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Global Employees
93
R&D Investment
64400000
This segment focuses on the research, development, and clinical trials of therapies for nonalcoholic steatohepatitis (NASH) and other liver diseases. The lead product candidate, pegozafermin, is a glycoPEGylated analog of fibroblast growth factor 21 (FGF21) designed to address the underlying metabolic dysfunction in NASH. Research and development activities include Phase 3 clinical trials (ENLIGHTEN-Fibrosis and ENLIGHTEN-Cirrhosis) to evaluate the efficacy and safety of pegozafermin. Technologies and methodologies include glycoPEGylation for improved drug half-life and efficacy, and advanced liver disease diagnostics. The segment aims to improve patient outcomes by reducing liver inflammation, fibrosis, and the risk of progression to cirrhosis and liver failure. Market positioning involves targeting a significant unmet medical need in NASH, with a competitive advantage in FGF21-based therapies. Future opportunities include expanding the use of pegozafermin to treat other liver diseases and exploring combination therapies. Regulatory and clinical aspects involve navigating FDA and EMA guidelines for NASH drug development. Partnerships include a collaboration agreement with BiBo Biopharma for commercial supply of pegozafermin.
This segment is dedicated to the development of therapies for cardio-metabolic diseases, specifically severe hypertriglyceridemia (SHTG). The primary focus is on utilizing pegozafermin to reduce elevated triglyceride levels and mitigate the associated cardiovascular risks. Research and development activities include preclinical studies and clinical trials to assess the efficacy and safety of pegozafermin in SHTG patients. Technologies and methodologies involve the use of FGF21 analogs and advanced lipid metabolism analysis. The segment aims to improve patient outcomes by reducing the risk of pancreatitis and cardiovascular events associated with SHTG. Market positioning involves addressing a significant unmet need in the treatment of SHTG, with a focus on innovative therapeutic approaches. Future opportunities include expanding the application of pegozafermin to other related metabolic disorders. Regulatory and clinical aspects involve obtaining regulatory approvals for SHTG indications. Partnerships and collaborations may be pursued to enhance the development and commercialization of therapies in this area.