XFOR

EX-99.1

2 ex-991pressreleasenovember.htm

EX-99.1

Document

Exhibit 99.1

X4 Pharmaceuticals Reports Third Quarter 2025 Financial Results and Provides Corporate Update

Company’s strategic focus and highest priority is now the advancement of the 4WARD Phase 3 chronic neutropenia trial

Previously announced workforce reductions resulted in $13M annualized cost savings

Successful completion of two financial transactions totaling $240.3M; cash runway now extends to the end of 2028

BOSTON, November 5, 2025 (GLOBAL NEWS WIRE) – X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare hematology diseases, today reported financial results for the third quarter ended September 30, 2025 and provided a corporate update.

“The third quarter of 2025 was a time of corporate restructuring at X4 with the start of a new leadership team and a renewed focus on chronic neutropenia,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “With a strengthened financial position through two successful financings totaling $240.3 million, our primary focus is now on the completion of the 4WARD Phase 3 pivotal trial of mavorixafor in patients with moderate and severe chronic neutropenia, which has a potential addressable market of 15,000 patients in the US. With a cash runway to the end of 2028, we are now positioned to unlock mavorixafor’s full potential and to establish X4 as a world-class rare hematology company.”

Recent Accomplishments and Updates

•Since early August, the Company has initiated a number of measures to restructure its operations:

◦A shift in the primary focus of the Company to the successful completion of the 4WARD Phase 3 pivotal trial of mavorixafor in patients with moderate and severe chronic neutropenia.

◦A deprioritization of the commercialization of mavorixafor (XOLREMDI) for patients with WHIM syndrome, while maintaining patient access.

◦A 50% reduction in the workforce (expected to generate approximately $13 million in annualized cost savings) with continued cost cutting measures.

◦An increase in the enrollment target for the pivotal Phase 3 4WARD study to 176 patients with enrollment now expected to be completed in third quarter of 2026.

◦The promotion of John Volpone to the role of Chief Operating Officer, in addition to his responsibilities as President.

◦Dr. Adam Craig expanded his role to include oversight of clinical development activities.

•Since August, the Company raised $240.3 million in gross proceeds from two successful financings: the closing of a $155.3 million underwritten public offering and an $85 million upsized private placement.

•With a strengthened cash runway to the end of 2028, X4 is now expected to be able to complete the 4WARD trial, file a potential sNDA for the chronic neutropenia indication, and, if successful, launch mavorixafor in this new indication by the end of 2028.

Third Quarter and Recent Financial Results

Net product sales of $1.6 million and $4.3 million for the three and nine months ended September 30, 2025, respectively, were entirely attributable to XOLREMDI product sales in the United States. Net product sales were $0.6 million and $1.1 million for the three and nine months ended September 30, 2024, respectively. License and other revenue of $0.2 million and $28.3 million for the three and nine months ended September 30, 2025, respectively, were entirely attributable to the Company's Norgine out-licensing agreement. Operating loss was $27.5 million and $34.5 million for the three months ended September 30, 2025 and 2024, respectively, and $63.2 million and $0.7 million for the nine months ended September 30, 2025 and 2024, respectively. The decrease in operating loss between the three-month periods ended September 30, 2025 and 2024 was primarily attributable to the impact of our 2025 Strategic Restructuring activities. Operating expenses for the nine months ended September 30, 2024 are net of a gain of $105.0

million realized on the sale of a priority review voucher. Exclusive of this gain, the decrease in operating loss between the nine-month periods ended September 30, 2025 and 2024 was primarily attributable to the impact of the Company's strategic restructuring activities undertaken in 2025.

Net loss for the three months ended September 30, 2025 was $29.8 million, or $0.69 for basic and diluted loss per share, compared to net loss of $36.7 million, or $5.48 for basic and diluted loss per share, for the same period in 2024. Net loss for the nine months ended September 30, 2025 was $55.3 million, or $2.87 for basic and diluted loss per share, compared to net income of $2.4 million, or $0.35 for basic and diluted income per share, for the same period in 2024.

