Pharma Update

Evrysdi: Becoming a global SMA market leader >11,000 patients treated worldwide USDm 600 500 400 300 200 100 0 Worldwide SMA sales* Q1 Q3 Q1 Q3 Q1 Q3 Q1 Q3 Q1 Q3 Q1 Q3 Q1 Q3 Q1 16 16 17 17 18 18 19 19 20 20 21 21 22 22 23 -Evrysdi Spinraza Zolgensma Roche Evrysdi •risdiplam 60mg Evrysdi performance update • Significant population of untreated adults remain in key markets • >40% of patients in the US are not on any DMT • Nearly half of Evrysdi new patients starts are treatment naïve • Patients continue to switch to Evrysdi for the following reasons: • tolerability concerns and lack/loss of efficacy (Spinraza) and hope of additional benefit (Zolgensma)1 ⚫ EU label expansion for infants under 2 months achieved in August Outlook • Continued global market share gains driven by switch and naïve • Access driving growth; Evrysdi approved in >100 countries globally Further potential to grow our SMA franchise: Ph II/III combination trial with anti-latent myostatin mAb (GYM329) ongoing *Evaluate pharma; 1. Chiriboga et al, CureSMA 2023; SMA-spinal muscular atrophy; DMT-disease modifying therapy; Evrysdi in collaboration with PTC Therapeutics and SMA Foundation 53 55

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