What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-08-11 compared with 2025-05-12 · 95 added, 60 removed, 145 unchanged (52% of the section changed)

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approval from the U.S.

Food and Drug Administration (“FDA”) in July 2019, and is currently approved and marketed in the U.S.

Food and Drug Administration in July 2019, and is currently approved and marketed in the U.S.

for the following indications:

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is managed by our partners in their respective territories.

XPOVIO/NEXPOVIO has received regulatory approval in various indications in over 45 countries outside the U.S.

XPOVIO/NEXPOVIO has received regulatory approval in various indications in 50 countries outside of the U.S.

and is commercially available in a growing number of countries as our partners continue to secure reimbursement approvals.

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As announced in January 2024, further clinical development of our eltanexor program continues to remain on hold in an effort to focus our resources on our prioritized late-stage programs.

As of March 31, 2025, we had an accumulated deficit of $1.6 billion.

We had net losses of $23.5 million and $37.4 million for the three months ended March 31, 2025 and 2024, respectively.

Based on our current business plan and current capital resources, combined with the uncertainty regarding the availability of additional funding, and considering our debt obligations, including the October 15, 2025 maturity date of our 3.00% convertible senior notes due 2025 (the “2025 Notes”) and a requirement of our other indebtedness to maintain cash, cash equivalents and investments of at least $25.0 million at all times, we have concluded that there is substantial doubt regarding our ability to continue as a going concern within one year after the date the accompanying condensed consolidated financial statements are issued.

As of June 30, 2025, we had an accumulated deficit of $1.6 billion.

We had net losses of $60.7 million and $13.6 million for the six months ended June 30, 2025 and 2024, respectively.

Based on our current business plan and current capital resources, given the uncertainty regarding the availability of additional funding or other strategic alternatives, and considering our debt service obligations, including our 3.00% convertible senior notes maturing on October 15, 2025 (the “2025 Notes”) with an aggregate principal amount of $24.5 million and a requirement of our Credit Agreement, as defined below, and the indenture governing our 6.00% convertible senior notes due 2029 (the “2029 Notes”) to maintain cash, cash equivalents and investments of at least $25.0 million at all times, we have concluded that there is substantial doubt regarding our ability to continue as a going concern within one year after the date the accompanying condensed consolidated financial statements are issued.

See “ Liquidity and Capital Resources ” below for a further discussion of our liquidity and the conditions that raise substantial doubt regarding our ability to continue as a going concern.

In May 2024, we entered into a series of transactions (the “Refinancing Transactions”) to limit our aggregate indebtedness, extend the maturity of certain of our indebtedness and provide us with additional working capital.

Pursuant to these transactions, we borrowed $100.0 million from existing lenders and certain entities managed by HealthCare Royalty Management, LLC (“HCRx”) under a new, senior secured term loan facility and used a portion of the proceeds of that loan to repay obligations under our existing financing arrangement with HCRx pursuant to an amendment that made other changes to our existing financing arrangement with HCRx.

We also exchanged, pursuant to privately negotiated agreements, an aggregate principal amount of $148.0 million of our existing 2025 Notes for (i) $111.0 million aggregate principal amount of our new 6.00% secured convertible senior notes due 2029 (the “2029 Notes”) and (ii) warrants to purchase up to 3.1 million shares of our common stock.

In addition, HCRx purchased $5.0 million aggregate principal amount of our 2029 Notes through satisfaction of $5.0 million of our existing obligations to HCRx.

Pursuant to these transactions, we borrowed $100.0 million from existing lenders and certain entities managed by HealthCare Royalty Management, LLC (“HCRx”), which was subsequently assigned by HCRx to KKR & Co.

(“KKR”) in connection with its acquisition of a majority ownership stake in HCRx in July 2025, under a new, senior secured term loan facility and used a portion of the proceeds of that loan to repay obligations under our existing financing arrangement with HCRx pursuant to an amendment that made other changes to our existing financing arrangement with HCRx.

We also exchanged, pursuant to privately negotiated agreements, an aggregate principal amount of $148.0 million of our existing 2025 Notes for (i) $111.0 million aggregate principal amount of our 2029 Notes and (ii) warrants to purchase up to 3.1 million shares of our common stock.

In addition, HCRx purchased $5.0 million aggregate principal amount of our 2029 Notes through satisfaction of $5.0 million of our existing obligations to KKR (as successor in interest to HCRx).

Please refer to Note 10 “ Long-Term Obligations ”, to the condensed consolidated financial statements contained within Part I, Item 1 of this Quarterly Report on Form 10-Q for additional details of the Refinancing Transactions.

