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Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

Day One Biopharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops and commercializes targeted therapies for patients with genomically defined cancers. Its lead product candidate is tovorafenib, an oral brain-penetrant type II pan-rapidly accelerated fibrosarcoma kinase inhibitor that is in Phase II clinical trial for pediatric patients with relapsed/ refractory low-grade glioma. The company is also developing Pimasertib, an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2. Day One Biopharmaceuticals, Inc. was incorporated in 2018 and is headquartered in Brisbane, California.

Chinook Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead clinical program is atrasentan, a Phase III endothelin receptor antagonist for the treatment of IgA nephropathy and other proteinuric glomerular diseases. Its product candidates also include BION-1301, an anti-APRIL monoclonal antibody is being evaluated in a Phase I/II trial for IgA nephropathy; and CHK-336, an oral small molecule LDHA inhibitor for the treatment of primary hyperoxaluria, as well as research programs for other rare and severe chronic kidney diseases. Chinook Therapeutics, Inc. is headquartered in Seattle, Washington. As of August 11, 2023, Chinook Therapeutics, Inc. operates as a subsidiary of Novartis AG.

Keros Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops and commercializes novel therapeutics for patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta family of proteins in the United States. The company's lead product candidate is KER-050, which is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia in patients with myelodysplastic syndromes, as well as in patients with myelofibrosis. It also develops KER-012, which is in Phase II clinical trial to treat pulmonary arterial hypertension and cardiovascular disorders; and KER-065 that is in Phase I clinical trial for the treatment of obesity and neuromuscular diseases. In addition, the company has collaboration and license agreement with Hansoh (Shanghai) Healthtech Co., Ltd. to develop, manufacture, and commercialize KER-050 and licensed products containing KER-050. Keros Therapeutics, Inc. was incorporated in 2015 and is headquartered in Lexington, Massachusetts.