X4 Pharmaceuticals, Inc
For the period ended Jun 30, 2026. Filed Aug 6, 2026.
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Jun 30, 2026 · Filed Aug 6, 2026
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Notes to Condensed Consolidated Financial Statements
- Description of Business and Summary of Significant Accounting Policies
X4 Pharmaceuticals Inc., together with its subsidiaries (the “Company”), is a biopharmaceutical company developing and commercializing novel therapeutics for the treatment of rare hematology diseases. The Company’s primary asset, mavorixafor, 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. In addition, the European Commission has granted marketing authorization for mavorixafor capsules for the treatment of patients with WHIM syndrome in the European Union.
The Company continues to progress its global, pivotal, 52-week 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. CN 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. Fast Track designation has been granted by the FDA to mavorixafor for the treatment of CN.
Basis of Presentation
The accompanying unaudited interim condensed consolidated financial statements for the three and six months ended June 30, 2026 and 2025, respectively have been prepared in accordance with accounting principles generally accepted in the U.S. for interim financial information and with the instructions to the Quarterly Report on Form 10-Q and Article 10 of Regulation S-X. Accordingly, certain information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) have been condensed or omitted pursuant to such rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. In the opinion of management, all adjustments, consisting only of normal recurring adjustments as necessary, for the fair statement of the Company’s condensed financial position, condensed results of its operations and cash flows have been made. Operating results for the three and six months ended June 30, 2026 are not necessarily indicative of the results that may be expected for the entire year or for any other subsequent interim period.
These unaudited interim condensed consolidated financial statements should be read in conjunction with the Company’s audited financial statements and the notes thereto for the year ended December 31, 2025 included in its Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 17, 2026. The condensed consolidated balance sheet at December 31, 2025 that is presented in these interim condensed consolidated financial statements was derived from audited financial statements but does not include all disclosures required by GAAP.
Principles of Consolidation
The condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, including X4 Pharmaceuticals (Austria) GmbH, which is incorporated in Vienna, Austria, and X4 Therapeutics, Inc. All intercompany accounts and transactions have been eliminated.
Use of Estimates
The preparation of the Company’s condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements, and the reported amounts of expenses during the reporting period. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual of research and development expenses, the accrual of operational and financial license milestones, the accrual of reserves for variable consideration related to product revenue, and the impairment or lack of impairment of long-lived assets including operating lease right-of-use assets and goodwill. The Company bases its estimates on historical experience, known trends and other market-specific or other relevant factors that it believes to be reasonable under the circumstances. On an ongoing basis, management evaluates its estimates when there are changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. As of the date of issuance of these condensed consolidated financial statements, the Company is not aware of any specific event or circumstance that would require the Company to update its estimates, assumptions and judgments or revise the carrying value of its assets or liabilities. Actual results could differ from those estimates, and any such differences may be material to the Company’s condensed consolidated financial statements.
Liquidity
The Company has evaluated whether there are certain conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date that the condensed consolidated financial statements are issued. Although the Company has an approved drug product, licensing and sales of the Company’s drug product over the next 12 months will not be sufficient to fund the Company’s operating expenses. Since inception, the Company has incurred significant operating losses and negative cash flows from operations, and the Company expects to continue to generate operating losses and negative cash flows from operations for the foreseeable future. For the six months ended June 30, 2026, the Company’s net loss was $36.4 million and net cash used in operating activities was $40.4 million. As of June 30, 2026, the Company had $208.0 million of cash, cash equivalents and short-term marketable securities, and an accumulated deficit of $631.0 million.
Based on its current operating plan, the Company believes (a) that its current cash, cash equivalents and short-term marketable securities will be sufficient to fund its operations and debt obligations for at least the next 12 months and (b) it will continue to comply with all covenants under the Hercules Loan Agreement, as defined and further described in Note 9, through at least the 12-month period from the issuance date of these condensed consolidated financial statements.
The Company is subject to risks common to companies in the biopharmaceutical industry including, but not limited to, uncertainties relating to conducting preclinical and clinical research and development, the manufacture and supply of products and product candidates for clinical and commercial use, obtaining and maintaining regulatory approvals and pricing and reimbursement for its products and product candidates, market acceptance, managing global growth and operating expenses, availability of additional capital, competition, obtaining and enforcing patents, stock price volatility, dependence on collaborative relationships and third-party service providers, dependence on key personnel, and from time to time government investigations, litigation, and potential product liability claims.
