X4 Pharmaceuticals, Inc
For the period ended Sep 30, 2025. Filed Nov 5, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 5, 2025
XFOR$3.18+3.6%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-08.
0001628280-25-039327
Item 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-05.
0001501697-25-000043
Item 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 79 previous paragraphs and 99 current paragraphs.
30 unchanged; 19 reworded; 49 added; 28 removed; 2 previous and 1 current paragraphs rearranged.
Tables are not compared: 4 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (49)
Current report
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.
Current report
Forward-Looking Statements
Current report
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:
Current report
•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;
Current report
•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;
Current report
•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;
Current report
•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;
Current report
•the timing of enrollment in, and our ability to successfully enroll, our clinical trials, including our Phase 3 4WARD clinical trial;
Current report
•the potential benefits, including clinical utility, that may be derived from XOLREMDI or any of our product candidates;
Current report
•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;
Current report
•our plans to research, develop, manufacture and commercialize XOLREMDI or our product candidates;
Current report
•the timing of our regulatory filings for our product candidates, along with regulatory developments in the United States and other foreign countries;
Current report
•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;
Current report
•the benefits of U.S. Food and Drug Administration and European Commission designations, including, without limitation, Fast Track, orphan and Breakthrough Therapy;
Current report
•our commercialization, marketing and manufacturing capabilities and strategy;
Current report
•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;
Current report
•our ability to attract and retain qualified employees and key personnel;
Current report
•our competitive position and the development of and projections relating to our competitors or our industry;
Current report
•our expectations regarding our ability to obtain and maintain intellectual property protection;
Current report
•the success of competing therapies that are or may become available;
Current report
•our estimates and expectations regarding future operations, financial position, revenues, costs, expenses, uses of cash, capital requirements or our need for additional financing;
Current report
•our plans to in-license, acquire, develop and commercialize additional product candidates;
Current report
•the impact of laws and regulations;
Current report
•our plans to identify additional product candidates with significant commercial potential that are consistent with our commercial objectives;
Current report
•our strategies, prospects, plans, expectations or objectives; and
Current report
•other risks and uncertainties, including those listed under the section titled “Risk Factors” in this Quarterly Report.
Current report
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.
Current report
Private Placement Financing and Management Changes
Current report
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.
Current report
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.
Current report
Q4 2025 Equity Financing
Current report
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.
Current report
Strategic Restructurings
Current report
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.
Current report
Regulatory Update and Out-License Agreements
Current report
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 million for the provision of research and development services. We had no license or other revenue during the three or nine months ended September 30, 2024.
Current report
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.
Current report
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.
Current report
Operating Cost and Expenses:
Current report
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.
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Removed paragraphs (28)
Previous report
Item 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
Previous report
The following information should be read in conjunction with our unaudited condensed consolidated financial statements and the notes thereto included in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K, which was filed with the Securities and Exchange Commission (“SEC”), on March 26, 2025, (the “Annual Report”). This discussion and analysis contains forward-looking statements that involve significant risks and uncertainties. Our actual results, performance or experience could differ materially from what is indicated by any forward-looking statement due to various important factors, risks and uncertainties, including, but not limited to, those set forth under “Risk Factors” included elsewhere in this Quarterly Report on Form 10-Q.
Previous report
We are a 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. FDA approved our NDA for mavorixafor, which is being marketed in the U.S. under the trade name XOLREMDI, 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 neutropenic disorder. Concurrent with the U.S. approval of XOLREMDI and pursuant to its Rare Pediatric Disease designation, the FDA granted us a PRV that we sold to another drug sponsor shortly thereafter.
Previous report
XOLREMDI Global Commercial Launch
Previous report
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.
Previous report
Phase 2 Clinical Study in Chronic Neutropenia
Previous report
Following positive results from a Phase 1b clinical study of a single dose of mavorixafor in people with idiopathic, cyclic, and congenital chronic neutropenia, we 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.
Previous report
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.
Previous report
Regulatory Updates and Progress of Phase 3 Clinical Trial in Chronic Neutropenia
Previous report
2025 Strategic Restructuring
Previous report
Reverse Stock Split
Previous report
On April 17, 2025, we held a special meeting where our stockholders approved an amendment to our Restated Certificate of Incorporation, as amended (“Certificate of Incorporation”) to combine outstanding shares of our common stock into a lesser number of outstanding shares, by a ratio of not less than one-for-fifteen and not more than one-for-thirty, with the exact ratio to be set within this range by our Board in its sole discretion.
