X4 Pharmaceuticals, Inc
For the period ended Jun 30, 2026. Filed Aug 6, 2026.
View on SEC.govAbout this filing
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.
Jun 30, 2026 · Filed Aug 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-03-31; filed 2026-05-06.
0001501697-26-000040
Item 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSCurrent report: 10-Q
Period ended 2026-06-30; filed 2026-08-06.
0001501697-26-000068
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 59 previous paragraphs and 61 current paragraphs.
40 unchanged; 15 reworded; 4 added; 4 removed; 0 previous and 2 current paragraphs rearranged.
Tables are not compared: 4 previous and 4 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 (4)
Current report
Other Income, Net
Current report
ATM Sales Agreement. On May 6, 2026, we entered into an Open Market Sale Agreement SM (“ATM”), under which we may sell up to $75.0 million of our common shares through an investment bank. Through August 6, 2026, there have been no sales of our common stock under the ATM. We terminated our previous ATM agreement on March 30, 2026.
Current report
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.
Current report
our common stock through our ATM and a common stock purchase agreement.
Removed paragraphs (4)
Previous report
Operating Cost and Expenses:
Previous report
Other Income (Expense), Net
Previous report
ATM Sales Agreement. We were a party to a Controlled Equity Offering SMSales Agreement (“ATM”), dated as of August 7, 2020, pursuant to which we periodically sold shares of our common stock through one or more investment banks. We have sold $24.2 million of our common stock, net of offering costs, under the ATM. We terminated the ATM effective March 30, 2026.
Previous report
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.
Reworded paragraphs (15)
Previous 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 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 by the end of 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.
Current wording
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.
Previous wording
In January 2025, the EMA validated for processing our Marketing Authorization Application 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.
Current wording
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 toour 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.
Previous wording
The following table summarizes the results of our operations for the three months ended March 31, 2026 and 2025:
Current wording
The following table summarizes the results of our operations for the three and six months ended June 30, 2026 and 2025:
Previous wording
Net product sales were $2.5 million and $0.9 million for the three months ended March 31, 2026 and 2025, respectively. Net product revenue growth over the prior year quarter 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 quarter of 2026. Co-pay assistance payments and rebates to U.S. government payors have comprised the majority of our gross-to-net revenue adjustments.
Current wording
Net product sales were $2.4 million and $1.7 million for the three months ended June 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 periods 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 half of 2026. Co-pay assistance payments and rebates to U.S. government payors have comprised the majority of our gross-to-net revenue adjustments.
Previous wording
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.
Current wording
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.
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, cost of manufacturing drug product sold, and sales-based or sublicense-based royalty payments due under our Genzyme license agreement. Cost of revenue decreased $4.1 million in thethree months ended March 31, 2026, as compared to the same period in the prior year, primarily due to royalties in the prior year associated with sublicense income from our Norgine Agreement that did not reoccur in the current period.
Current wording
Cost of revenue primarily consists of amortization of an intangible asset related to milestone payments associated with our Genzyme license agreement, cost of manufactured drug product sold, and sales-based or sublicense-based royalty payments due under our Genzyme license agreement. Cost of revenue increased $1.3 million in the three months ended June 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 thesix months ended June 30, 2026, as compared to the same period in the prior year, primarily due to prior year royalties due under our Genzyme license agreement associated with sublicense income from the Norgine Agreement that did not reoccur in the current period, partially offset by higher intangible amortization in the current period.
Previous 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.
Current wording
Research and development expenses consist primarily of costs incurred in connection with the development of our one product 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.
Previous wording
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.
Current wording
Substantially 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.
Previous wording
Research and development expenses decreased by $3.0 million in the three months ended March 31, 2026 as compared to the same period 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 2025 partially offset by higher clinical costs as we continue to progress our 4WARD trial.
Current wording
Research and development expenses decreased by $3.3 million and $6.4 million in the three and six months ended June 30, 2026, respectively, as compared to the same periods 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 the prior year, partially offset by higher clinical costs, primarily CRO costs related to our 4WARD trial.
Previous wording
General and administrative expenses decreased by $8.1 million in thethree months ended March 31, 2026 as compared to the same period in the prior year. The decrease was primarily due to a $4.0 million reduction in consulting fees, approximately $2.0 million in lower sales and marketing costs, $0.7 million of net 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.
Current wording
General and administrative expenses decreased by $1.0 million and $9.1 million for thethree and six months ended June 30, 2026, respectively, as compared to the same period in the prior year. The decrease for the three month period was primarily due to a reduction in legal, IT and sales and marketing costs, partially offset by higher 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.
Previous wording
Other income, net, decreased approximately $9.7 million in the three months ended March 31, 2026 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 ourmarketable security investment portfolio.
Current wording
Other income, net, decreased approximately $0.3 million and $10.0 million in the three and six months ended June 30, 2026, 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 ourmarketable security investment portfolio.
Previous wording
Public and Private Equity Offerings. Over the past several years, we have funded our operations from sales of common stock, warrants and prefunded warrants through both public offerings and private placements. For example, most recently in October 2025 we completed an underwritten public offering of approximately 52.8 million shares of our common stock and prefunded warrants to purchase up to 700,000 shares of our common stock for net proceeds of $145.6 million, after underwriting discounts and offering expenses.
Current wording
2025 we completed an underwritten public offering of approximately 52.8 million shares of our common stock and prefunded warrants to purchase up to 700,000 shares of our common stock for net proceeds of $145.6 million, after underwriting discounts and offering expenses.
Previous wording
During the three months ended March 31, 2026, cash provided by investing activities of $19.3 million was primarily due to net 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.
Current wording
During the six months ended June 30, 2026 and 2025, cash provided by investing activities of $18.3 million and $20.0 million, respectively, was primarily due to net maturities and sales of short-term marketable securities.
Previous wording
There were no significant cash flows from financing activities during the three months ended March 31, 2026 and 2025.
Current wording
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
Previous wording
Based on our cash, cash equivalents and marketable securities on hand as of May 6, 2026 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.
Current wording
Based on our cash, cash equivalents and marketable securities on hand as of August 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.
Rearranged current paragraphs (2)
Current report — wording carried from elsewhere
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. As part of a complete overhaul of the clinical operating infrastructure, we have transitioned management of the 4WARD 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”) to revisit the sample size of the 4WARD trial and expect to provide an update on our meeting with the FDA and the completion of enrollment by the end of the third quarter.
Current report — wording carried from elsewhere
Public and Private Equity Offerings. Over the past several years, we have funded our operations from sales of common stock, warrants and prefunded warrants through both public offerings and private placements. For example, most recently in October