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]

Core Insights - X4 Pharmaceuticals will report its financial results for Q1 2025 on May 1, 2025, and provide corporate updates [1] - A conference call and webcast will be held on the same day at 8:30 a.m. EDT for investors [2] Company Overview - X4 Pharmaceuticals focuses on developing innovative therapies for rare immune system diseases with significant unmet needs [3] - The company has developed mavorixafor, an orally available CXCR4 antagonist, currently marketed in the U.S. as XOLREMDI® [3] - X4 is conducting a global pivotal Phase 3 clinical trial (4WARD) for mavorixafor in patients with certain chronic neutropenic disorders [3]

When the reverse stock split becomes effective, every thirty (30) of the company's issued shares of Common Stock will be combined into one issued share of Common Stock, without any change to the par value per share. This will reduce the number of outstanding shares of Common Stock from approximately 173.6 million shares to approximately 5.8 million shares. BOSTON, April 24, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (NASDAQ:XFOR), a company driven to improve the lives of people with rare diseases of the im ...

PART I [Business](index=6&type=section&id=Item%201.%20BUSINESS) X4 Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare immune system diseases, with its lead product XOLREMDI® launched in the U.S [Overview and Commercial Product](index=6&type=section&id=Overview%20and%20Commercial%20Product) The company's first product, XOLREMDI® (mavorixafor), an oral CXCR4 antagonist, was approved by the U.S. FDA in April 2024 for treating WHIM syndrome - The U.S. FDA approved mavorixafor (trade name XOLREMDI®) in April 2024 as the first drug to treat WHIM syndrome in patients aged 12 and older, with the U.S. commercial launch commencing in May 2024[17](index=17&type=chunk) - Mavorixafor is an orally available, small-molecule selective antagonist of the CXCR4 receptor, designed to increase the mobilization of white blood cells from bone marrow into the bloodstream[16](index=16&type=chunk) - Upon FDA approval, the company was granted a **Priority Review Voucher (PRV)** due to the drug's Rare Pediatric Disease designation, which it sold in May 2024[23](index=23&type=chunk) [Pipeline and Clinical Development](index=8&type=section&id=Pipeline%20and%20Clinical%20Development) Clinical development focuses on expanding mavorixafor's indications, supported by a successful Phase 3 trial in WHIM syndrome and an ongoing pivotal trial in chronic neutropenia - The pivotal Phase 3 4WHIM trial for WHIM syndrome met its primary endpoint (p<0.0001), showing mavorixafor's superiority over placebo in increasing the time patients' absolute neutrophil count (ANC) remained above the severe neutropenia threshold[38](index=38&type=chunk) - In the 4WHIM trial, mavorixafor treatment led to a **statistically significant reduction of approximately 60%** in the annualized infection rate compared to placebo (p<0.01)[38](index=38&type=chunk) - The company has initiated a pivotal Phase 3 trial (the "4WARD" study) to evaluate mavorixafor in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia, aiming to enroll 150 participants[18](index=18&type=chunk)[56](index=56&type=chunk) - Preclinical candidates X4P-003 and X4P-002 are currently paused as part of the 2025 restructuring plan[57](index=57&type=chunk) [Commercial Strategy and Market Opportunity](index=7&type=section&id=Commercial%20Strategy%20and%20Market%20Opportunity) The company is targeting a U.S. WHIM syndrome market of up to 3,700 patients and a larger chronic neutropenia market while expanding internationally through partnerships - Market research and an AI-driven study of insurance claims suggest there may be **up to 3,700 WHIM patients** in the United States[28](index=28&type=chunk) - A retrospective analysis projects **up to 48,000 people** in the U.S. were living with a diagnosis of chronic neutropenia in 2021, suggesting a potential addressable market for mavorixafor of **approximately 15,000 individuals**[46](index=46&type=chunk)[47](index=47&type=chunk) - The company has partnered with **Norgine** for commercialization in Europe, Australia, and New Zealand, and with **taiba rare** for distribution in the Middle East and North Africa, contingent on regulatory approvals[19](index=19&type=chunk)[40](index=40&type=chunk)[41](index=41&type=chunk) [Manufacturing, Competition, and Intellectual Property](index=11&type=section&id=Manufacturing%2C%20Competition%2C%20and%20Intellectual%20Property) The company uses a virtual manufacturing model, faces no direct oral therapy competition for its indications, and relies on a patent portfolio expiring between 2024 and 2044 - X4 relies on third-party manufacturers, with **Evotec A.G.** producing the active pharmaceutical ingredient (API) for mavorixafor and **Catalent Inc.