Success for Phase III CHERISH study of Spinraza (nusinersen) for treatment of spinal muscular atrophy.- Biogen + Ionis Pharma

Biogen and Ionis Pharmaceuticals announced that Spinraza (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase III 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. Spinraza demonstrated a favorable safety profile in the study.

CHERISH is a fifteen-month study investigating Spinraza in 126 non-ambulatory patients with later-onset SMA (consistent with Type 2), including patients with the onset of signs and symptoms at greater than 6 months and an age of 2 to 12 years at screening. Results from the primary endpoint of the pre-specified interim analysis demonstrated a difference of 5.9 points (p= 0.0000002) at 15 months between the treatment (n=84) and sham-controlled (n=42) study arms, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE). From baseline to 15 months of treatment, patients who received Spinraza achieved a mean improvement of 4.0 points in the HFMSE, while patients who were not on treatment declined by a mean of 1.9 points. The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA, and a change of three points or greater in the HFMSE has previously been identified as clinically meaningful. Spinraza demonstrated a favorable safety profile.

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. With the positive interim analysis, the CHERISH study will be stopped and participants will be able to transition into the SHINE open-label extension study to receive Spinraza. Full study results will be presented at future medical congresses.

Comment:The FDA recently accepted the company’s New Drug Application (NDA) for Spinraza as a treatment for SMA and communicated they plan to act early on the NDA under an expedited review. Additionally, the European Medicines Agency (EMA) recently validated Biogen’s Marketing Authorization Application (MAA) in the EU. Biogen is preparing for the potential launch of Spinraza in the U.S. possibly as early as the end of 2016 or the first quarter of 2017.