Significant Accounting Policies
The Company’s significant accounting policies are disclosed in the audited consolidated financial statements and the notes thereto in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on March 17, 2026. Since the date of those consolidated financial statements, there have been no material changes to the Company’s significant accounting policies.
Restricted Cash
| (in thousands) | As of June 30, 2026 | As of December 31, 2025 |
|---|---|---|
| Letter of credit security: Vienna Austria lease | $— | $224 |
| Letter of credit security: Boston lease | 579 | 579 |
| Letter of credit security: Watertown lease | 95 | — |
| Total restricted cash | $674 | $803 |
In connection with the Company’s lease agreements for its facilities in Boston and Watertown, Massachusetts, the Company maintains letters of credit, which are secured by restricted cash, for the benefit of the respective landlords. As of December 31, 2025, restricted cash related to the Company’s former Vienna, Austria lease, which the Company terminated effective November 30, 2025, was included in other current assets and was subsequently converted to unrestricted cash during the six months ended June 30, 2026. Restricted cash related to the Boston and Watertown lease are included in other current assets and other assets, respectively.
In accordance with the Company’s Hercules Loan Agreement, the Company at all times must maintain a minimum level of cash of $15.0 million in an account or accounts in which Hercules has a first priority security interest.
Cash, Cash Equivalents, Restricted Cash and Marketable Securities
The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the condensed consolidated balance sheets to the total of amounts shown in the Company’s condensed consolidated statements of cash flows as of June 30, 2026 and December 31, 2025:
| (in thousands) | June 30, 2026 | December 31, 2025 |
|---|---|---|
| Cash and cash equivalents | $195,095 | $217,049 |
| Restricted cash, current (included within prepaid expenses and other current assets) | 579 | 803 |
| Restricted cash (included in other assets) | 95 | — |
| Total cash, cash equivalents and restricted cash | $195,769 | $217,852 |
The Company’s cash equivalents consisted of money market funds invested in U.S. Treasury securities. The money market funds were valued based on quoted prices in active markets for identical assets, which represents a Level 1 measurement. All marketable securities are classified as short-term investments as all are due within one year and include investments in U.S. Treasury notes, U.S. Treasury bills and federal government agency notes. The amortized cost of each investment, individually and in aggregate, approximated fair value. The Company has evaluated each marketable security for impairment that is other-than-temporary and concluded that no marketable security was impaired as of June 30, 2026 and December 31, 2025.
| Fair Value Measurements as of June 30, 2026 Using: | ||||
|---|---|---|---|---|
| (in thousands) | Level 1 | Level 2 | Level 3 | Total |
| Cash equivalents—money market funds | $191,613 | $— | $— | $191,613 |
| Marketable securities—federal government agency notes | — | 12,874 | — | 12,874 |
| $191,613 | $12,874 | $— | $204,487 | |
| Fair Value Measurements as of December 31, 2025 Using: | ||||
|---|---|---|---|---|
| (in thousands) | Level 1 | Level 2 | Level 3 | Total |
| Cash equivalents—money market funds | $123,838 | $— | $— | $123,838 |
| Marketable securities—U.S. Treasury notes, U.S. Treasury bills, and federal government agency notes | — | 35,949 | — | 35,949 |
| $123,838 | $35,949 | $— | $159,787 | |
The following table provides amortized cost, unrealized gains and losses and the carrying amount of available-for-sale debt marketable securities as of June 30, 2026:
| (in thousands) | Amortized Cost | Gross Unrealized Gains | Gross Unrealized Losses | Fair Value |
|---|---|---|---|---|
| Federal Government Agency securities | 12,878 | — | 4 | 12,874 |
| Total available-for-sale debt securities | $12,878 | $— | $4 | $12,874 |
The following table provides amortized cost, unrealized gains and losses and the carrying amount of available-for-sale debt marketable securities as of December 31, 2025:
| (in thousands) | Amortized Cost | Gross Unrealized Gains | Gross Unrealized Losses | Fair Value |
|---|---|---|---|---|
| U.S. Treasury securities | $7,959 | $3 | $— | $7,962 |
