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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.

Axsome Therapeutics, Inc., a biopharmaceutical company, engages in the development of novel therapies for central nervous system (CNS) disorders in the United States. The company's commercial product portfolio includes Auvelity (dextromethorphan-bupropion), a N-methyl-D-aspartate receptor antagonist with multimodal activity indicated for the treatment of major depressive disorder; and Sunosi (solriamfetol), a medication indicated to the treatment of excessive daytime sleepiness in patients with narcolepsy or obstructive sleep apnea. It is also developing AXS-05, which is in Phase III clinical trial to treat Alzheimer's disease agitation, as well as that has completed phase II clinical trial for the treatment of smoking cessation; AXS-07, an investigational medicine that has completed Phase III trials for the acute treatment of migraine; AXS-12, an investigational medicine, which is in Phase III trial to treat narcolepsy; AXS-14, a selective and potent norepinephrine reuptake inhibitor that has completed Phase III trial for the treatment of fibromyalgia and other conditions; and solriamfetol, a dual-acting dopamine and norepinephrine reuptake inhibitor, which has completed a Phase 2 trial for treating attention-deficit/hyperactivity disorder, and is in phase 2 major depressive, binge eating, and shift work disorder. Axsome Therapeutics, Inc. has a research collaboration agreement with Duke University for evaluating AXS-05 in smoking cessation. The company was incorporated in 2012 and is based in New York, New York.

Genmab A/S develops antibody therapeutics for the treatment of cancer and other diseases primarily in Denmark. The company markets DARZALEX, a human monoclonal antibody for the treatment of patients with multiple myeloma (MM); teprotumumab for the treatment of thyroid eye disease; and Amivantamab for advanced or metastatic gastric or esophageal cancer and NSCLC. Its products include daratumumab to treat MM, non-MM blood cancers, and AL amyloidosis; GEN1047; tisotumab vedotin for treating cervical, ovarian, and solid cancers; DuoBody-PD-L1x4-1BB, and DuoBody-CD40x4-1BB for treating solid tumors; Epcoritamab for relapsed/refractory diffuse large B-cell lymphoma and chronic lymphocytic leukemia; and HexaBody-CD38 and GEN3017 for treating hematological malignancies. In addition, the company develops Inclacumab, which is in Phase 3 trial for vaso-occlusive crises; Camidanlumab tesirine to treat hodgkin lymphoma and solid tumors; JNJ-64007957 and JNJ-64407564 to treat MM; PRV-015 for treating celiac disease; Mim8 for treating haemophilia A; and Lu AF82422 for treating multiple system atrophy disease. It operates various active pre-clinical programs. The company has a commercial license and collaboration agreement with Seagen Inc. to co-develop tisotumab vedotin. It also has a collaboration agreement with argenx to discover, develop, and commercialize novel therapeutic antibodies with applications in immunology and oncology; and AbbVie for the development of epcoritamab, as well as collaborations with BioNTech, Janssen, and Novo Nordisk A/S. Genmab A/S was founded in 1999 and is headquartered in Copenhagen, Denmark.

Intercept Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics to treat progressive non-viral liver diseases in the United States, Europe, and Canada. The company markets Ocaliva, a farnesoid X receptor agonist used for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults. It is also developing Ocaliva for various indications, including nonalcoholic steatohepatitis and NASH; and other product candidates in various stages of clinical and preclinical development. The company has a license agreement with Aralez Pharmaceuticals Canada Inc. to develop and commercialize bezafibrate in the United States. It markets its products through an internal commercial organization and third-party distributors. The company was incorporated in 2002 and is headquartered in Morristown, New Jersey. As of November 8, 2023, Intercept Pharmaceuticals, Inc. operates as a subsidiary of Alfasigma S.p.A..

MyoKardia, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Its lead product candidate is Mavacamten, an orally administered allosteric modulator of cardiac myosin, which is in Phase III clinical trial for the treatment of hypertrophic cardiomyopathy (HCM). The company also develops Danicamtiv, an orally administered small molecule, which completed Phase I and Phase IIa clinical trials on patients with dilated cardiomyopathy. Its preclinical programs include MYK-224, a small molecule program for the treatment of HCM targeting hypercontractility and impaired relaxation; ACT-1 for the treatment of diastolic dysfunction; and LUS-1 for the treatment of diastolic dysfunction. Myokardia, Inc. was founded in 2012 and is headquartered in Brisbane, California.