X4 Pharmaceuticals Inc.
11/05/2025 | Press release | Distributed by Public on 11/05/2025 07:25
Quarterly Report for Quarter Ending September 30, 2025 (Form 10-Q)
MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
The following discussion and analysis should be read in conjunction with our condensed consolidated financial statements and related notes in "Item 1. Financial Statements." References in this report to "X4," the "Company," "we," "our" and "us" are references to X4 Pharmaceuticals, Inc. and its subsidiaries.
Forward-Looking Statements
This Quarterly Report on Form 10-Q contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), that relate to future events or to our future operations or financial performance. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. These statements may be identified by such forward-looking terminology as "may," "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "potential," "continue" or the negative of these terms or other comparable terminology. Our forward-looking statements are based on a series of expectations, assumptions, estimates and projections about our company, are not guarantees of future results or performance and involve substantial risks and uncertainty. We may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements. These forward-looking statements are subject to a number of known and unknown risks, uncertainties and assumptions, including risks described in the section titled "Risk Factors" and elsewhere in this report, regarding, among other things:
•our ability to raise additional capital or achieve sufficient revenue to properly fund our business and operating plan as well as our ability to continue as a going concern;
•our expectations and goals for commercialization of XOLREMDI® , which has been approved for use as an oral, once-daily therapy to increase the number of circulating mature neutrophils and lymphocytes in patients 12 years of age and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome in the U.S. and that XOLREMDI, our one product approved for commercial sale, upon which we depend almost entirely on to produce revenue faces an unknown market size and growth potential and we have not generated significant revenue from product sales to date, and we may never achieve profitability;
•our expectations that the intended effects of the reverse stock split will occur and that we will be able to maintain compliance with Nasdaq's listing requirements and this will positively impact our ability to raise capital;
•the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and related preparatory work and the period during which the results of the trials will become available, as well as our research and development programs;
•the timing of enrollment in, and our ability to successfully enroll, our clinical trials, including our Phase 3 4WARD clinical trial;
•the potential benefits, including clinical utility, that may be derived from XOLREMDI or any of our product candidates;
•the timing of and our ability to obtain and maintain regulatory approval of our existing product XOLREMDI (mavorixafor) or any product candidates that we may develop in the future, and any related restrictions, limitations, or warnings in the label of any approved product candidates;
•our plans to research, develop, manufacture and commercialize XOLREMDI or our product candidates;
•the timing of our regulatory filings for our product candidates, along with regulatory developments in the United States and other foreign countries;
•the size and growth potential of the markets for XOLREMDI and our product candidates, if approved, and the rate and degree of market acceptance of XOLREMDI and our product candidates, including reimbursement that may be received from payors;
•the benefits of U.S. Food and Drug Administration and European Commission designations, including, without limitation, Fast Track, orphan and Breakthrough Therapy;
•our commercialization, marketing and manufacturing capabilities and strategy;
•our ability to execute and realize benefits from the strategic restructurings announced in February 2025 and September 2025, or any similar actions taken in the future;
•our ability to attract and retain qualified employees and key personnel;
•our competitive position and the development of and projections relating to our competitors or our industry;
•our expectations regarding our ability to obtain and maintain intellectual property protection;
•the success of competing therapies that are or may become available;
•our estimates and expectations regarding future operations, financial position, revenues, costs, expenses, uses of cash, capital requirements or our need for additional financing;
•our plans to in-license, acquire, develop and commercialize additional product candidates;
•the impact of laws and regulations;
•our plans to identify additional product candidates with significant commercial potential that are consistent with our commercial objectives;
•our strategies, prospects, plans, expectations or objectives; and
•other risks and uncertainties, including those listed under the section titled "Risk Factors" in this Quarterly Report.
OVERVIEW
We are a biopharmaceutical company developing, and commercializing novel therapeutics for the treatment of rare hematology diseases. We continue to progress our global, pivotal Phase 3 clinical trial, (the "4WARD" trial) to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enrollup to 176 patients, with full enrollment expected in Q3 2026. The FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia.Chronic neutropenia is defined as periods lasting more than three months persistently or intermittently where there are abnormally low levels of neutrophils circulating in the blood, and may be idiopathic (of unknown origin), cyclic (episodes typically occurring every three weeks), or congenital (of genetic causation). Chronic neutropenia disorders are rare blood conditions similarly characterized by increased risks of infections and cancer due to abnormally low levels of neutrophils in the body. In all cases, the CXCL12/CXCR4 pathway is the key regulator of neutrophil release from the bone marrow.
