XFOR 10-K & 10-Q changes, risk factors and insider trading
X4 Pharmaceuticals, Inc · Nasdaq · Biological Products, (No Diagnostic Substances) · CIK 1501697 · All filings on SEC.gov
At a glance
What changed in the latest 10-K
Risk Factors
New heading “Until we achieve profitability, our operations will require substantial additional funding. Our history of recurring losses and anticipated future expenditures could raise substantial doubts about our ability to continue as a going concern. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate any product development programs or commercialization efforts.”
New heading “Disruptions at the FDA, the SEC and other government agencies caused by funding shortages or global health concerns, in addition to substantial uncertainty regarding the Trump administration’s initiatives and staffing cuts and how these might impact the FDA, its implementation of laws, regulations, policies and guidance, and its personnel, could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.”
New heading “Significant political, trade, regulatory developments, economic downturns, inflation, increases in interest rates, natural disasters, public health crises, geopolitical events, such as the war in Ukraine and in Gaza and other circumstances beyond our control, could have a material adverse effect on our business, financial condition or results of operations.”
New heading “We may experience difficulties in managing the reduction in size of our organization due to our restructuring activities, and we may not achieve the expected benefits of such activities.”
New heading “We may be subject to adverse legislative or regulatory changes in tax laws that could negatively impact our financial condition.”
Removed heading “Our operating plan and liquidity position will require substantial additional funding. Our history of recurring losses and anticipated expenditures could raise substantial doubts about our ability to continue as a going concern. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate any product development programs or commercialization efforts.”
Removed heading “Inadequate funding for the FDA, the SEC and other government agencies could hinder their ability to hire and retain key personnel, and substantial leadership, personnel, and policy changes could prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.”
Removed heading “We will need to scale the size of our organization, and we may experience difficulties in managing this reduction in size.”
Removed heading “Our business could be adversely affected by economic downturns, inflation, increases in interest rates, natural disasters, public health crises, political crises, geopolitical events, such as the war in Ukraine and in Gaza, or other macroeconomic conditions, which have in the past and may in the future negatively impact our business and financial performance.”
Removed heading “If we fail to maintain an effective system of internal control over financial reporting, we may not be able to accurately report our financial results or prevent fraud. As a result, stockholders could lose confidence in our financial and other public reporting, which would harm our business and the trading price of our common stock.”
Removed heading “We may become involved in securities class action litigation or shareholder derivative litigation that could divert management’s attention and harm our business and insurance coverage may not be sufficient to cover all costs and damages.”
Largest changes
“Further, the global economy, including credit and financial markets, outside of recent tariffs, has experienced extreme volatility and disruptions, including, among other things, severely diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, supply chain shortages, increases in inflation rates, higher interest rates and uncertainty about economic stability. For example, the COVID-19 pandemic resulted in widespread unemployment, economic slowdown and extreme volatility in the capital markets. The U.S. …”see in full comparison
“The global economy, including credit and financial markets, has experienced extreme volatility and disruptions, including, among other things, severely diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, supply chain shortages, increases in inflation rates, higher interest rates and uncertainty about economic stability. For example, the COVID-19 pandemic resulted in widespread unemployment, economic slowdown and extreme volatility in the capital markets. The U.S. …”see in full comparison
“Our operating plan and liquidity position will require substantial additional funding. Our history of recurring losses and anticipated expenditures could raise substantial doubts about our ability to continue as a going concern. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate any product development programs or commercialization efforts.”see in full comparison
“In the past, securities class action or shareholder derivative litigation has often followed certain significant business transactions, such as the sale of a business division or announcement of a merger. This risk is especially relevant for us because biopharmaceutical companies have experienced significant stock price volatility in recent years. We may become involved in this type of litigation in the future. The outcome of litigation is necessarily uncertain, and we could be forced to expend significant resources in the defense of such suits, and we may not prevail. …”see in full comparison
“We may in the future discover weaknesses in our system of internal financial and accounting controls and procedures that could result in a material misstatement of our consolidated financial statements. Our internal control over financial reporting will not prevent or detect all error and all fraud. A control system, no matter how well designed and operated, can provide only reasonable, not absolute, assurance that the control system’s objectives will be met. …”see in full comparison
“Significant political, trade, regulatory developments, economic downturns, inflation, increases in interest rates, natural disasters, public health crises, geopolitical events, such as the war in Ukraine and in Gaza and other circumstances beyond our control, could have a material adverse effect on our business, financial condition or results of operations.”see in full comparison
Full comparison: every changed paragraph (148)
An investment in our securities involves a high degree of risk. You should carefully consider the following information about these risks, together with the other information appearing elsewhere in this Annual Report, including our audited consolidated financial statements and related notes hereto, before deciding to invest in our common stock. The occurrence of any of the following risks could have a material adverse effect on our business, financial condition, results of operations and future growth prospects, or cause our actual results to differ materially from those contained in forward-looking statements we have made in this report and those we may make from time to time. The risks and uncertainties described below are not the only ones facing us. Additional risks and uncertainties not presently known to us, or that we currently see as immaterial, may also adversely affect our business. In these circumstances, the market price of our common stock could decline, and you may lose all or part of your investment. We cannot assure you that any of the events discussed below will not occur. The disclosures in this section reflect our beliefs and opinions as to factors that could materially and adversely affect us in the future. References to past events are provided by way of example only and are not intended to be a complete listing or a representation as to whether or not such factors have occurred in the past. The information discussed below should be considered carefully with the other information contained in this Annual Report on Form 10-K and the other documents and materials we file with the SEC, as well as news releases and other information we publicly disseminate from time to time.
•We have incurred significant losses and have not generated significant revenue from product sales since our inception and we cannot predict whether or when and if we will be able to generate meaningful revenues from sales of XOLREMDI at levels or on timing necessary to support our operational costs and commercial goals. Our ability to generate revenue and become profitable depends upon our ability to successfully commercialize XOLREMDI and to obtain marketing approval and commercialize of our product candidates, including mavorixafor, or other product candidates that we may develop, in-license or acquire in the future. Even if we are able to successfully achieve regulatory approval for these potential product candidates, we are unable to predict the extent of any future losses and do not know when any of these potential product candidates will generate revenue for us, if at all. We expect to continue to incur losses for the foreseeable future, and we may never achieve or maintain profitability.
•OurUntil liquiditywe positionachieve raisesprofitability, our operations will require substantial doubtadditional funding. Our history of recurring losses and anticipated future expenditures could raise substantial doubts about our ability to continue as a going concern and we will require substantial additional funding.concern. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate any product development programs or commercialization efforts.
•We have incurred significant losses and have not generated significant revenue from product sales since our inception. We expect to continue to incur losses for the foreseeable future and we may never achieve or maintain profitability.
•Changes in estimates regarding fair value of intangible assets may result in an adverse impact on our results of operations.
•We have not generated significant revenues from product sales since inception and may never become profitable. We may never be able to generate meaningful revenues from sales of our product candidates and approved commercial products at levels or on timing necessary to support our investment and goals.
•We depend almost entirely on the success of our commercial product, XOLREMDI and on our development product candidate, mavorixafor, which we are advancing for the potential treatment of other chronic neutropenic disorders. We cannot be certain that we will be able to obtain regulatory approval for or successfully commercialize, mavorixafor for other chronic neutropenic disorders or any other product candidate for other indications.
