Core Insights - X4 Pharmaceuticals announced the acceptance of two abstracts for presentation at the 30th Annual Congress of the European Hematology Association, highlighting positive results from a Phase 2 trial of mavorixafor for chronic neutropenia [1][2] - Mavorixafor is positioned as a potential first oral treatment for chronic neutropenia in over 30 years, with ongoing Phase 3 trials supporting its efficacy and safety [2][8] Company Overview - X4 Pharmaceuticals focuses on developing therapies for rare immune system diseases, with mavorixafor being a key product currently marketed in the U.S. as XOLREMDI® [9] - The company is headquartered in Boston, Massachusetts, and is actively pursuing additional indications for mavorixafor [9] Clinical Trial Details - The Phase 2 trial was a six-month open-label study involving 23 participants with various forms of chronic neutropenia, assessing the safety and efficacy of mavorixafor [3][7] - The ongoing pivotal Phase 3 trial, named 4WARD, aims to evaluate mavorixafor's efficacy and safety in a larger cohort of 150 participants with chronic neutropenia [8] Research Findings - The Phase 2 study demonstrated meaningful increases in functional neutrophils, with many participants able to reduce their G-CSF dosing when treated with mavorixafor [1][6] - Mavorixafor acts as a CXCR4 antagonist, mobilizing neutrophils from the bone marrow into the bloodstream, which is crucial for managing chronic neutropenia [5][6]

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]

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 [19] Business Line Data and Key Metrics Changes - Cumulative sales of ZOLREMD reached $3.5 million since the mid-May launch, with Q1 sales slightly lower than Q4 due to inventory resupply timing [11][12] - The company is advancing the MAVERICK-four trial for chronic neutropenia, with over 90% of targeted global trial sites activated and enrollment ongoing in over 20 countries [7][10] 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][29] - The company is focusing on a high unmet need patient population for the MAVERICK-four trial, targeting those with moderate to severe chronic neutropenia [7][10] Company Strategy and Development Direction - The company aims to expand the global potential of MAVERICK-four for chronic neutropenia and has received a notice of allowance from the U.S. Patent Office for its application [10][11] - Partnerships with Norgene and Taberare have been established to commercialize MAVERICK in Europe, Australia, New Zealand, and the MENA region [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in delivering upcoming milestones in chronic neutropenia and highlighted the potential market opportunity of $1 billion to $2 billion in the U.S. alone [11] - The company anticipates full enrollment in the FORWARD trial by Q3 or Q4 2025, with top-line data expected in the second half of 2026 [10] Other Important Information - The company completed a one-for-thirty reverse stock split to address NASDAQ listing rules compliance [19] - The FORWARD trial is designed to demonstrate statistically significant increases in ANC response and decreases in annualized infection rates [8][10] Q&A Session Summary Question: Visibility into patient types in the study - Management noted that there is a clear demand for more patients to be included in the study, with many principal investigators eager to enroll additional patients [21][22] Question: Patient demographics for chronic neutropenia - The 50,000 diagnosed patients and 15,000 with high unmet needs are U.S. figures based on ICD-10 code analysis [29] Question: Label definition for the target population - The company anticipates a broad label for the entire chronic neutropenia population, supported by data from previous studies [31][33] Question: Commercialization agreement with Norgene - The agreement covers both WHIM and chronic neutropenia indications [34] Question: Patient compliance and persistency - Management reported higher than expected compliance and adherence rates for ZOLREMD, indicating strong patient engagement [38] Question: Patient prescription sizes - Most patients are currently receiving a month's supply of medication [42] Question: Patient dropout assumptions in the FORWARD trial - Management confirmed that the enrolled subjects' profiles are on track, and they are seeing the expected event rates early in the study [44]

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]

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]

Core Viewpoint - X4 Pharmaceuticals has announced a one-for-thirty reverse stock split to address compliance issues with Nasdaq's minimum bid price requirement and to potentially increase the market price of its Common Stock [1][11]. Summary by Sections Reverse Stock Split Details - The reverse stock split will take effect at 12:01 a.m. Eastern Time on April 28, 2025, with trading on a split-adjusted basis commencing on the same day [2]. - The split will consolidate every thirty shares into one, reducing the number of outstanding shares from approximately 173.6 million to about 5.8 million [3]. - Proportional adjustments will be made to equity incentive plans, stock options, and outstanding warrants in accordance with the reverse stock split [4]. Shareholder Impact - No fractional shares will be issued; instead, shareholders entitled to fractional shares will receive cash [5]. - Stockholders with shares held in book-entry form or through intermediaries will see the effects of the reverse stock split reflected in their accounts without needing to take action [6]. Compliance with Nasdaq - The reverse stock split was approved within the range disclosed at the 2025 Special Meeting of Stockholders held on April 17, 2025 [7]. - X4 received a deficiency letter from Nasdaq due to its stock price falling below the required minimum of $1.00 per share for 30 consecutive business days [8]. - The company was granted an additional 180-day compliance period until August 11, 2025, to regain compliance with the Bid Price Rule [10]. Company Strategy - X4 believes that the reverse stock split will help increase the market price of its Common Stock and address the deficiency in the Bid Price Rule [11].

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]