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BioCryst Announces Publication of Data from Open-label Extension of the APeX-2 Pivotal Trial of ORLADEYO® (berotralstat)
– Results published in JACI: In Practice show rapid and sustained reductions in HAE attacks and improved quality of life over 96 weeks of treatment with
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"– Results published in JACI: In Practice show rapid and sustained reductions in HAE attacks and improved quality of life over 96 weeks of treatment with ORLADEYO –\nRESEARCH TRIANGLE PARK, N.C., Dec. 19, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that data from the open-label extension (OLE) of the APeX-2 trial of oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older have been published online by the Journal of Allergy and Clinical Immunology: In Practice (JACI: In Practice). “As detailed in this manuscript, long-term prophylaxis with ORLADEYO has enabled patients with HAE to better manage their condition, including reducing the number of HAE attacks they experience and demonstrating clinically meaningful improvement in their quality of life. I have seen firsthand in my practice evidence that the longer patients remain on ORLADEYO, the better their outcomes are,” said William R. Lumry, M.D., clinical professor of internal medicine at the University of Texas Southwestern Medical School. “We are pleased to share long-term efficacy and safety data from APeX-2 as published in JACI: In Practice. These data further illustrate the potential lasting outcomes that can be appreciated by patients who are treated with oral, once-daily ORLADEYO. We continue to see long-term safety and effectiveness data that reinforce ORLADEYO as an important treatment option for patients with HAE, and we look forward to sharing additional real-world evidence at upcoming medical congresses,” said Dr. Ryan Arnold, chief medical officer of BioCryst. APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group, three-part study evaluating ORLADEYO versus placebo for the prevention of HAE attacks in 121 patients with HAE Type I or Type II. In part 3 of the study (weeks 49-96), all patients (n=81) were treated with open-label ORLADEYO at 150 mg. The primary endpoint of the OLE was long-term safety and tolerability, while secondary endpoints included HAE attack rates and quality of life (QoL). The authors concluded that ORLADEYO was generally well tolerated, provided rapid and sustained reductions in HAE attacks and improved QoL over the study duration of 96 weeks. The results from part 3 of APeX-2 were first presented at the Europ...