We have one commercially approved product, XOLREMDI ® (mavorixafor), which has received accelerated approval in the United States from the U.S. Food and Drug Administration ("FDA") for use as an oral, once-daily therapy in patients 12 years of age and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, to increase the number of circulating mature neutrophils and lymphocytes. WHIM syndrome is a rare combined primary immunodeficiency and chronic neutropenic disorder. In connection with the our long term strategy to successfully complete the 4WARD Phase 3 trial in patients with moderate and severe chronic neutropenia, we are no longer prioritizing investment in the WHIM indication.
Private Placement Financing and Management Changes
During the third quarter of 2025, we sold shares of common stock and pre-funded warrants to purchase common stock in a private placement that resulted in net proceeds of approximately $81.0 million, after deducting placement agent fees and other expenses. Pursuant to registration rights agreements, we registered the shares of common stock issued and issuable under pre-funded warrants under a registration statement on Form S-3 that was declared effective by the SEC on September 17, 2025.
Concurrent with the financing and effective August 12, 2025, our former President and Chief Executive Officer Paula Ragan, PhD, and Chief Financial Officer Adam Mostafa stepped down from their respective roles. Dr. Ragan also resigned from the Company's Board of Directors (the "Board"), and Michael Wyzga transitioned from Board Chair to Lead Independent Director. In their place, the Board appointed Adam R. Craig, M.D., Ph.D, as Executive Chair and Board Chair; John Volpone as President, and David Kirske as Chief Financial Officer.
Q4 2025 Equity Financing
In October 2025, we closed an underwritten public offering of our common stock and, in lieu of common stock to certain investors, pre-funded warrants, raising proceeds of $145.6 million, net of underwriting discounts and estimated offering
expenses.
Strategic Restructurings
In the first quarter of 2025, we implemented a strategic restructuring of our business operations, workforce and capital spending to focus efforts on advancing mavorixafor to treat those with chronic neutropenia. As part of this restructuring, we (i) implemented a net reduction of our employee headcount by 43 employees, or approximately 30% of our total workforce, including our U.S. commercial field team, (ii) commenced the closure of our research and development facility in Vienna, Austria, (iii) paused our pre-clinical drug candidate programs and (iv) streamlined other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia. We incurred charges of approximately $2.5 million for severance and other employee termination-related costs related to this strategic restructuring. We believe that this strategic restructuring will decrease our annual spending by $30 million to $35 million per year.
In September 2025, we announced an additional strategic restructuring designed to further sharpen operational focus and align resources with our long-term strategy to successfully complete the 4WARD Phase 3 trial in patients with moderate and severe chronic neutropenia. As part of this initiative, we further reduced our workforce by approximately 50%, a step anticipated to result in additional annualized cost savings of approximately $13 million. We incurred expenses of approximately $4.9 million during the third quarter for severance and other employee termination-related costs related to this strategic restructuring. This workforce reduction was substantially completed in the third quarter of 2025.
The estimate of costs that we expect to incur related to these workforce reduction as well as the decrease in spending, and the timing thereof are subject to a number of assumptions and actual results may differ. We may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the actions described above.
Regulatory Update and Out-License Agreements
In early 2025, we submitted a Marketing Authorization Application ("MAA") to the EMA seeking regulatory approval to commercialize mavorixafor for WHIM syndrome in the European Union. Such MAA was validated for processing by the EMA in January 2025. On January 13, 2025, we announced a License and Supply Agreement (the "Norgine Agreement") with Norgine Pharma UK ("Norgine"), pursuant to which Norgine was granted an exclusive license to distribute, market and sell our drug product for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia, and New Zealand.
