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In May 2019, the FDA followed with the approval of Zolgensma® (onasemnogene abeparvovic-xioi) for the treatment of spinal muscular atrophy (SMA). Also in 2019, the EMA approved Zynteglo® for the treatment of transfusion-dependent beta-thalassemia. Approval by the FDA is expected in 2020. From 1989 until December 2018, more than 2,900 gene therapy clinical trials were conducted, with more than 50% being phase 1 trials. In October 2019, there were 3,704 gene therapy clinical trials listed on clinicaltrials.gov. As everyone at Sarepta proudly recognizes, we are at the forefront of a revolution in medical treatment. However, efficacy and safety of a candidate treatment will not be sufficient to ensure the commercial viability of any gene therapy. Reimbursement will be a significant challenge – and to succeed, GMaX will continue to learn from both the triumphs and missteps of others who have come before Sarepta and to leverage new thinking into a successful path forward. Glybera, at one point, became the most expensive medication in the world, reaching a price of more than $1 million, which the manufacturer claimed was insufficient to cover its costs. Consequently, in 2017, the manufacturer chose to not seek the renewal of Glybera’s 5-year approval, highlighting the importance of accurate pricing models of new products in order to reflect commercial practicalities. Regarding Luxturna patients, the prevalence of LCA in the U.S. is estimated to be between 1,000 and 2,500 individuals. At a cost of $850,000 ($425,000 per eye), sales of Luxturna amounted to about $21 million over the first six months of 2019. In the United Kingdom, the approximately 180 individuals in England and Wales with LCA will soon be covered for treatment with Luxturna. In September, the National Institute for Health and Care Excellence (NICE) recommended the use of Luxturna, announcing a list price of $746,000 per patient. The manufacturers of Luxturna offered an undisclosed discount to secure a favorable recommendation, according to NICE. GMaX is meticulously tracking each step the manufacturer is taking to gain reimbursement in the U.S. While the incidence of DMD is reported to be 1 in 3,500 live male births, or approximately 540 new cases per CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE 8