WHIM Syndrome

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, May 01, 2025 (GLOBE NEWSWIRE) -- 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 wart areas.First-Ever WHIM Infection Burden Survey Results: Of the 20 WHIM patient respondents, 60% of those <18 years and 73% of those ≥18 years reported experiencing at least one infection in the previous 3 months, with 25% requiring overnight hospitalization due to infection. The study concludes that “the frequency and severity of infections requiring medical care and hospitalization underscores the urgency to proactively treat patients with WHIM syndrome.” 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), enabling possible approval in the first half of 2026.Ex-US Partnerships. In the first quarter of 2025, the company announced that it had entered into two ex-U.S. partnerships for the commercialization of mavorixafor: the first, an exclusive licensing and supply agreement under which Norgine Pharma UK will commercialize mavorixafor in Europe, Australia, and New Zealand and the second, with taiba rare to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in Saudi Arabia, United Arab Emirates, Qatar, Oman, Kuwait, Bahrain, and Egypt following the necessary regulatory approvals in those countries and/or regions. Strategic RestructuringIn February 2025, X4 announced a strategic restructuring to sharpen focus and maximize the opportunity for mavorixafor in chronic neutropenia while also optimizing its U.S. promotion of XOLREMDI. X4 continues to expect that these efforts will decrease spending by $30-35 million annually. Reverse Stock SplitOn April 24, 2025, X4 announced a one-for-thirty reverse stock split of the company’s common stock. The reverse stock split took effect at 12:01 a.m. ET on April 28, 2025, and the company’s common stock began trading on a split-adjusted basis on The Nasdaq Capital Market as of the opening of trading the same day under a new CUSIP number (98420X202). The reverse split reduced the number of outstanding shares of the company’s Common Stock from approximately 173.6 million shares to approximately 5.8 million shares. First-Quarter 2025 Financial Results Cash position: X4 had $87.7 million in cash, cash equivalents, restricted cash, and marketable securities as of March 31, 2025. The company continues to believe it has sufficient funds to support operations into the first half of 2026.Revenue and Cost of Revenue: For the first quarter ended March 31, 2025, X4 reported net revenue of $28.8 million and cost of revenue of $4.7 million. Revenue in the quarter included license and other revenues of $27.9 million associated with the completed Norgine agreement and XOLREMDI product revenue of $0.9 million. Cost of revenue included $4.5 million of sublicense royalties associated with the Norgine agreement.Research and Development (R&D) Expenses were $18.5 million for the first quarter ended March 31, 2025, as compared to $19.9 million for the comparable period in 2024. R&D expenses included $0.3 million of certain non-cash expenses for the first quarter ended March 31, 2025.Selling, General, and Administrative (SG&A) Expenses were $15.0 million for the first quarter ended March 31, 2025, as compared to $17.4 million for the comparable period in 2024. SG&A expenses included $0.3 million of certain non-cash expenses for the first quarter ended March 31, 2025.Net Income (Loss): X4 reported net income of $0.3 million for the first quarter ended March 31, 2025, as compared to a net loss of $51.8 million for the comparable period in 2024. Net income included $0.5 million of stock-based compensation expenses for the first quarter ended March 31, 2025. Conference Call and Webcast X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights. The conference call can be accessed by dialing 1-800-343-4849 from the United States or 1-203-518-9848 internationally, followed by the conference ID: X4PHARMA. The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the conclusion of the call, a webcast replay will be available on the website. About WHIM SyndromeWHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 receptor dysfunction that results in impaired mobilization of white blood cells from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis, although only a minority of patients experience all four manifestations in the acronym. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections. About XOLREMDI® (mavorixafor)XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U.S. as a once-daily oral treatment for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. XOLREMDI is the only treatment specifically approved for patients with WHIM syndrome in the U.S. About Chronic Neutropenia and MavorixaforChronic neutropenia is a primary, rare blood condition lasting more than three months, persistently or intermittently, and characterized by low levels of circulating neutrophils and increased risk of infections and reduced quality of life due to abnormally low levels of neutrophils circulating in the blood. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize functional neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC). About the 4WARD Clinical TrialThe 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people 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 150 participants with confirmed trough 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 endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response. About X4 PharmaceuticalsX4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging expertise in CXCR4 and immune system biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is currently being marketed in the U.S. as XOLREMDI® in its first indication. The company is also evaluating additional uses of mavorixafor and is conducting a global, pivotal Phase 3 clinical trial (4WARD) in people with certain chronic neutropenic disorders. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. 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 initiation, timing, progress, and results of our current and future preclinical studies and clinical trial; the timing and period during which the results of the trials will become available and reported, as well as our research and development programs; market opportunities for X4’s product candidates, including potential benefits from partnerships; expectations regarding the commercial potential of mavorixafor and ongoing engagement and feedback from regulatory authorities; and the sufficiency of X4’s cash resources and pro forma cash resources, including the potential impact of the 2025 strategic restructuring, and expectations regarding X4’s cash runway. 