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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.

Dynavax Technologies Corporation, a commercial stage biopharmaceutical company, focuses on developing and commercializing vaccines in the United States. It markets HEPLISAV-B, a hepatitis B vaccine for prevention of infection caused by all known subtypes of hepatitis B virus in age 18 years and older in the United States and Europe. The company also manufactures and sells CpG 1018, the adjuvant used in HEPLISAV-B. Dynavax Technologies Corporation has a collaboration agreement with Valneva Scotland Limited; Zhejiang Clover Biopharmaceuticals, Inc.; Clover Biopharmaceuticals (Hong Kong) Co., Limited; Biological E. Limited; PT Bio Farma; Medigen Vaccine Biologics; and Serum Institute of India Pvt. Ltd. The company was formerly known as Double Helix Corporation and changed its name to Dynavax Technologies Corporation in September 1996. The company was incorporated in 1996 and is headquartered in Emeryville, California.

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.

Satsuma Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, developing novel therapeutic products for the acute treatment of migraines. The company's lead product candidate is STS101, a drug-device combination of a proprietary dry-powder formulation of dihydroergotamine mesylate, which is in SUMMIT Phase 3 efficacy trials and can be self-administered with proprietary pre-filled, single-use, and nasal delivery device. Satsuma Pharmaceuticals, Inc. was incorporated in 2016 and is headquartered in South San Francisco, California.