BioCryst Pharmaceuticals (NASDAQ:BCRX) is a clinical-stage biotechnology company focused on the discovery and development of novel, orally administered small-molecule drugs for rare diseases and viral infections. The company applies structure-based drug design and high-throughput screening to identify enzyme inhibitors that address critical unmet medical needs. BioCryst’s scientific expertise spans the complement pathway, kinins, and viral polymerases, enabling a diversified pipeline of therapeutic candidates.
BioCryst’s flagship product, berotralstat (marketed as Orladeyo®), is the first once-daily oral prophylactic treatment for hereditary angioedema (HAE), a rare genetic disorder characterized by recurrent and potentially life-threatening swelling. In addition to berotralstat, BioCryst has progressed several investigational compounds, including BCX9930, an oral complement factor D inhibitor in development for complement-mediated diseases, and galidesivir, a broad-spectrum antiviral originally studied for filoviruses and coronaviruses. The company also owns global rights to peramivir (Rapivab®), an intravenous neuraminidase inhibitor for the treatment of acute influenza.
Since its founding in 1986 and its headquarters in Durham, North Carolina, BioCryst has established a global network of collaborations and partnerships with pharmaceutical companies, academic institutions, and government agencies. These alliances support clinical development, regulatory strategy, and potential commercialization in key markets across North America, Europe, and Asia. BioCryst’s integrated approach combines in-house discovery with external expertise to accelerate the translation of promising molecules into approved therapies.
Under the leadership of President and Chief Executive Officer Jon P. Stonehouse, BioCryst continues to expand its pipeline and strengthen its commercial infrastructure. Stonehouse, a veteran biopharmaceutical executive, joined the company in 2020 and has overseen the successful launch of Orladeyo as well as the advancement of multiple clinical-stage programs. The company’s board and management team bring extensive experience in drug development, regulatory affairs, and global market access, positioning BioCryst to address critical therapeutic gaps in both rare and infectious diseases.