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: X4’s restructuring activities may be more costly or time-consuming than expected or may not achieve their intended results and savings; X4’s future financial performance and position may not improve resulting in difficulties in implementing X4’s business strategy, and plans and objectives for future operations; the expected decrease in annual spending could negatively impact X4’s commercial plans and strategy for mavorixafor; the expected sufficiency of X4’s existing cash resources and runway may not be accurate resulting in the need for additional financing sooner than anticipated or unexpected liquidity constraints; the internal and external costs required for X4’s ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected, which may cause the company to use cash more quickly than expected or to change or curtail some of X4’s plans or both; the expected availability, content, and timing of clinical data from X4’s ongoing clinical trials of mavorixafor may be delayed or unavailable, including the ongoing Phase 3 clinical trial; trials, studies and research programs may be delayed and may not have satisfactory outcomes, additionally earlier trials and studies may not be predictive of later trials and studies; the design and rate of enrollment for current clinical trials may not enable successful completion of the trial(s); the commercial opportunity for mavorixafor may be smaller than anticipated; X4 may be unable to obtain and maintain regulatory approvals; adverse safety effects may arise from the testing or use of X4’s product and product candidates; the need to align with collaborators may hamper or delay development and commercialization efforts or increase costs; business may be adversely affected and costs may increase if any of the company’s key collaborators fails to perform its obligations or terminates the collaboration; X4’s may be unable to advance and commercialize and increase sales in mavorixafor to treat chronic neutropenia or to optimize the U.S. promotion of XOLREMDI® (mavorixafor), approved for the treatment of WHIM; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on X4’s Form 10-K , as well as in other filings X4 makes with the Securities and Exchange Commission, including its quarterly report 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. (Tables Follow) X4 PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in thousands, except per share amounts)(unaudited) Three Months Ended March 31, 2025 2024 License and other revenue $27,865 $— Product revenue, net 942 — Total revenue 28,807 — Costs and operating expenses: Cost of revenue 4,716 — Research and development 18,513 19,854 Selling, general and administrative 15,021 17,435 Total operating expenses 38,250 37,289 Loss from operations (9,443) (37,289)Other income (expense), net 9,759 (14,458)Income (loss) before provision for income taxes 316 (51,747)Provision for income taxes 34 19 Net income (loss) $282 $(51,766)Net income (loss) per share- basic $0.04 $(7.77)Weighted average common shares outstanding- basic 6,840 6,666 Net income (loss) per share- diluted $0.04 $(7.77)Weighted average common shares outstanding- diluted 6,869 6,666 X4 PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(in thousands)(unaudited) March 31,2025 December 31,2024Current assets: Cash and cash equivalents $40,257 $55,699 Accounts receivable 509 1,070 Marketable securities 46,694 46,361 Research and development incentive receivable 665 640 Inventory 3,089 2,817 Prepaid expenses and other current assets 4,483 5,588 Total current assets 95,697 112,175 Property and equipment, net 487 776 Goodwill 17,351 17,351 Intangible asset, net 9,813 10,000 Right-of-use assets 3,652 4,065 Other assets 3,013 2,080 Total assets $130,013 $146,447 Current liabilities: Accounts payable $11,115 $8,621 Accrued expenses 12,611 23,005 Deferred revenue 855 — Current portion of lease liability 1,303 1,251 Total current liabilities 25,884 32,877 Long-term debt, including accretion, net of discount 75,629 75,425 Lease liabilities 1,109 1,410 Warrant liability 2,925 13,755 Deferred revenue and other liabilities 1,522 831 Total liabilities 107,069 124,298 Total stockholders' equity 22,944 22,149 Total liabilities and stockholders' equity $130,013 $146,447 X4 Investor Contact:X4 Media Contact:Daniel FerryLifeSci Advisorsdaniel@lifesciadvisors.com(617) 430-7576Rhiannon JeselonisTen Bridge Communicationsrhiannon@tenbridgecommunications.com

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 , May 01, 2025 (GLOBE NEWSWIRE) -- 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 . 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. 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 wart areas. First-Ever WHIM Infection Burden Survey Results: Of the 20 WHIM patient respondents, 60% of those <18 years and 73% of those ≥18 years reported experiencing at least one infection in the previous 3 months, with 25% requiring overnight hospitalization due to infection. The study concludes that “the frequency and severity of infections requiring medical care and hospitalization underscores the urgency to proactively treat patients with WHIM syndrome.” 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), enabling possible approval in the first half of 2026. Ex-US Partnerships. In the first quarter of 2025, the company announced that it had entered into two ex- U.S. partnerships for the commercialization of mavorixafor: the first, an exclusive licensing and supply agreement under which Norgine Pharma UK will commercialize mavorixafor in Europe , Australia, and New Zealand and the second, with taiba rare to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in Saudi Arabia , United Arab Emirates , Qatar , Oman , Kuwait , Bahrain , and Egypt following the necessary regulatory approvals in those countries and/or regions. 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 wart areas. First-Ever WHIM Infection Burden Survey Results: Of the 20 WHIM patient respondents, 60% of those <18 years and 73% of those ≥18 years reported experiencing at least one infection in the previous 3 months, with 25% requiring overnight hospitalization due to infection. The study concludes that “the frequency and severity of infections requiring medical care and hospitalization underscores the urgency to proactively treat patients with 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), enabling possible approval in the first half of 2026. Ex-US Partnerships. In the first quarter of 2025, the company announced that it had entered into two ex- U.S. partnerships for the commercialization of mavorixafor: the first, an exclusive licensing and supply agreement under which Norgine Pharma UK will commercialize mavorixafor in Europe , Australia, and New Zealand and the second, with taiba rare to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in Saudi Arabia , United Arab Emirates , Qatar , Oman , Kuwait , Bahrain , and Egypt following the necessary regulatory approvals in those countries and/or regions. Strategic RestructuringIn February 2025 , X4 announced a strategic restructuring to sharpen focus and maximize the opportunity for mavorixafor in chronic neutropenia while also optimizing its U.S. promotion of XOLREMDI. X4 continues to expect that these efforts will decrease spending by $30-35 million annually. Reverse Stock SplitOn April 24, 2025 , X4 announced a one-for-thirty reverse stock split of the company’s common stock. The reverse stock split took effect at 12:01 a.m. ET on April 28, 2025 , and the company’s common stock began trading on a split-adjusted basis on The Nasdaq Capital Market as of the opening of trading the same day under a new CUSIP number (98420X202). The reverse split reduced the number of outstanding shares of the company’s Common Stock from approximately 173.6 million shares to approximately 5.8 million shares. First-Quarter 2025 Financial Results Cash position: X4 had $87.7 million in cash, cash equivalents, restricted cash, and marketable securities as of March 31, 2025 . The company continues to believe it has sufficient funds to support operations into the first half of 2026. Revenue and Cost of Revenue: For the first quarter ended March 31, 2025 , X4 reported net revenue of $28.8 million and cost of revenue of $4.7 million . Revenue in the quarter included license and other revenues of $27.9 million associated with the completed Norgine agreement and XOLREMDI product revenue of $0.9 million . Cost of revenue included $4.5 million of sublicense royalties associated with the Norgine agreement. Research and Development (R&D) Expenses were $18.5 million for the first quarter ended March 31, 2025 , as compared to $19.9 million for the comparable period in 2024. R&D expenses included $0.3 million of certain non-cash expenses