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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 positive results from a Phase 2 study of mavorixafor, a potential treatment for WHIM syndrome, published in Blood. The study demonstrated that mavorixafor at 400 mg once daily improved white blood cell counts and significantly reduced infection rates and wart burden. The company aims to accelerate mavorixafor's development in a pivotal Phase 3 trial, anticipating results in 2022. These findings bolster the clinical strategy and validate the therapy's potential in a severely underserved patient population.
X4 Pharmaceuticals (Nasdaq: XFOR) announced on August 31, 2020, that its Compensation Committee issued inducement awards to new employees under the 2019 Inducement Plan. The awards consist of options to purchase 61,750 shares at an exercise price of $8.38, equal to the closing stock price on that date. These options have a ten-year term and will vest over four years. X4 Pharmaceuticals focuses on developing therapies for diseases linked to the CXCR4 pathway, with its leading candidate, mavorixafor, currently undergoing Phase 3 trials for WHIM syndrome.
X4 Pharmaceuticals (Nasdaq: XFOR) announced management will present and hold one-on-one meetings at the Virtual Canaccord Genuity 40th Annual Growth Conference on August 12, 2020, at 2:30 PM ET. A live webcast of the presentation will be accessible on the investor section of the X4 Pharmaceuticals website and archived for 90 days.
The firm specializes in therapies targeting CXCR4 pathway dysfunction, with its lead candidate, mavorixafor, being evaluated in multiple clinical trials for rare diseases and cancers.
X4 Pharmaceuticals, Inc. (Nasdaq: XFOR) announced an inducement award of options to purchase 10,700 shares of its common stock to new employees under its 2019 Inducement Equity Incentive Plan. The options, set at an exercise price of $7.83 per share, will vest over four years, encouraging retention. This award aligns with Nasdaq Listing Rule 5635(c)(4). X4 is focused on therapies targeting CXCR4 pathway dysfunction, with its lead candidate, mavorixafor, undergoing various clinical trials.
X4 Pharmaceuticals, Inc. (Nasdaq: XFOR) reported second-quarter 2020 financial results, revealing a net loss of $15.1 million, up from $13.4 million in Q2 2019. The company highlighted progress in its Phase 3 trial for WHIM syndrome, supported by positive Phase 2 data presented at EHA 2020, which indicated substantial disease prevalence estimates exceeding 3,500 cases in the U.S. Additionally, initial data from the Phase 1b trial in Waldenström’s macroglobulinemia is expected later this year. X4 holds $105.6 million in cash, expected to fund operations into early 2022.
X4 Pharmaceuticals (Nasdaq: XFOR) announced plans to report its financial results for Q2 2020 on July 30, 2020. The earnings call will provide insights into recent business highlights and will take place at 8:30 a.m. ET. The company focuses on treating diseases linked to CXCR4 pathway dysfunction, with its lead candidate, mavorixafor, undergoing multiple clinical trials, including a Phase 3 trial for WHIM syndrome. Interested parties can access the live webcast via the company's investor relations webpage.
X4 Pharmaceuticals presented encouraging data from its ongoing Phase 2 trial of mavorixafor for WHIM syndrome, demonstrating significant reductions in yearly infection rates and wart burden. The trial observed sustained efficacy and safety trends for up to 28.6 months, with the 400 mg daily dose showing effective results in improving clinical outcomes. The data supports the ongoing pivotal Phase 3 trial, with top-line results anticipated in 2022. The company estimates WHIM syndrome affects over 3,500 individuals in the U.S., highlighting a crucial unmet medical need.
X4 Pharmaceuticals (Nasdaq: XFOR) has promoted Renato Skerlj, Ph.D., to Chief Scientific Officer. Dr. Skerlj, a co-inventor of the lead drug candidate mavorixafor, joined X4 in 2019 and has significantly contributed to the company's research efforts. His leadership is crucial as the company advances mavorixafor through clinical trials for WHIM syndrome and genetically-defined blood cancers. X4 continues to focus on therapies targeting the CXCR4 pathway for rare diseases, with ongoing global Phase 3 trials for mavorixafor.
X4 Pharmaceuticals (Nasdaq: XFOR) announced it will host a conference call on June 12, 2020, to discuss clinical efficacy and safety data from the ongoing Phase 2 trial of mavorixafor for WHIM syndrome. The data will be shared at the 25th European Hematology Association Annual Congress from June 11-14, 2020. Mavorixafor, a first-in-class treatment, is currently evaluated in a global Phase 3 trial for WHIM syndrome and in additional Phase 1b trials for other conditions. The conference call can be accessed via phone or webcast.
X4 Pharmaceuticals (Nasdaq: XFOR) announced an inducement award for new employees on May 29, 2020, as part of its 2019 Inducement Equity Incentive Plan. A total of 55,200 options to purchase common stock at an exercise price of $8.52 per share were granted. These options vest over four years, with 25% vesting after the first year and the remainder vesting monthly thereafter, contingent on continued employment. X4 is known for developing therapies targeting CXCR4 pathway dysfunction, focusing on rare diseases, with its lead candidate, mavorixafor, currently in Phase 3 trials.