Regulus Therapeutics
NASDAQ:RGLSRegulus Therapeutics Inc., a clinical stage biopharmaceutical company, focuses on discovery and development of drugs that targets microRNAs to treat a range of diseases in the United States. Its product candidates include RG-012, an anti-miR targeting miR-21 that is in Phase II clinical trial for the treatment of Alport syndrome, a life-threatening kidney disease; and RGLS8429, an anti-miR targeting miR-17, which is in Phase 1b clinical study for the treatment of autosomal dominant polycystic kidney disease. The company is also developing a pipeline of preclinical drug products for target organ-selective delivery strategies. Regulus Therapeutics Inc. was incorporated in 2007 and is headquartered in San Diego, 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.
Vanda Pharmaceuticals
NASDAQ:VNDAVanda Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of therapies to address high unmet medical needs worldwide. The company's marketed products include HETLIOZ to treat non-24-hour sleep-wake disorders; and Fanapt oral tablets for the treatment of schizophrenia. Its pipeline products include HETLIOZ (tasimelteon) to treat jet lag disorder, insomnia, delayed sleep phase disorder, sleep disturbances in autism spectrum disorder, and pediatric Non-24; Fanapt (iloperidone) for the treatment of bipolar I disorder and Parkinson's disease psychosis, as well as a long acting injectable (LAI) formulation to treat schizophrenia; and Tradipitant (VLY-686), a small molecule neurokinin-1 receptor (NK-1R) antagonist to treat gastroparesis, motion sickness, atopic dermatitis, and COVID-19 pneumonia. The company's pipeline products also include VTR-297, a small molecule histone deacetylase inhibitor to treat hematologic malignancies and with potential use as a treatment for various oncology indications; VQW-765, a small molecule nicotinic acetylcholine receptor partial agonist to treat performance anxiety and psychiatric disorders; VHX-896, an active metabolite of iloperidone; and antisense oligonucleotide molecules. In addition, it offers a portfolio of cystic fibrosis transmembrane conductance regulator activators and inhibitors that include VSJ-110 for the treatment of dry eye and ocular inflammation; and VPO-227 for the treatment of secretory diarrhea disorders comprising cholera. Vanda Pharmaceuticals Inc. was incorporated in 2002 and is headquartered in Washington, the District of Columbia.
Verastem
NASDAQ:VSTMVerastem, Inc., a development-stage biopharmaceutical company, focuses on developing and commercializing drugs for the treatment of cancer in the United States. Its product candidates are Avutometinib, an orally available small molecule RAF/MEK clamp that inhibits the ras sarcoma RAF/MEK, ERK mitogen activated pathway kinase pathway which is involved in cell proliferation, migration, transformation, and survival of tumor cells; and Defactinib, an oral small molecule inhibitor of FAK and proline-rich tyrosine kinase for various solid tumors. The company is involved in clinical studies, including RAMP 301, a randomized global confirmatory trial to evaluate the combination of Avutometinib and Defactinib for the treatment of patients with recurrent low-grade serous ovarian cancer; RAMP 201, an adaptive two-part multicenter, parallel cohort, randomized open label trial to evaluate the efficacy and safety of Avutometinib and in combination with Defactinib; and FRAME, an investigation of Avutometinib and Defactinib in patients with KRAS mutant cancers and subsequent analyses; and RAMP 204 and 205. It has license agreements with Chugai Pharmaceutical Co., Ltd. for the development, commercialization, and manufacture of products containing Avutometinib; and Pfizer Inc. to research, develop, manufacture, and commercialize products containing Pfizer's inhibitors of FAK for therapeutic, diagnostic, and prophylactic uses in humans. In addition, it has a clinical collaboration agreement with Amgen, Inc. to evaluate the combination of Avutometinib with Amgen's KRAS-G12C inhibitor LUMAKRAS which in Phase 1/2 trial entitled RAMP 203; and a discovery and development collaboration with GenFleet Therapeutics to advance new programs targeting RAS pathway-driven cancers. Verastem, Inc. was incorporated in 2010 and is headquartered in Needham, Massachusetts.