BOSTON, June 10, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to mavorixafor, an oral CXCR4 antagonist, for the treatment of chronic neutropenia (CN). The company is currently conducting a global, pivotal Phase 3 clinical trial (4WARD) evaluating mavorixafor in certain primary CN conditions. Mavorixafor was ...

Meaningful and durable increases in functional neutrophils observed over 6-month trial with oral once-daily mavorixafor Majority of physicians and chronic neutropenia participants chose to significantly reduce G-CSF dosing when using mavorixafor in combination therapy BOSTON, May 14, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced the acceptance of two abstracts for poster presentation at the ...

Mavorixafor Development and Commercialization - XOLREMDI® (mavorixafor) is approved and launched in the U S in May 2024 for WHIM syndrome, marking the first therapy for this condition[8, 32] - A Marketing Authorization Application (MAA) for WHIM syndrome has been submitted to and accepted by the EMA in January 2025, with potential EU approval and launch expected in the first half of 2026 via a partnership with Norgine[8] - The company anticipates full enrollment in the 4WARD trial for chronic neutropenia in the third or fourth quarter of 2025, with top-line data expected in the second half of 2026[8] Chronic Neutropenia (CN) Market and Unmet Needs - There are an estimated 50,000 chronic neutropenia patients in the U S , with approximately 15,000 representing a high unmet need and a minimum addressable market for mavorixafor[9, 49] - Currently, only injectable Granulocyte Colony-Stimulating Factor (G-CSF) is approved for severe chronic neutropenia, highlighting the need for innovation and alternative treatments[9, 10] - 67% of targeted WHIM hematologists in the U S would also be potential prescribers for chronic neutropenia if mavorixafor is approved for that indication[42] Clinical Trial Results and Analysis - Post-hoc analysis of the WHIM Phase 3 trial showed that 5 out of 13 mavorixafor-treated participants achieved the 4WARD "Responder" threshold, compared to 0 out of 16 in the placebo group[72] - In a Phase 2 study of mavorixafor in chronic neutropenia, mean Absolute Neutrophil Count (ANC) reached normal levels (≥ 1,500 cells/µL) at 3 and 6 months of treatment in the monotherapy group[59] - Physicians substantially reduced G-CSF dosage in 75% of eligible patients in the Phase 2 chronic neutropenia study, while maintaining normal mean ANC levels (>1,500 cells/µL)[68, 67] Financial Position - The company reported funds of $87 7 million as of March 31, 2025, which is expected to support operations into the first half of 2026[8]

X4 Pharmaceuticals (XFOR) Q1 2025 Earnings Call May 01, 2025 08:30 AM ET Company Participants Dan Ferry - Managing DirectorPaula Ragan - Founder, President, CEO & Board MemberAdam Mostafa - CFOMark Baldry - Chief Commercial OfficerChristophe Arbet-Engels - Chief Medical OfficerStephen Willey - Managing Director Conference Call Participants Edward Tenthoff - Sr. Research AnalystSwayampakula Ramakanth - Managing Director & Senior Equity Analyst Operator Please stand by. Your program is about to begin. Greetin ...

