Dr. Siedlecki reported that:
- Six out of 69 patients who started on Soliris therapy before transplant required dialysis, compared to 28 out of 78 who initiated Soliris treatment post-transplant. The adjusted hazard ratio was 3.0 (95% CI 1.2-7.7) comparing dialysis events in the two populations, indicating a three-fold increase in the likelihood of dialysis in the patients who initiated Soliris treatment post-transplant.
- In the sub-analysis of patients receiving labeled dosing, 4 out of 53 patients who started Soliris therapy before transplant required dialysis, compared to 23 out of 65 patients who initiated Soliris treatment post-transplant, indicating a nearly three-fold increase in likelihood of dialysis in the patients initiating Soliris treatment post-transplant. In addition, 1 out of 53 patients who started on Soliris therapy before transplant required chronic dialysis, compared to 9 out of 65 patients who initiated Soliris treatment post-transplant, indicating a four-fold increase in the likelihood of chronic dialysis in patients initiating Soliris treatment post-transplant.
Forward-Looking StatementsThis news release contains forward-looking statements, including statements related to potential medical benefits of Soliris ® (eculizumab) for the treatment of atypical hemolytic uremic syndrome (aHUS). Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including for example, risks and uncertainties of drug development, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Soliris for its current or potential new indications, delays in arranging satisfactory manufacturing capabilities for Soliris for aHUS, the possibility that results of clinical trials are not predictive of safety and efficacy results of Soliris in broader or different patient populations, the risk that third party payors (including governmental agencies) will not reimburse for the use of Soliris for the treatment of aHUS at acceptable rates or at all, the risk that estimates regarding the number of patients with aHUS and observations regarding the natural history of patients with aHUS are inaccurate, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended June 30, 2016 and in our other filings with the U.S. Securities and Exchange Commission. Alexion does not intend to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law. References 1. Siedlecki A, Isbel N, Vande Walle J, et al. Timing of Eculizumab Treatment and the Need for Dialysis in Patients with aHUS Who Receive a Kidney Transplant. Oral presentation at the American Society of Nephrology Annual Meeting, Chicago, November 19. Abstract TH-OR095. 2. Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens. 2010;19(3):242-247. 3. Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol. 2009;24:687-696. 4. Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23. 5. Schaefer F, Fakhouri F, Kupelian V, et al. Age at Onset, Complement Abnormality and Renal Survival in Patients with aHUS Enrolled in a Global Registry. Poster presentation at the American Society of Nephrology Annual Meeting, Chicago, November 19. Abstract SA-PO783. 6. Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens. 2010;19(3):242-7.
7. Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol. 2009;24:687-96.8. Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23. 9. Noris M, Caprioli J, Bresin E, et al. Relative Role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol. 2010;5:1844-59. 10. Caprioli J, Noris M, Brioschi S, et al; for the International Registry of Recurrent and Familial HUS/TTP. Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood. 2006;108:1267-1279. 11. Bresin E, et al. Combined Complement Gene Mutations in Atypical Hemolytic Uremic Syndrome Influence Clinical Phenotype. J Am Soc Nephrol. 2013;24: 475-486. 12. Fremeaux-Bacchi, et al. Genetics and Outcome of Atypical Hemolytic Uremic Syndrome: A Nationwide French Series Comparing Children and Adults. Clin J Am Soc Nephrol. 2013 Apr 5; 8(4): 554-562.