BOSTON, August 6, 2026

X4 Pharmaceuticals reported second quarter 2026 financial results while highlighting continued progress in the global Phase 3 4WARD clinical trial evaluating mavorixafor for chronic neutropenia. During the quarter, the company strengthened clinical operations by transitioning management of the pivotal study to a leading oncology Clinical Research Organization (CRO), supporting enrollment across more than 110 active trial sites worldwide. X4 also achieved a major regulatory milestone with the European Commission’s approval of XOLREMDI® (mavorixafor) for the treatment of WHIM syndrome, marking the first and only authorized therapy for the rare immunodeficiency disorder in the European Union. The company ended the quarter with $208 million in cash, providing an expected operating runway into 2029.

Phase 3 4WARD Trial Advances Toward Enrollment Completion

X4 continued expanding enrollment in the global Phase 3 4WARD trial, which is evaluating oral once-daily mavorixafor, with or without G-CSF, in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia. Management noted that the company’s restructuring of clinical operations and engagement of a premier oncology CRO have strengthened study execution and positioned the trial for successful enrollment completion. X4 expects to meet with the U.S. Food and Drug Administration (FDA) regarding potential sample size adjustments and plans to provide an update on both the regulatory discussions and enrollment progress by the end of the third quarter of 2026. The pivotal 52-week, randomized, placebo-controlled study is designed to enroll 176 patients aged 12 years and older, with primary endpoints focused on annualized infection rate reduction and absolute neutrophil count (ANC) response.

European Approval Expands XOLREMDI® Commercial Opportunity

During the quarter, XOLREMDI® (mavorixafor) received European Commission marketing authorization for the treatment of WHIM syndrome, following a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). Commercialization in Europe will be led by Norgine, with marketing authorization officially transferred to the partner during July 2026. Under the existing licensing agreement, X4 Pharmaceuticals remains eligible to receive up to €221 million in additional regulatory and commercial milestone payments, along with escalating double-digit royalties on future net sales across the licensed territories. The approval further expands the commercial potential of mavorixafor while supporting broader development across rare hematologic disorders.

Advertisement

cGxPJobs Banner

Financial Position Supports Long-Term Clinical Development

As of June 30, 2026, X4 reported $208 million in cash, providing sufficient resources to support operations into 2029. Second-quarter revenue totaled $8.8 million, including $2.4 million in U.S. XOLREMDI® product sales and $6.4 million in license revenue related to the company’s partnership with Norgine. Research and development expenses declined to $15.1 million, primarily reflecting workforce reductions following the company’s 2025 restructuring, partially offset by increased clinical trial costs associated with the 4WARD study. General and administrative expenses also declined to $8.5 million, while net loss improved to $16.2 million, or $0.13 per share, compared with $25.7 million, or $3.47 per share, during the same period in 2025. Supported by a strong balance sheet and continued execution of its clinical strategy, X4 remains focused on advancing mavorixafor across multiple rare hematology indications.

Source:X4 Pharmaceuticalspress release