Apellis Pharmaceuticals
NASDAQ:APLSApellis 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.
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.
Intercept Pharmaceuticals
NASDAQ:ICPTIntercept 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..
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.
Turning Point Therapeutics
NASDAQ:TPTXTurning 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.