Allakos
NASDAQ:ALLKAllakos Inc., a clinical stage biotechnology company, develops therapeutics that target immunomodulatory receptors present on immune effector cells in allergy, inflammatory, and proliferative diseases in the United States. The company's lead product candidate is AK006, which in a Phase I clinical trial for the treatment of chronic spontaneous urticaria (CSU) and other indications. The company was incorporated in 2012 and is headquartered in San Carlos, California.
Apellis Pharmaceuticals
NASDAQ:APLSApellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and SYFOVRE for treating geographic atrophy secondary to age-related macular degeneration and geographic atrophy (GA). The company also develops APL-3007, a small interfering RNA, or siRNA, which is in a Phase 1 clinical trial, as well as an oral complement inhibitor that is in preclinical development. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.
Arvinas
NASDAQ:ARVNArvinas, Inc., a clinical-stage biotechnology company, engages in the discovery, development, and commercialization of therapies to degrade disease-causing proteins. The company engineers proteolysis targeting chimeras (PROTAC) targeted protein degraders that are designed to harness the body's own natural protein disposal system to degrade and remove disease-causing proteins. Its product pipeline includes Bavdegalutamide and ARV-766, investigational orally bioavailable PROTAC protein degraders for the treatment of men with metastatic castration-resistant prostate cancer, which are in Phase 1/2 clinical trials; and ARV-471, an orally bioavailable estrogen receptor degrading PROTAC targeted protein degrader for the treatment of patients with locally advanced or metastatic estrogen receptor+/human epidermal growth factor receptor 2-breast cancer, which is Phase 3 clinical trial. Arvinas, Inc. has collaborations with Pfizer Inc., Genentech, Inc., F. Hoffman-La Roche Ltd., Carrick Therapeutics Limited, and Bayer AG. The company was founded in 2013 and is based in New Haven, Connecticut.
Cerevel Therapeutics
NASDAQ:CERECerevel Therapeutics Holdings, Inc., a clinical-stage biopharmaceutical company, develops various therapies for neuroscience diseases in the United States. It is developing Emraclidine, a positive allosteric modulator (PAM) that is in phase 1b clinical trials for the treatment of schizophrenia; and Darigabat, a PAM, which is in Phase 2 proof-of-concept trial in patients with drug-resistant focal onset seizures or focal epilepsy, as well as in phase 1 trial to treat panic symptoms model. The company's products also comprise Tavapadon, a selective dopamine D1/D5 partial agonist that is in phase 3 clinical trial for the treatment of early- and late-stage Parkinson's disease; CVL-871, a selective dopamine D1/D5 partial agonist, which is in Phase 2a clinical trial to treat dementia-related apathy; and CVL-354, a selective kappa-opioid receptor antagonist to treat major depressive disorder and substance use disorder. It is also involved in the development of an M4 agonist program for the treatment of psychosis and related indications; and PDE4 inhibitor for the treatment of psychiatric, neuroinflammatory, and other disorders. Cerevel Therapeutics Holdings, Inc. was founded in 2018 and is headquartered in Cambridge, Massachusetts.
Ionis Pharmaceuticals
NASDAQ:IONSIonis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, California.