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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.

Flexion Therapeutics, Inc. operates as a biopharmaceutical company, which engages in the development and commercialization of novel and local therapies. It specializes in the treatment of patients with musculoskeletal conditions including osteoarthritis. It offers products under the Zilretta brand, an extended-release corticosteroid approved to manage osteoarthritis knee pain. The company was founded by Michael D. Clayman and Neil Bodick on November 5, 2007 and is headquartered in Burlington, MA.

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.

Neoleukin Therapeutics, Inc., a biopharmaceutical company, develops immunotherapies for cancer, inflammation, and autoimmunity disorders using protein design technology. The company's lead product candidate is NL-201, a de novo protein designed to mimic the therapeutic activity of the cytokines interleukin (IL)-2/IL-15 for the treatment of various types of cancer, including renal cell carcinoma and melanoma. The company was formerly known as Aquinox Pharmaceuticals, Inc. and changed its name to Neoleukin Therapeutics, Inc. in August 2019. Neoleukin Therapeutics, Inc. was founded in 2003 and is headquartered in Seattle, Washington.

RAPT Therapeutics, Inc., a clinical-stage immunology-based biopharmaceutical company, focuses on discovery, development, and commercialization of oral small molecule therapies for patients with unmet needs in oncology and inflammatory diseases in the United States. The company's lead inflammation drug candidate is zelnecirnon (RPT193), a C-C motif chemokine receptor 4 (CCR4) antagonist that selectively inhibit the migration of type 2 T helper cells into inflamed tissues. Its lead oncology drug candidate is tivumecirnon (FLX475), an oral small molecule CCR4 antagonist that is in the Phase 1/2 clinical trial to investigate as a monotherapy and in combination with pembrolizumab in patients with advanced cancer. The company was formerly known as FLX Bio, Inc. and changed its name to RAPT Therapeutics, Inc. in May 2019. RAPT Therapeutics, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California. RAPT Therapeutics, Inc. operates as a subsidiary of Bristol-Myers Squibb Company