for the first quarter ended March 31, 2025 . Selling, General, and Administrative (SG&A) Expenses were $15.0 million for the first quarter ended March 31, 2025 , as compared to $17.4 million for the comparable period in 2024. SG&A expenses included $0.3 million of certain non-cash expenses for the first quarter ended March 31, 2025 . Net Income (Loss): X4 reported net income of $0.3 million for the first quarter ended March 31, 2025 , as compared to a net loss of $51.8 million for the comparable period in 2024. Net income included $0.5 million of stock-based compensation expenses for the first quarter ended March 31, 2025 . Conference Call and Webcast X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights. The conference call can be accessed by dialing 1-800-343-4849 from the United States or 1-203-518-9848 internationally, followed by the conference ID: X4PHARMA. The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the conclusion of the call, a webcast replay will be available on the website. About WHIM SyndromeWHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 receptor dysfunction that results in impaired mobilization of white blood cells from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis, although only a minority of patients experience all four manifestations in the acronym. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections. About XOLREMDI® (mavorixafor)XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U.S. as a once-daily oral treatment for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. XOLREMDI is the only treatment specifically approved for patients with WHIM syndrome in the U.S. About Chronic Neutropenia and MavorixaforChronic neutropenia is a primary, rare blood condition lasting more than three months, persistently or intermittently, and characterized by low levels of circulating neutrophils and increased risk of infections and reduced quality of life due to abnormally low levels of neutrophils circulating in the blood. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize functional neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC). About the 4WARD Clinical TrialThe 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people 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 150 participants with confirmed trough 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 endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response. About X4 Pharmaceuticals X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging expertise in CXCR4 and immune system biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is currently being marketed in the U.S. as XOLREMDI® in its first indication. The company is also evaluating additional uses of mavorixafor and is conducting a global, pivotal Phase 3 clinical trial (4WARD) in people with certain chronic neutropenic disorders. X4 is headquartered in Boston, Massachusetts . For more information, please visit www.x4pharma.com. 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 initiation, timing, progress, and results of our current and future preclinical studies and clinical trial; the timing and period during which the results of the trials will become available and reported, as well as our research and development programs; market opportunities for X4’s product candidates, including potential benefits from partnerships; expectations regarding the commercial potential of mavorixafor and ongoing engagement and feedback from regulatory authorities; and the sufficiency of X4’s cash resources and pro forma cash resources, including the potential impact of the 2025 strategic restructuring, and expectations regarding X4’s cash runway. 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: X4’s restructuring activities may be more costly or time-consuming than expected or may not achieve their intended results and savings; X4’s future financial performance and position may not improve resulting in difficulties in implementing X4’s business strategy, and plans and objectives for future operations; the expected decrease in annual spending could negatively impact X4’s commercial plans and strategy for mavorixafor; the expected sufficiency of X4’s existing cash resources and runway may not be accurate resulting in the need for additional financing sooner than anticipated or unexpected liquidity constraints; the internal and external costs required for X4’s ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected, which may cause the company to use cash more quickly than expected or to change or curtail some of X4’s plans or both; the expected availability, content, and timing of clinical data from X4’s ongoing clinical trials of mavorixafor may be delayed or unavailable, including the ongoing Phase 3 clinical trial; trials, studies and research programs may be delayed and may not have satisfactory outcomes, additionally earlier trials and studies may not be predictive of later trials and studies; the design and rate of enrollment for current clinical trials may not enable successful completion of the trial(s); the commercial opportunity for mavorixafor may be smaller than anticipated; X4 may be unable to obtain and maintain regulatory approvals; adverse safety effects may arise from the testing or use of X4’s product and product candidates; the need to align with collaborators may hamper or delay development and commercialization efforts or increase costs; business may be adversely affected and costs may increase if any of the company’s key collaborators fails to perform its obligations or terminates the collaboration; X4’s may be unable to advance and commercialize and increase sales in mavorixafor to treat chronic neutropenia or to optimize the U.S. promotion of XOLREMDI® (mavorixafor), approved for the treatment of WHIM; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on X4’s Form 10-K , as well as in other filings X4 makes with the Securities and Exchange Commission , including its quarterly report 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. (Tables Follow) Source: X4 Pharmaceuticals

In February 2025, the global pharmaceutical industry witnessed a series of milestone post-marketing collaboration deals, highlighting a deep integration of pharmaceutical innovation and market expansion that transcends geographical boundaries. From treatments for rare diseases to cancer management, and from improving sleep quality to alleviating chronic pain, drug development and distribution partnerships are advancing at an unprecedented pace. In February, Eisai joined forces with SciClone Pharmaceuticals to advance the application of tasurgratinib in the Greater China region, offering new hope to patients with cholangiocarcinoma. Meanwhile, Nxera Pharma’s partnership with Holling Bio-Pharma Corp. aims to introduce daridorexant to the Taiwanese market, providing a novel treatment option for insomnia sufferers. In another example of global collaboration, Bavarian Nordic and Biological E Limited announced a partnership to expand global supply of the chikungunya vaccine, underscoring the cooperative spirit of multinational companies in addressing public health challenges. 