Albireo Pharma
NASDAQ:ALBOAlbireo Pharma, Inc. is a clinical stage biopharmaceutical company, which engages in the development and commercialization of novel bile acid modulators for the treatment of orphan pediatric liver diseases and gastrointestinal disorders. Its pipeline products include Odevixibat, A3384, and Elobixibat. The company was founded by Jan Peter Mattsson and Per-Göran Gillberg on December 8, 2003 and is headquartered in Boston, MA.
Avadel Pharmaceuticals
NASDAQ:AVDLAvadel Pharmaceuticals plc operates as a biopharmaceutical company in the United States. Its lead product candidate is LUMRYZ, a formulation of sodium oxybate, which is in a Phase 3 clinical trial for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. The company was formerly known as Flamel Technologies SA and changed its name to Avadel Pharmaceuticals plc in January 2017. Avadel Pharmaceuticals plc was incorporated in 2015 and is headquartered in Dublin, Ireland.
GlycoMimetics
NASDAQ:GLYCGlycoMimetics, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of glycobiology-based therapies for cancers, including acute myeloid leukemia (AML) and inflammatory diseases with unmet needs in the United States. It is developing uproleselan, an E-selectin antagonist, which is used in combination with chemotherapy to treat AML, as well as in phase 3 trial to treat relapsed/refractory AML. The company also develops various other programs, including GMI-1687, an antagonist of E-selectin to treat vaso-occlusive crisis; and galectin-3 antagonists, a carbohydrate-binding protein. In addition, it is developing GMI-1359, which targets e-selectin and a chemokine receptor for the treatment of cancers that affect the bone and bone marrow, including solid tumors. The company has a cooperative research and development agreement with the National Cancer Institute; and a collaboration and license agreement with Apollomics (Hong Kong) Limited for the development and commercialization of uproleselan and GMI-1687. GlycoMimetics, Inc. was incorporated in 2003 and is headquartered in Rockville, Maryland.
IVERIC bio
NASDAQ:ISEEIVERIC bio, Inc., a biopharmaceutical company, focuses on the discovery and development of novel treatments for retinal diseases with unmet medical needs. The company develops Zimura, an inhibitor of complement factor C5 comprising GATHER1, which has completed Phase 2/3 clinical trial; GATHER2 that is in Phase 3 clinical trial for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD); and STAR or OPH2005 that is in Phase 2b clinical trial for the treatment of autosomal recessive Stargardt disease. Its preclinical product candidatures include IC-500, a high temperature requirement A serine peptidase 1 protein inhibitors for the treatment of GA and other age-related retinal diseases. The company also develops OPH2001 that has completed Phase 1/2a clinical trial of various doses of avacincaptad pegol (ACP) administered as a monotherapy for the treatment of GA; OPH2000, which has completed Phase 1/2a clinical trial of various doses of ACP administered in combination with Lucentis for the treatment of wet AMD; OPH2007 that has completed Phase 2a clinical trial for the treatment of wet AMD; and OPH2002, which has completed Phase 2a clinical trial for the treatment of idiopathic polypoidal choroidal vasculopathy. In addition, its minigene programs comprises miniCEP290 program for LCA10; miniABCA4 Program for STGD1; and miniUSH2A Program for usher syndrome type 2A-Related inherited retinal diseases (IRDs). The company was formerly known as Ophthotech Corporation and changed its name to IVERIC bio, Inc. in April 2019. The company was incorporated in 2007 and is based in Parsippany, New Jersey. As of July 11, 2023, IVERIC bio, Inc. operates as a subsidiary of Astellas US Holding, Inc.
NuCana
NASDAQ:NCNANuCana plc, a clinical-stage biopharmaceutical company, engages in the development of medicines to treat patients with cancer. It applies its ProTide technology to transform prescribed chemotherapy agents and nucleoside analogs into medicines. The company, through its technology, is developing ProTides medicines to overcome the limitations of nucleoside analogs and generate much higher concentrations of anti-cancer metabolites in cancer cells. Its pipeline includes NUC-3373, a chemical entity derived from the nucleoside analog 5-fluorouracil that is in Phase 1b/2 study in patients with metastatic colorectal cancer, Phase 2 clinical trial for the second-line treatment of patients with advanced colorectal cancer, and Phase 1b/2 modular clinical trial for patients with advanced solid tumors and lung cancer. It is also developing NUC-7738, a ProTide transformation of 3'-deoxyadenosine, which is in the Phase 2 part of a Phase 1/2 clinical trial for patients with advanced solid tumors. The company has an assignment, license, and collaboration agreement with Cardiff ProTides Ltd to discover, drug design, and in vitro screen purine and pyrimidine-based nucleosides as potential drug candidates. The company was formerly known as NuCana BioMed Limited and changed its name to NuCana plc in August 2017. NuCana plc was incorporated in 1997 and is headquartered in Edinburgh, the United Kingdom.