Biogen will present Phase 3 end of study SPINRAZA® (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy (SMA) compared to untreated children. The overall findings continue to support the robust efficacy and favorable safety profile of SPINRAZA across a broad range of individuals with SMA. SPINRAZA data will be presented at the American Academy of Neurology (AAN) annual meeting in Boston, Mass., April 22-28, 2017.
“The CHERISH study, conducted in collaboration with Ionis, further demonstrates the meaningful impact SPINRAZA can have in children with later-onset SMA, and reaffirms the benefit of treatment across SMA populations,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “Our clinical development program demonstrates the impact of early treatment, which is confirmed by NURTURE data showing significant motor milestone improvements generally consistent with normal development in presymptomatic infants treated with SPINRAZA.”
CHERISH: Later-onset SMA (Most Likely to Develop Type 2 or Type 3)
CHERISH is a Phase 3, multicenter, randomized, double-blind, sham-procedure controlled study to assess the efficacy and safety of SPINRAZA in children with later-onset SMA. The 15-month study investigated SPINRAZA in 126 non-ambulatory children 2 to 12 years old who experienced symptom onset at greater than 6 months of age.
In the CHERISH end of study analysis, children on SPINRAZA demonstrated a highly statistically significant and clinically meaningful improvement in motor function, as observed by the treatment difference of 4.9 points in the mean change from baseline to Month 15 in the Hammersmith Functional Motor Scale Expanded (HFMSE) score (p=0.0000001). When measuring changes from baseline, children who received SPINRAZA (n=84) achieved a 3.9 point mean improvement at Month 15, while children who were not on treatment (n=42) experienced a mean decline of 1.0 point. Primary endpoint results of the end of study analysis were consistent with results observed at the interim analysis.
Data from the other endpoints analyzed, including attainment of new motor milestones and upper limb motor function, were consistently in favor of children who received treatment.
SPINRAZA demonstrated a favorable safety profile. Treatment-emergent adverse events (AEs), severe AEs and serious AEs (SAEs) were reported less frequently in children treated with SPINRAZA than those not on treatment. No children discontinued the study due to AEs.
“In CHERISH, most children with later-onset SMA treated with SPINRAZA saw improvements in motor function and stabilization or slowing of disease progression,” said Dr. Richard Finkel, chief of neurology, Nemours Children’s Hospital, Orlando, Florida. “As a physician who has spent 37 years treating children with SMA, it’s incredibly encouraging to see some patients on SPINRAZA achieve milestones such as crawling and standing with assistance within the clinical trial. These kinds of clinically meaningful improvements are unprecedented and give new hope to individuals with SMA and their families.”
NURTURE: Presymptomatic Infants with SMA
Biogen will also present new interim data from the Phase 2, multicenter, open-label, single-arm NURTURE study evaluating SPINRAZA for the treatment of infants under six weeks old with genetically diagnosed SMA who were presymptomatic at treatment initiation. At the time of the interim analysis, infants (n=20) were enrolled for a median of 317.5 days, and all infants were alive and none required respiratory intervention (chronic non-invasive ventilation, invasive ventilation or tracheostomy). Further, most infants achieved motor milestone and growth parameter gains generally consistent with normal development, such as head control, independent sitting, standing and walking independently, as measured by validated scales.
Three infants experienced AEs considered possibly related to SPINRAZA by the investigator, all of which were resolved. No infants have discontinued or withdrawn from the study due to AEs, and no new safety concerns have been identified.
“The results from NURTURE are significant, as they continue to demonstrate the importance of beginning SPINRAZA treatment as soon as possible after an SMA diagnosis and the major impact that early treatment may have across a broad range of SMA populations,” said Sandrock.
For more information about SPINRAZA and U.S. prescribing information, visit www.SPINRAZA.com.
More About Spinraza
On December 23, the FDA announced that it had approved SPINRAZA™ (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.
From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into this therapeutic approach. We thank Drs. Ravindra Singh and Elliot Androphy of the University of Massachusetts Medical School for their work funded by Cure SMA in orginally indentifying the ISSN1 gene sequence, which is the sequence targeted by Spinraza. We acknowledge Drs. Adrian Krainer, Yimin Hua, and colleagues at Cold Spring Harbor Laboratory for generating the critical intellectual property. All this work was then licensed to Ionis Pharmaceuticals to created the antisense therapy, SPINRAZA.
The program was then licensed to Biogen. Together with Ionis, Biogen worked to develop and implement a comprehensive clinical testing program that would provide both the quickest route to approval and the high quality data necessary to support a broad label and access.