X4 Pharmaceuticals, Inc. Common Stock
X4 Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the research, development, and commercialization of novel therapeutics for the treatment of rare diseases of the immune system. It offers XOLREMDI, an orally bioavailable selective antagonist of chemokine receptor C-X-C chemokine receptor type 4 (CXCR4) for the treatment of patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome. The company also develops mavorixafor that is in Phase 3 4WARD clinical trial to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor with or without stable doses of G-CSF in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. It has a license agreement with Abbisko Therapeutics Co Ltd. to develop, manufacture, and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in oncology indications; and with Norgine to develop, manufacture, and commercialize mavorixafor in Europe, Australia, and New Zealand. The company is headquartered in Boston, Massachusetts.
Open XFOR dashboard ↗Zevra Therapeutics, Inc. Common Stock
Zevra Therapeutics, Inc., a commercial-stage company, focuses on addressing unmet needs for the treatment of rare diseases in the United States. The company develops its products through Ligand Activated Technology platform. Its lead product candidate is KP1077, consisting of KP1077IH, which is under Phase 2 clinical trial for the treatment of idiopathic hypersomnia, and KP1077N, which is under Phase 1/2 clinical trial to treat narcolepsy. The company is also developing Celiprolol, a prodrug product candidate that is under Phase 3 clinical trial for the treatment of vascular Ehlers Danlos syndrome. In addition, it offers AZSTARYS, a once-daily treatment for attention deficit and hyperactivity disorder in patients aged six years and older; OLPRUVA to treat urea cycle disorders; and MIPLYFFA for the treatment of niemann pick disease type C, an ultra-rare neurodegenerative lysosomal storage disorder. The company has collaboration and license agreement with Commave Therapeutics SA to develop, manufacture and commercialize the company's product candidates containing SDX and d-methylphenidate; and license agreement with Acer and Relief Therapeutics, Inc. to develop and commercialize rights for OLPRUVA. The company was formerly known as KemPharm, Inc. and changed its name to Zevra Therapeutics, Inc. in February 2023. Zevra Therapeutics, Inc. was incorporated in 2006 and is headquartered in Celebration, Florida.
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