| Federal Government Agency securities | 27,980 | 11 | 4 | 27,987 |
| Total available-for-sale debt securities | $35,939 | $14 | $4 | $35,949 |
Liabilities Measured at Fair Value
The following tables present information about the Company’s financial liabilities measured at fair value on a recurring basis and indicate the level of the fair value hierarchy used to determine such fair values:
| Fair Value Measurements as of June 30, 2026 Using: | ||||
|---|---|---|---|---|
| (in thousands) | Level 1 | Level 2 | Level 3 | Total |
| Liabilities: | ||||
| Embedded derivative liability | $— | $— | $10 | $10 |
| Class C warrant liability | — | — | 369 | 369 |
| $— | $— | $379 | $379 | |
| Fair Value Measurements as of December 31, 2025 Using: | ||||
|---|---|---|---|---|
| (in thousands) | Level 1 | Level 2 | Level 3 | Total |
| Liabilities: | ||||
| Embedded derivative liability | $— | $— | $10 | $10 |
| Class C warrant liability | — | — | 977 | 977 |
| $— | $— | $987 | $987 | |
The following table provides a roll-forward for the six months ended June 30, 2026 of the aggregate fair values of financial instruments for which fair values are determined using Level 3 inputs:
| (in thousands) | Embedded Derivative Liability | Class C Warrant Liability | Total |
|---|---|---|---|
| Balance as of December 31, 2025 | $10 | $977 | $987 |
| Change in fair value | — | (608) | (608) |
| Balance as of June 30, 2026 | $10 | $369 | $379 |
Valuation of Embedded Derivative Liability— The fair value of the embedded derivative liability recognized in connection with the Company’s Hercules Loan Agreement, which is associated with additional fees due to Hercules upon non-credit related events of default, was determined based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. The fair value of this embedded derivative liability, which is reported within other non-current liabilities on the condensed consolidated balance sheets, is estimated by the Company at each reporting date based, in part, on the results of third-party valuations, which were prepared based on a discounted cash flow model that considered the timing and probability of occurrence of a redemption upon an event of default, the potential amount of prepayment fees or contingent interest upon an event of default and the Company’s risk-adjusted discount rate of 17%. As of June 30, 2026 and December 31, 2025, the fair value of this derivative liability was $10 thousand.
Class C Warrant Liability—In December 2022, the Company issued Class C warrants (“Class C Warrants”) for the purchase of shares of its common stock in a public offering. The Class C Warrants are accounted for as a liability on the condensed consolidated balance sheet and are adjusted to fair value at period end through “change in fair value of warrant liability” on the condensed consolidated statements of operations and comprehensive (loss) income.
The Company calculated the fair value of the Class C Warrants using the Black-Scholes option pricing model, with the following inputs:
| June 30, 2026 | December 31, 2025 | |
|---|---|---|
| Common stock price | $4.67 | $4.00 |
| Risk-free interest rate | 4.0% | 3.5% |
| Expected term (in years) | 1.4 | 1.9 |
| Expected volatility | 114.2% | 136.0% |
| Expected dividend yield | —% | —% |
Net Loss Per Share
Basic net loss per common share is calculated based on net loss divided by the weighted average number of shares outstanding for the period. The calculation of diluted loss per common share excludes the impact of potential common stock as their inclusion would be to reduce net loss per common share and are thus antidilutive.
Basic and diluted net loss per share was calculated as follows:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| (in thousands, except share and per share data) | 2026 | 2025 | 2026 | 2025 |
| Numerator: | ||||
| Net loss | $(16,157) | $(25,741) | $(36,398) | $(25,459) |
| Denominator: | ||||
| Weighted average shares outstanding—basic and diluted | 126,344,020 | 7,412,990 | 126,316,523 | 7,095,846 |
| Net loss per share— basic and diluted | $(0.13) | $(3.47) | $(0.29) | $(3.59) |
Basic and diluted weighted average shares of common stock outstanding for the three and six months ended June 30, 2026 and 2025 includes the weighted average effect of pre-funded warrants for the purchase of shares of common stock, for which the remaining unfunded exercise price is less than or equal to $0.03 per share.