RESULTS OF OPERATIONS
The following table summarizes the results of our operations for the three and nine months ended September 30, 2025 and 2024:
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||||||||||||||
| (in millions) | 2025 | 2024 | Change | 2025 | 2024 | Change | |||||||||||||||||||||||||||||
| Revenue | $ | 1.8 | $ | 0.6 | $ | 1.2 | $ | 32.5 | $ | 1.1 | $ | 31.4 | |||||||||||||||||||||||
| Cost and operating expenses: | |||||||||||||||||||||||||||||||||||
| Cost of revenue | 0.4 | 0.2 | 0.2 | 5.4 | 0.5 | 4.9 | |||||||||||||||||||||||||||||
| Research and development | 17.3 | 19.2 | (1.9) | 54.2 | 59.9 | (5.7) | |||||||||||||||||||||||||||||
| Selling, general and administrative | 11.6 | 15.7 | (4.1) | 36.1 | 46.4 | (10.3) | |||||||||||||||||||||||||||||
| Gain on sale of non-financial asset | - | - | - | - | (105.0) | 105.0 | |||||||||||||||||||||||||||||
| Total operating expenses | 29.3 | 35.1 | (5.8) | 95.7 | 1.8 | 93.9 | |||||||||||||||||||||||||||||
| Loss from operations | (27.5) | (34.5) | 7.0 | (63.2) | (0.7) | (62.5) | |||||||||||||||||||||||||||||
| Total other (expense) income, net | (2.3) | (2.2) | (0.1) | 8.0 | 3.1 | 4.9 | |||||||||||||||||||||||||||||
| (Loss) income before income taxes | (29.8) | (36.7) | 6.9 | (55.2) | 2.4 | (57.6) | |||||||||||||||||||||||||||||
| Provision for income taxes | - | - | - | (0.1) | - | (0.1) | |||||||||||||||||||||||||||||
| Net (loss) income | $ | (29.8) | $ | (36.7) | $ | 6.9 | $ | (55.3) | $ | 2.4 | $ | (57.7) | |||||||||||||||||||||||
Revenue
License and Other
During the nine months ended September 30, 2025, we granted an exclusive license to Norgine to distribute, market and sell our product for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia and New Zealand following regulatory approval. For the nine months ended September, 2025, we recognized $27.6 million for the delivery of the
license and $0.7 millionfor the provision of research and development services. We had no license or other revenue during the three or nine months ended September 30, 2024.
Product Revenue, Net
We began recognizing product sales in June 2024 following FDA approval of XOLREMDI on April 29, 2024 and its subsequent commercial launch in the United States. Net product sales were as follows for the nine months ended September 30, 2025 and for each of the quarterly periods therein and the comparative quarter in the prior year.
| (in thousands) | 2025 | 2024 | |||||||||
| First quarter | $ | 942 | $ | - | |||||||
| Second quarter | 1,744 | 563 | |||||||||
| Third quarter | 1,566 | 560 | |||||||||
| Nine months ended September 30, | $ | 4,252 | $ | 1,123 | |||||||
Co-pay assistance payments and rebates to U.S. government payors have comprised the majority of our gross-to-net revenue adjustments. Gross-to-net adjustments have been approximately 10% in 2025 year to date.
Operating Cost and Expenses:
Cost of Revenue
Cost of revenue primarily consists of amortization of an intangible asset related to accrued and paid milestone payments associated with our Genzyme license agreement, and sales and sublicense-based royalty payments due under our Genzyme license agreement. Cost of revenue increased $4.9million in the nine months ended September 30, 2025, as compared to the same period in the prior year, primarily due to additional royalties in the current nine period associated with sublicense income from our Norgine Agreement.
Research and Development Expenses
Research and development expenses consist primarily of costs incurred in connection with the development of our product candidates, including employee salaries and related expenses, clinical development expenses, internal and third-party costs of manufacturing our drug products for use in our clinical trials. Research and development expenses also include costs related to compliance with regulatory requirements; and prior to the FDA's approval of our drug product in the U.S. in the second quarter of 2024, payments made under third-party licensing agreements were charged to research and development expense.
Following our strategic restructuring announced during the first quarter of 2025, substantially all of our research and development has been focused on our one product candidate, mavorixafor (X4P-001). The following table shows external costs incurred by product candidate (primarily external CRO costs) and unallocated research and development costs, primarily consisting of employee salaries and related expense for our research and development organization.