•The regulatory review and approval processes of the FDA and comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our commercial product, XOLREMDI and product candidates, including additional indications for mavorixafor, our business will be substantially harmed.
•We depend on license agreements, including a license agreement with Genzyme, to permit us to use patents and patent applications. Termination of these rights or the failure to comply with obligations under these agreements could materially harm our business and prevent us from developing or commercializing our product candidates.
•If the commercial opportunity for mavorixafor in WHIM syndrome and other chronic neutropenic (“CN”) disorders is smaller than we anticipate, our potential future revenue from mavorixafor for the treatment of any of these diseases may be adversely affected and our business may suffer.
•We depend almost entirely on the success of our future product candidate, mavorixafor. We cannot be certain that we will be able to obtain regulatory approval for, or successfully commercialize, mavorixafor for disorders other than WHIM, including CN, or any other product candidate.
•We may develop mavorixafor, and future product candidates, in combination with other therapies, which could expose us to additional risks.
•Interim top-line and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.
•Our product candidates that have received regulatory approval may still face future development and regulatory difficulties and any approved products will be subject to extensive post-approval regulatory requirements. Additionally, anyour productapproved candidateproduct, forXOLREMDI, whichand wefuture obtainapproved marketingproducts, approvalif any, could be subject to marketing restrictions or withdrawal from the marketmarket, and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products.
•The Food and Drug Administration (“FDA”) and other domestic and foreign regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses. If we are found or alleged to have improperly promoted off-label uses, we may become subject to significant liability.
•Our commercial success depends upon attaining significant market acceptance of our approved product or product candidates, if approved, among hospitals, physicians, patients and healthcare payors.
•If we are unable to maintain effective sales and marketing capabilities or to selectively enter into agreements with third parties to sell and market our product or product candidates, we may not be successful in commercializing our product candidates that have been approved.
•Our relationships with customers and third-party payors are subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to significant penalties, including administrative, civil and criminal penalties, contractual damages, reputational harm and diminished profits and future earnings.
•Current and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product or product candidates and affect the prices we may obtain.
•We are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws and other laws governing our operations. If we fail to comply with these laws, we could be subject to civil or criminal penalties, other remedial measures and legal expenses, which could adversely affect its business, results of operations and financial condition.
•Our commercial products may become subject to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, which would harm our business.
•We have limited experience manufacturing our product or product candidates on a large clinical or commercial scale and have no manufacturing facility. We are currently dependent on a single third-party manufacturer for the manufacture of mavorixafor, the active pharmaceutical ingredient (“API”) for mavorixafor, and a single manufacturer of mavorixafor finished drug product capsules. If we experience problems with these third parties, the manufacturing of mavorixafor could be delayed, which could harm our results of operations.
•We rely on third-party contractclinical research organizations (“CROs”) to conduct our preclinical studies and clinical trials. If these CROs do not successfully carry out their contractual duties or meet expected deadlines, we may not be able to obtain regulatory approval for or commercialize our product candidates and our business could be substantially harmed.
•Disruptions in our supply chain could delay the commercial sale of our product.
•Our employees, principal investigators, CROs, CMOs and consultants may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could have a material adverse effect on our business.
•Recent laws and rulings by U.S. courts make it difficult to predict how patents will be issued or enforced in our industry.
•We could be required to incur significant expenses to obtain our intellectual property rights, and we cannot ensure that we will obtain meaningful patent protection for our product candidates.
•Obtaining and maintaining our patent protection depends on compliance with various procedural, documentary, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
•Our future success depends on our ability to retain executives and to attract, retain and motivate key personnel in a competitive environment for skilled biotechnology personnel.
•We may experience difficulties in managing the reduction in size of our organization due to our restructuring activities, and we may not achieve the expected benefits of such activities.
•The pharmaceutical industry is highly competitive and is subject to rapid and significant technological change, which could render our technologies and products obsolete or uncompetitive.
•Our internal information technology systems and infrastructure, or those of our contractors, consultants, or other third parties, may be subject to cyber attacks, or data breaches, compromises, or other security incidents, which could result in additional costs, loss of revenue, significant liabilities, harm to our reputation, and disruption of our development programs and operations.
•Although we are currently in compliance with the Nasdaq continued listing requirements, if we are unable to maintain compliance with Nasdaq’s listing requirements, our securities could be delisted, which could affect our common stock’s market price and liquidity and reduce our ability to raise capital.
•Our stock price has been and is likely to continue to be volatile and fluctuate substantially.
•“Penny stock” rules may make buying or selling our securities difficult which may make our stock less liquid and make it harder for investors to buy and sell our securities.
•If securities analysts do not publish research or reports about our business or if they publish negative evaluations of our stock, the price of our stock could decline.
•Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
•Our term loan contains restrictions that limit our flexibility in operating our business.
Until we achieve profitability, our operations will require substantial additional funding. Our history of recurring losses and anticipated future expenditures could raise substantial doubts about our ability to continue as a going concern. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate any product development programs or commercialization efforts.
Our operations have consumed a large amount of cash since inception. To date, we have funded our operations primarily with proceeds from sales of common stock, warrants, prefunded warrants, and preferred stock, proceeds from the issuance of convertible debt and borrowings under loan and security agreements. We expect to continue to incur research and development expenses as we continue to advance the clinical development of our product candidates and prepare for the launch and commercialization of any product candidates for which we receive regulatory approval. We expect to incur significant commercialization expenses related to product sales, marketing, distribution and manufacturing. Furthermore, we expect to continue to incur costs associated with operating as a public company.
As of December 31, 2025, we have cash and cash equivalents of $217.0 million and short-term marketable securities of $35.9 million, which provides funding for our operations into 2028. Until we become profitable, our operations will require us to obtain additional funding in the future. If we are unable to obtain sufficient funding when needed in the future, our business, prospects, financial condition and results of operations will be materially and adversely affected, and we may be unable to continue as a going concern. Such additional funding may include raising funds through public or private equity or debt financings, third-party funding, marketing and distribution arrangements, as well as other collaborations, strategic alliances and licensing arrangements, or any combination of these approaches. If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, limit, reduce, restructure or terminate our product development or future commercialization efforts of one or more of our product candidates, or may be forced to reduce, restructure or terminate our operations. If we are unable to continue as a going concern, we may have to liquidate our assets and may receive less than the value at which those assets are carried on our audited financial statements, and it is likely that investors will lose all or a part of their investment. In our own future required quarterly assessments, we may again conclude that there is substantial doubt about our ability to continue as a going concern, and future reports from our independent registered public accounting firm may also contain statements expressing substantial doubt about our ability to continue as a going concern. If we seek additional financing to fund our business activities in the future and there exists substantial doubt about our ability to continue as a going concern, investors or other financing sources may be unwilling to provide additional funding to us on commercially reasonable terms, if at all.
Although we believe our current cash, cash equivalents and short-term marketable securities, together with our expected decrease in annual spending under our previously announced February 2025 Restructuring and September 2025 Restructuring, respectively, will be sufficient to support our operations into 2028, subject to our continued compliance with our Hercules Loan Agreement covenants, there can be no assurance that these initiatives will be successful or that any anticipated spending reductions will be adequate, which could materially affect our future operations.
