Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that five abstracts have been accepted for presentation at the 25th Congress of the European Hematology Association (EHA), which will be held virtually from June 11 to 14, 2020. During the meeting, results will be presented from an interim analysis of the Phase 2 ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL formulation open-label study extension period. The 100 mg/mL formulation of ULTOMIRIS—a long-acting C5 inhibitor administered every eight weeks offering immediate, complete and sustained complement inhibition—was evaluated to determine if participants with paroxysmal nocturnal hemoglobinuria (PNH) could achieve greater convenience by reducing infusion times, thus lessening demands on the healthcare system.
“ULTOMIRIS is an important new standard of care for patients with PNH and atypical hemolytic uremic syndrome,” said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. “An interim analysis of a PNH study evaluating the higher concentration formulation showed a 78-minute reduction in infusion time for adult patients in the 60-100kg cohort (representative of the majority of patients treated for PNH), and comparable safety, pharmacokinetics, and immunogenicity to the current formulation. This would reduce the time patients spend receiving their infusion—in clinic or in-home settings—lessening the overall burden on healthcare systems. We look forward to the opportunity to present these data during the virtually-held EHA meeting.”
The U.S. Food and Drug Administration (FDA) is currently reviewing the company’s supplemental biologics application for the ULTOMIRIS 100mg/mL formulation for the treatment of atypical hemolytic uremic syndrome (aHUS) and for adults with PNH. The agency has set a Prescription Drug User Fee Act (PDUFA) target action date of October 11, 2020. An application is also under review with the European Medicines Agency. The interim analysis that will be presented at EHA is from the extension period of the Phase 2 study evaluating the higher concentration of ULTOMIRIS in patients with PNH and is not inclusive of the aHUS patient population.
Additional data being presented at the meeting include analyses from the ongoing, observational real-world study of SOLIRIS® (eculizumab) as a treatment for PNH, along with a study evaluating the use of ALXN2040 (danicopan, formerly ACH-4471)—an investigational, oral, factor D inhibitor—used as an add-on therapy for the small, subgroup of PNH patients who are also diagnosed with extravascular hemolysis.
The accepted abstracts are listed below and are now available on the EHA website. All e-poster presentations will be available on the EHA website for the duration of the Congress.
An Interim Analysis of A Phase 2 Study Evaluating The Efficacy, Safety, and Pharmacokinetics of Intravenous Ravulizumab 100 mg/mL Formulation in Patients with Paroxysmal Nocturnal Hemoglobinuria. e-poster presentation, abstract ID#: EP862.
Development of A Composite Endpoint to Evaluate Treatment Benefit in Patients with Paroxysmal Nocturnal Hemoglobinuria. e-poster presentation, abstract ID#: EP845.
Effectiveness of Eculizumab Treatment in Patients Aged ≥65 Years with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from The International PNH Registry. e-poster presentation, abstract ID#: EP853.
Use of Prophylactic Antibiotics in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Eculizumab. e-poster presentation, abstract ID#: EP866.
Effects of Oral, Factor D Inhibitor Danicopan on Transfusion Rates in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients with A Sub-Optimal Response to Eculizumab: Phase 2 Study. E-poster presentation, abstract ID#: EP855.