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X4 Pharmaceuticals (Nasdaq: XFOR) is developing novel therapeutics designed to improve immune cell trafficking to treat rare diseases, including primary immunodeficiencies and certain cancers. The company`s oral small molecule drug candidates antagonize the CXCR4 pathway, which plays a central role in immune surveillance. X4`s most advanced product candidate, mavorixafor (X4P-001), is in a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease, and is currently also under investigation in combination with axitinib in the Phase 2a portion of an open-label Phase 1/2 clinical trial in clear cell renal cell carcinoma (ccRCC). X4 is also planning to commence clinical trials of mavorixafor in Severe Congenital Neutropenia (SCN) and Waldenström`s macroglobulinemia (WM) in 2019. The company was founded and is led by a team with extensive biopharmaceutical product development and commercialization expertise and is committed to advancing the development of innovative medicines on behalf of patients with limited treatment options. X4 is a global company that is headquartered in Cambridge, Massachusetts with research offices based in Vienna, Austria.
Corcept Therapeutics Incorporated, a pharmaceutical company, engages in the discovery, development, and commercialization of drugs for the treatment of metabolic, oncologic, and psychiatric disorders in the United States. The company focuses on disorders that are associated with a steroid hormone called cortisol. It offers Korlym (mifepristone) tablets as a once-daily oral medication for the treatment of hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing’s syndrome, who have type 2 diabetes mellitus or glucose intolerance, and have failed surgery or are not candidates for surgery. The company is also developing Korlym’s active ingredient, which is in a Phase 1/2 trial in combination with the chemotherapy drug eribulin (Halaven) to treat patients with GR-positive triple-negative breast cancer, a form of cancer with a particularly poor prognosis; and CORT 125134, a proprietary, selective GR antagonist that is in a Phase 1 clinical study to assess its safety, tolerability, and pharmacokinetics in healthy human volunteers. In addition, it discovers and develops three series of novel selective GR-II antagonists. The company has research and development agreements with Argenta Discovery Limited and Sygnature Discovery Limited. Corcept Therapeutics Incorporated was founded in 1998 and is headquartered in Menlo Park, California.
BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. The Company is building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms are based on deep understandings of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.
Regulatory & Clinical Research Institute, Inc. is a Minneapolis, MN-based company in the Healthcare, Pharmaceuticals, & Biotech sector.
ActivX Biosciences is a La Jolla, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.