While we have successfully raised capital in the past, our ability to raise capital in future periods is not assured. We may be required to raise additional capital to satisfy the cash covenant under our existing debt facility with Hercules that requires that we maintain a minimum level of cash at a level greater than 20% of our outstanding borrowings under the Hercules Loan Agreement and subject to certain operational covenants. Based on our current cash flow projections, excluding additional sources of external financing, we anticipate that we will be able to maintain the minimum cash required to satisfy this covenant for at least the next 12 month period following the issuance of these consolidated financial statements. See also the risk factor titled “Our term loan contains restrictions that limit our flexibility in operating our business” below.
We are a commercial-stage biopharmaceutical company. Investment in biopharmaceutical product development is highly speculative because it entails substantial upfront capital expenditures and significant risk that any potential product candidate will fail to demonstrate adequate effect or an acceptable safety profile, gain regulatory approval, become commercially viable, or maintain commercial viability. Since inception, other than during the three and nine months ended September 30, 2024, we have incurred significant operating losses. Our net losses were $37.5$79.2 million, $101.2$37.5 million, and $93.9$101.2 million for the years ended December 31, 2025, 2024, 2023, and 2022,2023. respectively, and weWe generated negative operating cash flows in each of these periods. As of December 31, 2024, we had an accumulated deficit of $515.4 million. To date, we have funded our operations to date 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. We have one product approved for commercial sale, XOLREMDI, upon which we depend almost entirely on to produce revenue. XOLREMDI, which has been approved for WHIM syndrome in the U.S., 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.
We expect to continue to incur significant expenses and operating losses for at least the next several years as we conduct additionalour global, pivotal Phase 3 clinical trialstrial for ourmavorixafor productfor candidates;the continuetreatment toof discoverCN and(the develop“4WARD” additional product candidates; acquire or in-license other product candidates and technologies; maintain, expand and protect our intellectual property portfolio; hire additional clinical, scientific and commercial personneltrial); establish a commercial manufacturing source and secure supply chain capacity sufficient to provide commercial quantities of any product candidates for which we may obtain regulatory approval; seek regulatory approvals for any product candidates that successfully complete clinical trials; further grow a sales, marketing and distribution infrastructure to commercialize XOLREMDIproduct and any other productscandidates for which we may obtain regulatory approval; and add operational, financial and management information systems and personnel, including personnel to support our product development and planned future commercialization efforts. We may encounter unforeseen expenses, difficulties, complications, delays, and/or other unknown factors that may adversely affect our business. The size of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenues.
•our ability to generate revenue from XOLREMDImavorixafor;
•the scope, number, progress, duration, endpoints, cost, results and timing of clinical trials and nonclinical studies of our current or potential future product candidates, including in particular the scope, progress, duration, endpoints, cost, results and timing for completion of our 4WARD Phase 3 clinical trial of mavorixafor for the treatment of chronic neutropenic disorders;
•our ability to manage our cash expenditures in line with our budget as approved by our Board of Directors, which reflects the cost savings we anticipate from our restructuring activities executed in 2025;
•our ability to hire additional clinical, regulatory and scientific personnel; and
•incurthe additionalextent of legal, accounting and other expenses associatedthat withwe operatingincur to continue to operate as a public company.
Our operating plan and liquidity position will require substantial additional funding. Our history of recurring losses and anticipated expenditures could raise substantial doubts about our ability to continue as a going concern. If we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate any product development programs or commercialization efforts.
We may be forced to delay or reduce the scope of our development programs and/or limit or cease our operations if we are unable to obtain additional funding to support our current operating plan.
Our operations have consumed a large amount of cash since inception. 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. We expect to continue to incur research and development expenses as we continue to advance the clinical development of our product candidates and prepare for the launch and commercialization of any product candidates for which we receive regulatory approval. We expect to incur significant commercialization expenses related to product sales, marketing, distribution and manufacturing. Furthermore, we expect to incur additional costs associated with operating as a public company.
As of December 31, 2024, we have cash and cash equivalents of $55.7 million and short-term marketable securities of $46.4 million. Although we have an approved drug product, sales of our drug product over the next 12 months will not be sufficient to fund our operating expenses. Our ability to continue as a going concern will require us to obtain additional funding. If we are unable to obtain sufficient funding, our business, prospects, financial condition and results of operations will be materially and adversely affected, and we may be unable to continue as a going concern. We will require additional capital to sustain our operations, and to carry out our business plans thereafter, which may include raising funds through public or private equity or debt financings, third-party funding, marketing and distribution arrangements, as well as other collaborations, strategic alliances and licensing arrangements, or any combination of these approaches. If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, limit, reduce, restructure or terminate our product development or future commercialization efforts of one or more of our product candidates, or may be forced to reduce, restructure or terminate our operations. If we are unable to continue as a going concern, we may have to liquidate our assets and may receive less than the value at which those assets are carried on our audited financial statements, and it is likely that investors will lose all or a part of their investment. In our own future required quarterly assessments, we may again conclude that there is substantial doubt about our ability to continue as a going concern, and future reports from our independent registered public accounting firm may also contain statements expressing substantial doubt about our ability to continue as a going concern. If we seek additional financing to fund our business activities in the future and there exists substantial doubt about our ability to continue as a going concern, investors or other financing sources may be unwilling to provide additional funding to us on commercially reasonable terms, if at all.
Although we announced in February 2025 that, as part of our 2025 Restructuring, we expect to decrease annual spending by approximately $30–35 million and believe this will provide sufficient funds to support operations into the first half of 2026, there can be no assurance that these initiatives will be successful or that the anticipated funding will be adequate, which could materially affect our future operations.
While we have successfully raised capital in the past, our ability to raise capital in future periods is not assured. We will also require additional capital to satisfy the covenant under our existing debt facility with Hercules Capital, Inc. and certain affiliated entities (“Hercules”) that requires that we maintain a minimum level of cash at a level greater than 20% of our outstanding borrowings under the Loan and Security Agreement, as most recently amended in August 2023 with Hercules Capital, Inc. (the “Hercules Loan Agreement”) and subject to certain operational covenants. Based on our current cash flow projections, excluding additional sources of external financing, we anticipate that we will not be able to maintain the minimum cash required to satisfy this covenant for at least the next 12 month period following the issuance of these consolidated financial statement. See also the risk factor titled “Our term loan contains restrictions that limit our flexibility in operating our business” below.
To finance our future operations, we will need to raise additional capital, which cannot be assured. We cannot be certain that additional funding will be available on acceptable terms, or at all. If we are unable to raise additional capital when needed or in sufficient amounts or on terms acceptable to us, we could be forced to delay, reduce or eliminate our research and development programs or any future commercialization efforts of one or more of our product candidates or one or more of our other research and development initiatives. In addition, when we need to secure additional financing, such additional fundraising efforts may divert our management from our day-to-day activities, which may adversely affect our ability to develop and commercialize our product candidates. Any of these events could significantly harm our business, financial condition and prospects, and our stockholders could lose all or part of their investment in our company.