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||||||||||||||
| 2025 | 2024 | Change | 2025 | 2024 | Change | ||||||||||||||||||||||||||||||
| (in millions) | |||||||||||||||||||||||||||||||||||
| Direct research and development expenses by product candidate: | |||||||||||||||||||||||||||||||||||
| Mavorixafor (X4P-001) | $ | 10.3 | $ | 9.7 | $ | 0.6 | $ | 28.2 | $ | 31.1 | $ | (2.9) | |||||||||||||||||||||||
| X4P-002 | - | - | - | - | 0.2 | (0.2) | |||||||||||||||||||||||||||||
| X4P-003 | - | - | - | - | 0.1 | (0.1) | |||||||||||||||||||||||||||||
| Unallocated expense | 7.0 | 9.5 | (2.5) | 26.0 | 28.5 | (2.5) | |||||||||||||||||||||||||||||
| Total research and development expenses | $ | 17.3 | $ | 19.2 | $ | (1.9) | $ | 54.2 | $ | 59.9 | $ | (5.7) | |||||||||||||||||||||||
Research and development expenses decreased by $1.9million and $5.7millionin the three and nine months ended September 30, 2025, respectively, as compared to the same periods in the prior yearprimarily due to decreases in spending associated with our 2025 strategic restructurings, including lower spending on non-clinical programs, lower consulting fees, lower drug substance manufacturing costs, and lower regulatory costs. For the three and nine months ended September 30, 2025, unallocated expense includes $1.5 million and $2.2 million, respectively, in severance charges for terminated employees.
Decreases in research and development expenses in the current three month period were partially offset by higher clinical costs associated with our 4WARD trial.
Selling, General and Administrative Expenses
Selling, general and administrative expenses consist primarily of salaries and related costs, including stock-based compensation, for personnel in sales and marketing, executive, finance, and administrative functions. Selling, general and administrative expenses also include direct and allocated facility-related costs as well as professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services.
Selling, general and administrative expenses decreased by approximately $4.1 million and $10.3million in the three and nine months ended September 30, 2025, respectively, as compared to the same periods in the prior year. Selling, general and administrative expenses include $3.4 million and $5.0 million in severance charges for terminated employees in the three and nine month periods ended September 30, 2025, respectively. Decreases to selling, general and administrative expense in each period were primarily due to a decreases in compensation expense due to lower head count in our general and administrative functions, a significant decrease in sales and marketing expenses in the current three and nine month periods as compared to the prior year, during which we incurred commercialization sales and marketing launch costs related to our drug product. These decreases in selling, general and administrative expenses were partially offset by higher severance costs associated with our 2025 strategic restructurings and higher legal costs in the current three and nine month periods.
Other (Expense) Income, Net
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||||||||||||||||||||||
| 2025 | 2024 | change | 2025 | 2024 | change | ||||||||||||||||||||||||||||||
| (in millions) | |||||||||||||||||||||||||||||||||||
| Interest income | $ | 0.9 | $ | 1.9 | $ | (1.0) | $ | 2.6 | $ | 4.5 | $ | (1.9) | |||||||||||||||||||||||
| Interest expense | (2.3) | (2.4) | 0.1 | (6.7) | (6.5) | (0.2) | |||||||||||||||||||||||||||||
| Change in fair value of Class C warrant liability | (0.7) | (1.9) | 1.2 | 12.8 | 4.6 | 8.2 | |||||||||||||||||||||||||||||
| Other (expense) income, net | (0.2) | 0.2 | (0.4) | (0.7) | 0.5 | (1.2) | |||||||||||||||||||||||||||||
| Total other (expense) income, net | $ | (2.3) | $ | (2.2) | $ | (0.1) | $ | 8.0 | $ | 3.1 | $ | 4.9 | |||||||||||||||||||||||
Other expense, net, increased approximately $0.1 million in the three months ended September 30, 2025 as compared to the same period in the prior year primarily due to lower interest income earned on our marketable securities, partially offset by lower losses in the current period on fair value adjustments related to our Class C warrants. Other expense, net, decreased $4.9 millionin the nine months ended September 30, 2025 as compared to the same period in the prior year primarily due to higher gains on fair value adjustments related to our Class C warrants in the current period, partially offset by lower interest income on our marketable security investment portfolio.
Provision for Income Taxes
Income tax provisions recorded for the three and nine months ended September 30, 2025 and 2024 were not significant and were primarily related to our Austrian subsidiary. We will continue to maintain a full valuation allowance against net deferred tax assets, including net operating loss carryforwards, until we are able to consistently generate sufficient taxable income to realize the benefit of our net deferred tax assets.
Liquidity and Capital Resources
Sources of Liquidity
To date, we have funded our operations primarily with proceeds from sales of common stock, warrants and prefunded warrants for the purchase of our preferred stock and our common stock, sales of preferred stock, proceeds from the issuance of convertible debt and borrowings under loan and security agreements.
