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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) reported its third-quarter 2022 financial results, highlighting key advancements in its lead candidate, mavorixafor, after positive Phase 1b data in chronic neutropenic disorders. The company anticipates top-line results from the pivotal Phase 3 WHIM syndrome trial in Q4 2022, potentially leading to a U.S. regulatory submission in H2 2023. X4 ended Q3 2022 with $81.1 million in cash, supporting operations into Q3 2023. Despite a net loss of $21.6 million, X4 is focused on advancing mavorixafor as a new treatment option for underserved patients.
X4 Pharmaceuticals has appointed Mark Baldry as the new Chief Commercial Officer, effective November 3, 2022. Baldry brings over 30 years of experience in launching specialty pharmaceuticals and will oversee pre-commercial and product launch efforts for mavorixafor, X4's lead candidate for chronic neutropenic disorders, including WHIM syndrome. The company is preparing to reveal Phase 3 data from the mavorixafor clinical trial, which holds significant potential as a first line therapy. This leadership change may strengthen the company’s market position.
X4 Pharmaceuticals (Nasdaq: XFOR) will report its Q3 financial results on November 3, 2022. The company will also host a conference call at 8:30 a.m. ET to discuss key milestones, particularly the results from its Phase 3 trial of mavorixafor for WHIM syndrome. This investigational therapy, a small molecule antagonist of CXCR4, aims to improve white blood cell mobilization for various immune system diseases. X4 is focusing resources on chronic neutropenic disorders and has additional studies ongoing for mavorixafor. More details can be found on their investor relations website.
X4 Pharmaceuticals announced promising results from its Phase 1b trial of mavorixafor, targeting chronic neutropenia (CN). All 25 participants in the study responded positively, achieving significant increases in absolute neutrophil count (ANC). Notably, the treatment normalized ANC levels in 100% of the neutropenic subjects, suggesting mavorixafor could become the first oral therapy for CN disorders. The trial included idiopathic, cyclic, and congenital variants, with an estimated patient population of 50,000 in the U.S. Further studies are planned to confirm the long-term efficacy and safety of mavorixafor.
X4 Pharmaceuticals (Nasdaq: XFOR) will host a webcast on September 27, 2022, at 8:00 a.m. ET to discuss data from its Phase 1b trial of mavorixafor in patients with neutropenia. The event will include insights from patients and clinical experts about treatment needs. Management will present data on 25 fully enrolled patients and explore mavorixafor's potential beyond WHIM syndrome. A Q&A session will follow. The trial aims to assess the safety and tolerability of mavorixafor, a novel small-molecule therapy for immune system diseases.
X4 Pharmaceuticals (Nasdaq: XFOR) will present a corporate overview at the Canaccord Genuity 42nd Annual Growth Conference in Boston, MA, on August 11, 2022, at 3:30 PM ET. The company focuses on developing novel CXCR4-targeted small molecule therapeutics for immune system diseases. A live webcast will be available on their website, with an archive accessible for 90 days post-event. Mavorixafor, their lead candidate, is under clinical trials for various conditions, including WHIM syndrome and chronic neutropenia.
X4 Pharmaceuticals reported its Q2 2022 financial results, revealing a net loss of $21.2 million, an increase from $19.6 million in Q2 2021. Cash and cash equivalents stood at $48.7 million as of June 30, 2022, bolstered by a recent PIPE financing raising approximately $56 million. The company anticipates significant milestones with data from its Phase 1b trial in chronic neutropenia expected in late September 2022, and pivotal Phase 3 results in WHIM syndrome expected in Q4 2022. X4 is now focusing its resources on chronic neutropenic disorders while pursuing partnerships for its oncology programs.
X4 Pharmaceuticals (Nasdaq: XFOR) has announced it will report its financial results for Q2 2022 on August 4, 2022. The company will also provide updates on notable business developments during a conference call at 8:30 a.m. ET, accessible via phone and a live webcast. X4's lead candidate, mavorixafor, is in a global Phase 3 trial for treating WHIM syndrome, a primary immunodeficiency disease. Mavorixafor shows promise in various conditions, including chronic neutropenic disorders and specific cancers. More details can be found on their website.
X4 Pharmaceuticals (Nasdaq: XFOR) focuses on clinical candidate mavorixafor for chronic neutropenic disorders like WHIM syndrome. Key milestones include pivotal Phase 3 trial results and Phase 1b outcomes expected in 2022. A recent capital raise of $55 million, along with cost reductions, extends the company's cash runway into Q3 2023 and accelerates the mavorixafor NDA filing to early 2023. The company is streamlining resources and reducing operating expenses by approximately $25 million over two years, prioritizing mavorixafor while limiting oncology program advancements to potential partnerships.
X4 Pharmaceuticals (Nasdaq: XFOR) announced on June 30, 2022, that its Compensation Committee granted inducement awards to new employees under the 2019 Inducement Equity Incentive Plan. These options total 21,000 shares, with an exercise price of $0.96 per share, aligning with the closing price on that date. The options will vest over four years, and this issuance is in compliance with Nasdaq Listing Rule 5635(c)(4). X4 focuses on developing CXCR4-targeted small molecule therapies, including its lead candidate mavorixafor for immune system dysfunction.
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