On July 11, 2025, we announced that our Board is evaluating potential financing transactions, along with strategic alternatives, which may include a potential merger or sale of the Company;

in or out of court restructurings;

repurchases, redemptions, exchanges or other refinancings of our existing debt;

among other potential alternatives.

We, with the assistance of our advisors, are exploring potential financing and strategic alternatives to enhance liquidity and maximize value.

However, there is no assurance that such additional funding, financing transactions, or strategic alternatives will be available on terms acceptable to us, or at all.

In August 2025, we announced that we expect to close new patient screening in our Phase 3 myelofibrosis trial in August 2025 and expect to report top-line data from this trial in March 2026.

CRITICAL ACCOUNTING ESTIMATES

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The following table summarizes our results of operations (in thousands, except for percentages):

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Product revenue, net

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Other income (expense), net

Loss before income taxes

(Loss) income before income taxes

Income tax provision

Net (loss) income

Product Revenue, net (in thousands, except for percentages)

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Product revenue, net

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sales of XPOVIO.

Net product revenue for the three months ended March 31, 2025 decreased by $5.0 million as compared to the three months ended March 31, 2024, due to an increase in the gross-to-net provision largely due to the increase in the product return reserve.

These atypical returns were primarily driven by expired 80 mg and 100 mg units returned from clinics and hospitals that had purchased these units following the 2020 approval of XPOVIO ® 100 mg in combination with bortezomib and dexamethasone.

The majority of XPOVIO ® that is prescribed today are 40 mg and 60 mg doses.

We expect net product revenue to increase in the second quarter of 2025 as compared to the first quarter of 2025 primarily due to gross-to-net favorability with product returns expected to be more in-line with historical averages in future quarters.

Net product revenue for the three months ended June 30, 2025 increased by $1.6 million as compared to the three months ended June 30, 2024, due to gross-to-net favorability mainly driven by lower 340B discounts, which are discounts provided under the federal 340B Drug Pricing Program for eligible safety-net providers, in the second quarter of 2025.

Net product revenue for the six months ended June 30, 2025 decreased by $3.3 million as compared to the six months ended June 30, 2024, primarily due to an increase in the gross-to-net provision largely due to the increase in the product return reserve, partially offset by gross-to-net favorability driven by lower 340B discounts in the six months ended June 30, 2025.

The product return reserve increased as a result of atypical returns in the first quarter of 2025, primarily driven by expired units returned from clinics and hospitals that had purchased these units following the 2020 approval of XPOVIO ®.

We expect net product revenue to increase in the second half of 2025 as compared to the first half of 2025 due to gross-to-net favorability, driven by lower product returns and lower 340B discounts than the first half of the year, coupled with increased demand.

License and Other Revenue (in thousands, except for percentages)

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Menarini Group ("Menarini")

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Total license and other revenue

License and other revenue for the three months ended March 31, 2025 increased by $1.8 million as compared to the three months ended March 31, 2024 primarily due to the timing of $1.2 million of revenue recognized for the reimbursement of development-related expenses from Menarini.

We expect license and other revenue to be relatively consistent in the second quarter of 2025 as compared to the first quarter of 2025 based on the expected timing and amount of revenue recognition for the reimbursement of development-related expenses from Menarini in the second quarter of 2025.

License and other revenue for the three and six months ended June 30, 2025 decreased as compared to the three and six months ended June 30, 2024 by $6.5 million and $4.7 million, respectively, primarily due to $6.0 million of non-recurring license-related revenue recognized during the three months ended June 30, 2024.

We expect license and other revenue to decrease in the second half of 2025 as compared to the first half of 2025 primarily because the reimbursement of development-related expenses from Menarini is limited to $1.5 million for the second half of 2025.

Operating Expenses (in thousands, except for percentages)

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Cost of sales

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Cost of Sales

Cost of sales for the three months ended March 31, 2025 and 2024 were relatively consistent.

We expect cost of sales to remain relatively consistent in the second quarter of 2025 as compared to the first quarter of 2025.

Cost of sales for the three and six months ended June 30, 2025 and 2024 were relatively consistent.

We expect cost of sales to remain relatively consistent in the second half of 2025 as compared to the first half of 2025.

Research and Development Expenses (in thousands, except for percentages)

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Clinical trial and related costs:

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These costs represent expenses incurred across multiple programs or to support our general research and development operations.

Research and development expenses for the three months ended March 31, 2025 decreased by $0.8 million as compared to the three months ended March 31, 2024.