The Company excluded the following potential shares of common stock from the computation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| (in thousands) | 2026 | 2025 | 2026 | 2025 |
| Options to purchase shares of common stock | 15,333 | 308 | 15,333 | 308 |
| Unvested restricted stock units | 714 | 333 | 714 | 333 |
| Warrants to purchase shares of common stock (excluding prefunded warrants, which are included in basic shares outstanding) | 2,546 | 2,546 | 2,546 | 2,546 |
| 18,593 | 3,187 | 18,593 | 3,187 | |
Recently Issued Accounting Standards Not Yet Adopted
In November 2024, the FASB issued ASU 2024-03, Disaggregation of Income Statement Expenses (Topic 220) (“ASU 2024-03”, or “DISE”) requiring that public business entities disclose additional information about specific expense categories in the notes to financial statements at interim and annual reporting periods. The amendments in DISE are effective for annual reporting periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. The disclosure requirements may be applied either prospectively to financial statements issued for reporting periods after the effective date or retrospectively to any or all prior periods presented in the financial statements. Although the Company continues to evaluate the impact of the future adoption of ASU 2024-03 to its consolidated financial statements, the Company anticipates that it will adopt ASU 2024-03 prospectively as of its fiscal year ended December 31, 2027 and that such adoption will result in an additional footnote disclosure to the consolidated financial statements that will include further details of specific line items on its consolidated statement of operations.
- Strategic Restructuring Liability
In 2025, the Company implementedtwo strategic restructuring actions designed to sharpen operational focus and align resources with the Company’s long‑term strategy to successfully complete the 4WARD trial in patients with moderate and severe CN. These strategic restructuring actions resulted in an approximately 65% reduction in employee headcount.
The following table summarizes the Company’s liability recognized in connection with severance benefits incurred under strategic restructuring initiatives:
| (in thousands) | |
|---|---|
| Balance as of January 1, 2026 | $1,526 |
| Severance and other employee-related expenses (of which $86 is included in general and administrative expense and $149 is included in research and development expense) | 235 |
| Cash payments | (1,096) |
| Balance as of June 30, 2026 (included in accrued expenses) | $665 |
- License Agreements
See Note 4 “License Agreements and Funding Arrangements” to the consolidated financial statements contained in the Company’s Annual Report on Form 10-K for a description of the Company’s license agreements. There have been no changes to license agreements and funding arrangements for the six months ended June 30, 2026, except as follows:
Under the Company’s license agreement with Genzyme (the “Genzyme Agreement”), the Company is obligated to make milestone payments contingent upon the achievement of certain regulatory and sales milestones with respect to licensed products. During the three and six months ended June 30, 2026, a$5.0 million regulatory milestone was paid after receipt of the European Medicines Agency’s (“EMA”) validation of the Company’s Marketing Authorisation Application (“MAA”) for processing. The payment was recorded as a definite-lived intangible asset.
As of June 30, 2026, the Company is obligated to make future one-time sales milestone payments of $0.5 million,$1.5 million and$3.0 million on cumulative net sales of$50.0 million,$150.0 million and$300.0 million, respectively. As of June 30, 2026, the Company accrued$0.5 million of sales-based milestones as a component of the definite-lived intangible asset, as management has concluded that the achievement of this milestone is probable.
- Revenue
Product Revenue, Net
During the three and six months ended June 30, 2026, the Company recorded net revenue of $2.4 million and $4.8 million, respectively for the sale of the Company’s drug product in the U.S. Net product revenue was $1.7 million and $2.7 million for the three and six months ended June 30, 2025, respectively.
The following table summarizes the balances and activity in each of the product reserve accounts for the six months ended June 30, 2026.
| (in thousands) | Rebates and Discounts | Co-Pay Assistance | Product Returns | Total |
|---|---|---|---|---|
| Beginning balance at December 31, 2025 | $418 | $30 | $10 | $458 |
| Provision related to revenue associated with sales processed in the six month period ended June 30, 2026 | 468 | 28 | 25 | 521 |
| Credits and payments made during the period | (353) | (58) | (22) | (433) |
| Balance as of June 30, 2026 | $533 | $— | $13 | $546 |
The provision for contractual discounts provided to the Company’s customer is recorded as a reduction of accounts receivable. The provisions for co-pay assistance payments, contractual rebates and product returns are classified within accrued expenses.
