Areas of focus
See the clinical trials for hereditary angioedema (HAE), Netherton syndrome (NS) and Diabetic Macular Edema (DME), 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-AActive, Not Recruiting
This Phase 3b open-label study is providing access to berotralstat for patients with HAE who were previously enrolled in berotralstat studies.
Netherton Syndrome
Phase 1 study evaluating BCX17725 Recruiting
This is a first-in-human, Phase 1, three-part study that includes the evaluation of safety, tolerability, pharmacokinetics, and immunogenicity of BCX17725.
Diabetic Macular Edema
BCX4161-111Recruiting
A Phase 1B Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Suprachoroidal Injection of Avoralstat in Participants With Diabetic Macular Edema
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Title
A Phase 1B Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Suprachoroidal Injection of Avoralstat in Participants With Diabetic Macular Edema
Study Description
The BCX4161-111 study is an open-label, single ascending dose study enrolling participants with DME, assessed over a 24-week follow-up. It will evaluate 3 cohorts, each including at least 3 participants, that will evaluate low, mid, and high doses of avoralstat.


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.

