Clinical Trials
Areas of focus
See the clinical trials for hereditary angioedema (HAE) and Netherton syndrome (NS), which use BioCryst medicines.
Clinical trials
Hereditary Angioedema
APeX-PActive, Not Recruiting
This Phase 3 study is evaluating the pharmacokinetics and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to <12 years old for prophylactic treatment to prevent attacks of HAE.
Visit ClinicalTrials.gov (NCT05453968) to learn more
APeX-AEnrolling by Invitation
This Phase 3b open-label study is providing access to berotralstat for patients with HAE who were previously enrolled in berotralstat studies.
Visit ClinicalTrials.gov (NCT04933721) to learn more
ALPHA-ORBIT Active, Not Recruiting
This Phase 3 trial is evaluating the safety and efficacy of subcutaneous administration of navenibart in adult and adolescent participants with type 1 or type 2 HAE.
Visit ClinicalTrials.gov (NCT06842823) to learn more
ALPHA-SOLAR Active, Not Recruiting
This Phase 2 open-label trial is assessing the long-term safety and efficacy of repeat dosing of navenibart in participants with HAE. Participants will receive repeat doses of navenibart for up to 5 years.
Visit ClinicalTrials.gov (NCT06007677) to learn more
ORBIT-EXPANSE Active, Enrolling by Invitation
This Phase 3 trial is evaluating the long-term safety and efficacy of navenibart in adult and adolescent participants with HAE who participated in ALPHA-ORBIT (NCT06842823). Part 1 provides all participants with navenibart in a dose-controlled fashion; part 2 introduces a personalized dosing option.
Netherton Syndrome
Phase 1 study evaluating BCX17725 Active, Recruiting
This is a first-in-human, Phase 1, four-part study that includes the evaluation of safety, tolerability, pharmacokinetics, and immunogenicity of BCX17725.
A growing pipeline of small-molecule and protein therapeutics
BioCryst development programs represent the potential to improve the well-being of people whose lives are currently limited by HAE and other rare diseases. We discover novel, small-molecule and protein therapeutics that treat diseases in which significant unmet medical needs exist.