The following table provides a rollforward of accounts receivable for the six months ended June 30, 2026.
| (in thousands) | Accounts Receivable |
|---|---|
| Beginning balance at December 31, 2025 | $573 |
| Increase in accounts receivable for drug product sales | 6,353 |
| Decrease in accounts receivable for cash collections | (4,879) |
| Balance as of June 30, 2026 | $2,047 |
License and Other
During the first quarter of 2025, the Company entered into a license and supply agreement (the “Norgine Agreement”) with Norgine Pharma UK Ltd. (“Norgine”). The Company analyzed the activities required under the Norgine Agreement and concluded that the arrangement was indicative of a vendor-customer relationship and would be accounted for under ASC 606. To date, the Company has received a one-time, non-refundable, up-front payment of €28.5 million, andregulatory milestone payments of€5.5 million, including a €5.0 million regulatory milestone achieved in the second quarter of 2026. The one-time upfront payment and regulatory milestone payments are included in the transaction price. All other future regulatory-based milestone payments, which represent variable consideration, have been fully constrained as these are not yet considered probable. The Company has also excluded from the transaction price future royalty payments that are based on units sold by Norgine and future cumulative revenue-based milestone payments under the applicable practical expedient.
Under the Norgine Agreement, the Company’s promises include a) the delivery of a license, b) research and development services for certain components of WHIM clinical studies, c) research and development services for the global Phase 3 trial of XOLREMDI for CN, and d) the option for delivery of commercial and early access drug supply.
The following table summarizes the allocation of transaction price to the performance obligations under the Norgine Agreement based on the weighting of estimated stand-alone selling price for these performance obligations at the inception of the agreement.
| Allocation of Transaction Price · Revenue Recognized | |||||
|---|---|---|---|---|---|
| (in thousands) | Six Months Ended June 30, | ||||
| Performance Obligation: · 2026 · 2025 | |||||
| License | $33,096 | $5,457 | $27,639 | ||
| Research and development services: WHIM | 373 | 5 | 25 | ||
| Research and development services: CN | 2,065 | 47 | 430 | ||
| Total | 35,534 | 5,509 | 28,094 | ||
| Drug product supply | 1,163 | — | |||
| Total license and other revenue | 6,672 | 28,094 | |||
The following table provides a rollforward of deferred revenue six months ended June 30, 2026.
| (in thousands) | Deferred Revenue |
|---|---|
| Beginning balance at December 31, 2025 | $1,085 |
| Increase for allocation of milestone transaction price to development services | 401 |
| Decrease for revenue recognized | (52) |
| Balance as of June 30, 2026 | $1,434 |
As of June 30, 2026, deferred revenue related to the Norgine Agreement was $1.4 million, of which $0.8 million was current.
- Inventory
Inventory consists of the following:
| (in thousands) | June 30, 2026 | December 31, 2025 |
|---|---|---|
| Raw materials | $1,582 | 1,382 |
| Work in process | 3,499 | 2,518 |
| Finished goods | 421 | 579 |
| Total inventory | $5,502 | $4,479 |
- Property and Equipment
Property and equipment, net consisted of the following:
| (in thousands) | June 30, 2026 | December 31, 2025 |
|---|---|---|
| Leasehold improvements | $228 | $228 |
| Furniture and fixtures | 1,081 | 1,035 |
| Computer equipment | 134 | 134 |
| Lab equipment | — | 17 |
| 1,443 | 1,414 | |
| Less: Accumulated depreciation | (1,332) | (1,232) |
| $111 | $182 |
Depreciation expense related to property and equipment was approximately $71 thousand and $457 thousand for the six months ended June 30, 2026 and 2025, respectively.
- Intangible Assets, Net
The Company’s net definite-lived intangible asset is comprised of the capitalization of certain milestone payments made or accrued related to its license agreement for the intellectual property contained in its drug product. As of June 30, 2026 and December 31, 2025, net definite-lived intangible assets included a gross intangible asset of$15.5 million and $10.5 million, respectively, less accumulated amortization of $2.4 million and $1.3 million, respectively, for a net intangible asset of $13.1 million and $9.3 million, respectively. The Company amortizes the intangible asset to cost of revenue over the remaining life of the underlying patent protecting the intellectual property through 2038.
As of June 30, 2026, amortization expense for the next five years and beyond is summarized as follows (in thousands):
| Year | Amortization expense |
|---|---|
| 2026 (remainder of year) | 554 |
| 2027 | 1,107 |
| 2028 | 1,107 |
| 2029 | 1,107 |
| 2030 | 1,108 |
| 2031 | 1,107 |
| Thereafter | 7,011 |
| Total | $13,101 |
The Company began amortizing its finite-lived intangible assets in April 2024 over a 14-year period based on the expected patent exclusivity period for XOLREMDI. Amortization expense totaled $1.0 million and $1.1 million for the three and six months ended June 30, 2026, respectively and $0.2 million and $0.4 million for the comparable prior year periods, respectively. Amortization expense is recorded as a component of cost of revenue on the condensed consolidated statements of operations and comprehensive loss.
