To present results from the SHINE open-label extension study (NCT02594124) for participants with later-onset SMA.

Several clinical trials have demonstrated a favorable benefit:risk profile for nusinersen and established clinically meaningful efficacy on motor function.

Participants from CS2/12, CHERISH and EMBRACE could enroll in SHINE. Following protocol amendment, all participants transitioned to the Modified Maintenance Dosing Regimen (MMDR; 12mg nusinersen every 4 months). Participants initiated the MMDR at the end of the loading dose period or 120 days after date of last visit. Endpoints were assessed from MMDR Day 1.

83 participants from the CHERISH nusinersen and 42 from the sham-procedure group transitioned to SHINE; 24 transitioned from CS2/12. Using the SHINE 15 October 2018 interim data cut, mean (±SD) Hammersmith Functional Motor Scale-Expanded (HFMSE) score at MMDR Day 1 (for participants with a MMDR Day 240 visit) was 26.0 (11.01) for those who received nusinersen in CHERISH/SHINE (n=61), and 21.2 (7.75) for those randomized to sham-procedure in CHERISH/nusinersen in SHINE (n=36). Mean (±SD) Revised Upper Limb Module (RULM) scores at MMDR Day 1 for these groups were 23.4 (5.54; n=61), and 21.2 (4.31; n=36), respectively. Mean (±SD) HFMSE at MMDR Day 1 (for participants with a MMDR Day 240 visit) was 33.2 (12.26) for those with SMA Type II who transitioned from CS2/12 (n=9) and 56.2 (6.85) for those with SMA Type III (n=13). Mean (±SD) RULM at MMDR Day 1 were 26.4 (4.81; Type II; n=8) and 36.7 (0.58; Type III; n=3), respectively. Data from the 2019 SHINE data cut for these cohorts and those who transitioned from EMBRACE, as well as an updated safety profile, will be presented.

Continued analysis of SHINE data will increase the information available on the long-term safety/tolerability and efficacy of repeated nusinersen doses in patients with later-onset SMA.

Study Supported by: Biogen