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Eton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.

Helix BioPharma Corp. operates as an immune-oncology company in Canada. It is developing L-DOS47 for the treatment of non-small cell lung cancer; and V-DOS47 that targets the vascular epithelial growth factor receptor 2. Helix BioPharma Corp. has collaboration agreements with Moffitt Cancer Center to investigate the pharmacodynamics of L-DOS47 and determine the benefits of combining L-DOS47 with immune checkpoint inhibitors; and with University Hospital Tuebingen to assess the therapeutic response of L-DOS47 in cancer models expressing CEACAM6, with advanced preclinical metabolic imaging. The company is headquartered in Toronto, Canada.

IDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.

Paratek Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of life-saving therapies for life-threatening diseases or other public health threats for civilian, government, and military use. Its products include NUZYRA, a once-daily oral and intravenous broad-spectrum antibiotic for the treatment of adult patients with community-acquired bacterial pneumonia, and acute skin and skin structure infections caused by susceptible pathogens; and SEYSARA, a once-daily oral therapy for the treatment of moderate to severe acne vulgaris. The company has license and collaboration agreements with Zai Lab (Shanghai) Co., Ltd. and Allergan plc; license agreement with Tufts University to develop and commercialize products for the treatment or prevention of bacterial or microbial diseases, or medical conditions; and contract with Biomedical Advanced Research and Development Authority to support the development of NUZYRA for the treatment of pulmonary anthrax. It also has license agreement with Tetraphase Pharmaceuticals, Inc. to develop, import, and sell the XERAVATM product, which is used for the treatment of complicated intra-abdominal infections caused by bacteria. The company was founded in 1996 and is headquartered in Boston, Massachusetts.

Strongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.