Q2 2021 Results - Reimagining Medicine
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
References
3 INNOVATION
ZolgensmaⓇ SPR1NT data (2-copy cohort) demonstrate transformative,
age-appropriate development when used presymptomatically
n=14 (2 copy cohort)
Achieved CHOP INTEND¹ score ≥58 points 100%
100%
CHOP INTEND scores
IN WA Ug
10
30
20
50
40
60
0
0
100%
Untreated natural history²
79%
15
20
20
5
10
Age (months)
Mean age at dosing: 20.6 days (8-34 days);
No serious, treatment-related adverse events
Presented at EAN 2021
Met primary endpoint:
Sitting independently for ≥30 seconds³
Nearly all patients (11/14) within the WHO window
for normal development
Met secondary endpoint:
Survival without permanent ventilation4
Standing alone for ≥3 seconds (Bayley)
Most (7/11) within WHO window
64%
Walking alone (Bayley)
Majority (5/9) within WHO window
Data reinforces ZolgensmaⓇ as foundational therapy for both presymptomatic and symptomatic children with SMA
1. CHOP INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders. 2. Natural history data from NeuroNEXT prospective natural history study in SMA infants with two copies of SMN2. 3. Functional independent
sitting for ≥30 seconds (Bayley-III item #26) at any visit up to 18 months of age. 4. Survival at age 14 months.
15 Investor Relations | Q2 2021 Results
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