** manufacturing the final capsule drug product[63](index=63&type=chunk)[64](index=64&type=chunk)[65](index=65&type=chunk) - The company is not aware of other companies developing oral therapies for WHIM syndrome or chronic neutropenia; the primary competition for chronic neutropenia is injectable G-CSF (filgrastim) and its biosimilars[62](index=62&type=chunk) - The company's intellectual property portfolio includes owned and exclusively licensed patents, with issued U.S. patents covering mavorixafor and its use expected to expire between **2024 and 2044**[89](index=89&type=chunk) [License Agreements](index=12&type=section&id=License%20Agreements) The company's operations depend on an in-license agreement with Genzyme for mavorixafor and key out-licensing agreements with Norgine and Abbisko for international commercialization Key License Agreement Financial Terms | Partner | Type | Key Financials | | :--- | :--- | :--- | | **Genzyme** | In-license | Paid $7.0M milestone in 2024. Up to $13.0M in future regulatory/sales milestones remain (as of 12/31/24). Tiered royalties (6%-12%) on net sales. | | **Norgine** | Out-license | Received €28.5M upfront payment (Jan 2025). Eligible for up to €226.0M in milestones and escalating double-digit royalties. | | **Abbisko** | Out-license | Eligible for up to $214.0M in milestones and tiered low double-digit royalties on net sales in Greater China. | [Government Regulation](index=17&type=section&id=Government%20Regulation) The company navigates extensive FDA and international regulations, benefiting from Orphan Drug exclusivity while facing challenges from payor cost-containment measures - The company's products are subject to extensive regulation by the FDA and other global authorities, covering research, development, manufacturing, labeling, marketing, and post-approval monitoring[96](index=96&type=chunk) - Mavorixafor received **Orphan Drug designation**, which provides **seven years of exclusive marketing** in the U.S. for the approved indication, and a Rare Pediatric Disease designation, which resulted in a Priority Review Voucher[127](index=127&type=chunk)[129](index=129&type=chunk) - Sales success depends on coverage and reimbursement from third-party payors, who are increasingly implementing cost-containment measures; recent legislation like the Inflation Reduction Act (IRA) could impact future pricing and reimbursement[152](index=152&type=chunk)[153](index=153&type=chunk)[161](index=161&type=chunk) [Human Capital](index=28&type=section&id=Human%20Capital) Following a February 2025 restructuring, the company's workforce was reduced from 143 to approximately 100 full-time employees - As of December 31, 2024, the company had **143 full-time employees**[169](index=169&type=chunk) - In February 2025, a restructuring resulted in a net reduction of 43 employees, leaving **approximately 100 employees** as of March 1, 2025[169](index=169&type=chunk) [Risk Factors](index=29&type=section&id=Item%201A.%20RISK%20FACTORS) The company faces substantial going concern risk due to a history of losses, dependence on a single product, and non-compliance with Nasdaq's minimum bid price rule - The company's history of significant losses and negative cash flows raises **substantial doubt about its ability to continue as a going concern**, and it will require substantial additional funding to continue operations[177](index=177&type=chunk)[183](index=183&type=chunk)[186](index=186&type=chunk) - The business depends almost entirely on the success of its commercial product, **XOLREMDI**, and its development for other indications; the commercial opportunity may be smaller than anticipated[176](index=176&type=chunk)[199](index=199&type=chunk)[201](index=201&type=chunk) - The company is currently **not in compliance with Nasdaq's minimum bid price requirement** of $1.00 per share, and failure to regain compliance could result in the delisting of its common stock[342](index=342&type=chunk)[343](index=343&type=chunk) - The company relies on **single third-party manufacturers** for both the active pharmaceutical ingredient (API) and the finished drug product for mavorixafor, creating significant supply chain risk[179](index=179&type=chunk)[280](index=280&type=chunk) [Unresolved Staff Comments](index=65&type=section&id=Item%201B.%20UNRESOLVED%20STAFF%20COMMENTS) The company reports that it has no unresolved staff comments from the SEC - None[370](index=370&type=chunk) [Cybersecurity](index=65&type=section&id=Item%201C.%20CYBERSECURITY) The company maintains a cybersecurity risk management program overseen by the Audit Committee, which has not materially impacted the business to date - The company has a cybersecurity program that is part of its overall enterprise risk management, with oversight provided by the **Audit Committee of the Board of Directors**[371](index=371&type=chunk)[377](index=377&type=chunk) - The program includes policies, monitoring, training, and an incident response plan, managed by the Director of Infrastructure, Operations and Cybersecurity[372](index=372&type=chunk)[376](index=376&type=chunk) [Properties](index=66&type=section&id=Item%202.