Research and development expenses for the three months ended June 30, 2025 decreased by $5.6 million as compared to the three months ended June 30, 2024, primarily due to the $1.7 million decrease in clinical trial and related costs for selinexor in multiple myeloma, which was primarily due to the reduced scope of our Phase 3 multiple myeloma trial, and the $2.1 million decrease in personnel and stock-based compensation costs, which was primarily due to a reduction in headcount and contractors for the three months ended June 30, 2025 as compared to the three months ended June 30, 2024 due to the realization of previously implemented cost reduction initiatives.

Research and development expenses for the six months ended June 30, 2025 decreased by $6.4 million as compared to the six months ended June 30, 2024.

The $3.8 million decrease in clinical trial and related costs for selinexor in multiple myeloma was primarily due to the reduced scope of our Phase 3 multiple myeloma trial.

The $1.9 million decrease in personnel and stock-based compensation costs was primarily due to a reduction in headcount and contractors for the three months ended March 31, 2025 as compared to the three months ended March 31, 2024 due to the realization of previously implemented cost reduction initiatives.

The $5.0 million increase in clinical trial and related costs for selinexor in myelofibrosis was primarily due to increased purchases of comparator drugs during the three months ended March 31, 2025 as compared to the three months ended March 31, 2024.

We expect our research and development expenses to be relatively consistent in the second quarter of 2025 as compared to the first quarter of 2025 as we continue to invest in our myelofibrosis and endometrial cancer Phase 3 clinical trials.

The $3.9 million decrease in personnel and stock-based compensation costs was primarily due to a reduction in headcount and contractors for the six months ended June 30, 2025 as compared to the six months ended June 30, 2024 due to the realization of previously implemented cost reduction initiatives.

The $4.1 million increase in clinical trial and related costs for selinexor in myelofibrosis was primarily due to increased purchases of comparator drugs during the six months ended June 30, 2025 as compared to the six months ended June 30, 2024.

We expect our research and development expenses to be relatively consistent in the second half of 2025 as compared to the first half of 2025 as we continue to invest in our myelofibrosis and endometrial cancer Phase 3 clinical trials.

Selling, General and Administrative Expenses (in thousands, except for percentages)

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Personnel costs

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Total selling, general and administrative expenses

Selling, general and administrative expenses for the three months ended March 31, 2025 decreased by $2.2 million as compared to the three months ended March 31, 2024.

The decrease in personnel and stock-based compensation costs of $2.2 million was primarily due to a reduction in headcount and contractors for the three months ended March 31, 2025 as compared to the three months ended March 31, 2024 primarily due to the realization of previously implemented cost reduction initiatives.

We expect our selling, general and administrative expenses to remain relatively consistent in the second quarter of 2025 as compared to the first quarter of 2025 as we continue to closely manage our spend.

Selling, general and administrative expenses for the three and six months ended June 30, 2025 decreased as compared to the three and six months ended June 30, 2024 by $2.6 million and $4.8 million, respectively, primarily due to a reduction in headcount and contractors resulting from previously implemented cost reduction initiatives.

Consulting, professional and other costs decreased $0.4 million for the three and six months ended June 30, 2025 as compared to three and six months ended June 30, 2024 due to a decrease in spend from cost reduction initiatives that was partially offset by an increase in legal expenses incurred in the three and six months ended June 30, 2025 in connection our recent evaluation of potential financings and other strategic transactions.

We expect our selling, general and administrative expenses to remain relatively consistent in the second half of 2025 as compared to the first half of 2025 due to our ongoing cost reduction initiatives, offset by fees to be incurred in connection with pursuit of strategic alternatives, such as efforts to extend our cash runway.

Other Income (Expense), net (in thousands, except for percentages)

For the Three Months Ended March 31,

For the Three Months Ended June 30,

For the Six Months Ended June 30,

Interest expense

Interest income

Gain on extinguishment of debt

Other (expense) income

Total other income (expense), net

Other income (expense), net for the three months ended March 31, 2025 increased by $13.4 million as compared to the three months ended March 31, 2024, primarily due to a $20.0 million non-cash gain from the remeasurement of embedded derivatives and liability classified common stock warrants.

These gains were partially offset by an increase in interest expense related to the senior secured term loan facility and 2029 Notes, both of which were issued in May 2024.

There was also a decrease in interest income resulting from lower investment balances during the three months ended March 31, 2025 as compared to the three months ended March 31, 2024.

We expect other income (expense), net to remain relatively consistent in the second quarter of 2025 as compared to the first quarter of 2025, however the future impact from remeasurements of the embedded derivatives and liability classified common stock warrants will depend on a variety of factors, including movements in our stock price.

Other income (expense), net for the three and six months ended June 30, 2025 decreased as compared to the three and six months ended June 30, 2024 by $64.8 million and $51.4 million, respectively, primarily due to a $44.7 million gain on extinguishment of debt from the Refinancing Transactions and a $14.3 million gain from the remeasurement of embedded derivatives and liability classified common stock warrants recognized during the three months ended June 30, 2024, both of which are non-cash items.

