BioCryst Pharmaceuticals Inc (BioCryst) discovers and develops oral, novel, and small-molecule medicines to treat various rare diseases. The company uses a structure-guided drug design process, which is a drug discovery approach to design synthetic compounds from the detailed structural knowledge of the active sites of enzyme targets related to particular diseases. The company’s products include peramivir injections: Rapivab, Rapiacta, Peramiflu, and Orladeyo for the treatment of patients with influenza. Peramivir is an intravenous neuraminidase inhibitor approved in various countries for the treatment of influenza. Peramivir is approved for commercial use in the US, Canada, and Australia under the name Rapivab, in the US, Australia, and Canada; in Japan and Taiwan as Rapiacta; and in South Korea as Peramiflu. Peramivir has been approved in the US for acute, uncomplicated influenza, and the drug has been approved in Japan and Korea for uncomplicated seasonal influenza. The company’s other products include Orladeyo (Berotralstat) in oral capsule form, which has been approved for the prophylactic treatment of HAE in the US and multiple global markets. The company’s other pipeline products include BCX17725 for Netherton syndrome and Avoralstat, a kallikrein inhibitor, with the help of Clearside Biomedical Inc.'s SCS Microinjector for diabetic macular edema (DME). Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and is delivered to the suprachoroidal space. Other complementary programs include oral C5 inhibitor, which could be the first targeted oral therapy with competitive efficacy to currently approved injected and infused anti-C5 therapies, such as eculizumab and ravulizumab; oral C2 inhibitor, a classical and lectin pathway complement inhibitor; and a bifunctional complement inhibitor, an anti-C2 monoclonal antibody to treat complex complement-mediated disease.

BioCryst's R&D initiatives focus on developing treatments for diseases caused by RNA pathogens, including Marburg virus disease, Yellow Fever, and Ebola virus disease. The company has a pipeline that includes ORLADEYO (berotralstat) for hereditary angioedema, BCX17725 for Netherton Syndrome, and avoralstat for diabetic macular edema, among others. The company also supports investigator-initiated studies and external collaborative research, providing funding and study drugs as needed. The R&D unit of BioCryst is located in Birmingham, Alabama. The R&D unit of BioCryst is responsible for conducting laboratory studies, developing manufacturing processes, manufacturing product candidates, and managing clinical trials. The unit also handles the discontinuation and closeout of development programs, including termination fees and other commitments. The R&D unit's role is crucial in advancing the company's clinical programs and ensuring compliance with regulatory requirements. In FY2025, the company spent US$166.1 million on R&D, which as a percentage of revenue, stood at 18.9% and declined 4.8% YoY. As of December 2025, the company has been issued approximately 39 US patents that expire between 2027 and 2040 and that relate to our kallikrein inhibitor compounds, neuraminidase inhibitor compounds, broad-spectrum antiviral (BSAV) compounds, PNP inhibitor compounds, and complement-mediated disease program compounds. Additionally, it also includes approximately 30 patent cooperation treaties or US patent applications pending related to kallikrein inhibitor compounds, neuraminidase inhibitor compounds, BSAV compounds, PNP inhibitor compounds, KLK5 program compounds, and complement-mediated disease program compounds.