Previous report
On April 17, 2025, our Board approved a 1-for-30 reverse stock split of its shares of Common Stock (the “Reverse Stock Split”). On April 24, 2025, we filed an amendment to our Certificate of Incorporation to effectuate the Reverse Stock Split effective as of 12:01 a.m. Eastern Time on April 28, 2025. As a result of the Reverse Stock Split, every 30 shares of our common stock issued or outstanding were automatically reclassified into one validly issued, fully-paid and non-assessable new share of common stock, subject to the treatment of fractional shares as described below, without any action on the part of the holders. Proportional adjustments were made to the number of shares of common stock awarded and available for issuance under our equity incentive plans, as well as the exercise price and the number of shares issuable upon the exercise or conversion of our outstanding stock options and other equity securities under our equity incentive plans. All outstanding warrants were also adjusted in accordance with their terms, which resulted, among other changes to the warrant terms, in proportionate adjustments being made to the number of shares issuable upon exercise of such warrants and to the exercise and redemption prices of such warrants. The shares of common stock outstanding following the Reverse Stock Split remain fully paid and non-assessable. The Reverse Stock Split did not affect the number of authorized shares of common stock or the par value of the common stock.
Previous report
No fractional shares were issued in connection with the Reverse Stock Split. Stockholders who would otherwise be entitled to receive fractional shares will automatically be entitled to receive cash in lieu of such fractional share.
Previous report
Comparison of the Three and Six Months Ended June 30, 2025 and 2024
Previous report
During the first quarter of 2025, we entered into the Norgine Agreement, pursuant to which Norgine was granted an exclusive license 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. See Note 4 to the condensed consolidated financial statements for a discussion of the accounting related to this agreement. Pursuant to the terms of the Norgine Agreement, we received an upfront payment of $29.2 million upon signing of the agreement and received an operational milestone payment of$0.4 million. No manufacturing services have been provided to Norgine in the three and six months ended June 30, 2025.
Previous report
Our performance obligations include the delivery of a license, which was delivered upon the closing of the Norgine Agreement, the delivery of commercial drug supply pursuant to a manufacturing agreement, and the provision of research and development services. We allocated the transaction price among these performance obligations based on our best estimate of stand-alone selling price for each distinct performance obligation. During the six months ended June 30, 2025, we recognized $27.6 million for the delivery of the license and$0.5 million for the provision of research and development services. We had no license or other revenue during the three or six months ended June 30, 2024.
Previous report
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. 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 been selling our drug product in the U.S. for one year, 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.
Previous report
Net revenue from the sale of our drug product increased$1.2 million and$2.1 millionin the three and six months ended June 30, 2025, respectively, as compared to the same periods in the prior year, due to an increase in prescribed patients on our drug product.
Previous report
Other Income (Expense), Net
Previous report
The income tax provision recorded for the three and six months ended June 30, 2025 and 2024 is primarily related to our Austrian subsidiary. We did not record a U.S. federal or state provision or benefit for income taxes in our condensed consolidated statement of operations and comprehensive (loss) income for the three and six months ended June 30, 2025 and 2024.
Previous report
We continue to maintain a valuation allowance against all remaining net deferred tax assets. We believe that it is more likely than not that we will not realize a future tax benefit of these attributes as we expect to continue to generate operating losses. Ultimate realization of any deferred tax asset is dependent on the our ability to generate sufficient future taxable income in the appropriate tax jurisdiction before the expiration of carryforward periods, if any. We will continue to monitor the valuation allowance assessment throughout the year.
Previous report
Lincoln Park Purchase Agreement— In June 2025, we entered into a common stock purchase agreement (“Purchase Agreement”) with Lincoln Park Capital Fund LLC (“Lincoln Park”) pursuant to which Lincoln Park has committed to purchase, at our request from time to time over a 24-month period, shares of our common stock having an aggregate offering price of up to $40.0 million, subject to certain limitations including a common share cap that will require shareholder approval to exceed. As of June 30, 2025, we raised $0.5 million through the Purchase Agreement.
Previous report
Going Concern—We have evaluated whether there are certain conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within one year after the date that the condensed consolidated financial statements are issued. 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. Since inception, we have incurred significant operating losses and negative cash flows from operations and we expect to continue to generate operating losses and negative cash flows from operations for the foreseeable future. As of June 30, 2025, we had $63.0 million of cash, cash equivalents and short-term marketable securities, and an accumulated deficit of $540.8 million. Net cash used in operating activities was $42.3 million for the six months ended June 30, 2025.
