“These results reinforce SPINRAZA’s unprecedented and compelling efficacy across a broad range of SMA populations, enabling patients to improve mobility and motor function – and, for the most severely affected, increase their chances of survival,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “We look forward to continuing to work with healthcare providers, institutions and SMA communities to provide access to SPINRAZA for those in need, no matter their age, disease severity or duration of the disease.”
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 3 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).
“This analysis demonstrates that participants improved their motor function and increased event-free survival time, whether they initiated treatment earlier, as in ENDEAR and continuing in SHINE, or later, after receiving sham-control in ENDEAR and beginning treatment in SHINE,” said Diana Castro, M.D., lead study author, UT Southwestern Medical Center, Dallas, Texas. “It also confirms that those who initiated SPINRAZA treatment earlier saw greater motor milestone performance that continued to improve over time, and that no new safety concerns were identified.”
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.”