Lead(ing)

We are redefining the standard of care for people living with PNH and aHUS with ULTOMIRIS, which has a unique value proposition that is supported by patient preference of going to the hospital every two months, for adults, instead of every two weeks. In 2020, we will continue leading with ULTOMIRIS as the market leader for PNH and aspire to the same outcome for people living with aHUS.

Jason

Living with PNH

Julia

Living with aHUS

Expand(ing)

2025 Ambition:
4x Expansion of U.S. Neurology Treated Patient Population
We continue our efforts to expand our C5 franchise into new areas, where there is great unmet need and opportunity to help even more patients and families.

1. Commercial estimate. 2. Prevalence of ALS-United States, 2015 MMWR Morb Mortal Wkly Rep. 2018 Nov 23; 67(46): 1285-1289. 3. Aligned with our Phase 3 PREVENT criteria. 4. Zimmerhackl, L.B., N. Besbas, T. Jungraithmayr, N. van de Kar, H. Karch, D. Karpman, D. Landau, C. Loirat, W. Proesmans, F. Prufer, G. Rizzoni and M. C. Taylor (2006). “Epidemiology, clinical presentation, and pathophysiology of atypical and recurrent hemolytic uremic syndrome.” Semin Thromb Hemost 32(2): 113–120. 5. Rosenthal, J., HCT-TMA: a review of pathophysiology, diagnosis, and treatment, 201.  

Diversify(ing)

Development Programs in Non-C5 Assets
We are diversifying our pipeline beyond C5 with new assets to help us serve even more patients in the future.



6. Poujois, A., et al. Characteristics and prevalence of Wilson’s disease: A 2013 observational population based study in France. Clin Res Hepatol Gastroenterol. 2018 Feb;42(1):57-6. 7. Risitano AM, et al. Blood. 2009;113(17):4094-4100 8. Quock, T. P., et al. Epidemiology of AL amyloidosis: a real-world study using US claims data. Blood Adv. 2018; 2(10):1046-1053 9. Eidos Therapeutics