Business Description

Our Business We are a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (“HAE”) and other rare diseases, driven by our deep commitment to improving the lives of people living with these conditions. We have built a robust commercial infrastructure to support the successful commercialization of ORLADEYO, an oral, once-daily therapy discovered and developed internally for the prevention of HAE attacks. Our business strategy includes leveraging this established commercial platform to successfully commercialize a pipeline of potential first-in-class or best-in class oral small molecule and injectable protein therapeutics targeting a range of rare diseases. These programs are being pursued through both internal discovery efforts and strategic business development. By utilizing our existing commercial capabilities and focusing on rare disease markets, we believe that we can more effectively optimize costs and strategically allocate resources to support long-term, sustainable growth. Molecules from our discovery and business development efforts that are commercially available or that are in active development are summarized in the table below and are discussed in further detail under “ Products and Product Candidates ” in this “ Part I—Item 1—Business ” section of this report. For a description of our relationships with third parties regarding our products and product candidates, see “ Business — Collaborations, License and Other Relationships .” In addition to the molecules referenced in the table below, we are pursuing certain pre-clinical medicines directed at other rare disease targets. Drug/Drug Candidate Drug Class Therapeutic Area(s) Phase ORLADEYO® (berotralstat) Oral Serine Protease Inhibitor Targeting Kallikrein (once-daily oral capsule treatment) Hereditary Angioedema Approved (United States and multiple global markets) Oral Serine Protease Inhibitor Targeting Kallikrein (once-daily oral pellets treatment for patients who are 2 to <12 years of age) Hereditary Angioedema Approved (United States) Navenibart (STAR-0215) Monoclonal Antibody Plasma Kallikrein Inhibitor Hereditary Angioedema Phase 3 BCX17725 Protein Therapeutic Netherton Syndrome Phase 1 Avoralstat Ocular Plasma Kallikrein Inhibitor Diabetic Macular Edema Phase 1 STAR-0310 Monoclonal Antibody OX40 Antagonist Atopic Dermatitis Phase 1a RAPIVAB® (peramivir injection) Intravenous Neuraminidase Inhibitor Acute Uncomplicated Influenza Approved (United States, Australia & Canada) RAPIACTA® (peramivir injection) Intravenous Neuraminidase Inhibitor Uncomplicated Seasonal Influenza Approved (Japan & Taiwan) PERAMIFLU® (peramivir injection) Intravenous Neuraminidase Inhibitor Uncomplicated Seasonal Influenza Approved (Korea) 5 Table of Contents Business Strategy Our business strategy is threefold: to serve patients, create stockholder value and increase profitability by (i) focusing our discovery efforts on potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics to target rare diseases, (ii) pursuing strategic external business development opportunities focusing on rare disease assets with disciplined and efficient use of capital, and (iii) successfully commercializing these products by leveraging our existing commercial infrastructure. By focusing primarily on rare disease markets, we believe that we can more effectively control the costs of, and our strategic allocation of financial resources toward, post-approval commercialization. We select disease targets and product candidates in which an orally-administered small-molecule or an injectable protein therapeutic has the potential to be best-in-class or would be the first to market. We strive to advance our product candidate portfolio from internal discovery and development and strategic business initiatives to commercial markets efficiently by utilizing talented and highly-skilled employees working in conjunction with strategic outsource partners. The principal elements of our strategy are: • Focusing on High Value-Added Structure-Guided Drug Design Technologies. We utilize structure-guided drug design in order to most efficiently develop new therapeutic product candidates.…