Biohaven
NYSE:BHVNBiohaven Ltd., together with its subsidiaries, focuses on discovering, developing, and commercializing therapies for immunology, neuroscience, and oncology worldwide. The company's pipeline products include Troriluzole, which is in Phase 3 clinical trial for the treatment of neurological and neuropsychiatric illnesses; BHV-5500 that blocks glutamate signaling mediated by post-synaptic NMDA receptors; Taldefgrobep Alfa, which is in Phase 3 clinical trial for the treatment of spinal muscular atrophy and obesity; BHV-7000, a candidate in Phase 2/3 clinical trials for the treatment of focal and generalized epilepsy, bipolar disorder, and major depressive disorder; BHV-2100 that is in Phase 1 clinical trials for the treatment of migraines and neuropathic pain; and BHV-8000, a product candidate in Phase 1 clinical trials for the treatment of early Alzheimer's and Parkinson's disease, sclerosis, and amyloid-related imaging abnormalities. It also offers BHV-1300, a product candidate in Phase 1 clinical trials to treat rheumatoid arthritis; BHV-1310 for the treatment of generalized myasthenia gravis and acute exacerbations or flares; BHV-1400 to treat IgA Nephropathy; and BHV-1600 for the treatment of dilated cardiomyopathy. In addition, the company develops BHV-1100, a product candidate in Phase 1a/1b clinical trials for multiple myeloma patients; BHV-1510, a preclinical product that targets carcinomas; and BHV-1500 for Hodgkin's lymphoma. It has license, development, and commercialization agreements with Yale University, AstraZeneca, University of Connecticut, Artizan Biosciences Inc., Reliant Glycosciences LLC, Katholieke Universiteit Leuven, BMS, and Highlightll Pharmaceutical Co. Ltd. The company was formerly known as Biohaven Research Ltd and changed its name to Biohaven Ltd. in September 2022. Biohaven Ltd. was incorporated in 2022 and is headquartered in New Haven, Connecticut.
Principia Biopharma
NASDAQ:PRNBPrincipia Biopharma Inc., a late-stage biopharmaceutical company, focuses on developing novel therapies for immunology and oncology. The company is developing PRN1008, an inhibitor that is in Phase III clinical trial for the treatment of pemphigus, a chronic skin disease, as well as in Phase II clinical trial to treat immune thrombocytopenic purpura. It is also developing PRN2246/SAR442168, an inhibitor, which has completed Phase I clinical trial for treating multiple sclerosis (MS) and other central nervous system (CNS) diseases; PRN1371, a drug candidate that is in Phase I clinical trial for the treatment of solid tumors; and oral small molecule inhibitors of the immunoproteasome. The company has a collaboration agreement with Genzyme Corporation to develop relapsing and progressive MS and other diseases of the CNS. Principia Biopharma Inc. was incorporated in 2008 and is headquartered in South San Francisco, California.
uniQure
NASDAQ:QUREuniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Ra Pharmaceuticals
NASDAQ:RARXRa Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapeutics for the treatment of diseases caused by excessive or uncontrolled activation of the complement system. The company's peptide chemistry platform enables the production of synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. Its lead product candidate is Zilucoplan, an injection into the tissue under the skin that has completed Phase II clinical trial for the treatment of generalized myasthenia gravis (gMG); has completed Phase II clinical trial for treating paroxysmal nocturnal hemoglobinuria (PNH); and has completed Phase Ib clinical trial to treat patients with renal impairment. The company's pre-clinical programs include Factor D inhibition for treating C3 glomerulonephritis and dense deposit disease; and inhibitors of other complement factors for renal, autoimmune, and central nervous system diseases. It has collaboration and license agreement with Merck & Co., Inc. to identify orally available cyclic peptides for non-complement program targets, and provide research and development services. Ra Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Cambridge, Massachusetts.
Synthorx
NASDAQ:THORSynthorx, Inc., a biopharmaceutical company, focuses on the development of cytokine Synthorin programs for the treatment of cancer and autoimmune disorders in the United States. The company's lead product candidate is THOR-707, a variant of recombinant human IL-2 that is in the development in various solid tumor types as a single agent and in combination with an immune checkpoint inhibitor. It also develops IL-2 Synthorin for autoimmune indications; IL-10 Synthorin, a naturally occurring immune cell growth factor in humans for the treatment of immuno-oncology (IO); and IL-15 Synthorins, an immunoregulatory cytokine to treat IO. In addition, the company develops other Synthorin programs targeting undisclosed cytokines that play critical roles in the orchestration of anti-tumor responses by innate and adaptive immune cells. The company was formerly known as Alinos, Inc. and changed its name to Synthorx, Inc. in March 2014. Synthorx, Inc. was founded in 2014 and is headquartered in La Jolla, California.