Cytokinetics
NASDAQ:CYTKCytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases. The company develops small molecule drug candidates primarily engineered to impact muscle function and contractility. Its drug candidates include omecamtiv mecarbil, a novel cardiac myosin activator that is in Phase III clinical trial in patients with heart failure. The company also develops CK-136, a novel cardiac troponin activator that is in Phase I clinical trial; CK-586, a small molecule cardiac myosin inhibitor, that is in Phase I clinical trial; and aficamten, a novel cardiac myosin inhibitor, which is in Phase III clinical trial for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy. Cytokinetics, Incorporated has a strategic alliance with Ji Xing Pharmaceuticals Limited. The company was incorporated in 1997 and is headquartered in South San Francisco, California.
Kura Oncology
NASDAQ:KURAKura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer. The company's pipeline consists of small molecule product candidates that target cancer. Its lead product candidates are ziftomenib, an orally bioavailable small molecule inhibitor of the menin-KMT2A interaction for the treatment of genetically defined subsets of acute leukemias, including acute myeloid leukemia and acute lymphoblastic leukemia; tipifarnib, an orally bioavailable farnesyl transferase inhibitor combination with alpelisib for patients with PIK3CA-dependent HNSCC; and KO-2806, a farnesyl transferase inhibitor for the treatment of solid tumors. It has a clinical collaboration with Novartis Pharma AG to evaluate the combination of tipifarnib and alpelisib in patients with head and neck squamous cell carcinoma whose tumors have HRAS overexpression and/or PIK3CA mutation and/or amplification. The company was founded in 2014 and is headquartered in San Diego, California.
Rhythm Pharmaceuticals
NASDAQ:RYTMRhythm Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases. The company's lead product candidate is IMCIVREE (setmelanotide), a rare melanocortin-4 receptor for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1, leptin receptor (LEPR) deficiency obesity, and Bardet-Biedl and Alström syndrome. It is in Phase 3 clinical trials for treating POMC or LEPR heterozygous deficiency obesities, steroid receptor coactivator 1 deficiency obesity, SH2B1 deficiency obesity, MC4 receptor deficiency obesity, and other MC4R disorders. The company has licensing agreements with LG Chem, Ltd; Ipsen Pharma S.A.S; Camurus; RareStone Group Ltd.; and LG Chem, Ltd. The company was formerly known as Rhythm Metabolic, Inc. and changed its name to Rhythm Pharmaceuticals, Inc. in October 2015. Rhythm Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Boston, Massachusetts.
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.
Wave Life Sciences
NASDAQ:WVEWave Life Sciences Ltd., a clinical-stage biotechnology company, designs, develops, and commercializes ribonucleic acid (RNA) medicines through PRISM, a discovery and drug development platform. The company's RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. It is developing WVE-006, a RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; siRNA clinical candidate for the treatment of obesity and other metabolic disorders; WVE-N531, a exon skipping oligonucleotide for the treatment of duchenne muscular dystrophy; and WVE-003, an antisense silencing oligonucleotide for the treatment of Huntington's disease (HD). The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was incorporated in 2012 and is based in Singapore.