1. Eisai and SciClone Partner to Advance the Development and Distribution of Tasurgratinib in Greater China On February 28, 2025, Eisai announced a significant licensing agreement with a subsidiary of SciClone Pharmaceuticals. The agreement grants SciClone exclusive rights to develop and commercialize tasurgratinib in Greater China, including Mainland China, Hong Kong, Macau, and Taiwan. Tasurgratinib is a novel tyrosine kinase inhibitor that selectively inhibits FGFR1, FGFR2, and FGFR3. It has already been approved in Japan for the treatment of unresectable cholangiocarcinoma with FGFR2 gene fusions or rearrangements that have progressed after chemotherapy, and it was successfully launched in November 2024. Through this collaboration, Eisai aims to leverage SciClone’s strong market presence and commercialization capabilities in the region to accelerate the local deployment of tasurgratinib, bringing hope to more patients. Under the terms of the agreement, Eisai will receive an upfront payment in recognition of the licensing rights. In addition, milestone payments will be made as the drug progresses through development stages and secures regulatory approvals. Following product launch, Eisai will also receive royalties based on net sales, providing a return on its R&D investment and reflecting confidence in the drug’s market performance. This financial structure is designed to promote close collaboration between both parties and maximize the value of tasurgratinib. Tasurgratinib, as an orally bioavailable tyrosine kinase inhibitor, exerts its effects through selective inhibition of FGFR1, FGFR2, and FGFR3. This selectivity allows tasurgratinib to target specific types of cancer cells effectively without interfering with other essential physiological processes. In particular, tumors associated with FGFR abnormalities—such as cholangiocarcinoma and certain breast cancers—present promising therapeutic targets. In addition to its approved use in cholangiocarcinoma in Japan, tasurgratinib is currently undergoing a Phase I clinical trial in Japan for patients with estrogen receptor-positive, HER2-negative breast cancer, further exploring its potential in broader oncology indications. 2. Nxera Pharma and Holling Collaborate to Commercialize Daridorexant in Taiwan On February 28, 2025, Tokyo- and Cambridge-based Nxera Pharma announced it had signed a licensing, supply, and commercialization agreement with Holling Bio-Pharma Corp., Taiwan’s largest pharmaceutical distributor, to bring daridorexant to the Taiwanese market. The collaboration seeks to address the growing demand for effective insomnia treatments in the region. Daridorexant is a new oral dual orexin receptor antagonist (DORA) that helps regulate overactive wakefulness signals by selectively binding to and inhibiting orexin receptors OX1R and OX2R, thereby promoting better sleep onset and maintenance. Under the agreement, Nxera will supply the drug product, while Holling will handle regulatory approval, commercialization, and distribution activities, retaining all regulatory rights. Holling is expected to submit a New Chemical Entity (NCE) application to the Taiwan Food and Drug Administration by mid-2025. If approved, daridorexant could be launched in Taiwan by mid-2026. Nxera will receive an upfront payment upon signing, along with additional milestone payments based on regulatory progress and sales performance. Nxera is also entitled to royalties on net sales from product supply, reflecting the mutual confidence in daridorexant’s market potential and supporting further development efforts. Daridorexant works by blocking the binding and activity of orexins—neuropeptides that promote wakefulness—through dual inhibition of their receptors. The drug has already been launched in Japan under the brand name QUVIVIQ™ following approval in September 2024 and a subsequent market launch in December 2024 by Nxera Pharma Japan in collaboration with Shionogi. Nxera is also conducting a Phase III clinical trial in South Korea to evaluate daridorexant’s efficacy and safety in adults with insomnia. Notably, QUVIVIQ™ has also received regulatory approval in the United States, Europe, and several other countries, where Idorsia Pharmaceuticals Ltd. is responsible for marketing activities. This broad international recognition further supports daridorexant’s status as a best-in-class treatment option. The development of daridorexant continues to progress positively. In addition to its successful launch in Japan, the Phase III trial in South Korea is ongoing, aiming to confirm the drug’s safety and efficacy in improving insomnia symptoms in adults. Given the significant number of insomnia patients in Taiwan, the introduction of daridorexant is expected to have a meaningful impact on clinical practice in the region. Furthermore, this collaboration is part of Nxera’s broader strategy to enhance access to innovative medicines across Japan and the wider Asia-Pacific region by partnering with leading regional commercial players, thus reaching a larger patient population while leveraging the company’s internal expertise in specialty and rare disease medicines. 3. Bavarian Nordic Partners with Biological E Limited to Expand Global Access to Chikungunya Vaccine On February 28, 2025, Bavarian Nordic announced a strategic partnership with Indian biopharmaceutical company Biological E Limited to enhance the global supply of its Chikungunya vaccine. This collaboration underscores both companies’ commitment to improving access to this important vaccine in developing countries. Through this partnership, Bavarian Nordic and Biological E Limited plan to co-invest in the further development of the vaccine and ensure its availability across more countries and regions. The product at the center of this agreement is a vaccine specifically designed to prevent Chikungunya virus infection. This innovative vaccine mimics the structural features of the virus—particularly its surface proteins—to induce a specific antibody response from the human immune system. These antibodies can recognize and neutralize the actual Chikungunya virus during infection, thereby preventing disease onset. Utilizing virus-like particle (VLP) technology, the vaccine delivers robust immune protection without containing live virus, significantly enhancing its safety profile. This Chikungunya VLP vaccine has already received its first regulatory approval in the United States for the prevention of Chikungunya fever. It has also been granted multiple special regulatory designations in both the U.S. and the European Union, including Accelerated Approval, Breakthrough Therapy Designation, Fast Track, and Priority Review—highlighting the recognized public health value of the vaccine. Beyond U.S. approval, additional clinical trials and regulatory submissions are underway in various other countries and regions to expand its global reach. Originally developed by PaxVax Holding Co., Ltd., the vaccine has achieved several critical development milestones. After Bavarian Nordic acquired the program, it advanced the clinical research and secured the initial U.S. approval. Through its new collaboration with Biological E Limited, the vaccine is expected to gain more rapid market access in Asia and other developing regions. Biological E Limited's deep market knowledge and extensive distribution network in these areas make it a key partner in achieving this objective. Moreover, early-stage development efforts were supported by Emergent BioSolutions, Inc. and the U.S. National Institute of Allergy and Infectious Diseases (NIAID), laying a solid foundation for the vaccine’s success. This partnership marks a significant step toward broader protection against Chikungunya fever worldwide. 