Biogen has revealed new data for its spinal muscular atrophy (SMA) treatment Spinraza, showing long-term benefit and new insights into the development of a higher dose of the treatment.
This includes new analysis of data from Biogen’s NURTURE study, which showed 92% of patients who started Spinraza (nusinersen) treatment as pre-symptomatic infants maintained the ability to swallow after a median of 3.8 years.
Typically, impaired swallowing is expected for SMA patients with two or three SMN2 copies, leading to an increased risk of aspiration pneumonia, choking and failure to thrive.
In addition, all participants in this study with three SMN2 and 73% of participants with two SMN2 copies were reported by their caregivers as being fed exclusively by mouth.
Further post-hoc data from the open-label CS2-CS12 and SHINE extension studies indicate children and teens with later-onset SMA also showed improvement in walking distance and stabilisation in fatigue over five years of Spinraza treatment.
In addition to the long-term efficacy data, Biogen also revealed new analysis of data from the phase 2 CS3A and phase 3 ENDEAR studies in children with infantile-onset SMA.
Using pharmacokinetic (PK)/pharmacodynamic (PD) modelling, the analysis suggests a higher dose of Spinraza may lead to clinically meaningful increase in the CHOP INTEND score ‘beyond that’ of the 12mg approved dose.
The CHOP INTEND test provides a method of examining the movement ability of infants with severe muscle weakness who can’t tolerate motor developmental or functional assessments.
Previously, Biogen reported safety data from part A of the DEVOTE study, which is evaluating a higher dose of Spinraza compared to the currently approved dose.
“Intervention with Spinraza can meaningfully impact the trajectory of SMA, and we remain relentless in our aim of improving outcomes for people with SMA,” said Alfred Sandrock, Jr, head of research and development at Biogen.
“We continue to better understand and explore Spinraza’s potential with our new and ongoing global clinical studies,” he added.