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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.

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.

KALA BIO, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapies for the treatment of eye diseases. The company's product candidates include KPI-012, which is in Phase 2b clinical trial for the treatment of persistent corneal epithelial defects; EYSUVIS for the short-term treatment of the signs and symptoms of dry eye disease; and INVELTYS, a topical twice-a-day ocular steroid for the treatment of post-operative inflammation and pain following ocular surgery. Its preclinical development product, including KPI-014 for the treatment of rare inherited retinal diseases. The company was formerly known as Kala Pharmaceuticals, Inc. and changed its name to KALA BIO, Inc. in August 2023. KALA BIO, Inc. was incorporated in 2009 and is headquartered in Arlington, Massachusetts.

Prothena Corporation plc, a late-stage clinical biotechnology company, focuses on discovery and development of novel therapies to treat diseases caused by protein dysregulation in the United States. The company is involved in developing birtamimab, an investigational humanized antibody that is in Phase III clinical trial for the treatment of AL amyloidosis; Prasinezumab, a humanized monoclonal antibody, for the treatment of Parkinson's disease and other related synucleinopathies which is in Phase IIb clinical trial; NNC6019 that is in Phase lI clinical trial for the treatment of ATTR amyloidosis; and BMS-986446 and PRX012, which is in Phase I clinical trial for the treatment of Alzheimer's disease. Its discovery and preclinical programs include PRX123, a dual Aß-Tau vaccine for the treatment and prevention of Alzheimer's disease; and PRX019 for the treatment of neurogenerative diseases, as well as TDP-43 for the treatment of amyotrophic lateral sclerosis. Prothena Corporation plc has a license, development, and commercialization agreement with F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. to develop and commercialize antibodies that target a-synuclein, including prasinezumab; and a collaboration agreement with Bristol-Myers Squibb to develop and commercialize antibodies targeting tau, TDP-43. The company was incorporated in 2012 and is based in Dublin, Ireland.

UroGen Pharma Ltd., a biotechnology company, engages in the development and commercialization of solutions for urothelial and specialty cancers. It offers RTGel, a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology to improve therapeutic profiles of existing drugs; and Jelmyto for pyelocalyceal solution. The company's lead product candidate is UGN-102 for the treatment of several forms of non-muscle invasive urothelial cancer that include low-grade upper tract urothelial cancer and low-grade intermediate risk non-muscle invasive bladder cancer (NMIBC). It is also developing UGN-301 for the treatment of high-grade NMIBC. The company has license agreement with Agenus Inc. to develop, make, use, sell, import, and commercialize products of Agenus for the treatment of cancers of the urinary tract via intravesical delivery; strategic research collaboration agreement with MD Anderson focusing on the sequential use of UGN-201 and UGN-301 for the treatment of NMIBC; and licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. to develop UGN-103 in low-grade intermediate risk NMIBC and UGN-104 in low-grade upper tract urothelial carcinoma. UroGen Pharma Ltd. was incorporated in 2004 and is based in Princeton, New Jersey.