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GMaX 2019 Value Learnings GMaX believes that the greatest chance of achieving a positive value assessment will be achieved by a multi-pronged approach, starting with providing payers information on the limitations of conventional value assessments as applied to one-time, high-cost gene therapies. This includes preparing articulate arguments to not only cast doubt about these traditional approaches, but to steer payers towards innovative methods for appropriately predicting the value for spend on gene-therapies. From there, once a fair value assessment is assured, we are working toward providing financing solutions that will facilitate market acceptability and adoption. Assessing the value of one-time, high cost therapies 11 CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE 1. 2. 3. 4. 5. 6. 7. Current value frameworks are inadequate to evaluate DMD treatments – requires new framework be built and adapted Pioneering financial models for microdystrophin have the potential to broaden access while addressing payer affordability. Cost of current gene therapies: less than 2019 price increase of Humira alone Innovative payer contracting has the potential to reduce CMS burden and spend for DMD Budget impact of current gene therapies: almost non-existent compared to other expensive chronic treatments Population-and outcomes-based-agreement approach reduces uncertainties and increases assessment confidence for payers and manufacturers Society’s perspective of the value of treating rare pediatric and fatal diseases is influenced by the effectiveness of treatments and rapid meaningful results