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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.

Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. The company's products in pipeline includes Plozasiran, which is in Phase 2b and one Phase 3 clinical trial to treat hypertriglyceridemia, mixed dyslipidemia, and chylomicronemia syndrome; Zodasiran that is in Phase 2b clinical trial for the treatment of dyslipidemia and hypertriglyceridemia; ARO-PNPLA3, which is in Phase 1 clinical trial to treat patients with non-alcoholic steatohepatitis; ARO-RAGE that is in Phase 1/2a clinical trial to treat inflammatory pulmonary conditions; and ARO-MUC5AC, which is in Phase 1/2a clinical trial to treat muco-obstructive pulmonary diseases. It also develops ARO-MMP7 that is in Phase 1/2a clinical trial for treatment of idiopathic pulmonary fibrosis; ARO-DUX4 for the treatment of facioscapulohumeral muscular dystrophy; ARO-SOD1 for the potential treatment of amyotrophic lateral sclerosis; and ARO-C3, which is in Phase 1/2a clinical trial for the treatment of patients with various complement mediated or complement associated renal diseases. In addition, the company is involved in the development of JNJ-3989, which is in Phase 2 clinical trial to treat chronic hepatitis B virus infection; Olpasiran that is in Phase 3 clinical trial to reduce the production of apolipoprotein A; GSK-4532990 that is in phase 2 clinical trial to treat liver diseases; HZN-457, which is in phase 1 clinical trial to treat uncontrolled gout; and Fazirsiran that is in Phase 3 clinical trial for the treatment for liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead Pharmaceuticals, Inc. has license and research collaboration agreements with Janssen Pharmaceuticals, Inc.; Takeda Pharmaceutical Company Limited; Horizon Therapeutics Ireland DAC; Amgen Inc.; and Glaxosmithkline Intellectual Property (No. 3) Limited. The company was founded in 2003 and is headquartered in Pasadena, California.

Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapies for unmet medical needs. It offers SKYTROFA for treating patients with growth hormone deficiency (GHD). The company is also developing a pipeline of three independent endocrinology rare disease product candidates in clinical development, as well as focuses on advancing oncology therapeutic candidates. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

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.

Zai Lab Limited develops and commercializes therapies to treat oncology, autoimmune disorders, infectious diseases, and neuroscience. Its commercial products include Zejula, an orally administered poly polymerase 1/2 inhibitor; Optune, a cancer therapy that uses electric fields tuned to specific frequencies to kill tumor cells; NUZYRA for acute bacterial skin and skin structure infections, and community acquired bacterial pneumonia; Qinlock to treat gastrointestinal stromal tumors, and VYVGART, a human IgG1 antibody fragment for myesthenia gravis. The company also develops Tumor Treating Fields, a portable device for delivery of electric fields; Repotrectinib, a tyrosine kinase inhibitor (TKI) to target ROS1 and TRK A/B/C in TKI-naïve- or -pretreated cancer patients; Tisotumab vedotin, an antibody drug conjugate; Adagrasib for treating KRAS-G12C-mutated NSCLC, colorectal cancer, and pancreatic cancer; and Bemarituzumab to treat gastric and gastroesophageal junction cancer patients. In addition, it develops Sulbactam/durlobactam, a combination of a beta-lactam antibiotic and a beta-lactamase inhibitor for the treatment of serious infections caused by Acinetobacter; KarXT for the treatment of psychiatric and neurological conditions. It has license and collaboration agreement with Tesaro, Inc. to develop, manufacture, and commercialize niraparib; NovoCure to develop and commercialize Tumor Treating Fields; Deciphera to develop and commercialize ripretinib; Paratek Bermuda Ltd. to develop, manufacture, and commercialize omadacycline; argenx, to develop and commercialize efgartigimod; BMS to develop and commercialize tisotumab vedotin and repotrectinib; Mirati to research, develop, manufacture, and commercialize adagrasib; Amgen to develop and commercialize bemarituzumab; and Innoviva to develop and commercialize Sulbactam-Durlobactam; Karuna to develop and commercialize KarXT. The company was incorporated in 2013 and is headquartered in Shanghai, China.