ALX Oncology
NASDAQ:ALXOALX Oncology Holdings Inc., a clinical-stage immuno-oncology company, focuses on developing therapies for cancer patients in the United States. The company's lead product candidate is Evorpacept, a CD47 blocking therapeutic biologic in development as a combination therapy with other anti-cancer agents, including ASPEN-06, under Phase 2 clinical study for treating Gastric/GEJ cancer; ASPEN-07, under Phase 1 clinical study for treating urothelial cancer; and ASPEN-03 and ASPEN-04, both under Phase 2 clinical study for treating head and neck squamous cell carcinoma. It also has collaboration agreement for Evorpacept combination programs comprising Jazz Pharmaceuticals plc for zanidatamab, under Phase 1 trial for the treatment of breast cancer and other solid tumors; Quantum Leap Healthcare collaborative with an ADC, fam-trastuzumab deruxtecan-nxki, under Phase 1 trial for the treatment of patients with breast cancer; MD Anderson Cancer Center with rituximab and lenalidomide for the treatment of patients with indolent and aggressive NHL; Sanofi with isatuximab and dexamethasone, under Phase 1/2 trial for the treatment of patients with relapsed or refractory multiple myeloma; Academic Gastrointestinal Cancer Consortium with pembrolizumab and cetuximab, under Phase 2 trial to treat refractory microsatellite stable metastatic colorectal cancer; and University of Pittsburgh with liposomal doxorubicin and pembrolizumab, under Phase 2 trial recurrent platinum-resistant ovarian cancer. In addition, the company has collaboration agreement with Tallac Therapeutics, Inc. for the development of ALTA-002, a potent immune activator targeted to myeloid cells in the tumor to promote innate and adaptive anti-cancer immune responses. It also has license agreements with Board of Trustees of the Leland Stanford Junior University, Selexis SA, and Crystal Bioscience, Inc. The company was incorporated in 2015 and is headquartered in South San Francisco, California.
Corcept Therapeutics
NASDAQ:CORTCorcept Therapeutics Incorporated engages in discovery and development of drugs for the treatment of severe endocrinologic, oncologic, metabolic, and neurologic disorders in the United States. It offers Korlym tablets medication for the treatment of hyperglycemia secondary to hypercortisolism in adult patients with endogenous cushing's syndrome; and who have type 2 diabetes mellitus or glucose intolerance and have failed surgery or are not candidates for surgery. The company is also developing relacorilant, which is in phase III clinical trial for the treatment of cushing's syndrome; treatment for adrenal cancer and cortisol excess which is in phase 1b clinical trial; treatment for prostate cancer which is in phase II clinical trial; and nab-paclitaxel in combination with relacorilant, which is in phase III clinical trial to treat platinum-resistant ovarian tumors. In addition, it develops dazucorilant, which is in phase II clinical trial for the treatment of amyotrophic lateral sclerosis; miricorilant, which is in phase IIb trial for the treatment of nonalcoholic steatohepatitis; and treatment for antipsychotic induced weight gain that is in phase I trial. The company was incorporated in 1998 and is headquartered in Menlo Park, California.
Amicus Therapeutics
NASDAQ:FOLDAmicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.
Rocket Pharmaceuticals
NASDAQ:RCKTRocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
Verve Therapeutics
NASDAQ:VERVVerve Therapeutics, Inc., a clinical stage genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases in the United States. The company's lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver; and VERVE-102, a product candidate that targets the PCSK9 gene for the treatment of HeFH. It engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.