Alexion Pharmaceuticals, Inc. ALXN announced that the phase III study of its long-acting C5 complement inhibitor Ultomiris, conducted in complement inhibitor-naive patients with atypical hemolytic uremic syndrome (aHUS), met its primary objective of complete thrombotic microangiopathy (TMA) response. The primary endpoint of complete TMA response was defined by hematologic normalization and improved kidney function.
Share price of Alexion has decreased 5.9% in the past year, narrower than the industry’s decline of 24.6%.
The study evaluated the safety and efficacy of Ultomiris (ravulizumab-cwvz/ALXN1210) administered by intravenous infusion in 56 adults (greater than or 18 years of age) who hadn’t been treated with a complement inhibitor before. In the initial 26-week treatment period, 53.6% of patients demonstrated complete TMA response. The study demonstrated 83.9% of patients treated with Ultomiris experience reduced thrombocytopenia as measured by normalization in platelet count. In the Ultomiris arm, 76.8% of patients experienced reduced hemolysis or the destruction of red blood cells, and 58.9% reported improved kidney function.
We note that aHUS is a chronic, ultra-rare disease that affects both children and adults, leading to potentially irreversible damage to kidneys and other vital organs, sudden or progressive kidney failure (requiring dialysis or transplant) and premature death. The results from the study prove that Ultomiris has the potential to become the new standard of care for patients with aHUS.
The company plans to file regulatory submission in the United States in the first half of 2019, followed by similar applications in the European Union and Japan.
We remind investors that last month the FDA approved Ultomiris for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), well ahead of its action date of Feb 18, 2019. Ultomiris is the first and the only long-acting C5 complement inhibitor to get an approval for this indication.
Alexion is also conducting a phase I study of subcutaneous ALXN1210, co-administered with Halozyme Therapeutics, Inc.’s HALO ENHANZE drug-delivery technology, PH20. Pending the co-formulation data, this next-generation subcutaneous formulation will be called ALXN1810 and has potential to further extend the dosing interval to once every two weeks or once a month.
Companies like Achillion Pharmaceuticals Inc. ACHN and Akari Therapeutics, Plc AKTX are also developing drugs for the treatment of PNH.
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