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ACADIA Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization innovative medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases in the United States. The company offers NUPLAZID (pimavanserin) for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis; and DAYBUE, a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 for treatment of Rett Syndrome. It also develops Pimavanserin that is in Phase III ADVANCE-2 study to treat the negative symptoms of schizophrenia; ACP-101 whixh is in Phase III for the treatment of hyperphagia in Prader-Willi syndrome; ACP-204 which is in Phase II for the treatment of Alzheimer's disease psychosis; ACP-2591 that is in Phase I for Rett syndrome and Fragile X syndrome; preclinical antisense oligonucleotide programs; and other programs for neuropsychiatric symptoms. It has a license agreement with Neuren Pharmaceuticals Limited to develop and commercialize trofinetide for Rett syndrome and other indications; and a license and collaboration agreement with Stoke Therapeutics, Inc. to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The company was formerly known as Receptor Technologies, Inc. and changed its name ACADIA Pharmaceuticals Inc. in 1997. ACADIA Pharmaceuticals Inc. was incorporated in 1993 and is headquartered in San Diego, California.

Amarin Corporation plc, a pharmaceutical company, engages in the development and commercialization of therapeutics for the treatment of cardiovascular diseases in the United States, European countries, Canada, Lebanon, and the United Arab Emirates. The company offers VASCEPA, a prescription-only omega-3 fatty acid product, used as an adjunct to diet for reducing triglyceride levels in adult patients with severe hypertriglyceridemia. It sells its products principally to wholesalers and specialty pharmacy providers. The company has a collaboration with Mochida Pharmaceutical Co., Ltd. to develop and commercialize drug products and indications based on the active pharmaceutical ingredient in Vascepa. The company was formerly known as Ethical Holdings plc and changed its name to Amarin Corporation plc in 1999. Amarin Corporation plc was incorporated in 1989 and is headquartered in Dublin, Ireland.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California.

Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products for unmet medical needs in the United States, Europe, and internationally. The company offers Xywav for cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia; Xyrem to treat cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut and Dravet syndromes, or tuberous sclerosis complex; Zepzelca to treat metastatic small cell lung cancer, or with disease progression on or after platinum-based chemotherapy; Rylaze for acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Defitelio to treat severe hepatic veno-occlusive disease; and Vyxeos for newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Zepzelca for the treatment of patients with select relapsed/refractory solid tumors based on limited response in three solid tumor cohorts; JZP815, a pan-RAF kinase inhibitor that targets components of the mitogen-activated protein kinase; JZP898, a conditionally-activated interferon alpha molecule; Epidiolex to treat LGS, DS, and TSC; Suvecaltamide to treat parkinson's disease tremor; JZP150, a fatty acid amide hydrolase inhibitor program to treat post-traumatic stress disorder; and JZP441 to treat narcolepsy, IH, and other sleep disorders. The company has licensing and collaboration agreements with XL-protein GmbH to extend the plasma half-life of selected asparaginase product candidates; Redx Pharma plc for preclinical activities Ras/Raf/MAP kinase pathway program; and Autifony Therapeutics Limited on discovering and developing drug candidates targeting two different ion channel targets associated with neurological disorders. The company was incorporated in 2003 and is headquartered in Dublin, Ireland.

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with duchenne with a confirmed mutation in the duchenne gene. The company is also developing SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Genevant Sciences; University of Florida; Dyno Therapeutics; Hansa Biopharma; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.