Investor Presentaiton
Company overview
Financial performance
Oncology
Financial review
2022 priorities
Appendix
References
Innovation: Pipeline overview
Innovation: Clinical trials
Abbreviations
Immunology
Ophthalmology
Neuroscience
Respiratory Disease
Cardio-Renal
Global Health
Biosimilars
ZolgensmaⓇ - SMN1 gene replacement therapy
NCT05089656 STEER (COAV101B12301)
Indication
Phase
Patients
Primary
Outcome
Measures
Arms
Intervention
Spinal muscular atrophy (IT administration)
Phase 3
125
1. Change from baseline in Hammersmith functional motor scale - Expanded
(HFMSE) total score at the end of follow-up period 1 in treated patients
compared to sham controls in the ≥ 2 to < 18 years age group
Arm 1: Experimental OAV101. Administered as a single, one-time intrathecal
dose
Arm 2: Sham Comparator: Sham control. A skin prick in the lumbar region
without any medication.
Patients Type 2 Spinal Muscular Atrophy (SMA) who are ≥ 2 to < 18 years of
age, treatment naive, sitting, and never ambulatory
Target
Patients
Read-out
Milestone(s)
2024
Publication
TBD
86 Investor Relations | Q2 2022 Results
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