ENDEAR primary endpoint: motor milestone response

ENDEAR

ENDEAR primary endpoint result: a higher proportion of patients with infantile-onset (Type 1) SMA treated with SPINRAZA® (nusinersen) met the HINE Section 2 response criteria compared with untreated patients.1,2

: Image shows 51% of patients treated with SPINRAZA achieved a substantial motor milestone response of their HINE Section 2
: Image shows 51% of patients treated with SPINRAZA achieved a substantial motor milestone response of their HINE Section 2

Final analysis was assessed
on day 394 of the trial.1

97% of patients treated with SPINRAZA either improved or maintained their HINE Section 2 score (N=78).2

Patients that continued with treatment and were present at the end of the study were included in this calculation.

Achievement of new motor milestones

Some patients with infantile-onset (Type 1) SMA treated with SPINRAZA achieved new improvements in motor milestones.1,2

Number of patients with an improvement in motor milestones from baseline3
Chart showing patients treated with SPINRAZA increased motor milestone score from baseline compared with untreated patients
Chart showing patients treated with SPINRAZA increased motor milestone score from baseline compared with untreated patients

*Ability to kick includes achievement of touching toes (n=5).3

Continued milestone gains over time

Patients with infantile-onset (Type 1) SMA treated with SPINRAZA showed continued improvement in mean total motor milestone score.4

Mean change in HINE Section 2 total motor milestone score over time4†
Chart showing patients treated with SPINRAZA increased in mean total motor milestone score compared with untreated patients
Chart showing patients treated with SPINRAZA increased in mean total motor milestone score compared with untreated patients

Baseline:
SPINRAZA: 1.29
Untreated: 1.54

HINE=Hammersmith Infant Neurological
Examination; SEM=standard error of the mean;
SMA=spinal muscular atrophy.
Excludes voluntary grasp.4

Milestones such as head control and rolling are rarely, if ever, achieved in untreated patients in the natural history of Type 1 SMA.5

Sofia
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA



 

Sofia
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA

Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.