Reimagining Medicine

Company overview Innovation: Pipeline overview Cardiovascular Financial review Immunology 2023 priorities Innovation: Clinical trials Neuroscience Oncology Appendix Abbreviations Other ZolgensmaⓇ - SMN1 gene replacement therapy ZolgensmaⓇ - SMN1 gene replacement therapy NCT05089656 STEER (COAV101B12301) Indication Phase Spinal muscular atrophy (IT administration) Phase 3 Patients 125 Primary Outcome Measures Arms Intervention Target 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 Patients Readout Milestone(s) 2024 Publication TBD 80 Investor Relations | Q4 2022 Results NCT05386680 STRENGTH (COAV101B12302) Spinal muscular atrophy (IT administration) Phase 3B Indication Phase Patients 28 Primary Outcome Measures Arms Intervention Number and percentage of participants reporting AEs, related AES, SAES, and AESIS [ Time Frame: 52 weeks ] Experimental: OAV-101 Single intrathecal administration of OAV101 at a dose of 1.2 x 10^14 vector genomes Participants with SMA who discontinued treatment With Nusinersen or Risdiplam (STRENGTH) Target Patients Readout Milestone(s) 2024 Publication TBD NOVARTIS | Reimagining Medicine

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