Novartis Q4 Results

Content Click below to navigate through the document ZolgensmaⓇ - SMN1 gene replacement therapy ZolgensmaⓇ - SMN1 gene replacement therapy ↓ ↑ Company overview Financial review Conclusions Appendix Innovation: Pipeline overview Financial performance Innovation: Clinical trials Cardiovascular, Renal and Metabolic Immunology > Neuroscience Oncology Other Global Health Abbreviations References NCT05089656 STEER (COAV101B12301) Spinal muscular atrophy (IT administration) Phase 3 Indication Phase Patients 125 Primary Outcome Measures Arms Intervention Target Patients 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 NCT05386680 STRENGTH (COAV101B12302) Spinal muscular atrophy (IT administration) Phase 3B Indication Phase Patients 28 Primary Outcome Measures Arms Intervention Target Patients 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) Readout Milestone(s) Publication 2024 TBD □ NOVARTIS Reimagining Medicine Readout Milestone(s) 2024 Publication TBD Novartis Q4 Results | January 31, 2024 68

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