Cash, cash equivalents and short-term investments totaled $122.2 million for the period ended September 30, 2025. On October 27, 2025, the Company completed a public offering with net proceeds of $145.6 million, which management believes w

EX-99.1

2 a991earningsreleasedatedma.htm

EX-99.1

Document

Exhibit 99.1

X4 Pharmaceuticals Reports First Quarter 2025 Financial Results and Provides Corporate Updates

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

Additional analyses of mavorixafor clinical trial data increase confidence in successful outcome of 4WARD trial

XOLREMDI® net U.S. revenues $3.5 million since May 2024 launch

Conference call and webcast today at 8:30 am ET

BOSTON, Mass., May 1, 2025 – X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the first quarter ended March 31, 2025, and highlighted key recent events and expected upcoming milestones.

“The first quarter of 2025 was an extremely productive and value-adding period for X4,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “Not only did we make significant progress in activating sites and enrolling participants in our ongoing mavorixafor Phase 3 trial in chronic neutropenia, but we also continued to support U.S. commercialization of XOLREMDI (mavorixafor) in WHIM syndrome, while also significantly expanding our global potential following acceptance of our EU regulatory submission in WHIM and the announcement of two international commercialization partnerships. We look forward to continuing to deliver on our milestones in the coming year toward our goal of maximizing the potential of mavorixafor to benefit patients with rare immunodeficiencies.”

Key First-Quarter 2025 and Recent Corporate Highlights

Advancing Mavorixafor in Chronic Neutropenia (CN)

•4WARD Trial Updates. X4 continues to enroll participants in its ongoing global, pivotal Phase 3 clinical trial (NCT06056297), evaluating oral, once-daily mavorixafor in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections.

◦As previously reported, an amendment to the 4WARD protocol has been implemented, focusing enrollment on those with the highest unmet needs and refining the definition of ANC response, making it consistent across all trial participants. Overall, the trial seeks to demonstrate statistically significant increases in ANC response and corresponding decreases in annualized infection rate between those on mavorixafor versus placebo.

◦To date, the demographics of the enrolled population are balanced and representative of the targeted commercial CN patient populations; baseline absolute neutrophil counts (ANC) and historical infection rates are consistent with this high unmet need population.

◦X4 continues to expect full enrollment in the trial in the third or fourth quarter of 2025, and disclosure of top-line data in the second half of 2026.

•Further Data Analyses Continue to Increase Confidence in Potential 4WARD Success. X4 recently applied the 4WARD trial ANC response criteria to individual participant results in the completed mavorixafor Phase 3 4WHIM trial and the Phase 2 CN trial to further assess the likelihood of 4WARD trial success. The company believes that the results of these analyses (details of which are available in the company’s updated investor presentation) further support the potential of mavorixafor to not only elevate ANC in the 4WARD trial participants, but that these ANC elevations should correspond with significant reductions in annualized infection rates in the mavorixafor-treated trial population.

•Mavorixafor Patent Update: In March 2025, X4 received a Notice of Allowance from the U.S. Patent and Trademark Office on its application 17/941,509, which claims include the use of mavorixafor in treating severe chronic, idiopathic, and autoimmune neutropenia in patients without a CXCR4 genetic variation. The patent is expected to expire in the U.S. in March of 2041; similar patent applications are pending in Europe, China, Japan, and Canada.

Commercializing XOLREMDI® (mavorixafor) in WHIM Syndrome, a Rare Primary Immunodeficiency

•Revenue Update: X4 has now generated $3.5 million in U.S. sales of XOLREMDI® (mavorixafor) from its mid-May 2024 launch through March 2025. The company also disclosed that newly identified patients on treatment are increasing in share of the overall current population on XOLREMDI, demonstrating the positive impact of ongoing WHIM education and awareness.

