A couple of biopharma companies were on the move Monday morning after they announced positive interim results from a late-stage trial. Ionis Pharmaceuticals Inc. (NASDAQ: IONS) announced, in conjunction with Biogen Inc. (NASDAQ: BIIB), that their treatment for spinal muscular atrophy (SMA) met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating Spinraza in later-onset (consistent with Type 2) SMA.
The analysis found that children receiving Spinraza experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. At the same time, the drug also demonstrated a favorable safety profile in the study.
The majority of the adverse events were considered to be either related to SMA disease, common events in the general population or events related to the lumbar puncture procedure. No patients discontinued the study.
Also the U.S. Food and Drug Administration (FDA) recently accepted the company’s New Drug Application (NDA) for Spinraza as a treatment for SMA and communicated that it plans to act early on the NDA under an expedited review.
Biogen is preparing for the potential launch of Spinraza in the United States, possibly as early as the end of 2016 or the first quarter of 2017.
Michael Ehlers, M.D., Ph.D., executive vice president, head of Research and Development at Biogen, commented:
These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients. 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.
Shares of Ionis closed Friday at $27.13, with a consensus analyst price target of $41.30 and a 52-week trading range of $19.59 to $62.68. Following the announcement, the stock was up 10% at $30.00 in early trading indications Monday.
Biogen closed at $277.00, with a consensus price target of $341.17 and a 52-week range of $223.02 to $333.65. In early trading indications, the stock was up over 3% at $286.00.