- Accrued Expenses
Accrued expenses consisted of the following:
| (in thousands) | June 30,2026 | December 31,2025 |
|---|---|---|
| Accrued employee compensation and benefits | $3,319 | 4,303 |
| Accrued external research and development expenses | 8,050 | 12,235 |
| Accrued royalty and milestone payments | 204 | 144 |
| Accrued professional fees | 1,246 | 816 |
| Other | 1,306 | 1,105 |
| $14,125 | $18,603 |
- Long-Term Debt
Long-term debt consisted of the following:
| (in thousands) | June 30,2026 | December 31,2025 |
|---|---|---|
| Principal amount of long-term debt | $75,000 | $75,000 |
| Debt discount, net of accretion | (283) | (412) |
| Cumulative accrual of end of term payments | 2,042 | 1,703 |
| Long-term debt | $76,759 | $76,291 |
Hercules Loan Agreement
The Company is party to the Second Amended and Restated Loan and Security Agreement, as amended, (the “Hercules Loan Agreement”) with Hercules Capital Inc., which provides for an aggregate term loan facility of up to $107.5 million, under which the Company has borrowed an aggregate of $75.0 million of term loans, representing the maximum borrowings allowable as of June 30, 2026. The Hercules Loan Agreement is described in Note 11 to the consolidated financial statements contained in the Company’s Annual Report on Form 10-K filed with the SEC on March 17, 2026.
The Company recognized interest expense under the Hercules Loan Agreement as follows:
| (in thousands) | Three Months Ended June 30, | Six Months Ended June 30, | ||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| Total interest expense | $2,163 | $2,232 | $4,296 | $4,433 |
| Non-cash interest expense | $239 | $212 | $469 | $416 |
The annual effective interest rate on the Hercules Loan Agreement as of June 30, 2026 was 11.2%. There wereno principal payments due or paid under the Hercules Loan Agreement during the three and six months ended June 30, 2026 and 2025. As of June 30, 2026, future principal and accrued end-of-term payments of $77.0 million under the Hercules Loan Agreement are due on July 1, 2027.
- Leases
The Company has a lease agreement for its facility in Boston, Massachusetts, which is the Company’s principal executive office. There are no restrictions or financial covenants associated with this lease agreement. The Company had an operating lease for approximately 1,200 square meters of laboratory and office space in Vienna, Austria that commenced in February 2021 for a term ofseven years. Effective November 30, 2025, the Company terminated the lease in accordance with a lease amendment entered into with the landlord. Accordingly, the Company accelerated the amortization of the right-of-use asset tozero and adjusted the remaining lease obligation tozero as of December 31, 2025.
On April 29, 2026, the Company entered into a lease agreement for approximately 11,000 square feet of office space in a building located in Watertown, Massachusetts (the “Watertown Lease”). The office space will replace the Company’s current headquarters located in Boston, Massachusetts. The Company intends to move into the premises in late 2026 or early 2027 upon the completion of construction, which is anticipated to commence in September 2026. Monthly rent payments under the lease will commence three months following the Company obtaining access to the space. Base rental payments will be approximately $0.3 million annually, plus certain operating expenses. The term of the lease will continue for 36 months following the commencement of rent payments, unless earlier terminated in accordance with the terms of the lease. The Company has the right to sublease the premises, subject to landlord consent. The Company also has the right to renew the lease for an additional 3 years at the then prevailing effective market rental rate. The Company was required to provide the landlord with a $0.1 million letter of credit, which is classified as restricted cash.
