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Amryt Pharma plc, a commercial-stage biopharmaceutical company, focuses on acquiring, developing, and commercializing various treatments to help improve the lives of patients with rare and orphan diseases. The company provides metreleptin, an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy; oral octreotide, a long-term maintenance therapy in acromegaly patients; and lomitapide, an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the Homozygous familial Hypercholesteraemia. Its development candidates include Oleogel-S10 for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults; and AP103, a pre-clinical gene therapy for patients with dystrophic EB. The company sells its products in the Americas, Europe, and the Middle East. Amryt Pharma plc was founded in 2015 and is headquartered in Dublin, Ireland.

CymaBay Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing and providing therapies to treat liver and other chronic diseases. Its lead product candidate is seladelpar (MBX-8025), a selective agonist of peroxisome proliferator activated receptor delta for the treatments of primary biliary cholangitis (PBC). The company also develops MBX-2982, a G protein-coupled receptor 119 (GPR119) agonist, in subjects with type 1 diabetes. It has a license agreement with ABW Cyclops SPV LP to support development of seladelpar for the treatment of PBC. The company was formerly known as Metabolex, Inc. CymaBay Therapeutics, Inc. was incorporated in 1988 and is headquartered in Fremont, California.

89bio, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for the treatment of liver and cardio-metabolic diseases. Its lead product candidate is pegozafermin, a glycoPEGylated analog of fibroblast growth factor 21 for the treatment of nonalcoholic steatohepatitis; and for the treatment of severe hypertriglyceridemia. The company was incorporated in 2018 and is headquartered in San Francisco, California.

Xenon Pharmaceuticals Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutics to treat patients with neurological disorders in Canada. Its clinical development pipeline includes XEN496, a Kv7 potassium channel opener that is Phase III clinical trials for the treatment of KCNQ2 developmental and epileptic encephalopathy; and XEN1101, a Kv7 potassium channel opener, which is in Phase II clinical trial for the treatment of epilepsy and other neurological disorders. The company's product candidates also comprise NBI-921352, a selective Nav1.6 sodium channel inhibitor that is in Phase II clinical trials for the treatment of SCN8A developmental and epileptic encephalopathy, and other indications, including adult focal epilepsy. It has a license and collaboration agreement with the Neurocrine Biosciences, Inc. to develop treatments for epilepsy. The company was incorporated in 1996 and is headquartered in Burnaby, Canada.

Zafgen, Inc., a clinical-stage biopharmaceutical company, develops therapies for patients suffering from metabolic diseases comprising type 2 diabetes, Prader-Willi syndrome (PWS), and other metabolically related disorders. The company's lead product candidate is ZGN-1061, a fumagillin-class methionine aminopeptidase 2 (MetAP2) inhibitor administered by subcutaneous injection, which is in Phase 2 clinical trial and profiled for its utility in the treatment of type 2 diabetes and other related metabolic disorders. It also develops ZGN-1258, a MetAP2 inhibitor for treatment of PWS. Zafgen, Inc. was founded in 2005 and is headquartered in Boston, Massachusetts.