RESEARCH TRIANGLE PARK, N.C.— BioCryst Pharmaceuticals, Inc. 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.
“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 European Academy of Allergy and Clinical Immunology (EAACI) Congress in July 2021.
About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies.
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