Positive interim results have been reported for the phase III trial of SpinrazaTM (nusinersen) in children with Type 2 SMA.
Following the announcement of the encouraging preliminary results from the ENDEAR (IONIS-CS3B) trial for infants less than 7 months of age with proximal SMA linked to SMN1, Biogen and IONIS Pharmaceuticals have announced positive interim results from the CHERISH trial.
The CHERISH trial
CHERISH is a 15-month study that evaluated the safety and clinical efficacy of SpinrazaTM (nusinersen) administration into the cerebrospinal fluid of 126 non-ambulatory children with later-onset SMA (consistent with Type 2) aged between 2 and 12 years old. In this trial, 84 participants were treated with SpinrazaTM and 42 with placebo.
Significant improvement in motor function
Preliminary results indicate a “highly significant” improvement in motor function (as measured by the Hammersmith Functional Motor Scale Expanded, an evaluation scale adapted to children with SMA) in patients treated with SpinrazaTM (5.9 points) compared to placebo-treated patients.
This improvement is such that, as with the ENDEAR trial, the double-blind CHERISH trial will be discontinued so that all participants (including those initially taking placebo) can receive SpinrazaTM as part of the open-label extension phase of this study (SHINE trial).
Early access to SpinrazaTM
To date, early access (EAP) to SpinrazaTM is being implemented for people with Type I SMA (following the results of the ENDEAR trial). In France, it is done via a nominative temporary use authorisation (TAU).
Biogen and IONIS Pharmaceuticals have indicated that they were exploring the possibility of including people with Type 2 SMA in this EAP. In France, it will also be done via an TAU.
> See the Biogen press release
> See the Biogen press release sent to associations of people with SMA
