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Amryt Pharma plc, a commercial-stage biopharmaceutical company, focuses on acquiring, developing, and commercializing various treatments to help improve the lives of patients with rare and orphan diseases. The company provides metreleptin, an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; oral octreotide, a long-term maintenance therapy in acromegaly patients; and lomitapide, an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the Homozygous familial Hypercholesteraemia. Its development candidates include Oleogel-S10 for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults; and AP103, a pre-clinical gene therapy for patients with dystrophic EB. The company sells its products in the Americas, Europe, and the Middle East. Amryt Pharma plc was founded in 2015 and is headquartered in Dublin, Ireland.

89bio, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for the treatment of liver and cardio-metabolic diseases. Its lead product candidate is pegozafermin, a glycoPEGylated analog of fibroblast growth factor 21 for the treatment of nonalcoholic steatohepatitis; and for the treatment of severe hypertriglyceridemia. The company was incorporated in 2018 and is headquartered in San Francisco, California.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Puma Biotechnology, Inc., a biopharmaceutical company, focuses on the development and commercialization of products to enhance cancer care in the United States and internationally. The company offers NERLYNX, an oral version of neratinib that is used to treat adult patients with early stage HER2-overexpressed/amplified breast cancer; and advanced or metastatic HER2-positive breast cancer when combined with capecitabine. It also develops alisertib, a small molecule inhibitor of aurora kinase A for the treatment of hormone receptor positive breast cancer, triple negative breast cancer, small cell lung cancer, and head and neck cancer. The company sells its products through specialty pharmacy and distributor networks. It has license agreements with Pfizer Inc. for the development, manufacture, and commercialization of neratinib (oral), neratinib (intravenous), PB357, and related compounds; and Takeda Pharmaceutical Company Limited for the research, development, and commercialization of alisertib, as well as sub-license agreements with Specialised Therapeutics Asia Pte Ltd., Medison Pharma Ltd., Pint Pharma International SA, Knight Therapeutics, Inc., Pierre Fabre Medicament SAS, and Bixink Therapeutics Co., Ltd. The company was founded in 2010 and is headquartered in Los Angeles, California.