This segment focuses on the development, commercialization, and ongoing research related to ORLADEYO, an oral serine protease inhibitor used to treat hereditary angioedema (HAE). Research and development activities include clinical trials to assess long-term efficacy and safety, as well as real-world evidence studies to demonstrate the drug's impact on patient outcomes. The technology platform centers around small-molecule drug design and development. The therapeutic area is HAE, a rare genetic disorder characterized by recurrent episodes of swelling. Patient impact is significant, as ORLADEYO provides a convenient oral treatment option, reducing the frequency of HAE attacks and improving quality of life. Market positioning is strong, with ORLADEYO being a key product. Future opportunities include expanding the drug's use and exploring new indications. Regulatory and clinical aspects involve ongoing post-marketing surveillance and adherence to FDA guidelines. Partnerships include collaborations with commercial partners for global distribution and market access.

This segment is dedicated to the research and development of BCX9930, an oral factor D inhibitor currently in Phase II clinical trials for complement-mediated diseases. Research activities include clinical trials to evaluate the safety and efficacy of BCX9930 in various complement-mediated disorders. The technology platform involves small-molecule drug discovery and development, targeting the complement system to address diseases like paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The therapeutic area focuses on rare diseases driven by complement dysregulation. The patient impact aims to provide effective treatments for conditions with high unmet medical needs. Market positioning is focused on addressing a niche market with significant growth potential. Future opportunities include expanding the clinical trial program and exploring additional indications. Regulatory and clinical aspects involve navigating the regulatory pathways for rare disease therapies. Partnerships may include collaborations with research institutions and patient advocacy groups.

This segment focuses on the development of antiviral therapies, including Galidesivir, a RNA-dependent RNA polymerase inhibitor. Research and development activities include Phase I clinical trials to evaluate the safety and efficacy of Galidesivir against various RNA viruses, such as Marburg, Yellow Fever, Ebola, and Zika. The technology platform centers around the development of small-molecule antiviral drugs. The therapeutic area covers infectious diseases caused by RNA viruses. The patient impact aims to provide effective treatments for life-threatening viral infections. Market positioning is focused on addressing unmet medical needs in the treatment of emerging and re-emerging viral diseases. Future opportunities include expanding the clinical trial program and exploring additional viral targets. Regulatory and clinical aspects involve navigating the regulatory pathways for antiviral drugs. Partnerships may include collaborations with government agencies and research institutions.