Until such time, if ever, as we can generate substantial product revenues,and licensing revenues that are in excess of our operating expense, we expect to finance our cash needs through public or private equity or debt financings, third-party funding, marketing and distribution arrangements, as well as other collaborations, strategic alliances and licensing arrangements, or any combination of these approaches. Other than our common stock purchase agreement with Lincoln Park Capital Fund LLC (“Lincoln Park”), pursuant to which Lincoln Park is obligated, subject to certain limitations and conditions, to purchase up to a remaining $47.0 million in the aggregate of shares of our common stock, weWe do not have any committed external sources of funds and may seek to raise additional capital at any time. To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a holder of our common stock. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, declaring dividends or other distributions, acquiring or licensing intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business and may result in liens being placed on additional assets such as intellectual property. For example, our debt facility with Hercules contains a minimum cash financial covenant. If we default on such indebtedness, with Hercules or a future lender, we could be required to pledge additional assets, or the lenders could enforce remedies on the current collateral.
If we raise additional funds through licensing, collaboration or similar arrangements with third parties, we may havebe required to relinquish valuable rights to our technologies, future revenue streams, research and development programs or product candidates or grant licenses on terms that are not favorable to us. If we are unable to raise additional funds through equity or debt financings or through licensing, collaboration or similar arrangements when needed, we may be required to delay, limit, reduce, restructure or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
We test our goodwill for impairment annually or more frequently if changes in circumstances or the occurrence of events suggest impairment exists. Any significant change in market conditions, including a sustained decline in our stock price, that indicate a reduction in carrying value may give rise to a significant impairment charge in the period that the change becomes known.
Management's Discussion & Analysis (MD&A)
New heading “Private Placement Financing and Management Changes”
New heading “Strategic Restructurings”
New heading “Regulatory Update and Out-License Agreements”
New heading “License and Other”
New heading “Operating Cost and Expenses:”
New heading “Operating Activities”
New heading “Investing Activities”
New heading “Financing Activities”
New heading “Capital Requirements”
Removed heading “For the discussion of the financial condition and results of operations for the year ended December 31, 2023 compared to the year ended December 31, 2022, refer to “Management’s Discussion and Analysis of Financial Condition and Results of Operations” to our Annual Report on Form 10-K filed with the SEC on March 21, 2024.”
Removed heading “XOLREMDI Commercial Launch”
Removed heading “Phase 2 Clinical Study in Chronic Neutropenia”
Removed heading “Phase 3 Clinical Trial in Chronic Neutropenia”
Removed heading “Norgine Agreement”
Removed heading “Comparison of the Years Ended December 31, 2024 and 2023”
Removed heading “Gain on Sale of Non-Financial Asset”
Removed heading “Operating Expenses”
Removed heading “Debt Obligations”
Removed heading “Lease Obligations”
Removed heading “Funding Requirements”
Removed heading “Recently Issued Accounting Pronouncements”
Largest changes
“We have a covenant (the “Minimum Cash Covenant”) under our Hercules Loan Agreement that effective January 31, 2025 requires that we maintain a minimum level of cash of $15 million, representing 20% of outstanding borrowings under the Hercules Loan Agreement. Based on our current operating plan, which includes estimates of anticipated cash inflows from product sales and cash outflows from operating expenses, we believe there is a risk that we will not meet the conditions of the Minimum Cash Covenant within the 12-month period from the issuance date of these consolidated financial statements. …”see in full comparison
•Insee in full comparisonFebruarythe first quarter of 2025, we implemented a strategic restructuring of our business operations, workforce and capital spending to focus efforts on advancing mavorixafor to treatthosepatients withchronic neutropenia, while also optimizing its U.S. promotion of XOLREMDI (the “2025 Restructuring”).CN. As part ofthethis2025 Restructuring,restructuring, we (i) implemented a net reduction of our employee headcount by 43 employees,orrepresenting approximately 30% of our totalworkforce.workforce,TheincludingstrategicourrestructuringU.S.activitiescommercialincludefield(i) discontinuing of research efforts,team, (ii)closingcommenced the closure of our research and development facility in Vienna, Austria, (iii)pausingpaused our pre-clinical drug candidateprograms,programs and (iv)scaling the U.S. commercial field team and supporting roles across our business and (v) streamliningstreamlined other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia. Weestimate that the workforce reduction will be substantially completed in the first quarter of 2025. We estimate that it will incurincurred charges of approximately$3.0$2.1 million for severance and other employee termination-relatedcosts, primarily in the first quarter of 2025. We expect the 2025 Restructuring will decrease annual spending by $30 to 35 million and believe it will have sufficient funds to support operations into the first half of 2026. The estimate ofcoststhat we expect to incurrelated tothethis2025strategicRestructuring as well as the decrease in annual spending, and the timing thereof are subject to a number of assumptions and actual results may differ.restructuring.
“•In the third quarter 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 CN. As part of this initiative, we further reduced our workforce by approximately 50%. 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. …”see in full comparison
“These strategic restructuring actions have allowed us to decrease our operating expenses, including research and development and general and administrative expenses, from $143.2 million in 2024 to $116.2 million in 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.”see in full comparison
“To the extent that we raise additional capital through future equity offerings or debt financings, the ownership interest of our stockholders may be materially diluted, and the terms of such securities could include liquidation or other preferences that adversely affect the rights of our stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include restrictive covenants that limit our ability to take specified actions, such as incurring additional debt, making capital expenditures or declaring dividends. …”see in full comparison
Full comparison: every changed paragraph (103)
The following discussion and analysis is intended to provide material information around events and uncertainties known to management that are relevant to an assessment of the financial condition and results of operations of X4 Pharmaceuticals and should therefore be read in conjunction with our audited Consolidated Financial Statements and the related notes thereto and other disclosures included as part of this Annual Report on Form 10-K, including the disclosures under Part I, Item 1A. Risk Factors.
You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and the related notes and the other financial information included elsewhere in this Annual Report. Some of the information contained in this discussion and analysis or set forth elsewhere in this Annual Report, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Risk Factors” section of this Annual Report, our actual results could differ materially from the results described in or implied by these forward-looking statements.
For the discussion of the financial condition and results of operations for the year ended December 31, 2023 compared to the year ended December 31, 2022, refer to “Management’s Discussion and Analysis of Financial Condition and Results of Operations” to our Annual Report on Form 10-K filed with the SEC on March 21, 2024.
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 granulocyte colony-stimulating factor (“G-CSF”) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia (“CN”) who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll up to 176 patients, with full enrollment expected in the third quarter of 2026. The U.S. Food and Drug Administration (“FDA”) has granted Fast Track designation to mavorixafor for the treatment of CN, which 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). CN 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 arehave aone biopharmaceutical company discovering, developing, and commercializing novel therapeutics for the treatment of rare diseases and those with limited treatment options, with a focus on conditions resulting from dysfunction of the immune system. On April 29, 2024, we announced that the U.S. FDAcommercially approved ourproduct, NDAXOLREMDI for® mavorixafor,(mavorixafor), which ishas beingreceived marketedaccelerated approval in the U.S.United underStates from the trade name XOLREMDI,FDA for use as an oral, once-daily therapy in patients aged 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 (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) is a rare combined primary immunodeficiency and chronic neutropenicCN disorder. ConcurrentIn connection with our long term strategy to successfully complete the U.S.4WARD approvalPhase of3 XOLREMDItrial in patients with moderate and pursuantsevere toCN, itswe Rareare Pediatricno Diseaselonger designation,prioritizing investment in the FDAWHIM granted us a PRV that we sold to another drug sponsor shortly thereafter.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 shares of 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 and their employment was terminated. Dr. Ragan also resigned from the Company’s Board of Directors (the “Board”), and Michael Wyzga transitioned from Board Chair to Lead Independent Director. The Board appointed Adam R. Craig, M.D., Ph.D, MBA as Executive Chairman, John Volpone as President and subsequently as Chief Operating Officer, and David Kirske as Chief Financial Officer.
