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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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BOSTON, Sept. 01, 2021 - X4 Pharmaceuticals (Nasdaq: XFOR) announced its participation in investor meetings at the Citi 16th Annual BioPharma Conference from September 8-10, 2021 and a fireside chat at the H.C. Wainwright 23rd Annual Global Investment Conference on September 13, 2021, at 7:00 AM ET. A live webcast of the fireside chat will be available on the company's investor section of their website for 90 days post-event. X4 specializes in therapies targeting diseases related to the CXCR4 pathway, with its lead candidate mavorixafor under evaluation in multiple clinical trials.
X4 Pharmaceuticals (Nasdaq: XFOR) will participate in a fireside chat at Canaccord Genuity’s 41st Annual Growth Conference on August 12, 2021, at 2:00 PM ET. The event will be accessible via a live webcast on the company's investor website, with an archive available for 90 days. X4 Pharmaceuticals focuses on developing therapies targeting the CXCR4 pathway, particularly for rare diseases. Their lead candidate, mavorixafor, is currently in various clinical trials, including a Phase 3 trial for WHIM syndrome.
On August 3, 2021, X4 Pharmaceuticals (XFOR) announced key milestones in its clinical trials for mavorixafor. Enrollment in the pivotal Phase 3 trial for WHIM syndrome surpassed the minimum requirement, with top-line data expected in Q4 2022. The Phase 1b trial for Waldenström’s macroglobulinemia has also met its minimum enrollment, with results anticipated in Q4 2021. Financially, the company reported a net loss of $19.6 million for Q2 2021, up from $15.1 million in Q2 2020, indicating rising research and development costs.
X4 Pharmaceuticals (Nasdaq: XFOR) will report its financial results for Q2 ended June 30, 2021, on August 3, 2021. The company will also provide updates on recent business highlights during a conference call scheduled at 8:30 a.m. ET on the same day. Investors can access the call by dialing (866) 721-7655 or internationally at (409) 216-0009, using conference ID 2236266. X4 is developing mavorixafor, a novel therapy targeting the CXCR4 pathway, currently being evaluated in multiple clinical trials for rare diseases and cancers.
X4 Pharmaceuticals (Nasdaq: XFOR) announced the issuance of inducement awards to new employees under the 2019 Inducement Equity Incentive Plan on June 30, 2021. The awards include options to purchase a total of 10,850 shares at an exercise price of $6.50, equal to the closing price on that date. The options will vest over four years, with 25% vesting after one year. X4 is focused on developing therapies targeting the CXCR4 pathway, including its lead candidate mavorixafor, currently in multiple clinical trials for rare diseases.
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.