Financial Performance - The company reported revenue of $28.8 million for the three months ended March 31, 2025, compared to no revenue in the same period in 2024, marking a significant increase [164]. - The company reported a net income of $0.3 million for the three months ended March 31, 2025, a significant improvement from a net loss of $51.8 million in the same period in 2024 [164]. - Net income for the three months ended March 31, 2025 was $0.3 million, compared to a net loss of $51.8 million in the same period of 2024 [183]. - Other income for the three months ended March 31, 2025 increased by approximately $25 million compared to the same period in 2024, primarily due to a significant gain from the change in fair value of Class C warrants [174]. Expenses - The cost of revenue for the same period was $4.7 million, primarily due to amortization of an intangible asset and royalty payments [168]. - Research and development expenses decreased by $2 million to $18 million for the three months ended March 31, 2025, compared to $20 million in the prior year, mainly due to reduced pre-clinical drug candidate programs [171]. - Selling, general and administrative expenses also decreased by approximately $2 million to $15 million, attributed to lower compensation expenses and reduced commercialization launch costs [173]. - Net cash used in operating activities for the three months ended March 31, 2025 was $12.4 million, a decrease from $33.6 million in the same period of 2024 [184]. - Cash used in investing activities for the three months ended March 31, 2025 was $3.1 million, down from $5.3 million in the same period of 2024 [185]. Strategic Initiatives - The company completed a strategic restructuring in February 2025, resulting in a 30% reduction in workforce, which is expected to decrease annual spending by $30 to $35 million [159]. - The company is progressing with a pivotal Phase 3 clinical trial (the "4WARD" study") for mavorixafor, aiming to enroll 150 participants and complete enrollment by Q3 or Q4 2025 [157]. - The company launched XOLREMDI in the U.S. for WHIM syndrome and is actively engaging with physicians and patient advocacy organizations to promote awareness [152]. Financing and Capital Needs - The Hercules Loan Agreement allows for a term loan facility of up to $107.5 million, with $75.0 million borrowed to date [179]. - The company anticipates needing to raise additional capital to fund operations and satisfy covenants, which may include equity offerings and debt financings [182]. - The company has a Minimum Cash Covenant requiring a minimum cash level of $15.0 million, which is 20% of outstanding borrowings under the Hercules Loan Agreement [181]. - The company expects operating expenses in 2025 to be lower than those in 2024, excluding the sale of non-financial assets [188]. Shareholder Actions - A reverse stock split of 1-for-30 was approved by shareholders on April 17, 2025, to reduce the number of outstanding shares [162]. Cash Position - The company had cash, cash equivalents, and short-term marketable securities of $87.0 million as of March 31, 2025, with an accumulated deficit of $515.1 million [180]. - The company continues to maintain a valuation allowance against all remaining net deferred tax assets due to expected ongoing operating losses [176].

Financial Data and Key Metrics Changes - The company ended Q1 2025 with just under $90 million in cash and cash equivalents, sufficient to support operations into the first half of 2026 [18] - Net revenues from ZOLREMD were just under $1 million for Q1 2025, bringing cumulative sales since the May 2024 launch to approximately $3.5 million [18] - R&D expenditures totaled $18.5 million, while SG&A expenses were $15 million for the first quarter [18] - The company reported a small net income due to $28 million in license and other revenue from the partnership with Norgene and a gain of $10.8 million on outstanding Class C warrants [18] Business Line Data and Key Metrics Changes - Cumulative sales of ZOLREMD reached $3.5 million since its launch, with Q1 sales slightly lower than Q4 due to inventory resupply timing [12][18] - The FORWARD trial for MAVERICK-four in chronic neutropenia is currently enrolling participants in over 20 countries, with more than 90% of targeted global trial sites activated [7] Market Data and Key Metrics Changes - Approximately 50,000 people are diagnosed with chronic neutropenia in the U.S., with an estimated 15,000 individuals experiencing high unmet needs [6] - The company anticipates a potential market opportunity of $1 billion to $2 billion in the U.S. for MAVERICK-four in chronic neutropenia [12] Company Strategy and Development Direction - The company is focused on advancing clinical trials for MAVERICK-four in chronic neutropenia and expanding the commercialization of ZOLREMD for WHIM Syndrome [5][12] - Partnerships with Norgene and Taberare aim to enhance the global reach of MAVERICK-four and ZOLREMD in Europe, Australia, New Zealand, and the MENA region [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving upcoming milestones in chronic neutropenia and emphasized the importance of long-term patent protection for MAVERICK-four [12] - The company is optimistic about the potential approval from the EMA for MAVERICK-four in WHIM Syndrome as early as Q1 2026 [14] Other Important Information - The company completed a one-for-thirty reverse stock split to address deficiencies with NASDAQ listing rules [19] - The FORWARD trial is designed to demonstrate statistically significant increases in ANC response and decreases in annualized infection rates [11] Q&A Session Summary Question: Visibility into patient types in the study - Management noted that there is a clear demand for including more patients in the study, with many principal investigators eager to enroll additional patients [21] Question: Patient diagnosis numbers - The 50,000 diagnosed patients and 15,000 with high unmet needs are specific to the U.S. based on ICD-10 code analysis [27] Question: Target population for the label - The company aims for a broad label that includes moderate and severe patients, supported by data from previous studies [29] Question: Commercialization agreement with Norgene - The agreement covers both WHIM and chronic neutropenia indications [32] Question: Patient compliance and persistency - Compliance rates are reportedly higher than expected for a daily oral medication, indicating a strong unmet need [36] Question: Patient prescription sizes - Most patients are currently receiving a month's supply of medication [39] Question: Patient dropout assumptions in the FORWARD trial - Management confirmed that the enrolled subjects' profiles are balanced and aligned with the trial's objectives, indicating confidence in the enrollment timeline [42]