4. RedHill and Hyloris Sign Global (Excluding North America) Commercialization Agreement for RHB-102 to Improve Quality of Life for Cancer and Gastrointestinal Patients On February 25, 2025, RedHill Biopharma Ltd. announced the signing of an exclusive development and commercialization licensing agreement with Hyloris Pharmaceuticals SA for RHB-102 (Bekinda®). Under this agreement, Hyloris gains the rights to develop and commercialize RHB-102 in all global markets except the United States, Canada, and Mexico. This collaboration reflects the shared commitment of both companies to provide effective treatment options to more patients worldwide, particularly those suffering from chemotherapy/radiation-induced nausea and vomiting (CINV/RINV), acute gastroenteritis, gastritis, and diarrhea-predominant irritable bowel syndrome (IBS-D). As part of the agreement, Hyloris will pay RedHill an upfront fee and milestone payments of up to $60 million based on the achievement of specific commercial targets. Additionally, Hyloris will pay mid-to-high double-digit percentage royalties on net sales, subject to certain cost deductions and inclusive of minimum annual payments. This financial structure not only provides RedHill with necessary funding support but also incentivizes Hyloris to actively promote RHB-102 and ensure its success in global markets. RHB-102 is a once-daily, dual-release, proprietary oral tablet formulation of ondansetron, a 5-HT3 receptor antagonist used to treat nausea and vomiting. It is available in two dosage strengths: 12 mg and 24 mg, aimed respectively at IBS-D and other indications such as acute gastroenteritis and gastritis. The extended-release profile allows for 24-hour symptom control, representing a significant therapeutic advancement for cancer patients undergoing chemotherapy or radiotherapy, as well as for those with gastrointestinal disorders. RHB-102 has completed multiple key clinical trials, including a Phase III study in the United States for acute gastroenteritis and gastritis (24 mg dose) and a Phase II study for IBS-D (12 mg dose), both of which met their primary endpoints. Recently, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) issued a positive opinion, paving the way for a Marketing Authorization Application (MAA) in the UK. If approved, RHB-102 would become the first 24-hour extended-release oral ondansetron formulation indicated for CINV/RINV. RedHill also plans to continue pursuing FDA approval for RHB-102 in the United States. 5. Pharmanovia and Er-Kim Reach Exclusive Distribution Agreement for Sunosi® in Eastern Europe In February 2025, Pharmanovia announced that it had entered into an exclusive distribution agreement with Er-Kim, a leading Turkish pharmaceutical company, for Sunosi® (solriamfetol) in Eastern Europe. This collaboration brings Sunosi® to the Eastern European market to meet the treatment needs of patients suffering from narcolepsy and obstructive sleep apnea (OSA). Although specific financial details have not been fully disclosed, it can be inferred that the agreement involves an upfront payment, milestone payments, and a royalty structure based on sales performance. This arrangement not only supports further development and marketing efforts for the product but also reflects both parties' confidence in the commercial potential of Sunosi®. Sunosi® is a medication indicated for the treatment of excessive daytime sleepiness associated with narcolepsy and OSA. Its active ingredient, solriamfetol, is a selective dopamine and norepinephrine reuptake inhibitor (DNRI). By increasing the concentrations of these neurotransmitters in the brain, Sunosi® effectively enhances wakefulness and alertness, helping patients regain a normal daily rhythm. This mechanism of action makes it an important treatment option, particularly for patients who do not respond well to or cannot tolerate traditional therapies. Sunosi® has been approved and is commercially available in several countries and regions. Originally developed by Jazz Pharmaceuticals, it received FDA approval in the United States in 2019 for the treatment of excessive daytime sleepiness in adults with narcolepsy or OSA. Since then, Sunosi® has gradually expanded its market presence globally. The partnership between Pharmanovia and Er-Kim marks a significant step toward deeper penetration into the Eastern European market, allowing more patients to benefit from the treatment. Ongoing monitoring of Sunosi®'s long-term safety and efficacy continues to ensure the provision of optimal treatment options for patients. Beyond its current indications, Sunosi® may also demonstrate potential efficacy in treating other types of excessive daytime sleepiness disorders, providing new directions for future research. Pharmanovia will continue to monitor the accumulation of clinical data for Sunosi® and explore broader application possibilities. Through its collaboration with Er-Kim, Pharmanovia aims to establish a strong market position in Eastern Europe while raising awareness of the disease and available treatment options. Furthermore, as the understanding of the pathophysiology of excessive daytime sleepiness deepens, Sunosi® is expected to introduce new research findings in the coming years, offering a stronger scientific foundation for its use. This partnership between Pharmanovia and Er-Kim not only enhances the accessibility of Sunosi® in Eastern Europe but also highlights both companies' shared commitment to improving public health. Leveraging Er-Kim's strong local presence and extensive distribution network will be key to achieving this goal. Such cross-border collaborations accelerate the entry of innovative therapies into emerging markets, offering patients more treatment choices. With increasing recognition and support for Sunosi® across more countries and regions, the medication is expected to significantly improve the quality of life for patients with excessive daytime sleepiness worldwide in the coming years. 6. Apotex Partners with Grünenthal to Bring Qutenza® to the Canadian Market On February 24, 2025, Apotex Inc., Canada’s largest pharmaceutical company, and Grünenthal, a global leader in pain management and related diseases, announced a strategic licensing agreement. Under the agreement, Apotex will obtain exclusive rights to Qutenza® in Canada. This partnership aims to introduce Qutenza® as an innovative treatment option for patients suffering from neuropathic pain in Canada. Although specific financial terms have not been fully disclosed, it can be inferred that Grünenthal will receive an upfront payment, additional milestone payments upon achieving specific regulatory milestones, and royalties based on sales. This financial arrangement not only supports further development and commercialization of the product but also reflects both parties’ confidence in the commercial potential of Qutenza®. Qutenza® is a topical, non-systemic, non-opioid pain treatment patch indicated for the management of neuropathic pain. Its active ingredient is high-concentration capsaicin (8%), a naturally occurring compound that is applied directly to the skin to alleviate pain. Capsaicin works by activating and subsequently desensitizing the TRPV1 receptors in sensory nerves, which play a key role in the transmission of pain signals. Through this mechanism, Qutenza® provides effective relief from chronic pain caused by nerve damage without causing systemic side effects or the risk of addiction. This treatment is particularly beneficial for patients who do not respond well to or cannot tolerate traditional therapies. Since acquiring global rights to Qutenza® in 2018, Grünenthal has continued to invest resources in the product’s development and expansion. Qutenza® has been commercialized in the European Union and the United States, with its indication broadened through relaunch and label expansion efforts. In the U.S. market, Qutenza® has experienced a significant brand resurgence, laying a strong foundation for its further promotion worldwide. Apotex’s subsidiary, Searchlight Pharma, will be responsible for seeking marketing authorization for Qutenza® in Canada and for its subsequent commercialization and distribution upon regulatory approval. This partnership is expected to further enhance the global availability and impact of Qutenza®. Since 