End results for CHERISH study of Spinraza for spinal muscular atrophy are published in NEJM.- Biogen + Ionis Pharma.

Biogen and Ionis Pharmaceuticals, Inc. announced end of study results from CHERISH, the Phase III study evaluating Spinraza (nusinersen) for the treatment of individuals with later-onset spinal muscular atrophy (SMA), were published in The New England Journal of Medicine. The full manuscript titled, �Nusinersen Versus Sham Control in Later-Onset Spinal Muscular Atrophy,� appears in the February 15 issue of The New England Journal of Medicine.

The pre-specified CHERISH primary endpoint was improvement in motor function, as defined by change from baseline in the Hammersmith Functional Motor Scale-Expanded (HFMSE). The HFMSE is a validated tool specifically designed to assess motor function in individuals with SMA. The final analysis demonstrated a highly statistically significant and clinically meaningful improvement in motor function in individuals treated with Spinraza versus the sham control, as observed by the treatment difference of 4.9 points in the mean change from baseline to Month 15 in the HFMSE score (p=0.0000001). When measuring changes from baseline, individuals who received Spinraza (n=84) achieved a 3.9 point mean improvement at Month 15, while individuals who were not on treatment (n=42) experienced a mean decline of 1.0 point. Primary endpoint results of the end of study analysis were consistent with the interim analysis. Data from the other endpoints analyzed, including attainment of new motor milestones and upper limb motor function, were consistently in favor of individuals who received treatment and were considered clinically significant. Upper limb function, as measured by the Revised Upper Limb Module (RULM), improved in individuals treated with Spinraza (4.2 points) from baseline to Month 15 compared to untreated individuals (0.5 points). The RULM is an important measure of motor function in non-ambulatory individuals.

Spinraza demonstrated a favorable benefit-risk profile. Safety data were consistent with those expected in the general SMA later-onset population and in individuals undergoing lumbar puncture and were similar to those reported in an open-label study in later-onset SMA. Following the positive interim analysis, Biogen ended the CHERISH study early so all participants could have the option to receive Spinraza in the SHINE open-label extension study. In addition to SHINE, Biogen continues to collect and evaluate data to provide a deeper understanding of the efficacy and safety of Spinraza across SMA populations. The Spinraza clinical development program includes more than five years of data and is the largest body of evidence for an interventional approach in SMA. End of study results from ENDEAR, the Phase III Spinraza study for the treatment of infantile-onset SMA, were published in the November 2, 2017 issue of The New England Journal of Medicine.

See-"Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy"-Eugenio Mercuri, M.D., Ph.D., Basil T. Darras, M.D., Claudia A. Chiriboga, M.D., M.P.H., John W. Day, M.D., Ph.D., Craig Campbell, M.D., et al., for the CHERISH Study Group-N Engl J Med 2018; 378:625-635 DOI: 10.1056/NEJMoa1710504.