Previous report
We have a Minimum Cash Covenant under our Hercules Loan Agreement that requires us to maintain a minimum level of cash of $15.0 million, which is equal to 20% of outstanding borrowings under the Hercules Loan Agreement. In addition, we must comply with a Performance Covenant, which includes maintaining a trailing six-month net product revenue of at least 55% of forecast as approved by the Board of Directors. Based on our current operating plan, which includes estimates of anticipated cash inflows from the sales and licensing of our product and cash outflows from operating expenses, we believe there is a risk that we will not meet the conditions of the Performance Covenant and the Minimum Cash Covenant within the 12-month period from the issuance date of these consolidated financial statements. In such case, Hercules could accelerate the principal payments on the our outstanding loans and we potentially would not have sufficient cash, cash equivalents and short-term marketable securities to settle such obligations. Accordingly, we have concluded that this condition meets the standard for raising substantial doubt about the our ability to continue as a going concern.
Previous report
Cash Flows
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Funding Requirements
Previous report
Hercules Loan Agreement
Reworded paragraphs (19)
Previous wording
In June 2025, we announced that the FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia.
Current wording
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 enroll up 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.
Previous wording
In February 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, while also optimizing U.S. promotion of XOLREMDI (the “2025 Strategic Restructuring”). As part of the 2025 Strategic Restructuring, we implemented a net reduction of our employee headcount by 43 employees, or approximately 30% of our total workforce. The strategic restructuring activities include (i) discontinuing of certain research efforts, (ii) closing of our facility in Vienna, Austria, (iii) pausing pre-clinical drug candidate programs, (iv) scaling the U.S. commercial field team and supporting roles across our business, and (v) streamlining other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia. The workforce reductions were substantially completed in the first quarter of 2025. During the first quarter of 2025, we incurred charges of approximately $2.5 million for severance and other employee termination-related costs. We expect the 2025 Strategic Restructuring will decrease annual spending by $30 to 35 million and believe we will have sufficient funds to support operations into the first half of 2026, subject to our continued compliance with our Loan and Security Agreement, amended from time to time, with Hercules Capital, Inc. (the “Hercules Agreement”). The estimate of costs that we expect to incur related to the 2025 Strategic 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.
Current wording
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.
Previous wording
We continue to execute our global launch of XOLREMDI in WHIM syndrome, including in the U.S. through 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 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 an MAA to the EMA in early 2025 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. In February 2025, we announced that we had entered into an agreement with Taiba Middle East Fz LLC (“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.
Current wording
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.
Previous wording
The following table summarizes the results of our operations for the three and six months ended June 30, 2025 and 2024:
Current wording
The following table summarizes the results of our operations for the three and nine months ended September 30, 2025 and 2024:
Previous wording
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. For the three months ended June 30, 2025, our cost of revenue remained constant as compared to the prior year period. Cost of revenue increased $4.7 million in thesix months ended June 30, 2025, as compared to the same period in the prior year, primarily due to additional royalties in the current six period associated with sublicense income from our Norgine Agreement.
Current wording
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 $4.9 million in thenine 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.
Previous wording
Research and development expenses consist primarily of costs incurred in connection with the discovery and development of our product candidates. Research and development costs include employee salaries and related expenses, preclinical and clinical development expenses, internal and third-party costs of manufacturing our drug products for use in our preclinical studies and clinical trials. Research and development expenses also include facility, depreciation and other expenses; 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.
Current wording
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.
Previous wording
Research and development expenses decreased by $2.5 million and$3.8 million in the three and six months ended June 30, 2025, respectively, as compared to the same periods in the prior year. These decreases were primarily due to decreases in spending associated with our 2025 Strategic Restructuring, including lower spending on non-clinical programs, lower consulting fees, lower drug substance manufacturing costs, and lower regulatory costs. These decreases were partially offset by higher clinical costs associated with our ongoing 4WARD trial.
Current wording
Research and development expenses decreased by $1.9 million and$5.7 million in the three and nine months ended September 30, 2025, respectively, as compared to the same periods in the prior year primarily 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.
Previous wording
Selling, general and administrative expenses decreased by approximately $3.8 million and$6.1 million in thethree and six months ended June 30, 2025, respectively, as compared to the same periods in the prior year. These decreases were primarily due to a decreases in compensation expense related to outstanding stock appreciation rights (“SARs”) that are measured at fair value, decreases in sales and marketing expenses as certain commercialization launch costs incurred in the prior year did not recur in the current year, lower salaries and benefits costs due to lower head count in our general and administrative functions and lower stock-based compensation costs. These decreases in selling, general and administrative expenses were partially offset by higher severance costs associated with our 2025 Strategic Restructuring and higher legal costs.