2017, Grünenthal has invested over €2 billion in successful mergers and acquisitions, continually creating synergies across manufacturing, logistics, and commercial activities by integrating acquired brands into its infrastructure. For Apotex, this partnership not only strengthens its ability to meet patient needs but also expands its global portfolio, which includes more than 550 branded, generic, and biosimilar products. With the launch of Qutenza® in the Canadian market, an effective new treatment option will soon be available for many patients suffering from neuropathic pain. 7. Daré Bioscience Partners with Theramex to Develop the First Biodegradable Long-Acting Contraceptive Implant, Casea S On February 20, 2025, Daré Bioscience, a company focused on innovation in women’s health, announced a co-development and licensing agreement with Theramex, a global specialty pharmaceutical company dedicated to women’s health. This collaboration aims to advance the development of a first-in-class, biodegradable, long-acting contraceptive implant based on etonogestrel. The agreement marks a significant partnership in promoting women’s health management. Etonogestrel is a small-molecule drug used for contraception. It was first approved on July 17, 2006, in the United States for contraceptive use. As the current Phase 1 study is foundation-funded, there are no direct development costs for either Daré or Theramex at this stage. Under the agreement, Daré receives a royalty-free, exclusive, fully paid-up, and sublicensable license to the U.S. patent for Casea S, recently acquired by Theramex. If Phase 1 results are positive, Daré will take responsibility for conducting Phase 2 studies in the U.S., and funding for future Phase 3 trials in the U.S. will be negotiated between Daré and Theramex based on market opportunity. Etonogestrel is a synthetic progestin that primarily works by inhibiting ovulation to prevent pregnancy. It also thickens cervical mucus, making it harder for sperm to reach the egg, and alters the endometrium to reduce the likelihood of implantation. As a long-acting reversible contraceptive (LARC), etonogestrel is typically delivered via a subdermal implant that can continuously release the hormone for up to three years, offering extended contraceptive protection. Due to its high efficacy and convenience, etonogestrel has become one of the preferred contraceptive options for many women. Since its initial approval, etonogestrel has been authorized for contraceptive use in multiple countries and regions worldwide. It has also been studied for other indications such as dysmenorrhea, bleeding disorders, lower urinary tract symptoms, and benign prostatic hyperplasia. Although some indications are no longer under investigation, the use of etonogestrel in contraception continues to evolve. Companies such as Organon NV are actively working to expand its applications and improve efficacy through ongoing research. 8. SK Chemicals Partners with Jeil Health Science to Boost Pharmacy Sales of Ginexin and Trast On March 17, 2025, SK Chemicals announced a co-marketing agreement with Jeil Health Science, a leading South Korean manufacturer of over-the-counter (OTC) pharmaceuticals. The partnership is aimed at increasing pharmacy sales of two SK Chemicals products—Ginexin-F Soft Cap 120mg and Trast Patch 30—by leveraging Jeil Health Science’s extensive distribution network. Previously, these products were distributed through general pharmaceutical wholesalers. Ginexin-F Soft Cap 120mg: This product is a circulation enhancer formulated with ginkgo biloba extract and comes in a soft capsule format, sold exclusively through pharmacies. Ginkgo biloba extract is renowned for its antioxidant properties and is widely used to improve blood circulation and alleviate symptoms related to poor circulation. Trast Patch 30: This is a pain relief patch available in packs of 7, 10, and 30 units, with the 30-pack being particularly suited for patients requiring repeated use. The patch is applied directly to the skin and delivers active ingredients to relieve localized pain, making it suitable for conditions such as muscle aches and arthritis. Both products are already on the market and have established brand recognition and customer bases. Ginexin enjoys a strong reputation as a natural-origin blood circulation booster, while Trast is favored by consumers for its convenient application and effective pain relief. The goal of the partnership is to further expand the market share of these two products in the pharmacy channel and to enhance consumer awareness and trust through more targeted marketing strategies. Through this collaboration, SK Chemicals aims to take advantage of Jeil Health Science’s powerful sales network—serving over 12,000 pharmacies directly—and its proven track record in promoting OTC products, especially in pain management with successful brands like Kefentech and Jeil Cool Pap. Han Sang-chul, CEO of Jeil Health Science, expressed his enthusiasm for the partnership, stating that they are honored to work with SK Chemicals to promote products that have earned long-term trust in the fields of blood circulation and pain relief. Together, the two companies hope to strengthen Ginexin and Trast’s positions as leading brands in their respective markets and to provide more therapeutic options for patients. This partnership not only aims to enhance the market standing of both companies but also to deliver higher-quality products and services to a broader consumer base. 9. X4 Pharmaceuticals and Taiba Rare Enter Exclusive Agreement to Advance Distribution and Commercialization of XOLREMDI® (Mavorixafor) in Select Middle Eastern Countries On February 19, 2025, X4 Pharmaceuticals, a company committed to improving the lives of patients with rare immunodeficiency diseases, announced an exclusive agreement with Taiba Rare, a division of Taiba Healthcare. The agreement grants Taiba Rare exclusive rights to distribute and commercialize XOLREMDI® (mavorixafor) in Saudi Arabia, the United Arab Emirates, Qatar, Oman, Kuwait, Bahrain, and Egypt, contingent upon obtaining local regulatory approvals. Although specific financial terms have not been disclosed, agreements of this nature typically include upfront payments, sales-based milestone payments, and royalties. These arrangements align the interests of both parties and incentivize Taiba Rare to actively promote XOLREMDI®. Given that XOLREMDI® is the first and only treatment approved for WHIM syndrome (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis), the therapy holds significant market potential, making the partnership attractive to both companies. XOLREMDI® (mavorixafor) is an oral, once-daily treatment approved by the U.S. Food and Drug Administration (FDA) in April 2024 for patients aged 12 and older with WHIM syndrome. It works by increasing the number of circulating mature neutrophils and lymphocytes, thereby enhancing the body's ability to fight infections. Mavorixafor functions by inhibiting the CXCR4 receptor, promoting the release of white blood cells from the bone marrow into the bloodstream. While XOLREMDI® has already received FDA approval in the U.S., its Marketing Authorization Application (MAA) is currently under review by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The collaboration with Taiba Rare will accelerate the product's introduction into Middle Eastern markets, offering new treatment options for patients with WHIM syndrome. Under the agreement, Taiba Rare will be responsible for XOLREMDI®’s distribution, promotion, marketing, and sales in the designated region and will jointly develop key strategic decisions with X4 Pharmaceuticals. 