Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of individuals with spinal muscular atrophy (SMA) over the long term.
“Intervention with SPINRAZA can meaningfully impact the trajectory of SMA, and we remain relentless in our aim of improving outcomes for people with SMA. We continue to better understand and explore SPINRAZA’s potential with our new and ongoing global clinical studies,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. “The data we are presenting at Cure SMA 2021 demonstrate the long-term benefits with SPINRAZA as individuals age. Additionally, a new analysis provides further support for the potential for a higher dose of SPINRAZA to offer even greater improvements in motor function for SMA patients.”
New Findings Support Continued Development of an Investigational Higher Dose of SPINRAZA
An analysis of data from the Phase 2 CS3A and Phase 3 ENDEAR studies in children with infantile-onset SMA used pharmacokinetic (PK)/pharmacodynamic (PD) modelling to predict the potential efficacy of an investigational higher dose regimen of SPINRAZA. This analysis suggests that a higher dose of SPINRAZA may lead to a clinically meaningful increase in the CHOP INTEND (Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders) score beyond that already observed with the 12 mg approved dose. These findings further reinforce the scientific rationale for the evaluation of a higher dose of SPINRAZA in the ongoing DEVOTE study.
DEVOTE is a Phase 2/3 study evaluating the safety, tolerability and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved. It is a three-part study that includes an open-label safety evaluation cohort (Part A), a pivotal, double-blind, randomized, active-controlled treatment cohort (Part B) and an open-label cohort of patients transitioning from the approved 12 mg dosing regimen of SPINRAZA to the higher dose regimen (Part C).
As previously reported,1 safety data from Part A (n=6; 28 mg) support continued development of a higher dose of SPINRAZA and an updated analysis presented at the meeting include data collected up to 10 months. Enrollment in the pivotal Part B of DEVOTE is ongoing and will evaluate a higher-dose regimen (two loading doses of 50 mg 14 days apart followed by 28 mg maintenance doses every four months) compared to the currently approved dosing regimen for SPINRAZA.2 More information about DEVOTE is available at ClinicalTrials.gov (NCT04089566).
Data Reinforce Long-term Efficacy of SPINRAZA in a Broad Range of People with SMA
An analysis of data from the NURTURE study (n=25) shows 92 percent of patients who initiated SPINRAZA treatment as pre-symptomatic infants maintained the ability to swallow after a median of 3.8 years. This is in contrast with the natural history of SMA where impaired swallowing is expected for people with 2 or 3 SMN2 copies3 and can lead to an increased risk of aspiration pneumonia, choking and failure to thrive. In this analysis, NURTURE study participants were consistently rated by their caregiver as, on average, never to rarely experiencing difficulty for the majority of measures related to general feeding, drinking liquids and eating solid foods. Additionally, all participants with 3 SMN2 copies and 73 percent (11 of 15) of participants with 2 SMN2 copies were reported by their caregiver as being fed exclusively by mouth.
In addition, post-hoc data from the open-label CS2-CS12 and SHINE extension studies indicate children and teens with later-onset SMA (n=14) showed improvement in walking distance over five years of SPINRAZA treatment and stabilization in fatigue.
In all analyses presented at the meeting, the safety profile of SPINRAZA was consistent with previously reported findings.
Featured SPINRAZA Data Presentations Include:
- Scientific Rationale for a Higher Dose of Nusinersen
- Part A Results From the Ongoing DEVOTE Study to Explore a Higher Dose of Nusinersen in SMA
- Preserved Swallowing Function in Infants Who Initiated Nusinersen Treatment in the Presymptomatic Stage of SMA: Results From the NURTURE Study
- Nusinersen in Later-onset Spinal Muscular Atrophy: Walking Distance and Fatigue in CS2/12 and SHINE Participants
These data are being presented at the Virtual SMA Researcher & Clinical Care Meeting taking place June 9-11, 2021.