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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.

Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Apellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and SYFOVRE for treating geographic atrophy secondary to age-related macular degeneration and geographic atrophy (GA). The company also develops APL-3007, a small interfering RNA, or siRNA, which is in a Phase 1 clinical trial, as well as an oral complement inhibitor that is in preclinical development. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.

Nektar Therapeutics, a biopharmaceutical company, focuses on discovering and developing medicines in the field of immunotherapy in the United States and internationally. The company is developing rezpegaldesleukin, a cytokine Treg stimulant that is in phase 2 clinical trial for the treatment of systemic lupus erythematosus and ulcerative colitis, as well as phase 2b clinical trial to treat atopic dermatitis and psoriasis; and NKTR-255, an IL-15 receptor agonist, which is in phase 1 clinical trial to boost the immune system's natural ability to fight cancer. It has collaboration agreements with Takeda Pharmaceutical Company Ltd.; AstraZeneca AB; UCB Pharma S.A.; F. Hoffmann-La Roche Ltd; Bausch Health Companies Inc.; Pfizer Inc.; Amgen Inc.; UCB Pharma (Biogen); Bristol-Myers Squibb Company; Merck KGaA; and SFJ Pharmaceuticals, Inc. The company was incorporated in 1990 and is headquartered in San Francisco, California.

Turning Point Therapeutics, Inc., a clinical-stage precision oncology biopharmaceutical company, engages in designing and developing therapies that target genetic drivers of cancer. It develops a pipeline of tyrosine kinase inhibitors (TKIs) that targets genetic drivers of cancer in TKI-naïve and TKI-pretreated patients. The company's lead drug candidate repotrectinib is being evaluated in an ongoing Phase 1/2 trial called TRIDENT-1 for the treatment of patients with ROS1+ advanced non-small-cell lung cancer (NSCLC) and patients with ROS1+ and NTRK+ advanced solid tumors. It also develops TPX-0022, a MET/SRC/CSF1R inhibitor, which is in Phase 1 SHIELD-1 clinical trial for patients with advanced solid tumors harboring genetic alterations in MET; TPX-0046, a RET inhibitor that is in 1/2 SWORD-1 clinical trial for patients with advanced solid tumors harboring RET genetic alterations; and TPX-0131, an ALK inhibitor, which is in Phase 1/2 FORGE-1 study for patient with advanced or metastatic TKI-pretreated ALK-positive NSCLC. Turning Point Therapeutics, Inc. was founded in 2013 and is headquartered in San Diego, California.