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Alexion's (ALXN) SC Ultomiris Meets Primary Goal in Phase III
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Alexion Pharmaceuticals, Inc. announced positive top-line results from a late-stage study on long-acting C5 complement inhibitor, Ultomiris (ravulizumab-cwvz).
The phase III study evaluated subcutaneous (SC) formulation of Ultomiris in adults with paroxysmal nocturnal hemoglobinuria (PNH), a serious, ultra-rare blood disorder. The study enrolled 136 adults with PNH who are clinically stable and have previously been treated with Soliris (eculizumab) for at least three months prior to study entry. The ongoing study met its primary objective of pharmacokinetic (PK)-based non-inferiority of Ultomiris SC versus the intravenous (IV) formulation of Ultomiris at day 71.
The study is ongoing to assess the secondary endpoints, including safety, immunogenicity and various PK/PD, quality of life, device performance, and efficacy measures.
Alexion intends to file for the approval of the SC formulation of the drug and device combination in PNH and atypical hemolytic uremic syndrome (aHUS) indications in the United States and the EU, once this study is completed and 12-month safety data are collected.
We note that Ultomiris is already approved in the United States, the EU and Japan as a treatment for adults with PNH. It is also approved for the treatment of aHUS in the United States to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. The drug is administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose.
Shares of Alexion have gained 3.5% in the year so far compared with the industry’s growth of 11.9%.
Ultomiris has gained traction since the approval, as the conversion rates of Soliris patients to Ultomiris have been encouraging. Applications for the approval of Ultomiris 100mg/mL formulation are under review in the EU and the United States. The FDA has set a Prescription Drug User Fee Act target action date of Oct 11, 2020. This higher concentration formulation is designed to reduce infusion time by more than 50% to approximately 45 minutes. Label expansion of the drug in additional indications will further boost performance.
Meanwhile, taking a lead from other biotechs like Incyte (INCY - Free Report) , Amgen (AMGN - Free Report) and Gilead Sciences, Inc. (GILD - Free Report) , Alexion has also initiated a phase III study on Ultomiris in a subset of adults suffering from COVID-19 and a positive outcome will boost prospects significantly.
Each was hand-picked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2020. Each comes from a different sector and has unique qualities and catalysts that could fuel exceptional growth.
Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.
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Alexion's (ALXN) SC Ultomiris Meets Primary Goal in Phase III
Alexion Pharmaceuticals, Inc. announced positive top-line results from a late-stage study on long-acting C5 complement inhibitor, Ultomiris (ravulizumab-cwvz).
The phase III study evaluated subcutaneous (SC) formulation of Ultomiris in adults with paroxysmal nocturnal hemoglobinuria (PNH), a serious, ultra-rare blood disorder. The study enrolled 136 adults with PNH who are clinically stable and have previously been treated with Soliris (eculizumab) for at least three months prior to study entry. The ongoing study met its primary objective of pharmacokinetic (PK)-based non-inferiority of Ultomiris SC versus the intravenous (IV) formulation of Ultomiris at day 71.
The study is ongoing to assess the secondary endpoints, including safety, immunogenicity and various PK/PD, quality of life, device performance, and efficacy measures.
Alexion intends to file for the approval of the SC formulation of the drug and device combination in PNH and atypical hemolytic uremic syndrome (aHUS) indications in the United States and the EU, once this study is completed and 12-month safety data are collected.
We note that Ultomiris is already approved in the United States, the EU and Japan as a treatment for adults with PNH. It is also approved for the treatment of aHUS in the United States to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. The drug is administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose.
Shares of Alexion have gained 3.5% in the year so far compared with the industry’s growth of 11.9%.
Ultomiris has gained traction since the approval, as the conversion rates of Soliris patients to Ultomiris have been encouraging. Applications for the approval of Ultomiris 100mg/mL formulation are under review in the EU and the United States. The FDA has set a Prescription Drug User Fee Act target action date of Oct 11, 2020. This higher concentration formulation is designed to reduce infusion time by more than 50% to approximately 45 minutes. Label expansion of the drug in additional indications will further boost performance.
Meanwhile, taking a lead from other biotechs like Incyte (INCY - Free Report) , Amgen (AMGN - Free Report) and Gilead Sciences, Inc. (GILD - Free Report) , Alexion has also initiated a phase III study on Ultomiris in a subset of adults suffering from COVID-19 and a positive outcome will boost prospects significantly.
Alexion currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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Each was hand-picked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2020. Each comes from a different sector and has unique qualities and catalysts that could fuel exceptional growth.
Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.
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