START Long-Term Follow-Up: ZOLGENSMA®(onasemnogene abeparvovec-xioi) demonstrates durability of effect over 4 years after treatment1

An ongoing, long-term follow-up study of ZOLGENSMA-treated patients from the START clinical trial (N=13)1,2

The long-term follow-up study of START began after the completion of the 24-month START trial and is planned to last 15 years. A total of 13 patients, 10 from the high-dose cohort and 3 from the low-dose cohort, are enrolled in the study.

A total of 10 out of 12 high-dose patients enrolled in an ongoing, observational, long-term follow-up of the START trial1

As of Dec 2019, the mean time since treatment was 4.5 years (range 4.1-5.2) and the mean age at last follow-up was 4.8 years (range 4.3-5.6).1,a

All 10 of the patients from the high-dose cohort who enrolled in the long-term follow-up were alive and free of permanent ventilation as of the Dec 2019 data cut1,b

a4 patients were on another disease-modifying agent.

bEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days.

cA 15-year, voluntary follow-up of patients in the START trial who received a high dose of ZOLGENSMA (n=10).1

dNatural history: The Pediatric Neuromuscular Clinical Research (PNCR) Network study population (n=23), 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

a4 patients were on another disease-modifying agent.

bEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days.

cA 15-year, voluntary follow-up of patients in the START trial who received a high dose of ZOLGENSMA (n=10).1

dNatural history: The Pediatric Neuromuscular Clinical Research (PNCR) Network study population (n=23), 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

ZOLGENSMA continues to provide durable efficacy over 4 years after treatment

There has been no loss of milestones achieved during START among 10 patients from the high-dose cohort who enrolled in the long-term follow-up (LTFU)1

aBayley-III, gross motor subtest item 20.1

bBayley-III, gross motor subtest item 22.1

cBayley-III, gross motor subtest item 26.1

dAlso includes patients who are observed sitting alone for ≥5, ≥10, or ≥30 seconds. Patients sitting ≥30 seconds are included in the total of ≥5 seconds.1

aBayley-III, gross motor subtest item 20.1

bBayley-III, gross motor subtest item 22.1

cBayley-III, gross motor subtest item 26.1

dAlso includes patients who are observed sitting alone for ≥5, ≥10, or ≥30 seconds. Patients sitting ≥30 seconds are included in the total of ≥5 seconds.1

START Trial

An open-label, single-arm, Phase 1, dose-escalation clinical trial supported the efficacy of ZOLGENSMA

SPR1NT Trial

A Phase 3 study of presymptomatic patients with 2 and 3 copies of SMN2