Poster #1654 highlights clinically meaningful and durable increases in circulating neutrophils observed with mavorixafor treatment +/- G-CSF over 6-month study Poster #1669 highlights findings that investigators were willing and able to reduce injectable G-CSF while maintaining ANC levels in the normal range Data continue to strengthen confidence in potential success of ongoing global, pivotal Phase 3 trial evaluating mavorixafor in primary chronic neutropenia BOSTON, June 16, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced the presentation of positive data from its completed Phase 2 trial of mavorixafor in the treatment of certain chronic neutropenic (CN) conditions at the 30th Annual Congress of the European Hematology Association (EHA) in Milan, Italy. “We are very proud to have sponsored this trial – the first of its kind – evaluating an oral agent for the chronic treatment of certain primary chronic neutropenic disorders,” said Christophe Arbet-Engels, M.D.
Meaningful and durable increases in functional neutrophils observed over 6-month trial with oral once-daily mavorixafor
4WARD Phase 3 chronic neutropenia trial in full swing; full enrollment on track for 3Q or 4Q 2025 and top-line data in 2H 2026
5 May 2025 Date | | - Cons. EPS | 0.04 EPS |
19 Mar 2025 Date | | - Cons. EPS | - EPS |
13 Nov 2024 Date | | - Cons. EPS | - EPS |
7 Nov 2024 Date | | - Cons. EPS | - EPS |
8 Aug 2024 Date | | 0.01 Cons. EPS | 0.45 EPS |
5 May 2025 Date | | - Cons. EPS | 0.04 EPS |
19 Mar 2025 Date | | - Cons. EPS | - EPS |
13 Nov 2024 Date | | - Cons. EPS | - EPS |
7 Nov 2024 Date | | - Cons. EPS | - EPS |
8 Aug 2024 Date | | 0.01 Cons. EPS | 0.45 EPS |
Biotechnology Industry | Healthcare Sector | Dr. Paula Ragan Ph.D. CEO | XSTU Exchange | US98420X1037 ISIN |
US Country | 143 Employees | - Last Dividend | 14 Mar 2019 Last Split | 16 Nov 2017 IPO Date |
X4 Pharmaceuticals, Inc. is a biopharmaceutical company engaged in late-stage clinical development, primarily focusing on crafting innovative therapeutics for the treatment of rare diseases. Positioned at the forefront of biomedical research, the company leverages its expertise in the domain of small molecule inhibitors, with a specific concentration on targeting the C-X-C chemokine receptor type 4 (CXCR4). Operating from its headquarters in Boston, Massachusetts, X4 Pharmaceuticals strives to address the unmet medical needs of patients suffering from complex conditions that are often overlooked in the broader pharmaceutical landscape. Through a steadfast commitment to scientific excellence and patient-centric research, the company endeavors to break new ground in the field of rare disease treatment.
1. Mavorixafor (Lead Product Candidate)
Mavorixafor stands at the forefront of X4 Pharmaceuticals' pipeline as a leading product candidate. It is a novel small molecule inhibitor targeting the chemokine receptor CXCR4. Currently in a Phase III clinical trial, mavorixafor is being evaluated for its efficacy in treating patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome (WHIM). Additionally, it is undergoing a Phase Ib clinical trial aimed at addressing chronic neutropenia and Waldenström's macroglobulinemia. This diverse application spectrum underscores mavorixafor's potential in transforming the therapeutic landscape for patients afflicted by these rare conditions.
2. X4P-002 (Research Program)
X4P-002 represents another critical component of X4 Pharmaceuticals’ product pipeline, developed as a CXCR4 antagonist for the potential treatment of brain cancers. By targeting specific pathways associated with tumor growth and spread, X4P-002 embodies the company's dedication to expanding the current treatment paradigms for cancer patients, especially those battling aggressive and difficult-to-treat brain malignancies.
3. X4P-003 (Research Program)
Further extending its research into the therapeutic potential of CXCR4 antagonism, X4 Pharmaceuticals is developing X4P-003 for treating a range of CXCR4 disorders and primary immunodeficiencies. This program emphasizes the company's commitment to harnessing its platform’s full potential in addressing various diseases tied to the CXCR4 receptor, promising new hope for patients with limited treatment options.
In addition to its robust internal pipeline, X4 Pharmaceuticals has established a strategic license agreement with Abbisko Therapeutics Co., Ltd. This partnership facilitates the development, manufacture, and commercialization of mavorixafor in combination with checkpoint inhibitors or other therapeutic agents across oncology indications, further amplifying the impact of X4 Pharmaceuticals’ innovative approach to drug development.