Core Insights - X4 Pharmaceuticals is making significant progress in its Phase 3 clinical trial for mavorixafor, targeting chronic neutropenia, with full enrollment expected by Q3 or Q4 2025 and top-line data anticipated in the second half of 2026 [1][5] - The company has generated $3.5 million in net U.S. revenues from XOLREMDI since its launch in May 2024, indicating a positive market response [1][4] - A strategic restructuring is underway to optimize the promotion of XOLREMDI and focus on mavorixafor, with expected annual savings of $30-35 million [6] Financial Performance - For Q1 2025, X4 reported net revenue of $28.8 million, with $27.9 million from license and other revenues and $0.9 million from product revenue [10][20] - Research and Development (R&D) expenses decreased to $18.5 million from $19.9 million in Q1 2024, while Selling, General, and Administrative (SG&A) expenses also fell to $15.0 million from $17.4 million [10][20] - The company achieved a net income of $0.3 million in Q1 2025, a significant improvement from a net loss of $51.8 million in the same period of 2024 [10][20] Clinical Developments - The 4WARD trial is a pivotal Phase 3 study evaluating mavorixafor in patients with chronic neutropenia, aiming to demonstrate significant increases in absolute neutrophil count (ANC) and reductions in infection rates [5][15] - Recent analyses of clinical trial data have bolstered confidence in the potential success of the 4WARD trial, suggesting that mavorixafor may effectively elevate ANC and reduce infection rates [5] - X4 has received a Notice of Allowance for a patent related to mavorixafor, which is expected to expire in March 2041, enhancing the company's intellectual property position [5] Market Expansion - X4 has entered into two international partnerships for the commercialization of mavorixafor, expanding its global reach [9] - The Marketing Authorization Application (MAA) for mavorixafor in the treatment of WHIM syndrome has been validated for review by the European Medicines Agency, with potential approval in the first half of 2026 [9] Stock and Cash Position - As of March 31, 2025, X4 had a cash position of $87.7 million, which is expected to support operations into the first half of 2026 [10] - A one-for-thirty reverse stock split was executed on April 28, 2025, reducing the number of outstanding shares significantly [7]

BOSTON, April 24, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that it will report financial results for the first quarter ended March 31, 2025 and provide corporate updates on Thursday, May 1, 2025. The company will host a conference call and webcast on the same day at 8:30 a.m. EDT. The conference call can be accessed by dialing 1-800-343-4849 from the United States or 1-203-518-9848 int ...

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X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q4 2024 Earnings Conference Call March 25, 2025 8:30 AM ET Company Participants Daniel Ferry - IR, LifeSci Advisors Paula Ragan - CEO Adam Mostafa - CFO Christophe Arbet-Engels - Chief Medical Officer Mark Baldry - CCO Conference Call Participants Stephen Willey - Stifel Ed Tenthoff - Piper Sandler Ayan Hussein - Cantor Fitzgerald Doug MacPherson - H.C. Wainwright David Bautz - Zacks Small-Cap Research Operator Greetings, and welcome to the X4 Pharmaceuticals Fourth Qu ...