ArQule
NASDAQ:ARQLArQule, Inc., a biopharmaceutical company, researches and develops therapeutics for the treatment of cancer and rare diseases in the United States. The company's pipeline includes ARQ 531, an orally bioavailable, potent and reversible dual inhibitor of wild type and C481S-mutant Bruton's tyrosine kinase that is in Phase I trial for patients with B-cell malignancies refractory to other therapeutic options; and miransertib (ARQ 092), a potent and selective inhibitor of the protein kinase B (AKT), a serine/threonine kinase, which is in Phase Ib in combination with the hormonal therapy and anastrozole in patients with advanced endometrial cancer. Its pipeline also comprises ARQ 75, a potent and selective inhibitor of AKT that is in Phase I clinical development for solid tumors harboring AKT, phosphoinositide 3-kinase or phosphatase, and tensin homolog loss mutations. In addition, the company's pipeline includes Derazantinib (ARQ 087), a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family of kinases that is in a registrational clinical trial in intrahepatic cholangiocarcinoma in patients with FGFR2 fusions. ArQule, Inc. has license agreements with Basilea Pharmaceutica Limited and Roivant Sciences Ltd. The company was founded in 1993 and is headquartered in Burlington, Massachusetts.
Insmed
NASDAQ:INSMInsmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product is ARIKAYCE® (amikacin liposome inhalation suspension), which is approved in the United States for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a rare and often chronic infection that can cause irreversible lung damage and can be fatal. Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and INS1009, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension.
Karuna Therapeutics
NASDAQ:KRTXKaruna Therapeutics, Inc., a clinical-stage biopharmaceutical company, creates and delivers transformative medicines for people living with psychiatric and neurological conditions. Its lead product candidate includes KarXT, an oral modulator of muscarinic receptors for the treatment of acute psychosis in patients with schizophrenia; and for the treatment of central nervous system disorders, such as negative and cognitive symptoms of schizophrenia and psychosis, as well as for the treatment of various peripheral tissues and dementia-related psychosis, including Alzheimer's disease. The company also focuses on developing other muscarinic-targeted drug candidates; and intends to develop lead candidature TRPC4/5 and KAR-2618 for the treatment of depressive disorder. Karuna Therapeutics, Inc. has a license agreement with Eli Lilly and Company, and Zai Lab (Shanghai) Co., Ltd; patent license agreement with PureTech Health LLC; and drug discovery partnership with Charles River Laboratories, as well as drug discovery collaboration with PsychoGenics, Inc. The company was formerly known as Karuna Pharmaceuticals, Inc. and changed its name to Karuna Therapeutics, Inc. in March 2019. Karuna Therapeutics, Inc. was incorporated in 2009 and is headquartered in Boston, Massachusetts.
Ra Pharmaceuticals
NASDAQ:RARXRa Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapeutics for the treatment of diseases caused by excessive or uncontrolled activation of the complement system. The company's peptide chemistry platform enables the production of synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. Its lead product candidate is Zilucoplan, an injection into the tissue under the skin that has completed Phase II clinical trial for the treatment of generalized myasthenia gravis (gMG); has completed Phase II clinical trial for treating paroxysmal nocturnal hemoglobinuria (PNH); and has completed Phase Ib clinical trial to treat patients with renal impairment. The company's pre-clinical programs include Factor D inhibition for treating C3 glomerulonephritis and dense deposit disease; and inhibitors of other complement factors for renal, autoimmune, and central nervous system diseases. It has collaboration and license agreement with Merck & Co., Inc. to identify orally available cyclic peptides for non-complement program targets, and provide research and development services. Ra Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Cambridge, Massachusetts.
TG Therapeutics
NASDAQ:TGTXTG Therapeutics, Inc., a commercial stage biopharmaceutical company, focuses on the acquisition, development, and commercialization of novel treatments for B-cell mediated diseases in the United States and internationally. It provides BRIUMVI, an anti-CD20 monoclonal antibody for the treatment of adult patients with relapsing forms of multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease in adults. The company's development pipeline comprises Umbralisib, an oral inhibitor of PI3K-delta inhibitor for the treatment of adult patients with relapsed or refractory marginal zone lymphoma and follicular lymphoma; TG-1701 is an orally available and covalently bound Bruton's tyrosine kinase (BTK) inhibitor that exhibits selectivity to BTK in vitro kinase screening; and TG-1801, a bispecific CD47 and CD19 antibody. Its research pipeline includes various investigational medicines. The company has license agreements with LFB Biotechnologies S.A.S; GTC Biotherapeutics; LFB/GTC LLC; Ildong Pharmaceutical Co. Ltd.; Rhizen Pharmaceuticals, S A. for the development and commercialization of umbralisib; Jiangsu Hengrui Medicine Co.; and Novimmune SA, as well as collaboration agreement with Checkpoint Therapeutics, Inc. for the development and commercialization of anti-PD-L1 and anti-GITR antibody research programs in the field of hematological malignancies. TG Therapeutics, Inc. was incorporated in 1993 and is based in Morrisville, North Carolina.