Q4 2021 Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
CRM
IHD
Neuroscience
Ophthalmology
Respiratory & Allergy
Oncology: Solid Tumors
Hematology
Biosimilars
Global Health
Abbreviations
ZolgensmaⓇ - SMN1 gene replacement therapy
Study
Indication
Phase
Patients
Primary Outcome
Measures
Arms Intervention
NCT05089656 STEER (COAV101B12301)
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
Isham 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)
Publication
2024
TBD
101 Investor Relations | Q4 2021 Results
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