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.
Eidos Therapeutics
NASDAQ:EIDXEidos Therapeutics, Inc., a clinical stage biopharmaceutical company, focuses on developing drugs to treat diseases caused by transthyretin (TTR) or amyloidosis (ATTR). It is developing AG10, which is in phase 3 clinical trial, is an orally-administered small molecule designed to stabilize tetrameric TTR, thereby halting at its outset the series of molecular events that give rise to ATTR. The company was founded in 2013 and is headquartered in San Francisco, California. Eidos Therapeutics, Inc. is a subsidiary of BridgeBio Pharma, Inc.
Amicus Therapeutics
NASDAQ:FOLDAmicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.
Ultragenyx Pharmaceutical
NASDAQ:RAREUltragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.
Zai Lab
NASDAQ:ZLABZai 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.