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Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need in the United States. The company's lead product candidate is AT-007 (also called govorestat) that has completed phase 3 for the treatment of galactosemia in healthy volunteers and adults, in pediatric clinical study for the treatment of galactosemia in kids, for treating enzyme sorbitol dehydrogenase, and for the treatment of phosphomannomutase enzyme-CDG. It also develops AT-001 (also called caficrestat) that is in phase 3 clinical trials to treat diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy; and AT-003, which is in preclinical studies for the treatment diabetic retinopathy. The company has exclusive license and supply agreement with Mercury Pharma Group Limited to commercialize drug products containing AT-007. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.

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.

Werewolf Therapeutics, Inc., a biopharmaceutical company, develops therapeutics engineered to stimulate the body's immune system for the treatment of cancer. The company, through its proprietary PREDATOR platform, designs conditionally activated molecules that stimulate adaptive and innate immunity for addressing the limitations of conventional proinflammatory immune therapies. Its lead product candidates are WTX-124, a conditionally activated Interleukin-2 INDUKINE molecule that is in Phase I/Ib clinical trial for the treatment of advanced or metastatic solid tumors; and WTX-330, a conditionally activated Interleukin-12 INDUKINE molecule, which is in Phase I clinical trial for the treatment of advanced or metastatic solid tumors and lymphoma. The company is also developing JZP898, a conditionally activated interferon alpha INDUKINE molecule for the treatment of cancer; and WTX-712, an activated Interleukin-21(IL-21) and WTX-518, an activated IL-18 (IL-18) INDUKINE molecule for the treatment of cancer. Werewolf Therapeutics, Inc. was incorporated in 2017 and is headquartered in Watertown, Massachusetts.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.

As of January 26, 2024, operates as a subsidiary of Pathos AI, Inc.