The components of lease expense for the three and six months ended June 30, 2026 and 2025 were as follows:
| (dollars in thousands) | Three Months Ended June 30, | Six Months Ended June 30, | ||
|---|---|---|---|---|
| Lease Cost | 2026 | 2025 | 2026 | 2025 |
| Fixed operating lease cost | $400 | $1,085 | $800 | $1,572 |
| Total lease expense | $400 | $1,085 | $800 | $1,572 |
| Other information | ||||
| Operating cash outflows from operating leases | $287 | 354 | $574 | $702 |
| Weighted-average remaining lease term—operating leases | 0.4 | 1.8 | 0.4 | 1.8 |
| Weighted-average discount rate—operating leases | 11.5% | 11.5% | 11.5% | 11.5% |
Maturities of lease liabilities due under lease agreements that have commenced as of June 30, 2026 are as follows (in thousands):
| Maturity of lease liabilities | OperatingLeases |
|---|---|
| 2026 (remainder of the year) | $478 |
| Less: interest | (13) |
| Total operating lease liabilities as of June 30, 2026 | $465 |
- Commitments and Contingencies
The Company has agreements with contract research organizations (“CROs”) pursuant to which the Company and the CROs are conducting clinical trials. The Company may terminate these agreements by providing notice pursuant to the contractual provisions of such agreements and would incur early termination fees. The Company has agreements with contract manufacturing organizations (“CMOs”) for the production of mavorixafor for use in clinical trials and for the commercial supply of XOLREMDI. The Company’s agreement with the CMO who produces batches of drug substance for use in the Company’s clinical and commercial drug supply contains cancellation provisions that would require the Company to pay up to the full contract value upon cancellation. As of June 30, 2026, the Company has approximately $1.3 millionof such commitments in place subject to cancellation provisions.
License Agreements
See Note 5 to the Company’s Annual Report on Form 10-K filed with the SEC on March 17, 2026 for a description of licensing agreements, which commit the Company to contingent milestone and royalty fees based on future operational events.
Indemnification Agreements
In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and its executive officers that will require the Company to, among other things, indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnification obligations. The Company is not currently aware of any indemnification claims and hasnot accrued any liabilities related to such obligations in its condensed consolidated financial statements as of June 30, 2026 or December 31, 2025.
Legal Proceedings
The Company is not a party to any litigation and does not have contingency reserves established for any litigation liabilities. At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company expenses as incurred the costs related to any legal proceedings.
- Equity Transactions
ATM Sales Agreement
On May 6, 2026, the Company entered into an Open Market Sale Agreement SM (“ATM”), under which it may sell up to $75.0 million of its common shares through an investment bank. Concurrently, the Company filed a registration statement on Form S-3 that registered the common shares issuable under the ATM. Through August 6, 2026 there have been no sales of common stock under the ATM. The Company terminated its previous ATM agreement on March 30, 2026.
- Stock-Based Compensation
As of June 30, 2026, there is an aggregate of approximately 2.3 million shares of common stock available for issuance under the Company’s equity incentive plans, including 0.1 million shares of common stock remaining available for issuance under the Amended and Restated 2017 Employee Stock Purchase Plan.
Stock Option Valuation
The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| Risk-free interest rate | 4.2% | 4.0% | 3.8% | 4.2% |
| Expected term (in years) | 6.1 | 6.1 | 6.0 | 6.1 |
| Expected volatility | 108.5% | 103.7% | 109.8% | 103.8% |
| Expected dividend yield | 0% | 0% | 0% | 0% |
As of June 30, 2026, total unrecognized compensation expense related to unvested stock options and restricted stock units was $18.9 million, which is expected to be recognized over a weighted average period of 2.6 years.
Stock Options
The following table summarizes the Company’s stock option activity for the six months ended June 30, 2026:
| Number ofShares | WeightedAverageExercisePrice | WeightedAverageContractualTerm (Years) | AggregateIntrinsicValue (in thousands) | |
|---|---|---|---|---|
| Outstanding as of December 31, 2025 | 12,080,718 | $2.34 | 9.6 | $28,329 |
| Granted | 4,020,000 | 4.01 | ||
| Forfeited and expired | (767,283) | 7.68 | $— | |
| Outstanding as of June 30, 2026 | 15,333,435 | $2.51 | 9.2 | $38,490 |
| Exercisable as of June 30, 2026 | 1,030,803 | $6.39 | 9.0 | $2,687 |
| Vested and expected to vest as of June 30, 2026 | 12,858,006 | $2.53 | 9.2 | $32,770 |
The aggregate intrinsic value of options is calculated as the difference between the exercise price of the stock options and the fair value of the Company’s common stock to the extent the stock option had a lower exercise price. There were no options exercised for the six months ended June 30, 2026 and 2025. The weighted average grant-date fair value per share of stock options granted during the six months ended June 30, 2026 and 2025 was $3.37 and $8.13, respectively.