%20PROPERTIES) The company leases its corporate headquarters in Boston, Massachusetts, and laboratory and office space in Vienna, Austria - Corporate headquarters are located in a leased **28,000 sq. ft.** office space in Boston, MA, with the lease expiring November 30, 2026[379](index=379&type=chunk) - The company leases approximately **1,200 square meters** of lab and office space in Vienna, Austria, with a lease expiring in March 2028[380](index=380&type=chunk) [Legal Proceedings](index=66&type=section&id=Item%203.%20LEGAL%20PROCEEDINGS) As of December 31, 2024, the company was not a party to any legal proceedings - As of December 31, 2024, the company was not party to any legal proceedings[381](index=381&type=chunk) [Mine Safety Disclosures](index=66&type=section&id=Item%204.%20MINE%20SAFETY%20DISCLOSURES) This item is not applicable to the company - Not applicable[382](index=382&type=chunk) PART II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=67&type=section&id=Item%205.%20MARKET%20FOR%20REGISTRANT'S%20COMMON%20EQUITY%2C%20RELATED%20STOCKHOLDER%20MATTERS%20AND%20ISSUER%20PURCHASES%20OF%20EQUITY%20SECURITIES) The company's common stock trades on the Nasdaq Capital Market under "XFOR", and the company has never paid cash dividends and does not plan to in the future - The company's common stock trades on the Nasdaq Capital Market under the ticker symbol **"XFOR"**[384](index=384&type=chunk) - The company has **never declared or paid cash dividends** and does not intend to do so in the foreseeable future[386](index=386&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=68&type=section&id=Item%207.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) The company recorded its first product revenue in 2024, but a history of losses and negative cash flow raises substantial doubt about its ability to continue as a going concern [Results of Operations](index=70&type=section&id=Results%20of%20Operations) In 2024, the company recorded its first product revenue of $2.6 million and significantly reduced its net loss to $37.5 million due to a $105.0 million gain from a PRV sale Consolidated Statements of Operations (2024 vs. 2023) | Metric | 2024 (in thousands) | 2023 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | **Product revenue, net** | **$2,557** | **$0** | **$2,557** | | Research and development | $81,643 | $72,017 | $9,626 | | Selling, general and administrative | $61,518 | $35,505 | $26,013 | | Gain on sale of non-financial asset | ($105,000) | $0 | ($105,000) | | Loss from operations | ($36,401) | ($107,522) | $71,121 | | **Net loss** | **($37,450)** | **($101,167)** | **$63,717** | - The increase in SG&A expenses by **$26.0 million** was primarily driven by a **$15.2 million increase in selling and marketing costs** to support the U.S. launch of XOLREMDI[410](index=410&type=chunk) [Liquidity and Capital Resources](index=72&type=section&id=Liquidity%20and%20Capital%20Resources) With $102.1 million in cash as of year-end 2024, management has substantial doubt about the company's ability to continue as a going concern due to an impending debt covenant breach - As of December 31, 2024, the company had cash, cash equivalents, and short-term marketable securities of **$102.1 million** and an accumulated deficit of **$515.4 million**[420](index=420&type=chunk) - Management has concluded that there is **substantial doubt about the company's ability to continue as a going concern** due to the risk of violating the Minimum Cash Covenant ($15 million) on its Hercules loan within the next 12 months[421](index=421&type=chunk) Cash Flow Summary (2024) | Cash Flow Activity | Amount (in thousands) | | :--- | :--- | | Net cash used in operating activities | ($130,901) | | Net cash provided by investing activities | $66,990 | | Net cash provided by financing activities | $20,294 | - The company expects the 2025 Restructuring to decrease annual spending by **$30 to $35 million**, which it believes will provide sufficient funds to support operations into the first half of 2026[400](index=400&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=78&type=section&id=Item%207A.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) As a smaller reporting company, X4 Pharmaceuticals is not required to provide this disclosure - As a smaller reporting company, we are not required to provide disclosure for this Item[449](index=449&type=chunk) [Financial Statements and Supplementary Data](index=78&type=section&id=Item%208.