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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 has appointed Diego Cadavid, M.D. as its new Chief Medical Officer. Dr. Cadavid will lead the strategy and execution of the company's clinical development programs. His expertise in advancing therapies for rare and immunological diseases is expected to positively influence ongoing programs, including the Phase 3 trial of mavorixafor for WHIM syndrome. With over 22 years of experience, including roles at Fulcrum Therapeutics and Biogen, Dr. Cadavid aims to leverage his skills to further develop mavorixafor and other preclinical candidates.
X4 Pharmaceuticals (Nasdaq: XFOR) announced its lead asset, mavorixafor, has received Rare Pediatric Disease (RPD) Designation from the FDA for treating WHIM syndrome, a rare primary immunodeficiency disease. This designation highlights the significant unmet medical need for effective therapies for WHIM patients, especially children who face serious infections. Mavorixafor is under investigation in a global Phase 3 clinical trial, 4WHIM, with anticipated results in 2022, aiming to provide a novel treatment option for over 3,500 diagnosed undiagnosed patients in the U.S.
X4 Pharmaceuticals (Nasdaq: XFOR) announced on November 30, 2020, that its Compensation Committee granted an inducement award to new employees under the 2019 Inducement Equity Incentive Plan. This includes options to purchase 125,950 shares of common stock at $6.60 each, the closing price on that day. Options will vest over four years, with 25% vesting after the first year. X4 focuses on therapies targeting CXCR4 pathway dysfunction, with its lead candidate mavorixafor undergoing clinical trials for various conditions, including WHIM syndrome.
X4 Pharmaceuticals (Nasdaq: XFOR) announced its participation in the Stifel 2020 Virtual Healthcare Conference on November 16, 2020, at 8:40 AM ET. This event will feature a fireside chat and one-on-one investor meetings. A live webcast of the chat will be available on X4's investor website and archived for 90 days. X4 specializes in developing therapies targeting the CXCR4 pathway, including its lead candidate, mavorixafor, currently in Phase 3 trials for treating WHIM syndrome and other rare diseases.
X4 Pharmaceuticals (XFOR) reported Q3 2020 financial results, revealing a net loss of $17.4 million, slightly improved from $17.7 million in Q3 2019. The company highlighted significant Phase 2 data for mavorixafor in WHIM syndrome published in 'Blood' and received FDA Fast Track Designation for the drug. The leadership team was strengthened with the appointment of Art Taveras as CSO and Alison Lawton to the Board of Directors. As of September 30, 2020, cash and equivalents stood at $90.7 million, which is expected to fund operations into early 2022.
X4 Pharmaceuticals (Nasdaq: XFOR) announced on October 30, 2020, that its Compensation Committee granted an inducement award under the 2019 Inducement Equity Incentive Plan to attract new employees. This grant consists of options to purchase 9,600 shares at an exercise price of $5.74 each, equal to the closing stock price on that date. The options will vest over four years, with 25% vesting after the first year. X4 focuses on developing therapies targeting CXCR4 dysfunction, with its lead candidate, mavorixafor, currently in Phase 3 trials for WHIM syndrome.
X4 Pharmaceuticals (Nasdaq: XFOR) has appointed Art Taveras, Ph.D., as its new Chief Scientific Officer. Dr. Taveras will lead research and non-clinical development for X4's pipeline, particularly the lead product candidate, mavorixafor, an oral therapy for rare genetic diseases. Dr. Taveras brings over 30 years of experience in small molecule drug discovery and has a strong background in chemokine-related chemistries. His leadership is expected to enhance X4's clinical programs and broaden its pipeline.
X4 Pharmaceuticals (Nasdaq: XFOR) will announce its third-quarter financial results on November 5, 2020. The company will provide updates on its business highlights during a conference call at 8:30 a.m. ET, accessible through U.S. and international dial-ins. X4 focuses on developing therapies targeting the CXCR4 pathway, with its lead candidate mavorixafor undergoing Phase 3 trials for WHIM syndrome and Phase 1b trials for other conditions. Interested parties can access the live webcast on the company’s investor relations page.
X4 Pharmaceuticals (Nasdaq: XFOR) announced that the FDA granted Fast Track Designation to its lead asset, mavorixafor, for treating WHIM syndrome, a rare genetic immunodeficiency disease. Mavorixafor is a first-in-class oral small molecule antagonist of the CXCR4 receptor, currently in a Phase 3 clinical trial. The Fast Track status will enable more frequent FDA meetings to discuss mavorixafor's development. Previous recognitions for mavorixafor include Breakthrough Therapy Designation and Orphan Drug status.
X4 Pharmaceuticals (Nasdaq: XFOR) announced the issuance of inducement equity awards to new employees under its 2019 Inducement Plan on September 30, 2020. These awards comprise options to purchase 30,500 shares of common stock at an exercise price of $6.77 per share, reflecting the closing price on that date. The options, which have a ten-year term, will vest over four years, contingent on the employees' ongoing employment. X4 develops therapies targeting the CXCR4 pathway, focusing on rare diseases, with its lead candidate, mavorixafor, currently in Phase 3 clinical trials.
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