Public and Private Equity Offerings. Over the past several years we have funded our operations primarily from sales of common stock, warrants and prefunded warrants through both public offerings and private placements. Most
recently in August 2025, we sold shares of common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of common stock in a private placement ("Q3 2025 PIPE") offering for net proceeds of $81.0 million, after placement agent fees and offering expenses. In addition, in Q4 2025 we completed an underwritten public offering of 52,844,000 shares of our common stock (inclusive of 6,984,000 shares pursuant to the exercise in full of the underwriters' option to purchase additional shares) at a public offering price of $2.90 per share and, in lieu of common stock to certain investors, prefunded warrants to purchase up to 700,000 shares of our common stock at a price of $2.899 per pre-funded warrant, for net proceeds of $145.6 million, after underwriting discounts and offering expenses.
ATM Sales Agreement. We are party to a Controlled Equity OfferingSM Sales Agreement ("ATM"), dated as of August 7, 2020, pursuant to which we may offer and sell shares of our common stock through one or more investment banks. To date and for the nine months ended September 30, 2025, we have sold $23.6 million and $9.0 million, respectively, of our common stock, net of offering costs, under the ATM. Pursuant to our Registration Statement on Form S-3 that became effective on August 24, 2023 and the related ATM prospectus contained therein, we may offer and sell shares of our common stock having an aggregate offering price of up to an additional $66.0 million.
Product Sales and License Revenue. We commercially launched XOLREMDI in the second quarter of 2024 following the approval of XOLREMDI by the FDA on April 29, 2024. To date, we have generated $6.8 million of net product revenue from the sale of XOLREMDI. In the first quarter of 2025, we generated $27.6 million in license revenue from the Norgine Agreement.
Hercules Loan Agreement. We are a party to a loan and security agreement (the "Hercules Loan Agreement"), which provides for a term loan facility of up to $107.5 million, under which we have borrowed an aggregate of $75.0 million of term loans to date, representing the maximum borrowings as of September 30, 2025. The term loan facility requires that we make interest-only payments through maturity on July 1, 2027 and requires that we meet certain operational and financial covenants. See Note 8 to the condensed, consolidated financial statements contained herein for a full description of our Hercules Loan Agreement.
Historical Cash Flows
The following table summarizes our cash flow activities for each of the periods presented:
| Nine Months Ended September 30, | |||||||||||
| 2025 | 2024 | ||||||||||
| (in thousands) | |||||||||||
| Net (loss) income | $ | (55.3) | $ | 2.4 | |||||||
| Adjustments to reconcile net (loss) income to net cash used in operating activities | (6.7) | (101.8) | |||||||||
| Changes in operating assets and liabilities | (8.1) | 1.5 | |||||||||
| Net cash used in operating activities | (70.1) | (97.9) | |||||||||
| Net cash (used in) provided by investing activities | (8.7) | 75.6 | |||||||||
| Net cash provided by financing activities | 92.5 | 20.3 | |||||||||
| Effect of exchange rate changes on cash, cash equivalents and restricted cash | 0.1 | - | |||||||||
| Net increase in cash, cash equivalents and restricted cash | 13.9 | (2.0) | |||||||||
| Cash, cash equivalents and restricted cash, beginning of period | 56.5 | 100.2 | |||||||||
| Cash, cash equivalents and restricted cash, end of period | $ | 70.4 | $ | 98.2 | |||||||
Operating Activities
During the nine months ended September 30, 2025, net cash used in operating activities was $70.1 million, primarily resulting from net losses of $55.3 million adjusted for net non-cash income of $6.7 million, primarily related to gains on changes to the fair value of our Class C warrants that are measured quarterly at fair value, and $8.1 million of changes to operating assets and liabilities primarily related to a reduction in accounts payable and accrued expenses. Net cash used in operating activities for the nine months ended September 30, 2024 was $97.9 million, primarily resulting from costs and operating expenses of $106.8 million, adjusted for non-cash expenses of $7.7 million and changes in our operating assets and liabilities of $1.5 million. Net cash used in operations for the nine months ended September 30, 2025 also excludes the gain on sale of a priority review
voucher, which is included in investing activities. Non-cash expenses primarily include stock-based compensation expense, non-cash lease expense, non-cash interest expense and change in fair value of financial liabilities.
