Mavorixafor

- Global 4WARD Phase 3 Chronic Neutropenia Trial On Track to Complete Enrollment by the end of Q3 2026 - - European Commission Approval of XOLREMDI® (Mavorixafor) Provides the First and Only Authorized Treatment for Patients with WHIM Syndrome in the European Union - - Balance Sheet Provides Cash Runway through 2028 - BOSTON, May 06, 2026 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company focused on improving the lives of people with rare hematology diseases, today reported financial results for the first quarter ended March 31, 2026 and provided a corporate update. “Our top priority remains the execution and full enrollment of the pivotal 4WARD Phase 3 trial of mavorixafor in chronic neutropenia,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “We remain on track to complete enrollment by the end of the third quarter of this year. In addition, we are pleased that the European Commission granted marketing authorization for XOLREMDI® (mavorixafor) for the treatment of patients with WHIM syndrome in the European Union, reinforcing the broader potential of our program to advance therapies for individuals affected by rare hematologic disorders. We look forward to a smooth transfer of the marketing authorization application to our European partner, Norgine, later this year as they proceed with commercialization plans throughout Europe.” Recent Accomplishments and Updates Continued to advance enrollment in the global 4WARD Phase 3 trial of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia, supported by several targeted initiatives: Expanded to over 110 active clinical trial sites worldwide, including more than 20 in the U.S., enhancing enrollment and regulatory alignment; Increased global Medical Affairs engagement including the implementation of a dedicated patient referral pathway for physicians; and, Leveraging data-driven approaches including database mining to identify potential participants. The European Commission granted marketing authorization for XOLREMDI® (mavorixafor) for the treatment of patients with WHIM syndrome in the European Union (EU). The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). European commercialization will be led by X4 Pharmaceuticals’ partner, Norgine. Under the terms of the license and supply agreement, X4 will receive up to €226 million contingent upon the achievement of certain regulatory and commercial milestones, in addition to escalating double-digit royalties of up to the mid-twenties on any future net sales in the licensed territories. First Quarter Financial Results Cash Position: As of March 31, 2026, the Company’s cash position was $233.7 million. Revenue: Revenue was $2.7 million and $28.8 million for the three months ended March 31, 2026 and 2025, respectively. Net product sales in both periods were primarily attributable to XOLREMDI product sales in the United States. Revenue for the three months ended March 31, 2025 included $27.9 million of license revenue attributable to the Norgine license and supply agreement. Research and Development (R&D) expenses: R&D Expenses were $15.5 million for the three months ended March 31, 2026 as compared to $18.5 million for the three months ended March 31, 2025. The decrease is primarily attributable to decreased headcount following the 2025 strategic restructuring. General and Administrative (G&A) expenses : G&A Expenses were $7.0 million for the three months ended March 31, 2026 as compared to $15.0 million for the three months ended March 31, 2025. The decrease is primarily attributable to decreased headcount following the 2025 strategic restructuring. Net (Loss) Income: Net loss for the three months ended March 31, 2026 was $(20.2) million, or $(0.16) per share, as compared to net income for the three months ended March 31, 2025 of $0.3 million or $0.04 per share. The difference was due to the Norgine license revenue recognized in the first quarter of 2025, partially offset by a significant reduction in operating expenses. About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a primary, rare blood condition characterized by abnormally low levels of circulating neutrophils in the blood lasting more than three months, persistently or intermittently. As a result, people with chronic neutropenia are at an increased risk of serious and life-threatening infections and reduced quality of life. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Mavorixafor is a small molecule delivered in a capsule for oral dosing as a selective antagonist of the chemokine receptor, CXCR4. Down-regulation of the CXCR4 receptor by mavorixafor has been shown to mobilize functional neutrophils from the bone marrow into the peripheral bloodstream across multiple disease states. The level of circulating neutrophils is typically determined by the absolute neutrophil count (ANC) obtained from a blood draw. About the 4WARD Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 176 patients aged 12 years and older with confirmed trough absolute neutrophil count (ANC) levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoints of the study are the reduction in annualized infection rate and positive ANC response. For more information, visit clinicaltrials.gov (NCT06056297). About X4 Pharmaceuticals X4 Pharmaceuticals is a company focused on improving the lives of people with rare hematology diseases by developing and commercializing innovative therapies in areas with significant unmet needs. Leveraging expertise in diseases of the immune system and CXCR4 biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is commercially available in the U.S. as