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X4 Pharmaceuticals, Inc

For the period ended Jun 30, 2025. Filed Aug 8, 2025.

View on SEC.gov

About 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, 2025 · Filed Aug 8, 2025

XFOR$3.18+3.6%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-03-31; filed 2025-05-01.

0001628280-25-021358

Item 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

Current 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 OPERATIONS

The 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 80 previous paragraphs and 79 current paragraphs.

48 unchanged; 23 reworded; 8 added; 7 removed; 2 previous and 0 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 (8)

Current report

In June 2025, we announced that the FDA has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia.

Current 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.

Current report

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 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.

Current report

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 report

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 report

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.
Removed paragraphs (7)

Previous report

We believe that successfully developing and commercializing mavorixafor to provide a new therapeutic option to individuals diagnosed with certain immunodeficiencies has the potential to revolutionize the current treatment landscape, which is principally served by injectable and infused therapies.

Previous report

We may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the actions described for the 2025 Strategic Restructuring above.

Previous report

Research and development expenses decreased by $2 million in the three months ended March 31, 2025, as compared to the same period in the prior year. The decrease was primarily due to a decrease inpre-clinical drug candidate programs.

Previous report

During the three months ended March 31, 2025, net cash used in operating activities 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. Net cash used in operating activities for the three months ended March 31, 2024 was $33.6 million, primarily resulting from our net losses of $51.8 million, adjusted for noncash expenses of $16.1 million.

Previous report

During the three months ended March 31, 2025, cash used in investing activities of $3.1 million included net investments in short-term marketable securities. During the three months ended March 31, 2024, cash used in investing activities of $5.3 million also included investments in short-term marketable securities.

Previous report

There were no cash used in financing activities during the three months ended March 31, 2025 and 2024.
Reworded paragraphs (23)

Previous wording

We are currently executing our U.S. launch of XOLREMDI in WHIM syndrome, including 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 entered into agreements with a third-party logistics organization and a specialty pharmacy to support the distribution of XOLREMDI in the U.S., to mitigate barriers to product access, and to provide a suite of patient support services to help patients through their treatment journey. We submitted 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

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

Regulatory Updates and Progress of Phase 3 Clinical Trial in Chronic Neutropenia

Previous wording

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. We believe we are on track to complete enrollment in the 4WARD study in the third or fourth quarter of 2025.

Current wording

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 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. 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 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.

Previous wording

On April 17, 2025, we held a special meeting (the “Special Meeting”) to vote for the approval of 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 (the “Reverse Stock Split Proposal”). The Reverse Stock Split Proposal was approved by our shareholders at the Special Meeting.

Current wording

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 wording

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 the Company’s 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 the Company’s equity incentive plans, as well as the exercise price and the number of shares issuable upon the exercise or conversion of the Company’s outstanding stock options and other equity securities under the Company’s 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.

Current wording

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 wording

Comparison of the Three Months Ended March 31, 2025 and 2024

Current wording

Comparison of the Three and Six Months Ended June 30, 2025 and 2024

Previous wording

The following table summarizes the results of our operations for the three months ended March 31, 2025 and 2024:

Current wording

The following table summarizes the results of our operations for the three and six months ended June 30, 2025 and 2024:

Previous wording

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 mavorixafor 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. Pursuant to the terms of the Norgine Agreement, we received an upfront payment of€28.5 million ($29.2 million) upon signing of the agreement and received an operational milestone payment of€0.5 million ($0.4 million). No manufacturing services were provided to Norgine in the first quarter of 2025.

Current wording

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 wording

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 regulatory and 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. We recognized $27.6 million for the delivery of the license and $0.3 million for the provision of regulatory, and research and development services during the first quarter of 2025. We had no license or other revenue in the first quarter of 2024.

Current wording

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 wording

Net revenue from the sale of our drug product was $0.9 million for the three months ended March 31, 2025. There was no product revenue during the same period in the prior year. 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 less than 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.

Current wording

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 wording

For the three months ended March 31, 2025, our cost of revenue of $4.7 million primarily consisted of amortization of an intangible asset related to accrued and paid milestone payments associated with our Genzyme license agreement, sales and sublicense-based royalty payments due under our Genzyme license agreement. Cost of revenue also included $0.01 million of drug product manufacturing costs. There was no cost of revenue during the same period in the prior year.

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. 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.

Previous wording

Selling, general and administrative expenses decreased by approximately $2 million in the three months ended March 31, 2025, as compared to the same period in the prior year. The decrease was primarily due to a decrease in compensation expense related to outstanding stock appreciation rights (“SARs”) that are measured at fair value, as well as decreases in commercialization launch costs incurred in the prior year in preparation for the commercial launch of our drug product in the U.S.

Current 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.

Previous wording

Other income (expense), net, for three months ended March 31, 2025 increased approximately $25 million, as compared to the same period in the prior year. The increase in other income in the three months ended March 31, 2025 as compared to the prior period was primarily due to a significant gain recognized in the current 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 loss in the prior period. These Class C warrants will continue to be measured at fair value and may continue to generate gains or losses each quarter, until they are exercised.

Current 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.

Previous wording

The income tax provision recorded for the three months ended March 31, 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 income (loss) for the three months ended March 31, 2025 and 2024.

Current wording

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 wording

warrants for the purchase of our preferred stock and our common stock, sales of preferred stock, proceeds from the issuance of

Current wording

To date, we have funded our operations primarily with proceeds from sales of common stock, warrants and prefunded warrants for the purchase of our preferred stock and our common stock, sales of preferred stock, proceeds from the issuance of convertible debt and borrowings under loan and security agreements.

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 $14.3 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 $75.0 million.

Current 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.

Previous wording

Hercules Loan Agreement—We have a Loan and Security Agreement, amended from time to time, with Hercules Capital, Inc. (the “Hercules Loan Agreement”). The Hercules Loan Agreement 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 March 31, 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 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.

Previous wording

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 March 31, 2025, we had $87.0 million of cash, cash equivalents and short-term marketable securities, and an accumulated deficit of $515.1 million. Net cash used in operating activities was $12.4 million for the three months ended March 31, 2025.

Current wording

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 wording

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 its forecast as approved by our 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.

Current wording

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 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.

Current 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.

Previous wording

Based on our cash, cash equivalents and marketable securities on hand as of March 31, 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, we will be required to raise additional capital, which may be through a combination of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements, or collaborations and strategic alliances.

Current 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.
Rearranged previous paragraphs (2)

Previous report — wording carried elsewhere

To date, we have funded our operations primarily with proceeds from sales of common stock, warrants and prefunded

Previous report — wording carried elsewhere

convertible debt and borrowings under loan and security agreements.