Q1 2023 Results

Company overview Innovation: Pipeline overview Cardiovascular Financial review Financial performance Immunology Conclusions Appendix Innovation: Clinical trials Neuroscience Oncology References Abbreviations Other ZolgensmaⓇ - SMN1 gene replacement therapy ZolgensmaⓇ - SMN1 gene replacement therapy NCT05089656 STEER (COAV101B12301) Indication Phase Patients Primary Outcome Measures Arms Intervention Target 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 Patients Readout Milestone(s) 2024 Publication TBD 67 Investor Relations | Q1 2023 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) Publication 2024 TBD NOVARTIS | Reimagining Medicine

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