Investing Activities
During the nine months ended September 30, 2025, cash used in investing activities of $8.7 million, primarily include net sales of short-term marketable securities. During the nine months ended September 30, 2024, cash provided by investing activities of $75.6 million included $105.0 million in cash proceeds from the sale of a priority review voucher obtained as a result of the approval of our drug product by the FDA, partially offset by cash outflows of $7.0 million in license payments classified as the acquisition of an intangible assets, $22.1 million of net investments of short-term marketable securities and the acquisition of equipment.
Financing Activities
During the nine months ended September 30, 2025, cash provided by financing activities of $92.5 million was primarily due to proceeds of $81.2 million, net of cash paid for issuance costs, for the sale our common stock and pre-funded warrants in our Q3 2025 PIPE, $9.0 million in sales of our common stock through our ATM program and $2.2 million in sales of our common stock through our common stock purchase agreement with Lincoln Park Capital Fund LLC. Cash provided by financing activities for the nine months ended September 30, 2024 included $20.0 million of new borrowings on our loan facility.
Capital Resources
Based on our cash, cash equivalents and marketable securities on hand as of November 5, 2025 and our current operating plan, we believe that our cash, cash equivalents and marketable securities will allow us to fund operations for at least the next 12 months.
Capital Requirements
October 27, 2025, we closed an underwritten offering of shares of our common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of our common stock, for net proceeds of approximately $145.6 million, after deducting underwriting discounts and commissions and estimated offering expenses, which further extends our ability to fund our operations and financial obligations. Until we reach profitability, we will need to raise additional capital, which cannot be assured, to fund our operations and meet our financial obligations beyond this period. Such additional capital could be raised through a combination of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements, or other collaborations and strategic alliances. If we are unable to obtain funding, we could be forced to delay, reduce, or eliminate some or all of our research and development programs, product portfolio expansion or commercialization efforts, which would adversely affect our business prospects, or we may be unable to continue operations and may need to restructure our obligations in a court-supervised process or otherwise.
Due to the numerous risks and uncertainties associated with the future sale of our approved drug product and the research, development, and commercialization of future product candidates, we are unable to estimate the exact amount of our funding requirements. Our short-term and long-term funding requirements will depend on and could increase significantly as a result of many factors, including:
•the scope, number, initiation, progress, timing, costs, design, duration, any potential delays, and results of clinical trials and nonclinical studies for our current or future product candidates, particularly our Phase 3 clinical trial of mavorixafor for the treatment of individuals with chronic neutropenic disorders;
•the outcome, timing and cost of regulatory reviews, approvals or other actions to meet regulatory requirements established by the FDA and comparable foreign regulatory authorities, including the potential for the FDA or comparable foreign regulatory authorities to require that we perform more studies for our product candidates than those that we currently expect;
•our ability to obtain marketing approval for our product candidates;
•the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights covering our product and product candidates, including any such patent claims and intellectual property rights that we have licensed from Genzyme pursuant to the terms of our license agreement with Genzyme;
•our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product or product candidates;
•our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement;
•the success of any other business, product or technology that we acquire or in which we invest;
•the costs of acquiring, licensing or investing in businesses, product candidates and technologies;
•the effect of competing technological and market developments; and
•the costs to continue operating as a public company
CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES
Our condensed consolidated financial statements are prepared in accordance with generally accepted accounting principles in the United States. The preparation of our condensed consolidated financial statements and related disclosures requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, costs and expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions.
During the three months ended September 30, 2025, there were no material changes to our critical accounting policies as reported for the year ended December 31, 2024 as part of our Annual Report on Form 10-K other than as updated in Note 2 of these condensed consolidated financial statements. Also see Note 2 under the heading "Recently Adopted Accounting Pronouncements" for new accounting pronouncements or changes to the accounting pronouncements during the three months ended September 30, 2025.
Smaller Reporting Company Status
We are a smaller reporting company ("SRC") as defined by Rule 12b-2 of the Exchange Act and Item 10(f)(1) of Regulation S-K. We may take advantage of certain of the scaled disclosures available to smaller reporting companies for so long as (i) our voting and non-voting common stock held by non-affiliates is less than $250.0 million measured on the last business day of our second fiscal quarter or (ii) our annual revenue is less than $100.0 million during the most recently completed fiscal year and our voting and non-voting common stock held by non-affiliates is less than $700.0 million measured on the last business day of our second fiscal quarter.
X4 Pharmaceuticals Inc. published this content on November 05, 2025, and is solely responsible for the information contained herein. Distributed via Edgar on November 05, 2025 at 13:25 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]