Crinetics Pharmaceuticals
NASDAQ:CRNXCrinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.
Mirum Pharmaceuticals
NASDAQ:MIRMMirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.
NextCure
NASDAQ:NXTCNextCure, Inc., a clinical-stage biopharmaceutical company, engages in discovering and developing novel immunomedicines to treat cancer and other immune-related diseases by restoring normal immune function. The company is developing NC410, an immunomedicine designed to block immune suppression mediated by LAIR-1; NC762, a monoclonal antibody that binds specifically to B7-H4, a protein expressed on multiple tumor types; and NC525, a monoclonal antibody that binds to LAIR-1 to functionally kill AML blast cells and leukemic stem cells while preserving normal blood cells, including hematopoietic stem cells. Its discovery and research programs include an antibody in preclinical evaluation of other potential novel immunomodulatory molecules. The company has a license agreement with Yale University. NextCure, Inc. was incorporated in 2015 and is headquartered in Beltsville, Maryland.
UroGen Pharma
NASDAQ:URGNUroGen Pharma Ltd., a biotechnology company, engages in the development and commercialization of solutions for urothelial and specialty cancers. It offers RTGel, a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology to improve therapeutic profiles of existing drugs; and Jelmyto for pyelocalyceal solution. The company's lead product candidate is UGN-102 for the treatment of several forms of non-muscle invasive urothelial cancer that include low-grade upper tract urothelial cancer and low-grade intermediate risk non-muscle invasive bladder cancer (NMIBC). It is also developing UGN-301 for the treatment of high-grade NMIBC. The company has license agreement with Agenus Inc. to develop, make, use, sell, import, and commercialize products of Agenus for the treatment of cancers of the urinary tract via intravesical delivery; strategic research collaboration agreement with MD Anderson focusing on the sequential use of UGN-201 and UGN-301 for the treatment of NMIBC; and licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. to develop UGN-103 in low-grade intermediate risk NMIBC and UGN-104 in low-grade upper tract urothelial carcinoma. UroGen Pharma Ltd. was incorporated in 2004 and is based in Princeton, New Jersey.
Viking Therapeutics
NASDAQ:VKTXViking Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of novel therapies for metabolic and endocrine disorders. The company's lead drug candidate is VK2809, an orally available tissue and receptor-subtype selective agonist of the thyroid hormone receptor beta (TRß), which is in Phase IIb clinical trials to treat patients with biopsy-confirmed non-alcoholic steatohepatitis, as well as NAFLD. It also develops VK5211, an orally available non-steroidal selective androgen receptor modulator that is in Phase II clinical trials for the treatment of patients recovering from non-elective hip fracture surgery; VK0612, an orally available Phase IIb-ready drug candidate for type 2 diabetes; VK2735, a novel dual agonist of the glucagon-like peptide, which is in Phase 1 SAD/MAD clinical trial, and VK0214, an orally available tissue and receptor-subtype selective agonist of the TRß for X-linked adrenoleukodystrophy. The company was incorporated in 2012 and is headquartered in San Diego, California.