START Long-Term Follow-Up: ZOLGENSMA®(onasemnogene abeparvovec-xioi) demonstrates durability of effect over 5 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 June 2020, the mean time since treatment was 5.0 years (range 4.6-5.6) and the mean age at last follow-up was 5.2 years (range 4.7-6.1).1,a,b

aBased on adverse event reporting death, withdrawal, or permanent ventilation. There were no in-person assessments between December 2019 and June 2020.1

bA 15-year, voluntary follow-up of patients in the START trial who received a high dose of ZOLGENSMA (n=10). In long-term follow-up, 4 patients had used another disease-modifying therapy.1

As of the June 2020 data cut1:

Event-free survival

100% (10/10) of patients

were alive and free of permanent ventilation1,a,b

ZOLGENSMA continues to provide durable efficacy over 5 years after treatment1

The long-term follow-up study is an ongoing follow-up of the Phase 1 START trial.

All 10 patients from the START high-dose cohort who enrolled in the long-term follow-up were alive and free of permanent ventilation as of June 20201,b

aAs of the last visit prior to December 2019 data cut.

bBased on adverse event reporting death, withdrawal, or permanent ventilation. There were no in-person assessments between December 2019 and June 2020.

aAs of the last visit prior to December 2019 data cut.

bBased on adverse event reporting death, withdrawal, or permanent ventilation. There were no in-person assessments between December 2019 and June 2020.

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