BioCryst Pharmaceuticals, Inc. is a clinical‐stage biotechnology company headquartered in Durham, North Carolina, that focuses on the discovery and development of novel, oral small‐molecule medicines for rare and serious diseases. Since its founding in 1986, the company has leveraged structure‐based drug design to advance a pipeline of targeted therapeutics designed to address underlying disease mechanisms rather than just treat symptoms.
The company’s first commercial product, Orladeyo (berotralstat), is an oral kallikrein inhibitor approved for the prophylactic treatment of hereditary angioedema (HAE) in both the United States and Europe. Orladeyo represents BioCryst’s entry into the specialty pharmaceutical market and offers an alternative to injectable prophylactic therapies, with the goal of reducing the frequency of HAE attacks and improving patient convenience.
In addition to its approved product, BioCryst is advancing a pipeline of investigational candidates, including BCX9930, an oral factor D inhibitor being studied for complement‐mediated diseases, and BCX10013, a novel kallikrein inhibitor under development for the treatment of acute HAE attacks. The company’s research and development efforts extend from preclinical stages through multiple phases of clinical trials, with a focus on expanding its small‐molecule platform to address additional rare and serious conditions.
Under the leadership of President and CEO Jon P. Stonehouse, BioCryst maintains a global orientation, collaborating with regulatory authorities and commercial partners in North America, Europe, and select international markets. The company’s headquarters in Durham support its research, development, and administrative functions, while strategic alliances and distribution agreements help facilitate patient access to its therapies worldwide.
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