on a positive readout for nusinersen (Spinraza) for late-onset SMA. The first round of promising results dropped three months ago for early-onset cases and Biogen says the FDA has already signaled that it plans to act early on its application to start selling the drug.
As a result of the preliminary Phase III success, investigators are stopping the trial early and transferring patients over to the extended study. Biogen shares surged 6% and Ionis stock jumped 18% in pre-market trading.
Using the Hammersmith Functional Motor Scale Expanded report card for patients’ motor functions, the drug arm witnessed a mean 4.0 improvement on scores while the placebo arm dropped by 1.9 points, a 5.9 point overall spread in the drug’s favor.
That amounts to a “highly statistically significant improvement,” investigators reported, which further backs up their expectations for winning a near-term approval to sell the drug. Biogen says it expects to get a green light from the FDA to start marketing the drug either later this quarter or in early 2017.
That’s welcome news for Biogen investors, who have been dealing with a variety of setbacks over the course of the year as its Tecfidera franchise begins to wane. Back in May, Ionis was sent reeling after it revealed that evidence of thrombocytopenia—severe platelet declines — had derailed one of its late-stage efforts. That makes this success particularly critical. And coming early with the second round of Phase III data didn’t hurt at all.
“The timing and result materially increases the assurance that the initial label for nusinersen will include all, or at least most forms of SMA and thus gives the drug even more near term revenue potential than we have modeled previously,” noted Leerink’s Geoffrey Porges.
Said Michael Ehlers, MD, PhD, executive vice president, head of research and sevelopment at Biogen:
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.
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