10. Beihai Biotechnology Partners with Zydus Lifesciences to Commercialize BEIZRAY® (BH009) in the U.S. On February 18, 2025, Beihai Biotechnology announced a strategic partnership with Zydus Lifesciences. Under the agreement, Beihai grants Zydus exclusive commercialization rights for its novel oncology drug BEIZRAY® in the United States. Following the agreement, Beihai will receive an upfront payment of USD 15 million, with an additional USD 10 million upon the first product delivery—amounting to nearly RMB 200 million in total. Beihai will also benefit from future sales-based milestone payments and a high double-digit percentage share of profits. This payment structure not only provides Beihai with early financial support but also motivates both parties to ensure the commercial success of the product. BEIZRAY® (BH009) is a proprietary, improved formulation of docetaxel developed by Beihai Biotechnology for the treatment of various solid tumors, including breast cancer, non-small cell lung cancer, prostate cancer, gastric cancer, and head and neck squamous cell carcinoma. It acts by stabilizing microtubules and preventing their depolymerization, arresting the cell cycle in the mitotic phase and thereby inhibiting tumor cell proliferation. BEIZRAY® is distinguished by its use of targeted molecular delivery technology that eliminates the use of polysorbate 80—a common component in traditional docetaxel formulations—significantly reducing adverse reactions and improving patient safety. BEIZRAY® received FDA approval in October 2024, making it the first clinically validated and demonstrably superior docetaxel reformulation approved in nearly 30 years. Clinical trials have shown that BEIZRAY® reduces the risk of hematologic toxicity and severe allergic reactions, thereby enhancing the benefit-risk profile for patients. This partnership will leverage Zydus Lifesciences’ robust sales infrastructure in the U.S. to expedite market access and deliver this innovative treatment to a broader patient population. 11. Baheal Pharmaceutical Partners with Roche to Advance Commercialization of Rituximab in China On February 13, 2025, Baheal Pharmaceutical announced that it had entered into a collaboration agreement with Roche. Under this agreement, Baheal has obtained the exclusive rights to promote Roche’s well-known oncology-targeted therapy Rituximab in mainland China. According to the agreement, Roche will pay Baheal a quarterly service fee for its promotional efforts. This arrangement ensures that while Baheal bears the promotional costs, it also receives a corresponding economic return based on sales performance. Additionally, the agreement stipulates that Roche may not appoint any third party to promote the product in the region without prior notice to Baheal, ensuring Baheal’s exclusivity in the market. Rituximab is the world’s first monoclonal antibody specifically targeting the CD20 antigen and marked the beginning of a new era in targeted cancer therapy. It is primarily used for the treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, and other related diseases. Rituximab binds to the CD20 antigen on the surface of B lymphocytes, inducing immune responses against the cancer cells and promoting their apoptosis, thereby inhibiting tumor growth. Since its initial approval in the U.S. in 1997, Rituximab has accumulated over two decades of clinical use and has benefited more than 6.8 million patients worldwide. In China, Rituximab was approved in April 2000, and with approvals for multiple indications—including maintenance therapy for treatment-naïve follicular lymphoma and combination chemotherapy for chronic lymphocytic leukemia—its market potential continues to grow. 12. Chong Kun Dang Secures Exclusive Distribution and Sales Rights for Bayer’s Nexavar and Stivarga in South Korea Chong Kun Dang Pharmaceutical has announced that it has reached an agreement with German pharmaceutical giant Bayer to exclusively distribute and sell two of Bayer’s key oncology drugs—Nexavar and Stivarga—in the South Korean market. This partnership aims to leverage Chong Kun Dang’s robust market network and sales capabilities to expand the local market share of both therapies. Although the specific financial terms have not been disclosed, such agreements typically involve an upfront payment, milestone payments based on sales performance, and profit-sharing arrangements. These structures align the interests of both parties and incentivize Chong Kun Dang to actively promote the products and expand their market presence. Nexavar: A multi-kinase inhibitor indicated for the treatment of advanced renal cell carcinoma, hepatocellular carcinoma, and radioactive iodine-refractory differentiated thyroid cancer. It works by inhibiting several intracellular protein kinases involved in tumor growth and angiogenesis. Stivarga: Also a multi-kinase inhibitor, Stivarga is indicated for metastatic colorectal cancer, gastrointestinal stromal tumors (GIST), and hepatocellular carcinoma. It shares a similar mechanism of action with Nexavar but is approved for different indications and is sometimes used as a follow-up treatment after Nexavar. Both Nexavar and Stivarga are globally approved and have been widely used in clinical practice across many countries and regions. They have demonstrated significant efficacy in treating specific types of cancer and have accumulated extensive real-world usage data. With Chong Kun Dang now taking over the distribution and sales of these two drugs in South Korea, the accessibility of these innovative therapies for local patients is expected to increase substantially. For Chong Kun Dang, this partnership strengthens its presence in the oncology sector by adding two well-recognized therapeutic options to its portfolio, potentially creating new growth drivers for the company. For Bayer, choosing Chong Kun Dang as its local partner reflects confidence in the latter’s commercial capabilities and market expertise in South Korea. This strategic alliance is expected to accelerate market penetration of Nexavar and Stivarga in South Korea, ultimately benefiting more patients with access to advanced cancer treatments. Furthermore, it highlights the growing trend of multinational pharmaceutical companies partnering with strong local firms to optimize resource allocation and achieve mutually beneficial outcomes in global market expansion. 13. Lotus Pharmaceutical Partners with Formycon to Advance Commercialization of FYB203/AHZANTIVE® in the Asia-Pacific Region On February 5, 2025, Klinge Biopharma GmbH, the exclusive global commercialization rights holder of FYB203/AHZANTIVE® (Aflibercept), announced an exclusive licensing agreement with multinational pharmaceutical company Lotus Pharmaceutical. Under the terms of the agreement, Lotus will be responsible for the commercialization of FYB203/AHZANTIVE® in the Asia-Pacific region, including Indonesia, Malaysia, the Philippines, Singapore, Taiwan, Thailand, Vietnam, and the Hong Kong Special Administrative Region. Concurrently, Formycon also signed a finished product supply agreement with Lotus. According to the agreement, Klinge will receive an upfront payment from Lotus and is eligible for additional milestone payments tied to product launch and sales achievements. Furthermore, Klinge will receive royalties based on Lotus’ net sales, with Formycon sharing in Klinge’s revenues at a rate ranging from mid-single to low-double-digit percentages. This payment structure not only provides Klinge with initial financial support but also incentivizes all parties to collaborate closely toward the successful commercialization of the product. FYB203/AHZANTIVE® is a biosimilar to Eylea® (Aflibercept), used for the treatment of neovascular age-related macular degeneration (nAMD) and other severe retinal diseases. The drug works by inhibiting vascular endothelial growth factor (VEGF), thereby reducing the formation of abnormal blood vessels in the retina. VEGF is a protein that promotes the formation of new blood vessels, but its overexpression in certain eye diseases can lead to vision loss or even blindness. By blocking the action of VEGF, FYB203/AHZANTIVE® helps stabilize or improve patients’ vision. FYB203/AHZANTIVE® has achieved significant progress in development, receiving approval from the U.S. Food and Drug Administration (FDA) in June 2024, followed by European Commission approval in January 2025. Currently, Formycon and Lotus are working closely together to prepare submissions to regulatory authorities in the Asia-Pacific region, in accordance with local regulations, to facilitate the timely launch of FYB203/AHZANTIVE® in these markets. This process includes the consolidation of clinical data and adaptations to meet local market requirements. 