XOLREMDI Commercial Launch
We are currently engaged in our U.S. launch of XOLREMDI in WHIM syndrome, continuing our engagements with physicians and rare disease patient advocacy organizations and our disease-awareness campaign to further the understanding of WHIM syndrome and educate patients and physicians on the importance and benefits of early diagnosis. We have entered into agreements with a third-party logistics organization and a specialty pharmacy to support the distribution of XOLREMDI in the U.S., to mitigate barriers to product access, and to provide a suite of patient support services to help patients through their treatment journey. We submitted a Marketing Authorisation Application (“MAA”) to the European Medicines Agency (“EMA”) in early 2025 seeking regulatory approval to commercialize mavorixafor for WHIM syndrome outside of the U.S. Such MAA was accepted for processing by the EMA in January 2025. As discussed further below, on January 13, 2025, we entered into a License and Supply Agreement (the “Norgine Agreement”) with Norgine Pharma UK Limited (“Norgine”), pursuant to which Norgine is 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. In February 2025, we announced that we had entered into an agreement with taiba rare to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in select Middle East and North African countries. We continue to explore additional potential commercial and distribution opportunities in geographies where we may be able to efficiently leverage any of our regulatory approvals.
The U.S. approval of XOLREMDI in the WHIM syndrome indication is the first for mavorixafor, which is an orally active, selective antagonist of chemokine receptor CXCR4, a key regulator of the movement of immune cells throughout the body. Due to its ability to increase the mobilization of white blood cells from the bone marrow into the bloodstream, we believe that mavorixafor has the potential to provide therapeutic benefit across a variety of immune system disorders in addition to WHIM syndrome.
Phase 2 Clinical Study in Chronic Neutropenia
Following positive results from a Phase 1b clinical study of a single dose of mavorixafor in people with idiopathic, cyclic, and congenital chronic neutropenia (“CN”), we recently completed and announced positive results from a Phase 2 clinical study evaluating the durability of effect, safety, and tolerability of chronic dosing of once-daily oral mavorixafor with or without concurrent treatment with injectable granulocyte colony-stimulating factor (“G-CSF”) in the same patient population.
The results from the completed six-month study showed that once-daily oral mavorixafor was generally well tolerated and durably increased participants’ absolute neutrophil counts (“ANC”) both as a monotherapy and in combination with G-CSF, the only therapy approved in the U.S. for severe chronic neutropenia. In addition, when tested in combination with G-CSF, mavorixafor treatment enabled physicians to substantially reduce G-CSF dosing while maintaining normal mean ANC levels.
Phase 3 Clinical Trial in Chronic Neutropenia
We continue to progress our global, pivotal Phase 3 clinical trial, (the “4WARD” study) 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 CN who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants. We believe we are on track to complete enrollment in the 4WARD study in the third or fourth quarter of 2025.
We believe that successfully developing and commercializing mavorixafor to provide a new therapeutic option to individuals diagnosed with certain immunodeficiencies has the potential to revolutionize the current treatment landscape, which is principally served by injectable and infused therapies.
Q4 2025 RestructuringEquity 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 to purchase shares of common stock, raising net proceeds of $145.6 million, net of underwriting discounts and estimated offering expenses.
Strategic Restructurings
•In Februarythe 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 thosepatients with chronic neutropenia, while also optimizing its U.S. promotion of XOLREMDI (the “2025 Restructuring”).CN. As part of thethis 2025 Restructuring,restructuring, we (i) implemented a net reduction of our employee headcount by 43 employees, orrepresenting approximately 30% of our total workforce.workforce, Theincluding strategicour restructuringU.S. activitiescommercial includefield (i) discontinuing of research efforts,team, (ii) closingcommenced the closure of our research and development facility in Vienna, Austria, (iii) pausingpaused our pre-clinical drug candidate programs,programs and (iv) scaling the U.S. commercial field team and supporting roles across our business and (v) streamliningstreamlined other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia. We estimate that the workforce reduction will be substantially completed in the first quarter of 2025. We estimate that it will incurincurred charges of approximately $3.0$2.1 million for severance and other employee termination-related costs, primarily in the first quarter of 2025. We expect the 2025 Restructuring will decrease annual spending by $30 to 35 million and believe it will have sufficient funds to support operations into the first half of 2026. The estimate of costs that we expect to incur related to thethis 2025strategic Restructuring as well as the decrease in annual spending, and the timing thereof are subject to a number of assumptions and actual results may differ.restructuring.
•In the third quarter 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 CN. As part of this initiative, we further reduced our workforce by approximately 50%. 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.
These strategic restructuring actions have allowed us to decrease our operating expenses, including research and development and general and administrative expenses, from $143.2 million in 2024 to $116.2 million in 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. In February 2026, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending the grant of marketing authorization, under exceptional circumstances, for mavorixafor for the treatment of WHIM syndrome in the European Union. The positive opinion has been submitted to the European Commission for review, and we expect the European Commission to issue a final approval decision in the second quarter of 2026. 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.
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 for the 2025 Restructuring above.
Norgine Agreement
On January 13, 2025, we entered into the Norgine Agreement, pursuant to which Norgine is granted an exclusive license to distribute, market and sell our product mavorixafor for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia and New Zealand following regulatory approval. Pursuant to the terms of the Norgine Agreement, we received an upfront payment of €28.5 million upon signing of the agreement and could receive up to €226.0 million upon the achievement of certain regulatory, commercial and sales milestones. We will also receive escalating, double-digit royalties of up to mid-twenties on any future net sales of our drug product by Norgine. Norgine will be responsible for all market access and commercialization activities and will eventually hold all marketing authorizations in the licensed territories. We will manufacture and supply drug product to Norgine for a supply price derived from our product manufacturing cost plus a low double-teen digit margin.
Comparison of the Years Ended December 31, 2024 and 2023
The following table summarizes the results of our operations for the periodsyears indicatedended December 31, 2025 and 2024:
Revenue
License and Other
In January 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 year ended December 31, 2025, we recognized $27.6 million for the delivery of the license and $1.0 million for the provision of research and development services to Norgine. We had no license or other revenue during the year ended December 31, 2024.
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 years ended December 31, 2025 and 2024, and for each of the quarterly periods therein.
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 were approximately 10% and 9% for the years ended December 31, 2025 and 2024, respectively.
Operating Cost and Expenses:
Net revenue from the sale of our drug product was $2.6 million for the year ended December 31, 2024. There was no product revenue in the prior periods. We sell our approved drug product in the U.S. to a specialty pharmacy that dispenses the product to patients who have been prescribed our drug product by their health-care providers. We also sell our drug product through international distributors. There were no sales to international distributors for the year ended December 31, 2024. Net revenue includes reserves for distributor discounts, estimated rebates that we may owe to U.S. government payors, future co-pay assistance payments that we may owe for patients who enroll in our patient assistance program, and for potential product returns. As we have just recently launched our first drug product in the U.S. in 2024, we have limited history of returns or downstream rebates or co-pay assistance payments. Therefore, we expect to adjust these estimates quarterly as new information becomes available.
