New findings from studies of Spinraza for both infantile- and later-onset spinal muscular atrophy.- Biogen.

Biogen announced new findings detailing the benefits that Spinraza (nusinersen) demonstrates for both infantile- and later-onset spinal muscular atrophy (SMA) populations, including improvement in motor function as well as increased survival for the most severely affected. These findings are based on interim results from the SHINE open-label extension study and an analysis of Spinraza�s effects on mobility and fatigability in later-onset participants from the CS2/CS12 studies. The research will be presented at the American Academy of Neurology (AAN) Annual Meeting on April 21-27, 2018, in Los Angeles, California.

The SHINE analysis reported interim results as of June 30, 2017, from the open-label extension study for patients (n=89) with infantile-onset SMA (most likely to develop Type 1) who transitioned from the Phase III ENDEAR study. Participants either initiated Spinraza treatment in ENDEAR and continued treatment through SHINE (n=65) or transitioned from the sham-control arm in ENDEAR to active treatment with Spinraza in SHINE (n=24). The interim results showed that participants who initiated Spinraza in ENDEAR and continued in SHINE, as well as those who received sham in ENDEAR and initiated Spinraza in SHINE, experienced improvements in HINE-2 motor milestones and general motor function as measured by CHOP INTEND. The median time to death or permanent ventilation for participants who initiated Spinraza in ENDEAR and continued in SHINE was 73 weeks. Among participants who received sham, the median time to death or permanent ventilation was 22.6 weeks within ENDEAR. The majority of subjects who were alive and did not require permanent ventilation after they received sham in ENDEAR remained event-free after receiving Spinraza in SHINE for a median time of 9.2 months.

An additional analysis � which was led by researchers at Columbia University Medical Center with support from Biogen � evaluated a subset of data from CS2 and CS12, two multicenter, open-label clinical trials, to assess the change in participants� performance during the Six-Minute Walk Test (6MWT) and measures of fatigue. The analysis examined the walking ability and fatigability of ambulatory participants (n=14) ages two to 15 years with SMA Type 2 (n=1) or Type 3 (n=13) at study enrollment. Participants� baseline median distance walked was 250.5 meters and baseline median fatigue level was 14.8 percent. Following Spinraza treatment, their walking distance increased (a median increase of 98 meters) while simultaneously, their fatigue level remained stable or decreased (a median decrease of 3.8 percent) over nearly 3 years.

"With Spinraza treatment, not only were participants able to walk longer distances but they experienced a stabilization or decrease in fatigue while doing so � both of which are meaningful, real-world benefits for individuals with SMA,� said Jacqueline Montes, P.T., Ed.D., N.C.S., Assistant Professor, lead study author, Columbia University Irving Medical Center, New York. �Furthermore, the analysis illustrates that Spinraza�s benefits continue to grow over time for Type 2 and 3 SMA populations.�.

Comment; Spinraza was approved in the US on 23 December 2016 and in the EU on 1 June 2017 for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.