Ascendis Pharma A/S, a biopharmaceutical company, focuses on developing therapies for unmet medical needs. It offers SKYTROFA for treating patients with growth hormone deficiency (GHD). The company is also developing a pipeline of three independent endocrinology rare disease product candidates in clinical development, as well as focuses on advancing oncology therapeutic candidates. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

Rhythm Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases. The company's lead product candidate is IMCIVREE (setmelanotide), a rare melanocortin-4 receptor for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1, leptin receptor (LEPR) deficiency obesity, and Bardet-Biedl and Alström syndrome. It is in Phase 3 clinical trials for treating POMC or LEPR heterozygous deficiency obesities, steroid receptor coactivator 1 deficiency obesity, SH2B1 deficiency obesity, MC4 receptor deficiency obesity, and other MC4R disorders. The company has licensing agreements with LG Chem, Ltd; Ipsen Pharma S.A.S; Camurus; RareStone Group Ltd.; joint research collaboration with Axovia Therapeutics for developing in bardet-biedl syndrome; and LG Chem, Ltd. The company was formerly known as Rhythm Metabolic, Inc. and changed its name to Rhythm Pharmaceuticals, Inc. in October 2015. Rhythm Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Boston, Massachusetts.

Crinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

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.

Lexicon Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of pharmaceutical products. Its orally-delivered small molecule drug candidates under development comprise Sotagliflozin that completed Phase III clinical trials for the for the treatment of heart failure and type 1 diabetes; and LX9211, which is in Phase II clinical development for the treatment of neuropathic pain and LX2761, which is in Phase I clinical development for gastrointestinal tract. Lexicon Pharmaceuticals, Inc. has strategic collaboration and license agreements with Bristol-Myers Squibb Company and Genentech, Inc. The company was incorporated in 1995 and is headquartered in The Woodlands, Texas.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. It has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

Aileron Therapeutics, Inc., a biopharmaceutical company, focuses on developing medicines to address unmet medical needs in orphan pulmonary and fibrosis indications in the United States. The company's lead product candidate is LTI-03, a peptide, which is in Phase 1b clinical trial for the treatment of idiopathic pulmonary fibrosis (IPF). Its second product candidate is LTI-01, a proenzyme that has completed Phase 1b and Phase 2a clinical trials in LPE patients for the treatment of loculated pleural effusion. The company's pipeline also includes preclinical programs targeting cystic fibrosis and a peptide program focused on the caveolin-1 protein for systemic fibrosis indications. The company was formerly known as Renegade Therapeutics, Inc. and changed its name to Aileron Therapeutics, Inc. in February 2007. Aileron Therapeutics, Inc. was incorporated in 2001 and is headquartered in Austin, Texas.

Eiger BioPharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the development and commercialization of targeted therapies for rare and ultra-rare diseases in the United States and internationally. Its lead product candidate is Lonafarnib, an orally bioavailable, small molecule, which is in Phase III clinical trials to treat hepatitis delta virus infection. The company's product candidate also include Lambda, which targets type III interferon receptors that has completed Phase II clinical trials; Lonafarnib for the treatment of progeria and progeroid laminopathies; and Avexitide for the treatment of congenital hyperinsulinism, as well as has completed Phase II clinical trials to treat post-bariatric hypoglycemia. Eiger BioPharmaceuticals, Inc. was founded in 2008 and is headquartered in Palo Alto, California.