For the year ended December 31, 2024, cost of revenue was $0.8 million. There was no cost of revenue in prior periods.
Cost of revenue primarily consists of $0.5 million of amortization of an intangible asset related to accrued and paid milestone payments associated with our license agreement (the “Genzyme licenseAgreement”) agreement,with $0.2Genzyme millionCorporation (“Genzyme”), a wholly owned subsidiary of sales-basedSanofi, and sales and sublicense-based royalty payments accrueddue andthereunder. paidCost underof revenue increased $5.0 million in the year ended December 31, 2025, as compared to the prior year, primarily due to additional royalties in the current year associated with sublicense income from our GenzymeNorgine license agreement, and $0.1 million of drug product costs.Agreement.
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 XOLREMDI 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. The following table shows external costs incurred by product candidate (primarily external contract research organization costs) and unallocated research and development costs, primarily consisting of employee salaries and related expense for our research and development organization.
Research and development expenses consist primarily of costs incurred in connection with the development of our product candidates, including employee salaries and related expenses for personnel in our clinical operations, biostatistics, medical affairs, manufacturing and technical, quality and regulatory affairs departments; external expenses incurred in connection with the clinical development of our product candidates, including under agreements with third parties, such as consultants and contract research organizations (“CROs”); the cost of manufacturing drug products for use in our clinical trials, including under agreements with third parties, such as consultants and contract manufacturing organizations (“CMOs”); facilities, depreciation and other expenses, which include direct or allocated expenses for rent and maintenance of facilities and insurance; and costs related to compliance with regulatory requirements. We expense research and development costs as incurred. We capitalized as intangible assets certain milestone payments due under our key in-license agreement.
Research and development expenses increaseddecreased by $9.6$8.9 million forin the year ended December 31, 2024,2025 as compared to the prior year primarily due to an increasedecreases 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 year ended December 31, 2025, unallocated expense. Unallocated expense increasedincludes primarily$2.0 due to an increasemillion in personnelseverance withincharges ourfor terminated employees. The overall decrease in research and development functions, primarilyexpenses in ourthe current year was partially offset by higher clinical operations,costs biostatistics,associated medicalwith affairs,our manufacturing4WARD and technical, and quality organizations.trial.
We expect that our research and development expenses, particularly for our mavorixafor programs, will be relatively consistent over the next several years as we continue to conduct our clinical trials of mavorixafor in chronic neutropenic disorders. Research and development expenses related to our X4P-002 and X4P-003 programs was not significant in 2024 or 2023 relative to our overall research and development expenses.
Selling, General and Administrative Expenses
Selling, generalGeneral and administrative expenses consist primarily of salaries and related costs, including stock-based compensationcompensation, for personnel in sales and marketing, executive, financefinance, and administrative functions. Selling, generalGeneral and administrative expenses also include direct and allocated facility-related costs,costs as well as professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services.
General and administrative expenses decreased by approximately $18.1 million in the year ended December 31, 2025 as compared to the prior year. General and administrative expenses include $5.0 million in severance charges for terminated employees in the year ended December 31, 2025. The decrease to general and administrative expenses was primarily due to an $8.0 million decrease in compensation expense due to lower head count in our sales, general and administrative functions, and a decrease of $9.0 million in sales and marketing expenses in the current year as compared to the prior year during which we incurred commercialization sales and marketing launch costs related to XOLREMDI. These decreases in general and administrative expenses were partially offset by higher severance costs associated with our 2025 strategic restructurings and higher legal costs in the current year.
Selling, general and administrative expenses increased by $26.0 million to $61.5 million for the year ended December 31, 2024 as compared to $35.5 million in the prior year. The increase in selling, general and administrative expense was primarily due to an increase of $15.2 million in selling and marketing expenses, including increases in sales and marketing personnel and external marketing costs to support the ongoing launch activities of our approved product, XOLREMDI in the U.S. Selling, general and administrative expenses also increased due to an increase of $10.8 million in general and administrative expenses, primarily due to higher legal, corporate communications, accounting, information technology, and professional service costs associated with the ongoing commercialization of our drug product.
Gain on Sale of Non-Financial Asset
During the year ended December 31, 2024, we entered into contractual arrangement with a third party that transferred the rights to a Priority Review Voucher (“PRV”) awarded to the Company as a result of the FDA’s approval of XOLREMDI. The PRV was accounted for as an intangible asset with no accounting cost basis. The third party purchased the PRV for $105.0 million. As a result of the transfer of control of the PRV to the third party, the Company derecognized the associated intangible asset and recorded a gain through “gain on transfer of nonfinancial assets.” There was no such transaction in year ended December 31, 2023 and we do not expect similar PRV sales in the future.
Operating Expenses
Our operating expenses decreased by $68.6 million for the year ended December 31, 2024 as compared to the prior year. Excluding the impact of the PRV sale, operating expenses increased $36.4 million in the current year as compared to the prior year, primarily due to an increase in head count to support our U.S. launch of XOLREMDI and to support our 4WARD clinical trial. As a result of the strategic restructuring of our workforce and capital spending to focus efforts on advancing mavorixafor to treat those with chronic neutropenia, while also optimizing its U.S. promotion of XOLREMDI, we expect that our operating expenses will decrease by approximately $30.0 million to $35.0 million in 2025.
Other Income (Expense) Income,, Net
Other income (expense), net, increased approximately $8.4 million in the year ended December 31, 2025 as compared to the prior year primarily due to higher gains in the current year on fair value adjustments related to our Class C warrants, partially offset by lower interest income earned on our marketable security investment portfolio.
The increase in other (expense) income, net, of $7.2 million for the year ended December 31, 2024 as compared to 2023 was primarily due (i) lower income resulting from the decrease in the fair value of outstanding Class C warrants, which are accounted for as a liability and are remeasured to fair value each period, as compared to the prior year and, (ii) an increase in interest expense associated with the Hercules Loan agreement due to more borrowings outstanding during the current period. These increases were partially offset by higher interest income earned in the current year on our marketable security portfolio as compared to the prior year.
Provision for Income Taxes
Our income tax provision of $41.0 thousand for the year ended December 31, 2025, which was primarily related to our Austrian subsidiary, was lower than our income tax provision in the prior year. Our income tax provision for the year ended December 31, 2024 of $0.3 million reflected U.S. federal and state taxable income that included the sale of a priority review voucher, generating $105.0 million of taxable income, partially offset by available deductions, net operating loss carryforwards, which were limited under IRC 382 due to several qualifying ownership changes, and available research and development credits. 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.
For the year ended December 31, 2024, we recorded an income tax provision of $0.3 million related to our U.S. operations and related to our Austrian subsidiary. Our tax provision in the year ended December 31, 2023 was related to our U.S. security corporation, which holds our investment portfolio, and our Austrian subsidiary. During the current year, our U.S. entity generated taxable income due to the sale of a PRV voucher, which generated $105.0 million of taxable income, and product revenue, partially offset by available deductions, net operating loss carryforwards and available research and development credits. Our net operating loss carryforwards were limited under IRC 382 due to several qualifying changes in ownership resulting from certain of our equity financings and therefore we were not able to fully offset our taxable income in the current year. We do not expect to record a significant income tax benefit or expense for several years until such time as we begin to generate meaningful and sustained taxable income in our U.S. jurisdiction.
