Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that four abstracts have been accepted for presentation at the American Society of Nephrology’s Kidney Week 2020, taking place virtually from October 22 to 25, 2020. New data will be presented from Alexion’s pivotal Phase 3, single-arm trial evaluating the efficacy and safety of ULTOMIRIS® (ravulizumab-cwvz) to resolve thrombotic microangiopathy (TMA) in pediatric patients with atypical hemolytic uremic syndrome (aHUS). The data demonstrate that ULTOMIRIS improved hematologic and renal outcomes by 50 weeks in 94 percent of pediatric patients who had not previously received treatment with a complement inhibitor, without any unexpected safety concerns, when administered every four or eight weeks (depending on body weight). Additionally, new data will be presented that shows results from a switch study involving pediatric patients with aHUS from stable treatment with SOLIRIS® (eculizumab) to treatment either every four or eight weeks (depending on body weight) with ULTOMIRIS. The study demonstrated continued efficacy, presented no additional safety concerns and the benefit of reduced dosing frequency.
Alexion also plans to present on the comparative efficacy of ULTOMIRIS and SOLIRIS in the treatment of adults with aHUS. Data from Phase 3 clinical trials show there are no significant differences in outcomes between patients treated with SOLIRIS verses those treated with ULTOMIRIS at 26 weeks, after balancing patient characteristics between the study groups.
“ULTOMIRIS is quickly emerging as the new standard of care for patients living with atypical hemolytic uremic syndrome,” said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. “The data we are presenting during the American Society of Nephrology’s virtual meeting reinforce our commitment to advancing the understanding of the safety and efficacy of ULTOMIRIS among different patient populations—including pediatric patients—regardless of their previous treatment experience. We look forward to sharing these data at the upcoming Congress, which continues to provide the scientific community with valuable insights into ongoing research in nephrology, despite the meeting being held virtually this year.”
The accepted abstracts are listed below and are now available on the ASN website:
Characteristics and Outcomes of Pregnancy-Triggered Atypical Hemolytic-Uremic Syndrome: Global aHUS Registry Analysis. Abstract ID #SU-OR40 – oral presentation, October 25, 2020, 5:00 p.m. ET.
Efficacy and Safety of Ravulizumab in Pediatric Patients with Atypical Hemolytic Uremic Syndrome Naïve to Complement Inhibitor Treatment: 26-week and 1-year Data. Abstract ID #PO2358 – poster presentation, October 22, 2020, 10:00 a.m. ET.
Efficacy and Safety of Ravulizumab in Pediatric Patients with Atypical Hemolytic Uremic Syndrome Previously Treated with Eculizumab: 26–week and 1–year Data. Abstract ID #PO2331 – poster presentation, October 22, 2020, 10:00 a.m. ET.
Comparative Efficacy of Ravulizumab and Eculizumab in the Treatment of Atypical Hemolytic Uremic Syndrome: An Indirect Comparison Using Clinical Trial Data. Abstract ID #PO1851 – poster presentation, October 22, 2020, 10:00 a.m. ET.