Achillion Pharmaceuticals
NASDAQ:ACHNAchillion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes small molecule drug therapies for immune system disorders. Its lead drug candidate is ACH-4471, an inhibitor of factor D that is Phase II clinical trials for patients with paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy/immune complex membranoproliferative glomerulonephritis. The company is also developing ACH-5228, a factor D inhibitor that is in Phase I clinical trials; and ACH-5548, a factor D inhibitor, which is in Phase I clinical trials for the treatment of PNH and other complement mediated diseases. It has license agreements with Ora, Inc. for the development and commercialization of ACH-702; and GCA Therapeutics, Ltd for elvucitabine, a nucleoside reverse transcriptase inhibitor for the treatment of hepatitis B infection and human immunodeficiency virus infection. The company was founded in 1998 and is based in New Haven, Connecticut.
Akero Therapeutics
NASDAQ:AKROAkero Therapeutics, Inc., together with its subsidiary, engages in the development of treatments for patients with serious metabolic diseases in the United States. The company's lead product candidate is efruxifermin (EFX), which is in Phase 3 clinical trials that protects against cellular stress and regulates the metabolism of lipids, carbohydrates, and proteins throughout the body for the treatment of biopsy-confirmed metabolic dysfunction-associated steatohepatitis (MASH) patients. It has a license agreement with Amgen Inc. to develop, manufacture, use, distribute, and sell EFX as a treatment for MASH and other metabolic diseases. The company was formerly known as Pippin Pharmaceuticals, Inc. and changed its name to Akero Therapeutics, Inc. in May 2018. Akero Therapeutics, Inc. was incorporated in 2017 and is headquartered in South San Francisco, California.
Arcturus Therapeutics
NASDAQ:ARCTArcturus Therapeutics Holdings Inc., a late-stage clinical messenger RNA medicines and vaccine company, focuses on the development of infectious disease vaccines and other products within liver and respiratory rare diseases. Its technology platforms include LUNAR lipid-mediated delivery and STARR mRNA. The company is developing ARCT-810 (LUNAR-OTC), a mRNA-based therapeutic candidate, which is in Phase 2 clinical trial for treating ornithine transcarbamylase deficiency; and ARCT-154 (LUNAR-COV19), a mRNA vaccine candidate that is in Phase 3 arm of a Phase 1/2/3 study in Vietnam for the treatment of COVID-19, as well as ARCT-032 (LUNAR-CF), a mRNA therapeutic candidate for cystic fibrosis. Its product pipeline includes, ARCT-2301 for bivalent: ancestral/omicron which is in Phase 3; ARCT-2303 for monovalent that is in Phase 3; ARCT-2138 for quadrivalent which is in Phase 1; and LUNAR-FLU which is in pre-clinical trial. Arcturus Therapeutics Holdings Inc. was founded in 2013 and is headquartered in San Diego, California.
Nabriva Therapeutics
NASDAQ:NBRVNabriva Therapeutics plc, a biopharmaceutical company, engages in the development and commercialization of novel anti-infective agents to treat serious infections. The company's product includes SIVEXTRO, an oxazolidinone-class antibacterial for the treatment of acute bacterial skin and skin structure infection (ABSSSI); and XENLETA, a semi-synthetic pleuromutilin antibiotic for oral and IV administration. It also develops XENLETA that is in Phase I clinical trial for the treatment of pediatric infections, as well as sexually transmitted infections, cystic fibrosis, ABSSSI, ventilator-associated bacterial pneumonia, complicated intra-abdominal infections, hospital-acquired bacterial pneumonia, acute bacterial skin and skin structure infections, osteomyelitis, and prosthetic joint infections. In addition, the company develops CONTEPO, an epoxide antibiotic for use in treating complicated urinary tract infections, as well as is in Phase I clinical trial for peri-operative prophylaxis. The company was formerly known as Nabriva Therapeutics Forschungs GmbH and changed its name to Nabriva Therapeutics plc in 2007. Nabriva Therapeutics plc was incorporated in 2005 and is headquartered in Dublin, Ireland.
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.