To date, we have funded our operations primarily with proceeds from sales of common stock, warrants andwarrants, 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.
ATM Sales Agreement — On August 7, 2020, we have entered into a Controlled Equity OfferingSM Sales Agreement (“ATM Sales Agreement”), with B. Riley Securities, Inc., Cantor Fitzgerald & Co., and Stifel, Nicolaus & Company, Incorporated (collectively the “Sales Agents”), pursuant to which we may offer and sell, at our sole discretion through one or more of the Sales Agents, shares of our common stock having an aggregate offering price of up to $75 million. To date, we have sold approximately $14.3 million of our common stock, net of offering costs, under the ATM Sales Agreement.
What changed in the latest 10-Q
Risk Factors
Our business is subject to various risks, including those described in Part I, Item 1A, “Risk Factors” of our Annual Report on Form 10-K for the year ended December 31, 2025. There have been no material changes from the risk factors disclosed in Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2025.
No wording changes found in this section.
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Management's Discussion & Analysis (MD&A)
Removed heading “Operating Cost and Expenses:”
Largest changes
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 granulocyte colony-stimulating factor (“G-CSF”)) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia (“CN”) who are experiencing recurrent and/or serious infections.see in full comparisonTheAs52-weekparttrial isof arandomized,completedouble-blind, placebo-controlled, multicenter study aiming to enroll up to 176 patients, with full enrollment expected by the endoverhaul of thethirdclinicalquarteroperating infrastructure, we have transitioned management of2026.theThe4WARD trial to a premiere Clinical Research Organization (CRO) to further strengthen the study execution and support completion of enrollment. We plan to meet with the U.S. Food and Drug Administration (“FDA”)hastograntedrevisitFasttheTracksampledesignationsize of the 4WARD trial and expect tomavorixaforprovideforan update on our meeting with thetreatmentFDA and the completion ofCN,enrollmentwhichbyisthedefined as periods lasting more than three months persistently or intermittently where there are abnormally low levelsend ofneutrophils circulating intheblood,thirdand may be idiopathic (of unknown origin), cyclic (episodes typically occurring every three weeks), or congenital (of genetic causation). CN 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.quarter.
“The FDA has granted Fast Track designation to mavorixafor for the treatment of CN, which 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). CN disorders are rare blood conditions similarly characterized by increased risks of infections and cancer due to abnormally low levels of neutrophils in the body. …”see in full comparison
see in full comparisonFollowing our strategic restructuring announced during the first quarter of 2025, substantiallySubstantially all of our research and development has been focused on our one product candidate, mavorixafor (X4P-001)., specifically for the CN indication in the U.S. 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.
“During the six months ended June 30, 2026, net cash used in operating activities was $40.4 million, primarily resulting from our net losses of $36.4 million adjusted for non-cash items of $7.2 million, primarily consisting of stock-based compensation, and changes in operating assets and liabilities of $11.2 million, primarily due to increases is in accounts receivable due to increases in revenue, and decreases in accounts payable and accrued expenses due to timing of vendor payments. …”see in full comparison
“During the three and six months ended June 30, 2026, license and other revenue under the Norgine Agreement was primarily comprised of $5.5 million of license revenue associated with the achievement of a regulatory milestone and $1.0 million and $1.2 million, respectively, of sales of drug supply to Norgine for early-access programs and future commercial sales. License and other revenue was not significant in the three months ended June 30, 2025. …”see in full comparison
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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 granulocyte colony-stimulating factor (“G-CSF”)) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia (“CN”) who are experiencing recurrent and/or serious infections. TheAs 52-weekpart trial isof a randomized,complete double-blind, placebo-controlled, multicenter study aiming to enroll up to 176 patients, with full enrollment expected by the endoverhaul of the thirdclinical quarteroperating infrastructure, we have transitioned management of 2026.the The4WARD trial to a premiere Clinical Research Organization (CRO) to further strengthen the study execution and support completion of enrollment. We plan to meet with the U.S. Food and Drug Administration (“FDA”) hasto grantedrevisit Fastthe Tracksample designationsize of the 4WARD trial and expect to mavorixaforprovide foran update on our meeting with the treatmentFDA and the completion of CN,enrollment whichby isthe defined as periods lasting more than three months persistently or intermittently where there are abnormally low levelsend of neutrophils circulating in the blood,third and may be idiopathic (of unknown origin), cyclic (episodes typically occurring every three weeks), or congenital (of genetic causation). CN 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.quarter.
The FDA has granted Fast Track designation to mavorixafor for the treatment of CN, which 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). CN 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.
In January 2025, the EMA validated for processing our Marketing Authorization Application (“MAA”) seeking regulatory approval to commercialize mavorixafor for WHIM syndrome in the European Union. On April 29, 2026, the European Commission granted marketing authorization for XOLREMDI® (mavorixafor) capsules for the treatment of patients with WHIM syndrome in the European Union. Pursuant to our license and supply agreement (the “Norgine Agreement”) with Norgine Pharma UK Ltd. (“Norgine”), Norgine elected that we transfer the approved MAA to them. We completed this transfer in July 2026.
The following table summarizes the results of our operations for the three and six months ended MarchJune 31,30, 2026 and 2025:
Net product sales were $2.5$2.4 million and $0.9$1.7 million for the three months ended MarchJune 31,30, 2026 and 2025, respectively, and were $4.8 million and $2.7 million for the six months ended June 30, 2026 and 2025, respectively. Net product revenue growth over the prior year quarterperiods was primarily due to an increase in the number of patients who have been prescribed the Company’s drug product. Gross-to-net adjustments were approximately 10% in the first quarterhalf of 2026. Co-pay assistance payments and rebates to U.S. government payors have comprised the majority of our gross-to-net revenue adjustments.
During the three and six months ended June 30, 2026, license and other revenue under the Norgine Agreement was primarily comprised of $5.5 million of license revenue associated with the achievement of a regulatory milestone and $1.0 million and $1.2 million, respectively, of sales of drug supply to Norgine for early-access programs and future commercial sales. License and other revenue was not significant in the three months ended June 30, 2025. For the six months ended June 30, 2025, license and other revenue was primarily comprised of $27.6 million in license revenue in connection with the delivery of a license upon the execution of the Norgine Agreement and $0.5 million for the provision of research and development services thereunder.
During the three months ended March 31, 2026, other revenue under our license and supply agreement (the “Norgine Agreement”) with Norgine Pharma UK Ltd. (“Norgine”) was comprised of $0.2 million for the provision of research and development services and delivery of drug product supply to Norgine. For the three months ended March 31, 2025, we recognized $27.6 million in license revenue in connection with the delivery of the license and $0.3 million for the provision of research and development services under the Norgine Agreement.
