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.
Pliant Therapeutics
NASDAQ:PLRXPliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States. The company's lead candidate is bexotegrast, an oral, small-molecule, dual selective inhibitor of avß6 and avß1 integrins, which is in phase 2b trials for idiopathic pulmonary fibrosis and in phase 2a trial for primary sclerosing cholangitis. It also develops PLN-1474, an oral, small-molecule selective inhibitor of avß1 for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis; PLN-101095, a dual inhibitor of integrins avß8 and avß1 for the treatment of solid tumors; and PLN-101325 for treatment of muscular dystrophies. Pliant Therapeutics, Inc. was incorporated in 2015 and is based in South San Francisco, California.
Cassava Sciences
NASDAQ:SAVACassava Sciences, Inc., a clinical stage biotechnology company, develops drugs for neurodegenerative diseases. Its lead therapeutic product candidate is simufilam, a small molecule drug, which is completed Phase 2 clinical trial; and investigational diagnostic product candidate is SavaDx, a blood-based biomarker/diagnostic to detect Alzheimer's disease. The company was formerly known as Pain Therapeutics, Inc. and changed its name to Cassava Sciences, Inc. in March 2019. Cassava Sciences, Inc. was incorporated in 1998 and is based in Austin, Texas.
Y-mAbs Therapeutics
NASDAQ:YMABY-mAbs Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of antibody based therapeutic products for the treatment of cancer in the United States and internationally. It offers DANYELZA, a monoclonal antibody in combination with granulocyte-macrophage colony-stimulating factor for the treatment of pediatric patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow. The company is also developing DANYELZA for the treatment of patients with second-line relapsed osteosarcoma and is in Phase II clinical study; GD2-SADA, which is in Phase I clinical trial for the treatment of GD2 positive solid tumor; and Omburtamab, a murine monoclonal antibody for the treatment of central nervous system/leptomeningeal metastases from neuroblastoma, as well as SADA PRIT technology platform. In addition, it is engages in the developing of CD38-SADA and GD2-GD3 Vaccine. The company has a license agreement with Memorial Sloan Kettering Cancer Center and Massachusetts Institute of Technology to develop and commercialize licensed products. Y-mAbs Therapeutics, Inc. was incorporated in 2015 and is headquartered in New York, New York.