%20FINANCIAL%20STATEMENTS%20AND%20SUPPLEMENTARY%20DATA) This section contains the audited financial statements, for which the auditor's report expresses substantial doubt about the company's ability to continue as a going concern - The Report of Independent Registered Public Accounting Firm from PricewaterhouseCoopers LLP includes a paragraph highlighting **substantial doubt about the Company's ability to continue as a going concern**[491](index=491&type=chunk) Key Balance Sheet Data (as of Dec 31, 2024) | Account | Amount (in thousands) | | :--- | :--- | | Cash and cash equivalents | $55,699 | | Marketable securities | $46,361 | | **Total Assets** | **$146,447** | | Long-term debt, net | $75,425 | | **Total Liabilities** | **$124,298** | | **Total Stockholders' Equity** | **$22,149** | [Changes in and Disagreements with Accountants on Accounting and Financial Disclosures](index=78&type=section&id=Item%209.%20CHANGES%20IN%20AND%20DISAGREEMENTS%20WITH%20ACCOUNTANTS%20ON%20ACCOUNTING%20AND%20FINANCIAL%20DISCLOSURES) The company reports no changes in or disagreements with its accountants on accounting and financial disclosures - None[451](index=451&type=chunk) [Controls and Procedures](index=78&type=section&id=Item%209A.%20CONTROLS%20AND%20PROCEDURES) Management concluded that the company's disclosure controls and internal control over financial reporting were effective as of December 31, 2024 - Management concluded that the company's disclosure controls and procedures were **effective** as of December 31, 2024[453](index=453&type=chunk) - Management concluded that the company's internal control over financial reporting was **effective** as of December 31, 2024[456](index=456&type=chunk) [Other Information](index=79&type=section&id=Item%209B.%20OTHER%20INFORMATION) No directors or officers adopted, modified, or terminated any Rule 10b5-1 trading plans during the fourth quarter of 2024 - No directors or officers adopted, modified, or terminated any Rule 10b5-1 trading plans during the three months ended December 31, 2024[459](index=459&type=chunk) PART III [Directors, Executive Officers and Corporate Governance](index=80&type=section&id=Item%2010.%20DIRECTORS%2C%20EXECUTIVE%20OFFICERS%20AND%20CORPORATE%20GOVERNANCE) Information for this item is incorporated by reference from the company's 2025 Proxy Statement - Information for this item is incorporated by reference from the company's 2025 Proxy Statement[463](index=463&type=chunk) [Executive Compensation](index=80&type=section&id=Item%2011.%20EXECUTIVE%20COMPENSATION) Information regarding executive compensation is incorporated by reference from the company's 2025 Proxy Statement - Information for this item is incorporated by reference from the company's 2025 Proxy Statement[466](index=466&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=80&type=section&id=Item%2012.%20SECURITY%20OWNERSHIP%20OF%20CERTAIN%20BENEFICIAL%20OWNERS%20AND%20MANAGEMENT%20AND%20RELATED%20STOCKHOLDER%20MATTERS) Information regarding security ownership is incorporated by reference from the company's 2025 Proxy Statement - Information for this item is incorporated by reference from the company's 2025 Proxy Statement[467](index=467&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=80&type=section&id=Item%2013.%20CERTAIN%20RELATIONSHIPS%20AND%20RELATED%20TRANSACTIONS%2C%20AND%20DIRECTOR%20INDEPENDENCE) Information regarding related party transactions and director independence is incorporated by reference from the company's 2025 Proxy Statement - Information for this item is incorporated by reference from the company's 2025 Proxy Statement[468](index=468&type=chunk) [Principal Accountant Fees and Services](index=80&type=section&id=Item%2014.%20PRINCIPAL%20ACCOUNTANT%20FEES%20AND%20SERVICES) Information regarding principal accountant fees and services is incorporated by reference from the company's 2025 Proxy Statement - Information for this item is incorporated by reference from the company's 2025 Proxy Statement[469](index=469&type=chunk) PART IV [Exhibit and Financial Statement Schedules](index=81&type=section&id=Item%2015.%20EXHIBIT%20AND%20FINANCIAL%20STATEMENT%20SCHEDULES) This section lists the financial statements and exhibits filed as part of the Annual Report - This section provides an index of the financial statements and a list of all exhibits filed with the Form 10-K[471](index=471&type=chunk)[474](index=474&type=chunk) [Form 10-K Summary](index=84&type=section&id=Item%2016.%20FORM%2010-K%20SUMMARY) The company indicates that there is no Form 10-K summary - None[479](index=479&type=chunk)

