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Aeglea BioTherapeutics, Inc., a clinical-stage biotechnology company, designs and develops human enzyme therapeutics for the treatment of rare metabolic diseases. The company's therapeutic candidates include pegtarviliase, a polyethylene glycol modified, which is in Phase I/II clinical trial for the treatment of patient with homocystinuria; and pegzilarginase, a recombinant human Arginase 1 that is in Phase III PEACE trial to evaluate the safety and efficacy for the treatment of Arginase 1 deficiency. In addition, its preclinical pipeline includes AGLE-325 for the treatment of cystinuria, as well as other research programs. The company was formerly known as Aeglea BioTherapeutics Holdings, LLC and changed its name to Aeglea BioTherapeutics, Inc. in March 2015. Aeglea BioTherapeutics, Inc. was founded in 2013 and is headquartered in Austin, Texas.

Avadel 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.

NuCana 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.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, acute leukemias, and solid tumor; and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD) and idiopathic pulmonary fibrosis (IPF). The company is also developing Entinostat. It has an agreement with Eddingpharm International Company Limited for licensing, development, and commercialization of Entinostat. Syndax Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Waltham, Massachusetts.

Xeris Biopharma Holdings, Inc., a biopharmaceutical company, engages in developing and commercializing therapies in Illinois. The company offers Gvoke, a ready-to-use liquid-stable glucagon for the treatment of severe hypoglycemia pediatric and adult patients; Keveyis, a therapy for the treatment of hyperkalemic, hypokalemic, and related variants of primary periodic paralysis; and Recorlev, a cortisol synthesis inhibitor proved for the treatment of endogenous hypercortisolemia in adult patients with Cushing's syndrome. It is also developing XP-8121, a once-weekly subcutaneous injection of levothyroxine that is in phase I clinical trial for the treatment of hypothyroidism; and non-aqueous XeriSol and XeriJect technologies for various therapies. The company was incorporated in 2005 and is headquartered in Chicago, Illinois.