•Upcoming WHIM Clinical and Survey Data Presentations: Published abstracts accepted for poster presentation at the upcoming meeting of the Clinical Immunology Society (CIS) summarize the following results:

◦Phase 3 Open Label Extension (OLE) Wart Data: Two-year data from the OLE phase of the 4WHIM Phase 3 clinical trial evaluating once-daily oral mavorixafor in people with WHIM syndrome revealed marked clinical improvement in wart severity as assessed by Clinical Global Impression of Severity across 70 defined

EX-99.1

2 a991earningsreleasedatedma.htm

EX-99.1

Document

Exhibit 99.1

X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial

Results and Provides Corporate Update

Phase 3 4WARD trial in chronic neutropenia now activated at ~90% of targeted trial sites;

full enrollment expected in 3Q or 4Q 2025; top-line data anticipated in 2H 2026

4WARD trial protocol refined, increasing confidence in successful outcome

2024 XOLREMDI® net revenues $2.6 million since May launch; company expects ramp up in 2025 as targeted physician outreach increases patient finding and pull-through

Conference call and webcast today at 8:30 am ET

BOSTON, Mass., March 25, 2025 – X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the fourth quarter and full year ended December 31, 2024, and highlighted key 2024 and recent events and expected upcoming milestones.

“As expected, 2024 was a transformative year for the company and our momentum has continued into 2025,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “With the U.S. approval and launch of our first product, XOLREMDI® (mavorixafor) in WHIM syndrome, we are now a fully integrated company advancing our mission to serve those with rare immunodeficiencies and few treatment options. As we await word from the EU regulatory authority on the potential approval of mavorixafor for WHIM in that region, we’ve continued to expand our potential reach to the global WHIM community through our recently completed commercialization partnerships in the EU, Australia, New Zealand, and the Middle East and North Africa (MENA) territories.”

Dr. Ragan continued: “In addition, we continue to make meaningful progress in our efforts to develop mavorixafor for the larger immunodeficiency population with chronic neutropenia (CN). Following positive results from our Phase 2 trial of mavorixafor in certain CN populations, we are currently conducting a global, pivotal Phase 3 trial in CN. With a large number of sites now activated and global screening ongoing, we expect full trial enrollment in the third or fourth quarter of this year and top-line trial data in the second half of 2026.”

Dr. Ragan concluded: “Market research and patient testimonials continue to strengthen our belief that there remains significant untapped potential for XOLREMDI for the U.S. and possibly global WHIM patient populations. We are equally confident in mavorixafor’s future potential to address unmet needs in the global CN community.”

Key 2024 and Recent Corporate Highlights

Commercializing XOLREMDI® (mavorixafor) in WHIM Syndrome, a Rare Primary Immunodeficiency

•FDA Approval and Launch of XOLREMDI: In May 2024, X4 launched XOLREMDI® (mavorixafor) following approval by the U.S. Food and Drug Administration (FDA) for its use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. Concurrent with the approval of XOLREMDI, X4 received a priority review voucher (PRV), which it subsequently sold to another drug developer for $105 million.

•U.S. Launch Update: X4 is continuing to execute on its commercialization of XOLREMDI in the U.S., generating $2.6 million in sales from its mid-May launch through December 2024. During the year, the company advanced disease awareness by engaging with physicians and rare disease patient advocacy groups through a combination of in-person and targeted digital education campaigns. The company’s suite of patient services, including its X4Connect™ and nurse educator programs, continue to provide access and support for patients prescribed XOLREMDI.

•Clinical Data Publications and Presentations: Journal publications and presentations of clinical data results at top medical meetings, including those of the American Society of Hematology (ASH), the American Academy of Allergy, Asthma, and Immunology (AAAAI), and the Clinical Immunology Society (CIS), have provided further visibility on the XOLRMEDI approval.

•Maximizing the Global Opportunity for Mavorixafor in WHIM Syndrome:

MAA Acceptance. In January 2025, X4 announced that its submitted Marketing Authorization Application (MAA) for mavorixafor in the treatment of WHIM syndrome was validated for review by the European Medicines Agency (EMA), meeting an important corporate milestone. Given a typical 12- to 15-month review process, the company believes approval to be possible in the first half of 2026.

EU/ANZ Partnership. Also in January 2025, the company announced that it had entered into an exclusive licensing and supply agreement under which Norgine Pharma UK will commercialize mavorixafor in Europe, Australia, and New Zealand following any regulatory approvals in those territories. X4 received €28.5 million as an upfront payment and is eligible to r