STR1VE: ZOLGENSMA significantly increased event-free survival in patients with SMA Type 1 compared with natural history1

91% (20/22) of patients were alive, free of permanent ventilation, and continuing in the study2,c

aEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hour of respiratory assistance per day continuously for ≥14 days.2
bNatural history: The Pediatric Neuromuscular Clinical Research (PNCR) Network study population, with bi-allelic deletion of SMN1 gene, 2 copies of SMN2, and onset of SMA symptoms at age ≤6 months, was used as a matched control cohort for START and STR1VE studies.3
cOne patient died at 7.8 months from respiratory failure, which was deemed unrelated to treatment. One patient withdrew consent at 11.9 months of age; this patient was determined by the investigator to have required permanent ventilation at the time of discontinuation.2
dResults from the March 2019 data cut of STR1VE (N=22) an ongoing, open-label, single-arm, clinical trial of symptomatic patients with SMA. One patient was initially classified as presymptomatic and removed from the intention to treat (ITT) data set included in the prescribing information. The patient was later confirmed to be symptomatic at baseline and will be included in the final ITT analysis.2
eAt most recent visit prior to March 2019 data cut.2

STR1VE: Motor milestones were achieved
and maintained with ZOLGENSMA1,2

Patients continue to attain new milestones and have maintained existing milestones at successive data cuts. In natural history, motor milestones are not achieved and maintained.2

aResults from the March 2019 data cut of STR1VE (N=22), an ongoing, open-label, single-arm clinical trial of symptomatic patients with SMA Type 1. One patient was initially classified as presymptomatic and removed from the ITT data set included in the prescribing information. The patient was later confirmed to be symptomatic at baseline and will be included in the final ITT analysis.2
bTwo patients reached the milestone of head control at the first screening visit (prior to ZOLGENSMA dosing), therefore n=20 was used for this calculation.2
cAssessment is based on the Bayley Scales: Gross and Fine Motor Skills Subtexts, item 26, “Sits Without Support Series: 30 seconds.”

50% (11/22) of patients

were sitting ≥30 seconds, a coprimary endpoint, at a mean of
8.2 months post treatment (range 4.9–12.7)2,a-c

aResults from the March 2019 data cut of STR1VE (N=22), an ongoing, open-label, single-arm clinical trial of symptomatic patients with SMA Type 1. One patient was classified as presymptomatic and removed from the ITT data set including the prescribing information. The patient was later confirmed to be symptomatic at baseline and will be included in the final ITT analysis.2
bAssessment is based on the Bayley Scales: Gross and Fine Motor Skills Subtexts, item 26, “Sits Without Support Series: 30 seconds.”2
cPatients were at a mean age of 11.9 months (range 9.2--16.9).2

STR1VE: ZOLGENSMA resulted in rapid and sustained motor function improvements2

CHOP INTEND scores as of March 2019 data cut2,a,b

Improvements in CHOP INTEND from baseline were seen as early as 1 month post infusion (March 2019 data cut)2

At baseline, the average CHOP INTEND score was 32 points (range 18-52).

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
aResults from STR1VE (N=22), an ongoing, open-label, single-arm clinical trial of symptomatic patients with SMA Type 1. One patient was initially classified as presymptomatic and removed from the ITT data set included in the prescribing information. The patient was later confirmed to be symptomatic at baseline and will be included in the final ITT analysis.2
bScores on the CHOP INTEND scale of motor function range from 0 to 64, with higher scores indicating better function.6

Understand the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)

CHOP INTEND was specifically developed to assess motor function in patients with SMA. The CHOP INTEND score is measured in points, which are assigned based on a patient’s ability to perform specific motor skills. The test consists of 16 measures, each scored on a scale 0-4, with the highest possible score being 64 points.6

95% (21/22)

of patients achieved a clinically meaningful CHOP INTEND score
of ≥402,a,b

vs

In natural history, children with SMA Type 1 who were 6 months of age or older do not achieve and maintain CHOP INTEND scores of 40 points or higher4

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
aResults from the March 2019 data cut of STR1VE (N=22), an ongoing, open-label, single-arm clinical trial of symptomatic patients with SMA Type 1. One patient was initially classified as presymptomatic and removed from the ITT data set included in the prescribing information. The patient was later confirmed to be symptomatic at baseline and will be included in the final ITT analysis.2
bScores on the CHOP INTEND scale of motor function range from 0 to 64, with higher scores indicating better function.6