14. Daewon Pharmaceutical Partners with AstraZeneca Korea to Promote Asthma Treatment Products In early February 2025, Daewon Pharmaceutical announced a collaboration agreement with AstraZeneca Korea to take on the distribution responsibilities for AstraZeneca’s asthma treatment products in the South Korean market. This partnership aims to leverage Daewon’s robust sales network and market expertise to broaden the reach of these essential medications, ensuring that more patients have access to effective treatment options. The agreement includes an upfront payment, sales-based milestone payments, and royalties calculated based on net sales. This structure ensures mutual benefit: AstraZeneca Korea can rapidly expand market share for its products through Daewon’s extensive distribution channels, while Daewon Pharmaceutical gains financial returns from successful product promotion and strengthens its presence in the respiratory disease market. Budesonide/Formoterol (Symbicort) is one such combination product included in this partnership. It consists of a long-acting β2-agonist (LABA) and an inhaled corticosteroid (ICS), designed for long-term control of asthma symptoms. Budesonide helps reduce airway inflammation, while Formoterol relaxes the airway smooth muscles, making breathing easier. This combination therapy effectively alleviates asthma symptoms and reduces the risk of acute exacerbations. These asthma treatment products have been approved globally and have been marketed in numerous countries for years, backed by extensive clinical experience and safety data. For certain products within this collaboration, additional registration steps or labeling adjustments may be required to comply with Korean regulatory standards. However, given their proven efficacy and safety profiles in other markets, it is expected that they will quickly gain recognition and be widely adopted in South Korea. 15. MITEM Pharma Acquires FLISINT® from Sanofi, Committed to Safeguarding the Interests of Patients with Rare and Life-Threatening Diseases In February 2025, MITEM Pharma announced the successful acquisition of the specialty drug FLISINT® from Sanofi. This acquisition underscores MITEM Pharma’s ongoing mission to improve the quality of life for patients suffering from rare and life-threatening diseases. Through this collaboration with Sanofi, MITEM Pharma not only expands its product portfolio but also strengthens its expertise in targeted therapeutic areas. FLISINT® is a specialty medication designed for the treatment of specific rare diseases. Although its exact indication was not disclosed, the name suggests that it may be intended for the treatment of a genetic disorder or metabolic condition requiring specialized attention. The mechanism of action of FLISINT® likely involves addressing enzyme deficiencies or abnormal protein expression caused by genetic mutations, thereby alleviating symptoms and slowing disease progression. For example, it may function through enzyme replacement therapy or by modulating intracellular signaling pathways to restore normal physiological functions. FLISINT® has successfully completed multiple phases of clinical trials and has been approved by regulatory authorities in several countries and regions. This confirms the drug's safety and efficacy, offering an effective treatment option for patients with specific rare conditions. However, with ongoing scientific advancements and the accumulation of new data, further studies may be necessary to explore broader applications or to optimize the formulation for enhanced efficacy and reduced side effects. Conclusion The series of partnership agreements and acquisitions in February 2025 reveals an overarching trend: globalization, specialization, and technological advancement are reshaping the pharmaceutical industry. Whether it's deepening market penetration through local collaboration (such as the partnership between Daewon Pharmaceutical and AstraZeneca Korea) or entering emerging markets (such as X4 Pharmaceuticals' agreement with Taiba Rare for the Middle East), these partnerships demonstrate how pharmaceutical companies are leveraging each other's strengths for mutual benefit. More importantly, they share a common goal—to enhance healthcare access and outcomes for patients worldwide. It is noteworthy that these collaborations go beyond traditional sales and distribution agreements and extend into R&D partnerships. For instance, MITEM Pharma’s acquisition of FLISINT® from Sanofi, as well as Daré Bioscience’s co-development of the biodegradable long-acting contraceptive implant Casea S with Theramex, highlight the growing importance of long-term strategic alliances in driving innovation and accelerating product development in today's rapidly evolving healthcare environment. In summary, the pharmaceutical collaborations of February 2025 mark the dawn of a new era—one characterized by cooperation, resource sharing, and a focus on addressing complex health challenges. As technology continues to evolve and societal awareness of health deepens, we can expect to see more such partnerships emerge, further advancing the frontier of medicine and delivering new therapeutic possibilities to patients around the globe. How to get the latest progress on drug deals? If you would like to access the latest transaction event information, you can click on the 'Deal' module from the homepage of the Synapse database. Within the Deal module, you can search for global pharmaceutical transaction information using labels such as Drugs, Organization, Target, Drug Type, Deal Date.  Furthermore, you can obtain the original link to the transaction coverage by clicking on the "Deal Name." In the analysis view, you can see the most active assignors, assignees, popular targets, and other dimensions of analysis, as well as the distribution of research and development statuses at the time of the transaction, to help you better understand the search results. The Synapse database also supports the ability to view current transactions from the dimension of "drugs" (by selecting "drugs" from the "Adjust Dimension" dropdown menu above). Targeting transactions involving renowned pharmaceutical companies that are of interest to the industry, such as Merck, Roche, etc., Synapse has identified a group of "leading companies" through drugs that have achieved global sales exceeding 1 billion US dollars in 2022. Transactions involving drugs from these leading companies can be filtered by clicking on the "Leading Company" tag on the left-hand side. In addition to the drug transaction module, you can also view related transaction history on the drug detail page and the institution detail page. Click on the image below to explore new pharmaceutical funding transactions!