Albireo Pharma
NASDAQ:ALBOAlbireo Pharma, Inc. is a clinical stage biopharmaceutical company, which engages in the development and commercialization of novel bile acid modulators for the treatment of orphan pediatric liver diseases and gastrointestinal disorders. Its pipeline products include Odevixibat, A3384, and Elobixibat. The company was founded by Jan Peter Mattsson and Per-Göran Gillberg on December 8, 2003 and is headquartered in Boston, MA.
Clovis Oncology
NASDAQ:CLVSClovis Oncology, Inc, a biopharmaceutical company, focuses on acquiring, developing, and commercializing anti-cancer agents in the United States, Europe, and internationally. Its commercial product includes Rubraca (rucaparib) tablet, a small molecule poly ADP-ribose polymerase inhibitor, used as monotherapy for the treatment of patients with deleterious BRCA mutation associated advanced ovarian cancer, who have been treated with two or more chemotherapies, and selected for therapy by an FDA-approved companion diagnostic for Rubraca. As of 4/6/18, Rubraca® (rucaparib) is also approved by the FDA for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. FDA granted regular approval for Rubraca in this second, broader and earlier-line indication on a priority review timeline based on positive data from the phase 3 ARIEL3 clinical trial. Biomarker testing is not required for patients to be prescribed Rubraca in this maintenance treatment indication.
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.
Rigel Pharmaceuticals
NASDAQ:RIGLRigel Pharmaceuticals, Inc., a biotechnology company, engages in discovering, developing, and providing therapies that enhance the lives of patients with hematologic disorders and cancer. The company's commercialized products include Tavalisse, an oral spleen tyrosine kinase inhibitor for the treatment of adult patients with chronic immune thrombocytopenia; Rezlidhia, a non-intensive monotherapy for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test; and GAVRETO, a once daily, small molecule, oral, kinase inhibitor for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer, as well as for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer. It also develops R289, an oral IRAK1/4 Inhibitor, which is in Phase 1b clinical trials for the treatment of hematology-oncology, autoimmune, and inflammatory diseases; and a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor program in clinical development with partner Eli Lilly and Company. In addition, the company has product candidates in clinical development with partners BerGenBio ASA and Daiichi Sankyo. The company has strategic development collaboration with The University of Texas MD Anderson Cancer Center for the development of REZLIDHIA (Olutasidenib) in acute myeloid leukemia (AML) and other hematologic cancers. The company was incorporated in 1996 and is headquartered in South San Francisco, California.