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ENDEAR primary endpoint: proportion of HINE Section 2 motor milestone responders

Patients with infantile-onset (Type 1) spinal muscular atrophy treated with SPINRAZA achieved a substantial motor milestone response on their HINE Section 2 score compared with untreated patients.1-3

INTERIM ANALYSIS*

Median time on treatment: 8.7 months

40% SPINRAZA (n=52)
vs P<0.0001
0% Untreated (n=30)
FINAL ANALYSIS

Median time on treatment: 9.3 months for SPINRAZA; 6.2 months for untreated

51% SPINRAZA (n=73)
vs P<0.0001
0% Untreated (n=37)

HINE=Hammersmith Infant Neurological Exam.

*A planned interim efficacy analysis was conducted based on 82 patients who completed at least 183 days of treatment, withdrew from the study, or died. For subjects who were alive and ongoing in the study, the efficacy data were calculated at the later of days 183, 302, or 394.1,2

The interim analysis in the Finkel et al publication indicates a motor milestone responder rate of 41%. This reflects 78 patients, excluding 4 patients who died and were not enrolled early enough to reach the day 183 cutoff.1-3

Milestone gains with SPINRAZA

Some patients with infantile-onset (Type 1) spinal muscular atrophy treated with SPINRAZA saw improvements in motor milestones.1-3

Net change from baseline in HINE Section 2 total motor milestone score2‡

In the chart above, only patients who were alive and ongoing at end of study are shown. Patients who withdrew or died are not included.2

For subjects who were alive and ongoing in the study, the efficacy data were calculated at the later of days 183, 302, or 394.1

Excludes voluntary grasp.3

Key Icon

97% of patients treated with SPINRAZA either improved or maintained their HINE Section 2 scores.2

Milestone gains over time with SPINRAZA

Patients with infantile-onset (Type 1) spinal muscular atrophy 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‡

The mean HINE Section 2 total motor milestone score at baseline was 1.29 for SPINRAZA compared with 1.54 for untreated patients.2

SEM=standard error of the mean.
Excludes voluntary grasp.

Key Icon

Patients treated with SPINRAZA demonstrated continued improvements over time, while untreated patients saw a decline in motor milestones.4

SPINRAZA may unlock new abilities

Some patients with infantile-onset (Type 1) spinal muscular atrophy treated with SPINRAZA achieved new motor milestones.2,4

Maximum score achieved in HINE Section 2 motor milestone categories2,4

§Independent sitting includes HINE Section 2 score categories: stable sit (5%) and pivots (rotates) (3%).4
Standing includes HINE Section 2 score categories: stands with support (1%) and stands unaided (0).4

Key Icon

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

image of Sofia aged 2.5 years old with Type 1 SMA taking a bath
Sofia
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA

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

INDICATION

SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

IMPORTANT SAFETY INFORMATION

Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.

In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.

Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).

Laboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.

Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.

Cases of rash were reported in patients treated with SPINRAZA.

SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.

The most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.

Please see full Prescribing Information for additional Important Safety Information.

As a courtesy, our full Prescribing Information is also available en Español. For prescribing decisions, please refer to official approved labeling.