Shares of Ionis Pharmaceuticals (IONS 2.44%) are down 13% at 12:45 p.m. EDT without any direct news from the biotech.
However, there were a couple of indirect news items from competitors and collaborators that seem to have investors taking profits on Ionis' shares that were up 87% over the last year through yesterday.
First, Novartis (NVS 0.46%) announced interim phase 3 data for its gene therapy Zolgensma, which treats spinal muscular atrophy (SMA), the same disease treated by Ionis' and Biogen's (BIIB 1.05%) Spinraza. Biogen is only down 1%, but Spinraza sales contribute a smaller portion of Biogen's overall revenue than Spinraza royalties do for Ionis.
Of the seven treated infants with type 1 SMA that had reached 10.5 months or older, six of them were alive and not requiring permanent ventilation. There wasn't a control arm of untreated patients, but typically, 50% of babies do not survive or will require permanent ventilation by the time they reach 10.5 months of age. Novartis also released data on the infants' development milestones -- turning over or sitting unassisted, for example -- which appeared fairly similar to the positive phase 1 data.
The phase 3 study isn't scheduled to conclude until next year, but Novartis has already submitted marketing applications to regulators. The FDA is expected to make a decision next month, while decisions in the EU and Japan are expected later in the year. On the plus side for Ionis and Biogen, Zolgensma is likely to only be approved for type 1 SMA patients, while Spinraza is also approved for the less severe type 2 and type 3 SMA, so the immediate impact will be muted.
Roche also added fuel to the fire with positive comments on its SMA drug risdiplam, which can be used for all three types of SMA, on its first-quarter conference call. Management also noted that it wouldn't be presenting updated data for RG6042 at the American Academy of Neurology meeting next month; Roche licensed RG6042, which treats Huntington's disease, from Ionis, which will get royalties on sales if the drug is approved.
None of the news today seems like a major issue for Ionis. We've known about the competition for Spinraza, and no update on RG6042 doesn't mean Roche isn't moving the drug through the clinic as quickly as possible.
Nevertheless, it's understandable that any slightly negative news might cause investors to take some profits considering the sustained run Ionis' shares have been on over the last year. Long-term investors looking to pick up additional shares should consider the buying opportunity.