Biogen recently presented new data on Spinraza for the treatment of spinal muscular atrophy (SMA) during the 70th American Academy of Neurology (AAN) Annual Meeting, currently taking place from April 21-27 in Los Angeles, California. Both platform and poster presentations highlighted the benefits Spinraza provides for individuals with spinal muscular atrophy (SMA) across the age and disease spectrum.
Several analyses illustrating Spinraza’s effectiveness were presented, including part one of the Phase 2 EMBRACE study as well as an interim analysis of the SHINE open-label extension study. Featured in AAN’s Emerging Science program, the SHINE analysis examined the longer-term safety and efficacy of SPINRAZA in infantile-onset SMA patients.
EMBRACE Part 1 is a phase 2, double-blind, sham-procedure controlled study assessing the safety/tolerability of intrathecal nusinersen in symptomatic infants/children with spinal muscular atrophy (SMA) who were not eligible to participate in the ENDEAR or CHERISH studies. EMBRACE Part 1 was terminated early based on positive results from the phase 3 ENDEAR study. The safety/tolerability profile of nusinersen in EMBRACE was similar to that observed in previous trials. A larger proportion of nusinersen-treated (11/14; 79%) vs. sham procedure–treated individuals (2/7; 29%) were HINE motor milestone responders. Between the Day 183 and 302 visits, hours of ventilator use changed by a mean (SD) of +1.236 (3.712) hours in nusinersen-treated individuals (12 patients) and by +2.123 (3.023) hours in sham procedure–treated individuals (7 patients).
The SHINE analysis reported interim results as of June 30, 2017, from the open-label extension study of 89 patients with infantile-onset SMA (most likely to develop Type 1) who transitioned from the Phase 3 ENDEAR study. Participants either initiated Spinraza treatment in ENDEAR and continued treatment through SHINE (65 patients) or transitioned from the sham-control arm in ENDEAR to active treatment with SPINRAZA in SHINE (24 patients).
Participants in the treatment group, who either continued on SPINRAZA from the Phase 3 ENDEAR study or who transitioned to SPINRAZA after receiving sham-control in ENDEAR, experienced improved motor function and improved event-free survival time. Those who initiated treatment earlier showed the greater motor performance. However, improvements in motor function were also seen in type 1 patients for whom treatment was initiated later after receiving sham-control in ENDEAR, albeit to a lesser extent. The median time to death or permanent ventilation in the group who received SPINRAZA in ENDEAR and continued in SHINE was 73.0 weeks, significantly longer than 22.6 weeks for those who received sham-control before SPINRAZA. The majority of patients who were alive and did not require permanent ventilation after receiving sham in ENDEAR remained event-free for a median of 9.2 months after initiating Spinraza therapy.
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