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which is expected to support operations into the first half of 2026 [24] - Net revenues from XOLREMDI were reported at $1.4 million for the fourth quarter and $2.6 million for the full year 2024 [25] - R&D expenditures totaled $21.7 million for the fourth quarter and $81.6 million for the full year, while SG&A expenses were $15.1 million and $61.5 million respectively [25] - The net loss for the fourth quarter was $39.8 million, and for the full year, it was $37.5 million, reflecting a one-time sale of a priority review voucher for $105 million in May of the previous year [25][26] Business Line Data and Key Metrics Changes - XOLREMDI sales topped $2.5 million for the 7.5 months since its launch in mid-May 2024 [10] - The company has successfully engaged with top-tier immunologists and hematologists, which is expected to continue to increase patient finding and shorten the time to prescription [11] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of mavorixafor, with the EMA accepting the submitted MAA for review, potentially leading to approval as early as Q1 2026 [12] - The company entered into a partnership with Norgine for commercialization in Europe, Australia, and New Zealand, receiving EUR 28.5 million upfront and potential milestone payments of up to EUR 226 million [13] Company Strategy and Development Direction - The company aims to maximize the opportunity for mavorixafor in chronic neutropenia, which is viewed as a larger market opportunity [22] - A strategic restructuring is expected to decrease annual spending by about $30 million to $35 million, implemented to sharpen focus on chronic neutropenia [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for the trial design [32][34] - The company is focused on building awareness and education around WHIM syndrome and chronic neutropenia, with plans to pivot efforts towards the patient community [86] Other Important Information - The company has initiated a peer-to-peer speaker program to enhance physician education regarding WHIM syndrome [7] - The 4WARD trial for chronic neutropenia has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment by Q3 or Q4 of this year [22] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold is not expected to impact trial pace [32][33] Question: Inventory and discounting trends - The company noted that inventory reflects stocking up in Q4, and there are no current trends in discounting as they have a tight distribution channel [43][49] Question: Patient numbers and price increase for XOLREMDI - Management did not disclose patient numbers but confirmed a 7% price increase for the new year [54][56] Question: Commercialization experiences since launch - The company has successfully engaged physicians and identified additional patients, with ongoing efforts to build demand through conferences and education [70][71] Question: Expected patient numbers for WHIM and CN - Management reiterated the guidance of approximately 1,000 diagnosed WHIM patients in the U.S. and about 50,000 total chronic neutropenia patients, targeting the refractory severe end of that population [78]

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which includes the EUR 28.5 million received from Neurogene and the expected financial impact of the strategic restructuring [17] - Net revenues for Zolremdi were $1.4 million for Q4 and $2.6 million for the full year 2024 [17] - R&D expenditures totaled $21.7 million for Q4 and $81.6 million for the full year, with SG&A expenses at $15.1 million for Q4 and $61.5 million for the full year [17] - The net loss was $39.8 million for Q4 and $37.5 million for the full year, reflecting a one-time sale of a priority review voucher for $105 million [17] Business Line Data and Key Metrics Changes - Zolremdi sales topped $2.5 million, covering the 7.5 months since its launch in mid-May [8] - The company has engaged with top-tier immunologists and hematologists, expecting continued patient finding and shorter time to prescription [8] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of MAVERICKS-four, with an MAA submitted to the EMA and accepted for review [9] - Two international partnerships were established, one with Neurogene for commercialization in Europe, Australia, and New Zealand, and another with TABA Rare for the MENA region [10][11] Company Strategy and Development Direction - The company aims to maximize the opportunity for MAVERICKS-four in chronic neutropenia, believing it represents a larger market opportunity [16] - A strategic restructuring was announced to decrease spending by approximately $30 million to $35 million annually [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for trial design and success [20][24] - The company is focused on building awareness and education around WIM syndrome, with plans to pivot efforts towards the patient community [62] Other Important Information - The FORWARD trial for MAVERICKS-four has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment in Q3 or Q4 of this year [15] - A slight price increase of 7% for Zolremdi was implemented at the start of 2025 [37] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold was aligned with this [20][21] Question: Impact of tightening eligibility criteria on enrollment pace - Management does not expect the tightening of eligibility criteria to slow down enrollment, as the targeted patient population remains consistent [25][26] Question: Current inventory status and discounting trends - The company noted that inventory metrics reflect stocking up in Q4, and there is no discounting occurring at this time [28][33] Question: Patient numbers and price increase for Zolremdi - Management refrained from disclosing specific patient numbers but confirmed a 7% price increase for the new year [36][38] Question: Screening progress for Phase III trial - Management reported that site activations are at about 90%, and screening is ramping up as expected [43][44] Question: Commercialization experiences since launch - The company has focused on building awareness and engaging physicians, successfully enrolling patients from the clinical trial onto the product [50][51] Question: Sales guidance for 2025 - Management is striving for continued demand increase but is not providing formal sales guidance [60][62] Question: Impact of primary outcome change on labeling for chronic neutropenia - Management does not believe the change in primary outcome will affect the labeling for chronic neutropenia [66]

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which includes the EUR 28.5 million received from Neurogene and the expected financial impact of the strategic restructuring [19] - Net revenues for Zolremdi were $1.4 million for Q4 and $2.6 million for the full year 2024 [19] - R&D expenditures totaled $21.7 million for Q4 and $81.6 million for the full year, including non-cash expenses [19] - SG&A expenses were $15.1 million for Q4 and $61.5 million for the full year, also including non-cash expenses [19] - The net loss was $39.8 million for Q4 and $37.5 million for the full year, with the annual figure reflecting a one-time sale of a priority review voucher for $105 million [19] Business Line Data and Key Metrics Changes - Zolremdi sales topped $2.5 million, covering 7.5 months since its launch in mid-May [9] - The company has engaged with top-tier immunologists and hematologists to increase patient finding and shorten the time to prescription [9] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of MAVERICKS-four, with an MAA submitted to the EMA for review [10] - The company entered into two international partnerships to commercialize MAVERICKS-four and Zolremdi in Europe, Australia, New Zealand, and the MENA region [11][12] Company Strategy and Development Direction - The company aims to maximize the opportunity for MAVERICKS-four in chronic neutropenia, which is viewed as a larger market opportunity [17] - A strategic restructuring is expected to decrease annual spending by $30 million to $35 million [17] - The commercialization strategy focuses on disease awareness and strengthening relationships with patient advocacy groups [6] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for trial success [23][26] - The company is focused on building awareness and education around WIM syndrome to drive demand for Zolremdi [52][64] - Management is confident in the enrollment trajectory for the FORWARD trial, expecting full enrollment by Q3 or Q4 of 2025 [45] Other Important Information - A slight price increase of 7% for Zolremdi was implemented at the start of 2025 [39][41] - The company is not currently engaging in discounting due to a tight distribution channel [35] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold does not impact the trial's case [21][24] Question: Impact of tightening eligibility criteria on enrollment pace - Management does not expect the tightening of eligibility criteria to slow down enrollment, as the targeted patient population has been consistent [28] Question: Current inventory status and discounting trends - The company noted that inventory reflects stocking up in Q4, and there are no current trends in discounting as they have a tight distribution channel [30][35] Question: Patient numbers and price increase for Zolremdi - Management is not disclosing patient numbers yet but confirmed a 7% price increase for Zolremdi [38][41] Question: Screening progress for Phase III trial - Management reported that site activations are at about 90% and screening is progressing well [45][46] Question: Commercialization learnings since launch - The company has focused on building awareness and engaging physicians, successfully enrolling patients from the clinical trial onto the product [52][53] Question: Sales goals for 2025 - Management is striving for continued demand increase but is not providing formal sales guidance [62]