XOLREMDI® in its first indication. The Company is currently conducting a global, pivotal Phase 3 clinical trial (4WARD) evaluating mavorixafor in chronic neutropenic disorders. The U.S. FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia. X4 is headquartered in Boston, Massachusetts. For more information, please visit . X4 Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the Company’s ability to obtain and maintain regulatory approval for its product candidates, plans for the commercialization of XOLREMDI in the European Union by Norgine, the potential achievement of milestones and receipt of royalties under the Company’s licensing and supply agreement with Norgine, the expected design and enrollment of the Company’s clinical trials, including expected timing for full enrollment in 4WARD, the sufficiency of the Company’s cash resources and its expected cash runway, and future plans for the Company. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that even if approved, mavorixafor may not ultimately be commercially successful; the Company is unable to initiate and complete its clinical trials, including the 4WARD trial; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on Form 10-K, as well as in other filings X4 makes with the Securities and Exchange Commission, including its Quarterly Reports on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. X4 Investor Contact: Remy Bernarda Jenny Kobin IR Advisory Solutions IR@X4pharma.com Source: X4 Pharmaceuticals, Inc. (Tables Follow) X4 PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except per share amounts) (unaudited) Three Months Ended March 31, 2026 2025 License and other revenue $ 239 $ 27,865 Product revenue, net 2,469 942 Total revenue 2,708 28,807 Costs and operating expenses: Cost of revenue 598 4,716 Research and development 15,450 18,513 General and administrative 6,966 15,021 Total operating expense 23,014 38,250 Loss from operations (20,306 ) (9,443 ) Other income, net 65 9,759 (Loss) income before provision for income taxes (20,241 ) 316 Provision for income taxes — 34 Net (loss) income $ (20,241 ) $ 282 Net (loss) income per share: basic $ (0.16 ) $ 0.04 Weighted average shares outstanding: basic 126,289 6,840 Net (loss) income per share: diluted $ (0.16 ) $ 0.04 Weighted average shares outstanding: diluted 126,289 6,869 Balance Sheet Data (unaudited) (amounts in thousands) March 31, 2026 December 31, 2025 Cash and cash equivalents $ 216,908 $ 217,049 Accounts receivable 1,113 573 Marketable securities 16,812 35,949 Working capital 218,211 235,820 Total assets 270,281 290,461 Current portion of long-term debt — — Total stockholders’ equity 168,450 186,290

Norgine announced that the European Commission has granted marketing authorization for Xolremdi (mavorixafor) for WHIM syndrome, making it the first therapy authorized for this condition in the European Union. The authorization, which follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use, was granted under exceptional circumstances given the ultra-rare nature of the disease. Norgine will lead commercialization across Europe under a licensing agreement with X4 Pharmaceuticals, the US-based company that originated and holds intellectual property rights to the molecule. Xolremdi is indicated in patients aged 12 years and older for the treatment of WHIM syndrome — an acronym for warts, hypogammaglobulinemia, infections, and myelokathexis — to increase the number of circulating mature neutrophils and lymphocytes. The condition is caused by gain-of-function mutations in the CXCR4 receptor, which impairs the release of white blood cells from bone marrow into peripheral circulation, leaving affected individuals vulnerable to recurrent and severe infections. The EC authorization was granted under exceptional circumstances, a regulatory pathway applied when applicants cannot provide comprehensive efficacy and safety data due to the rarity of a condition; additional post-authorization data will be required. The authorization is supported by results from NCT03995108, a Phase III randomized, double-blind, placebo-controlled, 52-week multicenter study that enrolled 31 patients aged 12 years and older with confirmed WHIM syndrome. The trial evaluated the efficacy and safety of mavorixafor against placebo, with results published in Blood in 2024 by Badolato and colleagues. The study met its primary endpoint, demonstrating that mavorixafor increased the duration of time that absolute neutrophil and lymphocyte counts remained above defined thresholds compared with placebo. WHIM syndrome has until now been managed primarily through off-label use of filgrastim, a recombinant granulocyte colony-stimulating factor originally developed by Amgen that raises neutrophil counts but has no effect on lymphocytes, monocytes, or the wart burden associated with the condition. Plerixafor, a subcutaneously administered CXCR4 antagonist approved for stem cell mobilization, was evaluated in a Phase III crossover trial against G-CSF in WHIM syndrome but was not advanced to regulatory submission for this indication. Mavorixafor, as an oral once-daily CXCR4 antagonist that mobilizes both neutrophils and lymphocytes, occupies a distinct position as the only therapy carrying an approved label specifically for WHIM syndrome in either the US or the EU. X4 Pharmaceuticals received US FDA approval for Xolremdi in 2024. Beyond WHIM syndrome, mavorixafor is under active Phase III investigation in congenital and acquired chronic neutropenic disorders, with trial NCT06056297 currently recruiting. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website