Restricted Stock Units
The following table summarizes the Company’s restricted stock unit activity for the six months ended June 30, 2026:
| Number ofShares | Weighted-Average Grant Date Fair Value | |
|---|---|---|
| Unvested as of December 31, 2025 | 73,381 | $15.48 |
| Granted | 773,016 | 4.50 |
| Vested | (115,403) | 5.06 |
| Forfeited | (16,759) | 20.05 |
| Unvested as of June 30, 2026 | 714,235 | $5.25 |
Outstanding restricted stock units include performance-based restricted stock units (“PRSUs”), which will vest based on the achievement of an operational milestone. The Company considers the achievement of the remaining operational milestone related to outstanding PRSUs to be probable. Stock-based compensation expense has been recognized for these awards using the accelerated attribution model based on the fair value of the awards as of the date of grant and management’s best estimate of the date the probable operational milestone will be achieved. The Company updates its estimates related to the probability and timing of achievement of the operational milestones each period until the award either vests or is forfeited.
Stock-Based Compensation
Stock-based compensation expense was classified in the condensed consolidated statements of operations and comprehensive loss as follows:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| (in thousands) | 2026 | 2025 | 2026 | 2025 |
| Research and development expense | $484 | $340 | $1,199 | $599 |
| General and administrative expense | 2,602 | 408 | 4,300 | 668 |
| Total stock-based compensation | $3,086 | $748 | $5,499 | $1,267 |
- Income Taxes
The Company did not record a material U.S. federal, state or international provision or benefit for income taxes in its condensed consolidated statement of operations and comprehensive loss for the six months ended June 30, 2026 and 2025. The Company continues to maintain a valuation allowance against all remaining net deferred tax assets. The Company believes that it is more likely than not that it will not realize a future tax benefit of these attributes as the Company expects to continue to generate operating losses. Ultimate realization of any deferred tax asset is dependent on the Company’s ability to generate sufficient future taxable income in the appropriate tax jurisdiction before the expiration of carryforward periods, if any. The Company will continue to monitor the valuation allowance assessment throughout the year.
- Segment Information
The Company has defined its Chief Operating Decision Maker (“CODM”) as the Executive Chairman. The CODM manages the Company’s operations as a single operating segment, which comprises its single reportable segment, for the purposes of assessing performance and making operating decisions. The Company’s focus is on the research, development and commercialization of novel therapeutics for the treatment of rare diseases. The Company’s research, development and commercialization efforts are focused on its lead molecule, mavorixafor, which is being marketed in the U.S. under the trade name XOLREMDI. The CODM uses the Company’s condensed consolidated net loss to monitor actual results as compared to forecast in assessing segment performance and allocation of resources.
The Company derives its revenue from the sale of XOLREMDI in the U.S. and from licensed rights and product sales of mavorixafor for other non-U.S. territories. The Company recognized $2.4 million and $4.8 million of revenue from its U.S. customer and $6.4 million and $6.7 million of revenue from a customer in the United Kingdom for the three and six months ended June 30, 2026, respectively. The Company recognized $1.7 million and $2.7 million of revenue from its U.S. customer and $0.2 million and $28.1 million of revenue from its customer in the United Kingdom for the three and six months ended June 30, 2025, respectively.
The measure of profit for the segment is net loss and consisted of the following for the three and six months ended June 30, 2026, and 2025:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| (in thousands) | 2026 | 2025 | 2026 | 2025 |
| Revenue from external customers | $8,806 | $1,973 | $11,514 | $30,780 |
| Compensation expense, excluding stock-based compensation, stock appreciation rights compensation expense and severance expense | 4,820 | 9,903 | 9,900 | 21,473 |
| Direct research and development program expenses (X4P-001, mavorixafor) | 10,428 | 9,413 | 20,824 | 17,825 |
| Other segment items (a) | 9,715 | 8,398 | 17,188 | 16,941 |
| Net loss (measure of segment profit) | $(16,157) | $(25,741) | $(36,398) | $(25,459) |
(a) Other segment items primarily include cost of revenue, non-compensation departmental costs within sales, general and administrative departments, certain unallocated external costs within research and development, stock-based compensation expense, stock appreciation rights compensation expense, severance expense, other income (expense), and provision for income taxes.
The CODM only receives and reviews information regarding segment assets at the consolidated level. Certain other entity-wide disclosures are included elsewhere in these condensed consolidated financial statements. As of June 30, 2026, the Company’s single operating segment had long-lived assets, including property and equipment and right-of-use assets, of $0.8 million, which were located in the U.S. As of December 31, 2025, the operating segment’s long-lived assets were $1.6 million, all of which were located in the U.S.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.