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Calliditas Therapeutics AB (publ), a commercial-stage specialty pharmaceutical company, focused on identifying, developing, and commercializing pharmaceuticals products for treatments in orphan indications with initial focus on renal and hepatic diseases. It offers Nefecon, an oral formulation of budesonide that is an immunosuppressant for the treatment of the autoimmune renal disease immunoglobulin A nephropathy. The company's lead product candidate is Setanaxib, a NOX inhibitor that is in Phase 2b/3 clinical trial for the treatment of primary biliary cholangitis; and in Phase 2 clinical trial for the treatment of squamous cell carcinoma of the head and neck cancer and idiopathic pulmonary fibrosis, as well as for type 1 diabetic kidney disease. It also develops Budenofalk 3 mg oral capsules for the treatment of autoimmune hepatitis. The company was founded in 2004 and is headquartered in Stockholm, Sweden.

Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

Protagonist Therapeutics, Inc., a biopharmaceutical company, develops peptide-based drugs for hematology and blood disorders, and inflammatory and immunomodulatory diseases. It is developing Rusfertide (PTG-300), an injectable hepcidin mimetic that completed phase 2 clinical trials for the treatment of polycythemia vera and other blood disorders; and JNJ-2113, an orally delivered investigational drug to block biological pathways that completed phase 2b clinical trials for the treatment of moderate-to-severe plaque psoriasis; and PN-943, an orally delivered, gut-restricted alpha 4 beta 7 specific integrin antagonist completed a phase 2 clinical trials in patients with moderate to severe ulcerative colitis. The company has a license and collaboration agreement with Takeda to commercialize rusfertide; and JNJ Innovative Medicines to co-develop Interleukin-23 receptor antagonist compound JNJ-2113. Protagonist Therapeutics, Inc. was incorporated in 2006 and is headquartered in Newark, California.

Syros Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development of treatment for hematologic malignancies. The company's lead product candidates are Tamibarotene, a selective retinoic acid receptor alpha agonist, which is in Phase III clinical trial for genomically defined subset of patients with myelodysplastic syndrome and Phase II clinical trial for patients with acute myeloid leukemia; SY-2101, a novel oral form of arsenic trioxide for treating patients with acute promyelocytic leukemia; and SY-5609, a cyclin-dependent kinase 7 inhibitor, which is in a Phase I clinical trial in patients with select advanced solid tumors. The company was formerly known as LS22, Inc. and changed its name to Syros Pharmaceuticals, Inc. in August 2012. Syros Pharmaceuticals, Inc. was incorporated in 2011 and is headquartered in Cambridge, Massachusetts.

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.