OUR PURPOSE PIONEERING SCIENCE PATIENTS OUR PEOPLE ENVIRONMENT COMMUNITY REPORTING 22 ENHANCING OUR STRATEGIC PORTFOLIO BIOGEN 2021 YEAR IN REVIEW SPINRAZA Celebrating 5 years of possibility for the SMA community 2012 Biogen licensed the giobal rights from lonis to develop nusinersen as the first potential treatment for spinal muscular atrophy (SMA). Biogen embarked on the largest clinical development program of its kind in SMA, comprised of no photal clinical trials in both infantile-onset (ENDEAR) and later- onset SMA (CHERISH). 2014 The first SMA patients were treated in the Phase 3 studies. 2015 Biogen initiated the first clinical trial to assess SMA treatment in presymptomatic infants, called NURTURE. SHINE, the largest, long-term extension study of people with SMA receiving a disease-modifying therapy, began. 2016 Positive interim analyses from the pivotal studies prompted Biogen to file marketing applications for SPINRAZA in the U.S. and Europe. SPINRAZA was approved by the U.S. Food and Drug Administration (FDA) just three months later. 2017 The European Commission granted marketing authorization for SPINRAZA. Phase 3 data from ENDEAR were published in the New England Joumal of Medicine. 2018 Biogen was awarded the Best Biotechnology Product from the Prix Galien Foundation. Phase 3 CHERISH results were published in the New England Journal of Medicine. Biogen accepted over 800 participants with infantile-onset SMA to its expanded access program across 10 countries, making it one of the largest in rare disease history. SPINRAZA reported $1.7 billion in revenues, with 5,000+ people treated worldwide. 2019 10,000+ people treated with SPINRAZA worldwide. 2020 The first person was treated in the Phase 2/3 DEVOTE study evaluating the potential for even greater efficacy with an investigational higher dose of nusinersen. Results from an investigational, observational study supporting the safety and efficacy of SPINRAZA in adults with SMA were published in Lancet Neurology. Results presented from NURTURE show that after up to 4.8 years of continuous treatment with SPINRAZA, 100% of children treated pre- symptomatically were alive and none required permanent ventilation. 2021 The first person was treated in the Phase 4 RESPOND study evaluating the potential benefit of SPINRAZA in infants and children with a suboptimal clinical response to Zolgensma* (onasemnogene abeparvovec). Biogen announced plans to initiate the Phase 3b ASCEND study to evaluate an investigational higher dose of nusinersen in children, teens and adults with later-onset SMA previously treated with Evrysdi® (risdiplam). SPINRAZA is approved in 58 countries with 11,000+ patients treated worldwide. 2012 2014 2015 2016 Pictured (from left): Marci, Andrea, Alexa and Matilda, all living with SMA.