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X4 Pharmaceuticals, Inc. (Nasdaq: XFOR) is a late-stage clinical biopharmaceutical company committed to the development of novel therapeutics aimed at improving immune cell trafficking and reversing immune suppression. The company's core focus is on treating rare diseases and conditions resulting from immune system dysfunction, including cancer and genetic primary immunodeficiency diseases.
X4’s lead clinical candidate, mavorixafor, is a selective, small-molecule antagonist of the chemokine receptor CXCR4. Designed as an oral, once-daily therapy, mavorixafor targets immune cell mobilization and has shown promising results in clinical trials. It is currently under priority review by the FDA for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare primary immunodeficiency. The company’s recent NDA submission is supported by positive results from the global, pivotal 4WHIM Phase 3 clinical trial, which demonstrated mavorixafor's ability to reduce infection rates and improve immune cell counts without serious adverse events.
In addition to mavorixafor for WHIM syndrome, X4 is advancing its pipeline with ongoing investigations for mavorixafor in chronic neutropenic disorders and other immunodeficiencies. The company has also initiated preclinical development of other CXCR4 antagonists, including X4P-002 and X4P-003, to address different properties and mechanisms within the immune system.
Headquartered in Boston, Massachusetts, with a research center in Vienna, Austria, X4 leverages its deep understanding of CXCR4 biology to innovate and bring transformative therapies to patients with limited treatment options. The company's mission is driven by a commitment to improve the lives of those affected by rare and severe immune diseases through rigorous scientific research and strategic investment in its proprietary pipeline.
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X4 Pharmaceuticals (Nasdaq: XFOR) announced it will report its first-quarter financial results for 2023 on May 4, 2023. The company is known for developing small molecule therapeutics aimed at treating immune system diseases. A conference call will take place at 8:30 a.m. ET on the same day to discuss the results and recent business developments. X4's lead candidate, mavorixafor, is in late-stage clinical trials for chronic neutropenic disorders, including WHIM syndrome. Following positive results from a pivotal Phase 3 trial, the company is preparing a U.S. regulatory submission for mavorixafor. X4 aims to enhance patient treatment options by mobilizing white blood cells, showcasing its commitment to innovative immune therapies.
X4 Pharmaceuticals (XFOR) has announced a virtual investor event on May 16, 2023, to discuss new data from its Phase 3 trial of mavorixafor for patients with WHIM syndrome. This follows the acceptance of a late-breaking abstract for oral presentation at the Annual Meeting of the Clinical Immunology Society (CIS) on May 21, 2023. The 4WHIM trial met its primary and key secondary endpoints, showing mavorixafor to be well tolerated with no serious treatment-related adverse events. The company will also provide updates on its U.S. regulatory plans for a New Drug Application submission, expected in H2 2023. Mavorixafor has received multiple designations including Breakthrough Therapy and Orphan Drug status, emphasizing its potential in treating WHIM syndrome, a rare immunodeficiency disorder.
X4 Pharmaceuticals issued inducement awards to new employees under the 2019 Inducement Equity Incentive Plan on March 31, 2023. The awards included options for an aggregate of 595,000 shares of common stock with an exercise price of $0.87 per share, matching the closing stock price on the grant date. These options will vest over four years, with 25% vesting after the first year and the remainder monthly thereafter, contingent on continued employment. X4 is advancing its lead candidate, mavorixafor, aimed at treating chronic neutropenic disorders, and is preparing for a U.S. regulatory submission.