Current wording
Selling, general and administrative expenses decreased by approximately $4.1 million and$10.3 million in thethree 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.
Previous wording
Other income, net, decreased approximately$19.2 million in the three months ended June 30, 2025 as compared to the same period in the prior year primarily due to a significant gain recognized in the prior period due to a change in the fair value of our Class C warrants, which are accounted for as a liability at fair value, relative to a lower gain in the current period. Other income, net, increased$5.1 million in thesix months ended June 30, 2025 as compared to the same period in the prior year primarily due to higher gains due to the change in fair value of our Class C warrants in the current period, partially offset by lower other income, net, which includes unrealized foreign currency losses and other adjustments to the fair value of financial instruments as described in Note 5 to the condensed consolidated financial statements.
Current wording
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 million in thenine 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.
Previous wording
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.
Current wording
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
Previous wording
ATM Sales Agreement — We have entered into a Controlled Equity Offering SMSales Agreement (“ATM Sales Agreement”), with certain investment banks (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. To date, we have sold approximately $19.5 million of our common stock, net of offering costs, under the ATM Sales Agreement. 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 $69.8 million.
Current wording
ATM Sales Agreement. We are party to a Controlled Equity Offering SMSales 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.
Previous wording
Hercules Loan Agreement—We are a party to 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 June 30, 2025. The term loan facility allows for an additional tranche of up to $32.5 million, which will be available subject to approval by Hercules Capital, Inc. (“Hercules”) in its sole discretion.
Current wording
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.
Previous wording
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. Net cash used in operating activities for the six months ended June 30, 2024 was $63.9 million, primarily resulting from operating expenses of $71.1 million, adjusted for non-cash expenses of $5.0 million and changes in our operating assets and liabilities of $3.1 million. 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.
Current wording
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.
Previous wording
During the six months ended June 30, 2025, cash provided by investing activities of $20.0 million primarily include net sales of short-term marketable securities. During the six months ended June 30, 2024, cash provided by investing activities of $91.5 million included cash proceeds of $105.0 million in proceeds from the sale of a priority review voucher obtained as a result of the approval of our drug product by the FDA and cash outflows of $7.0 million in license payments classified as the acquisition of an intangible assets and $6 million of net investments of short-term marketable securities.
Current wording
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.
Previous wording
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 Purchase Agreement. Cash provided by financing activities for the six months ended June 30, 2024 included $20.0 million of new borrowings on our loan facility.
Current wording
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.
Previous wording
Based on our cash, cash equivalents and marketable securities on hand as of June 30, 2025 and our current operating plan, we believe that our cash, cash equivalents and marketable securities will not allow us to fund operations for at least the next 12 months. In order to fund operations and satisfy the Minimum Cash Covenant in the Hercules Loan Agreement, which includes a Performance Covenant that is waived if we maintain cash, cash equivalents and marketable securities subject control accounts accessible by the lender in the event of default in an amount greater than 75% of outstanding principal, or $56 million as of June 30, 2025. If we are unable to raise additional capital in the near term, which may be through a combination of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements, or collaborations and strategic alliances, we may not be in compliance with the Minimum Cash Covenant, which would cause an event of default on our Hercules Loan Agreement. In such case, the lender could either redeem the entire amount outstanding or begin to charge a default interest of 4% of on the entire outstanding balance, due on demand. 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 statements.
Current wording
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.
Previous wording
To alleviate the risk that the we violate the Performance Covenant and the Minimum Cash Covenant, we will need to raise additional capital, which cannot be assured, and/or modify such Performance Covenant and Minimum Cash Covenant. Unless and until we reach profitability in the future, we will require additional capital to fund our operations, which 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.
Current wording
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.
Previous wording
During 2025, assuming no changes to our current operational expectations, which include the impact of the strategic restructuring we announced in the first quarter of 2025, we expect our operating expenses to be lower than the operating expenses, we generated in 2024, excluding the sale of non-financial assets. Because of 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:
Current wording
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:
Previous wording
During the three months ended June 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 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 June 30, 2025.
Current wording
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.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
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.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
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 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 enroll 150 participants.
Previous report — wording carried elsewhere
See Note 10 to the notes to our condensed consolidated financial statements for a full description of our Hercules Loan Agreement.