Operating Cost and Expenses:
Cost of revenue primarily consists of amortization of an intangible asset related to accrued and paid milestone payments associated with our Genzyme license agreement, cost of manufacturingmanufactured drug product sold, and sales-based or sublicense-based royalty payments due under our Genzyme license agreement. Cost of revenue decreasedincreased $4.1$1.3 million in the three months ended MarchJune 31,30, 2026 as compared to the same period in the prior year due to an increase in net product sales and an increase in drug supply sales. Cost of revenue decreased $2.8 million in the six months ended June 30, 2026, as compared to the same period in the prior year, primarily due to royalties in the prior year royalties due under our Genzyme license agreement associated with sublicense income from ourthe Norgine Agreement that did not reoccur in the current period, partially offset by higher intangible amortization in the current period.
Research and development expenses consist primarily of costs incurred in connection with the development of our one product candidates,candidate, including employee salaries and related expenses, clinical development expenses, and 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.
Following our strategic restructuring announced during the first quarter of 2025, substantiallySubstantially all of our research and development has been focused on our one product candidate, mavorixafor (X4P-001)., specifically for the CN indication in the U.S. 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.
Research and development expenses decreased by $3.0$3.3 million and $6.4 million in the three and six months ended MarchJune 31,30, 20262026, respectively, as compared to the same periodperiods in the prior year. The decrease was primarily due to lower compensation and related benefit costs as a result of strategic restructuring actions implemented in 2025the prior year, partially offset by higher clinical costs, primarily CRO costs as we continuerelated to progress our 4WARD trial.
General and administrative expenses decreased by $8.1$1.0 million inand $9.1 million for the three and six months ended MarchJune 31,30, 20262026, respectively, as compared to the same period in the prior year. The decrease for the three month period was primarily due to a $4.0 million reduction in consultinglegal, fees,IT approximately $2.0 million in lowerand sales and marketing costs, $0.7partially millionoffset ofby nethigher stock-based compensation expense. The decrease for the six month period was primarily due to a significant reduction in sales and marketing expenses related to our approved drug product, a reduction in outside legal expenses, and reductions in compensation costs due to lower head count in general and administrative functions, and lower professional services costs in the current period as compared to the prior year.functions.
Other Income (Expense),Income, Net
Other income, net, decreased approximately $9.7$0.3 million and $10.0 million in the three and six months ended MarchJune 31,30, 20262026, respectively, as compared to the same period in the prior year primarily due to lower gains in the current period on fair value adjustments related to our Class C warrants, partially offset by higher interest income on our marketable security investment portfolio.
ATM Sales Agreement. WeOn wereMay a6, party2026, towe aentered Controlledinto Equityan OfferingSMOpen SalesMarket AgreementSale AgreementSM (“ATM”), dated as of August 7, 2020, pursuant tounder which we periodicallymay soldsell sharesup ofto our common stock through one or more investment banks. We have sold $24.2$75.0 million of our common stock,shares netthrough an investment bank. Through August 6, 2026, there have been no sales of offeringour costs,common stock under the ATM. We terminated theour previous ATM effectiveagreement on March 30, 2026.
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 MarchJune 31,30, 2026. 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 11 to our Annual Report on Form 10-K as filed with the SEC on March 17, 2026 for a full description of our Hercules Loan Agreement.
During the six months ended June 30, 2026, net cash used in operating activities was $40.4 million, primarily resulting from our net losses of $36.4 million adjusted for non-cash items of $7.2 million, primarily consisting of stock-based compensation, and changes in operating assets and liabilities of $11.2 million, primarily due to increases is in accounts receivable due to increases in revenue, and decreases in accounts payable and accrued expenses due to timing of vendor payments. During the six months ended June 30, 2025, net cash used in operating activities was $42.3 million, primarily resulting from net losses of $25.5 million adjusted for net non-cash income (expense) of $9.3 million, which includes a $13.5 million gain for the adjustment of our Class C warrant liability to fair value. Net cash used in operating activities also includes $7.5 million of changes to operating assets and liabilities primarily related to a reduction in accrued expenses.
During the three months ended March 31, 2026, net cash used in operating activities was $19.6 million, primarily resulting from our net losses of $20.2 million. Net cash used in operating activities for the three months ended March 31, 2025 was $12.4 million, primarily resulting from operating expenses of approximately $38.2 million, approximately $2.5 million in severance and related payments, and $6.1 million for the payment of our annual bonuses, partially offset by license and net product sale receipts of $31.2 million and other changes in working capital.
During the threesix months ended MarchJune 31,30, 2026,2026 and 2025, cash provided by investing activities of $19.3$18.3 million and $20.0 million, respectively, was primarily due to net maturities and sales of short-term marketable securities. During the three months ended March 31, 2025, cash used in investing activities of $3.1 million was due to net investment in short-term marketable securities.
There were no significant cash flows from financing activities during the six months ended June 30, 2026. During the six months ended June 30, 2025, cash provided by financing activities of $5.6 million was primarily due to sales of our common stock through our ATM and a common stock purchase agreement.
There were no significant cash flows from financing activities during the three months ended March 31, 2026 and 2025.
Based on our cash, cash equivalents and marketable securities on hand as of MayAugust 6, 2026 and our current operating plan, we believe that our cash, cash equivalents and marketable securities will allow us to fund our operations and debt obligations for at least the next 12 months.
During the three months ended MarchJune 31,30, 2026, there were no material changes to our critical accounting policies as reported for the year ended December 31, 2025 as part of our Annual Report on Form 10-K. See Note 1 to the unaudited interim condensed consolidated financial statements included in this Quarterly Report on Form 10-Q under the heading Recently Issued Accounting Standards Not Yet Adopted for new accounting pronouncements or changes to the accounting pronouncements during the three months ended MarchJune 31,30, 2026.
XFOR insider buying and selling (Form 4)
Since 2026-04-11, insiders reported open-market purchases in 0 Form 4 filings and open-market sales in 5 filings (3 insiders, 4 trade dates, 109,283 shares, about $462.5K). Net open-market shares: -109,283 (purchases minus sales); net value about -$462.5K.Totals add up every open-market (code P and S) line in those filings, using the prices reported in the filings. Awards, option exercises, tax withholding and gifts are listed below but not counted.
| Trade date | Insider | Transaction | Shares | Price | Value |
|---|---|---|---|---|---|
| 2026-10-01 | Kirske David |
Open-market sale | 11,767 | $2.86 | $33.7K |
| 2026-08-12 | Volpone John |
Open-market sale | 24,576 | $4.44 | $109.1K |
| 2026-08-12 | Craig Adam R |
Open-market sale | 47,436 | $4.44 | $210.6K |
| 2026-06-30 | Kirske David |
Open-market sale | 8,543 | $4.26 | $36.4K |
| 2026-05-12 | Kirske David |
Open-market sale | 16,961 | $4.29 | $72.8K |
Well-known investors holding XFOR (13F)
| Investor | Quarter | Shares | Reported value | % of their 13F | Change vs prior quarter |
|---|---|---|---|---|---|
| Millennium Management (Israel Englander) | 2026-06-30 | 1,210,514 | $5.0M | — | Sold out |
| Point72 Asset Management (Steve Cohen) | 2026-06-30 | 41,072 | $191.8K | 0.0% | New position |
| Two Sigma Investments | 2026-06-30 | 19,426 | $90.7K | 0.0% | Added 40% |
| Renaissance Technologies | 2026-06-30 | 18,100 | $84.5K | 0.0% | New position |