Financial Performance - X4 Pharmaceuticals reported net revenues of $2.6 million for XOLREMDI® since its launch in May 2024, with expectations for increased sales in 2025 [3]. - X4 reported a net loss of $37.5 million for the full year 2024, compared to a net loss of $101.2 million in 2023 [20]. - Product revenue for Q4 2024 was $1.434 million, compared to $0 in Q4 2023, indicating a significant increase [28]. - The net loss for Q4 2024 was $39.821 million, compared to a net loss of $19.130 million in Q4 2023, representing a 108.5% increase in losses [28]. Expenses - Research and Development (R&D) expenses for the full year 2024 were $81.6 million, up from $72.0 million in 2023 [15]. - Selling, General, and Administrative (SG&A) expenses for the full year 2024 were $61.5 million, compared to $35.5 million in 2023 [15]. - Research and development expenses for Q4 2024 were $21.702 million, up from $15.272 million in Q4 2023, reflecting a 42.5% increase [28]. - Selling, general and administrative expenses rose to $15.145 million in Q4 2024 from $9.927 million in Q4 2023, a 52.5% increase [28]. - A strategic restructuring announced in February 2025 is expected to reduce spending by $30-35 million annually [12]. Cash and Assets - X4 had a cash position of $102.8 million as of December 31, 2024, and expects sufficient funds to support operations into the first half of 2026 [15]. - Total current assets decreased to $112.175 million as of December 31, 2024, down from $122.076 million as of December 31, 2023 [30]. - Cash and cash equivalents decreased to $55.699 million from $99.216 million year-over-year, a decline of 43.8% [30]. - The company reported a total stockholders' equity of $22.149 million as of December 31, 2024, down from $51.099 million a year earlier, a decrease of 56.7% [30]. - Total liabilities increased to $124.298 million as of December 31, 2024, compared to $96.159 million as of December 31, 2023, marking a 29.3% increase [30]. Clinical Trials and Regulatory Challenges - The company anticipates full enrollment in the Phase 3 4WARD trial for chronic neutropenia by Q3 or Q4 2025, with top-line data expected in the second half of 2026 [1]. - The 4WARD trial is now activated at approximately 90% of targeted sites worldwide [14]. - The ongoing Phase 3 clinical trial for mavorixafor may face delays or unsatisfactory outcomes, impacting future commercialization efforts [25]. - X4 Pharmaceuticals anticipates potential challenges in obtaining and maintaining regulatory approvals for mavorixafor, which could affect its market strategy [25]. Licensing and Partnerships - X4 entered into a licensing agreement with Norgine Pharma UK, receiving €28.5 million upfront and potential milestone payments of up to €226 million [6]. - The company recognized a gain of $105 million from the sale of a priority review voucher (PRV) in 2024 [20].

Core Insights - X4 Pharmaceuticals has activated approximately 90% of targeted sites for its Phase 3 4WARD trial in chronic neutropenia, with full enrollment expected in Q3 or Q4 2025 and top-line data anticipated in the second half of 2026 [1][8] - The company reported net revenues of $2.6 million for XOLREMDI® since its launch in May 2024, with expectations for increased sales in 2025 as physician outreach improves [1][3] - A strategic restructuring was announced in February 2025 to focus on mavorixafor in chronic neutropenia, aiming to reduce annual spending by $30-35 million [9][12] Company Developments - X4 Pharmaceuticals launched XOLREMDI® (mavorixafor) in May 2024 after receiving FDA approval for treating WHIM syndrome, marking a significant milestone for the company [2][3] - The company has entered into commercialization partnerships in the EU, Australia, New Zealand, and MENA regions to expand its reach for mavorixafor [2][4][5] - The Phase 2 trial of mavorixafor showed positive results, leading to the ongoing Phase 3 4WARD trial, which aims to enroll 150 participants with chronic neutropenia [11][18] Financial Performance - For the fourth quarter and full year ended December 31, 2024, X4 reported net product revenue of $1.4 million and $2.6 million, respectively, with a net loss of $39.8 million for Q4 and $37.5 million for the full year [17][23] - The company had a cash position of $102.8 million as of December 31, 2024, which is expected to support operations into the first half of 2026 [10][12] - X4 recognized a gain of $105 million from the sale of a priority review voucher in 2024, contributing to its financial results [17][23]

Core Insights - X4 Pharmaceuticals will report its financial results for Q4 and the full year ended December 31, 2024, on March 25, 2025 [1] - A conference call and webcast will be held on the same day at 8:30 a.m. ET for investors [2] - The company focuses on developing therapies for rare immune system diseases, with its lead product mavorixafor currently marketed in the U.S. as XOLREMDI [3] Company Overview - X4 Pharmaceuticals is dedicated to improving the lives of patients with rare immune system diseases and significant unmet medical needs [3] - The company has developed mavorixafor, an orally available CXCR4 antagonist, and is exploring additional indications for this drug [3] - X4 is conducting a global pivotal Phase 3 clinical trial (4WARD) for mavorixafor in patients with chronic neutropenic disorders [3]