Biogen (BIIB) today announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA® (nusinersen) among pre-symptomatic infants with spinal muscular atrophy (SMA). In NURTURE, all infants treated with SPINRAZA were alive, did not require permanent ventilation and showed improvement in motor function and motor milestone achievements as of July 5, 2017, compared to the disease’s natural history. This study, along with a case series demonstrating SPINRAZA’s effectiveness among teens and young adults, will be presented at the Muscular Dystrophy Association (MDA) Clinical Conference on March 11-14, 2018, in Arlington, Virginia.

“These results reinforce SPINRAZA’s effectiveness as the first and only approved treatment for SMA and demonstrate once again the benefit it can bring to individuals with SMA, including infants, teens and young adults,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “SPINRAZA is supported by the largest well-controlled SMA clinical development program conducted to date and we are committed to ongoing research in SMA. Biogen looks forward to working with healthcare providers and institutions in order to provide teens and young adults with SMA access to SPINRAZA, a treatment that has demonstrated significant benefit in the maintenance of motor function for these patients.”

In the NURTURE study, SPINRAZA was administered to infants six weeks old or younger (n=25), who were in the pre-symptomatic stage, genetically-diagnosed with SMA and had two or three copies of the SMN2 gene (n=15 for two copies (most likely to develop Type 1 SMA); n=10 for three copies (most likely to develop Type 2 SMA)). At the time of this interim analysis, infants had been followed for up to 25.6 months – well beyond the typical timeframe when most infants with Type 1 SMA would have required permanent ventilation or died. The interim analysis, titled, “Nusinersen in Infants Who Initiate Treatment in a Pre-Symptomatic State of SMA: Interim Efficacy and Safety Results from the Phase 2 NURTURE Study,” showed that all infants were alive and none required tracheostomy or permanent ventilation.

“The NURTURE findings document the continuing benefits that SPINRAZA provides for patients with SMA who initiated treatment in early infancy while clinically pre-symptomatic, including age-appropriate developmental gains in motor function and motor milestone achievements,” said Dr. Darryl C. De Vivo, M.D., lead study author, Columbia University Medical Center, New York. “The treated infants in the NURTURE study had genetic SMA and were likely to clinically develop Type 1 or 2, yet with enough observation time they have all achieved independent sitting and the majority have developed the ability to walk.”

NURTURE participants also achieved a mean CHOP INTEND score, which measures general motor function among infants with SMA, of 58.4 at last visit (out of a maximum score of 64). Many continue to improve and maintain these scores beyond a point in time at which untreated individuals with Type 1 SMA would experience a significant decline. Overall, the study showed that SPINRAZA was well-tolerated and no new safety concerns were identified.

Also presented at the MDA Clinical Conference was a case series, “Nusinersen Experience in Teenagers and Young Adults With Spinal Muscular Atrophy,” which showcased SPINRAZA’s stabilizing or improving effect on teens and young adults with Type 2 or 3 SMA.

In the case series, participants (n=5) were 14 to 15 years old at the start of Study CS2, and 17 to 19 years old at the time of their last visit in the extension Study CS12. One participant was Type 2 and four were Type 3, and all received multiple doses of SPINRAZA over 2.5 years of observation. The results included improvement on the Hammersmith Functional Motor Scale–Expanded (HFMSE); stabilization on the Upper Limb Module (ULM); improvement in the Six-Minute Walk Test (6MWT); and stable or improved scores on the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND).

“The case series demonstrated SPINRAZA’s effectiveness in teens and young adults with SMA Type 2 or 3,” said Dr. John Day, M.D., Ph.D., lead study author, Stanford University Medical Center, California. “The study participants demonstrated stable or slightly improved motor function and quality of life during two years of treatment – and even afterward, we have continued to see improved and maintained stability and motor benefits with SPINRAZA. This differs importantly from untreated teens and young adults with SMA, who experience a decline in motor function, specifically in reduced walking distance and upper limb activities, as well as in health-related quality of life.”