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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.
For more updates and detailed information, visit X4 Pharmaceuticals.
X4 Pharmaceuticals (Nasdaq: XFOR) announced promising preliminary efficacy and safety results from its Phase 1b clinical trial of mavorixafor combined with ibrutinib for Waldenström’s macroglobulinemia patients with MYD88 and CXCR4 mutations. Patients demonstrated robust serum IgM reductions of 60%-75%, exceeding previous monotherapy results of 38%-45%. Furthermore, all patients showed increases in hemoglobin levels, indicating reduced cancer burden. The trial continues with patient enrollment and dose escalation for further evaluation.
X4 Pharmaceuticals (Nasdaq: XFOR) announced a conference call and webcast to discuss initial efficacy and safety data from the Phase 1b trial of mavorixafor combined with ibrutinib in Waldenström’s Macroglobulinemia patients. This data will be presented during the European Hematology Association Annual Congress on June 11, 2021. The company aims to highlight the potential benefits of mavorixafor, a novel therapy targeting CXCR4 dysfunction, which is currently also evaluated in a Phase 3 trial for WHIM syndrome.
X4 Pharmaceuticals (Nasdaq: XFOR) announced management participation in two significant events: the Cowen 2nd Annual Virtual Oncology Innovation Summit on May 20, 2021, at 2:00 PM ET, and the Oppenheimer Rare & Orphan Disease Summit on May 21, 2021, at 12:25 PM ET. A live webcast will be available for the latter, hosted on their investor website and archived for about 90 days. X4 is focused on developing therapies targeting diseases caused by CXCR4 pathway dysfunction, with its lead candidate, mavorixafor, currently in Phase 3 trials for WHIM syndrome and Phase 1b trials for other conditions.
X4 Pharmaceuticals announced initial efficacy and safety data from a Phase 1b trial of mavorixafor combined with ibrutinib for treating Waldenström’s macroglobulinemia. Key findings show the combination was well tolerated, with a median serum IgM decrease of 51% in patients who completed three treatment cycles. This promising data will be presented as an e-Poster at the European Hematology Association Congress on June 11, 2021. The trial supports further exploration of mavorixafor's role in sensitizing specific genetic mutations to treatment, enhancing therapeutic options for patients with limited responses.
X4 Pharmaceuticals (Nasdaq: XFOR) reported significant clinical advancements in its lead candidate, mavorixafor, targeting rare diseases. The company announced a net loss of $18.7 million for Q1 2021, compared to $11.1 million in Q1 2020. Cash reserves stand at $116.7 million, expected to fund operations into Q4 2022, following a $55 million PIPE financing. The company is set to present initial data from its Phase 1b trial at the EHA Meeting in June 2021 and anticipates an enrollment update for its Phase 3 WHIM trial by mid-2021.
X4 Pharmaceuticals (Nasdaq: XFOR) recently announced the issuance of an inducement award on April 30, 2021, under its 2019 Inducement Equity Incentive Plan. The award includes options to purchase 17,000 shares of common stock at an exercise price of $8.38 per share, reflecting the closing price on the grant date. These options will vest over four years and are aimed at attracting new talent. X4 focuses on developing therapies targeting CXCR4 pathway dysfunction, with its lead candidate, mavorixafor, currently undergoing Phase 3 clinical trials for WHIM syndrome.
X4 Pharmaceuticals (Nasdaq: XFOR) will report its Q1 2021 financial results on May 6, 2021. A conference call is scheduled for 8:30 a.m. ET, where updates on recent business highlights will also be discussed. The company is focused on developing therapies targeting CXCR4 pathway dysfunction, with its lead candidate mavorixafor undergoing Phase 3 trials for WHIM syndrome and Phase 1b trials for Waldenström macroglobulinemia and severe congenital neutropenia. Investors can access the call via phone or live webcast on its website.
X4 Pharmaceuticals (Nasdaq: XFOR) is set to present a corporate overview at the 20th Annual Needham Virtual Healthcare Conference on April 12, 2021, at 2:15 PM ET. The presentation will focus on their innovative therapies targeting the CXCR4 pathway, particularly the lead candidate, mavorixafor, a unique treatment aimed at rare diseases. A live webcast will be available on the company's website, with an archive accessible for 90 days following the event.
X4 Pharmaceuticals (Nasdaq: XFOR) announced on March 31, 2021, that its Board's Compensation Committee granted inducement awards under the 2019 Inducement Equity Incentive Plan. The awards consist of options to purchase a total of 58,000 shares at an exercise price of $8.61 per share, matching the stock's closing price on the grant date. These options will vest over four years, helping attract new employees to the company. X4 focuses on developing therapies targeting diseases related to the CXCR4 pathway, with its lead candidate being mavorixafor.
X4 Pharmaceuticals (Nasdaq: XFOR) announced a private investment in public equity (PIPE) financing, raising approximately $55 million by selling 6,321,837 shares of common stock at $8.70 per share. New and existing institutional investors participated, including Bain Capital Life Sciences and Abingworth. The funds will support the clinical development of X4's lead candidate, mavorixafor, aimed at treating rare diseases. Following the transaction, X4 intends to file a registration statement with the SEC for resale of shares.
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