There was also an increase in interest expense related to the senior secured term loan facility and 2029 Notes, both of which were issued in May 2024 and a decrease in interest income resulting from lower investment balances during the three and six months ended June 30, 2025 as compared to the three and six months ended June 30, 2024.

We expect other income (expense), net to remain relatively consistent in the second half of 2025 as compared to the first half of 2025, however the future impact from remeasurements of the embedded derivatives and liability classified common stock warrants will depend on a variety of factors, including movements in our stock price.

LIQUIDITY AND CAPITAL RESOURCES

We have historically financed our operations primarily through a combination of proceeds from (i) product revenue sales, (ii) public and private placements of equity securities, (iii) the issuance of convertible debt, (iv) a term loan, (v) our deferred royalty obligation, (vi) at the market offerings and (vii) business development activities.

As of March 31, 2025, our principal source of liquidity was $69.9 million of cash, cash equivalents and investments.

We have had recurring losses since inception and incurred a loss of $23.5 million for the three months ended March 31, 2025.

As of June 30, 2025, our principal source of liquidity was $51.7 million of cash, cash equivalents and investments.

We have had recurring losses since inception and incurred a loss of $60.7 million for the six months ended June 30, 2025.

We anticipate that we will continue to incur significant operating losses in the foreseeable future.

Based on our current business plan and current capital resources, combined with the uncertainty regarding the availability of additional funding and considering our debt obligations, including the October 15, 2025 maturity date of our 2025 Notes and a requirement of our other indebtedness to maintain cash, cash equivalents and investments of at least $25.0 million at all times, we have concluded that there is substantial doubt regarding our ability to continue as a going concern within one year after the date the accompanying condensed consolidated financial statements are issued.

We expect that our cash, cash equivalents and investments as of March 31, 2025 will fund our current operating plans and debt obligation requirements into early fourth quarter of 2025.

See “ Liquidity and Capital Resources – Funding Requirements ” below and Note 1 “ Nature of Business ” to the condensed consolidated financial statements included under Part I, Item I of this Quarterly Report on Form 10-Q for a further discussion of our liquidity and the conditions that raise substantial doubt regarding our ability to continue as a going concern.

Based on our current business plan and current capital resources, given the uncertainty regarding the availability of additional funding or other strategic alternatives and considering our debt service obligations, including the October 15, 2025 maturity date of our 2025 Notes, with an aggregate principal amount of $24.5 million and a requirement of our Credit Agreement, as defined below, and the indenture governing our 6.00% convertible senior notes due 2029 (the “2029 Notes”) to maintain cash, cash equivalents and investments of at least $25.0 million at all times, we have concluded that there is substantial doubt regarding our ability to continue as a going concern within one year after the date the accompanying condensed consolidated financial statements are issued.

We currently expect that cash, cash equivalents and investments as of June 30, 2025 as well as cash flow from net product revenue and license and other revenue, will fund our current operating plans and debt obligation requirements into October 2025 given the $24.5 million aggregate principal amount of the 2025 Notes and $25.0 million minimum liquidity covenant.

Excluding repayment of the 2025 Notes and minimum liquidity covenant, we expect that our cash runway would be sufficient to fund planned operations into January 2026.

See “ Liquidity and Capital Resources – Funding Requirements ” below and Note 1 “ Nature of Business, Basis of Presentation and Segment Information ” to the condensed consolidated financial statements included under Part I, Item I of this Quarterly Report on Form 10-Q for a further discussion of our liquidity and the conditions that raise substantial doubt regarding our ability to continue as a going concern.

The following table provides information regarding our cash flows (in thousands):

For the Three Months Ended March 31,

For the Six Months Ended June 30,

Net cash used in operating activities

Net cash provided by investing activities

Net cash provided by financing activities

Effect of exchange rates on cash, cash equivalents and restricted cash

Net decrease in cash, cash equivalents and restricted cash

Net (decrease) increase in cash, cash equivalents and restricted cash

Operating activities.

The $4.7 million decrease in net cash used in operating activities for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 was primarily driven by changes in the timing of payments made for drug product.

The $24.5 million decrease in net cash used in operating activities for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was primarily driven by changes in working capital resulting primarily from decreased spend year-over-year as a result of the realization of previously implemented cost reduction initiatives.

Investing activities.

The $6.7 million decrease in net cash provided by investing activities for the three months ended March 31, 2025 compared to the three months ended March 31, 2024 was driven by a $38.5 million decrease in proceeds from the maturities of investments, partially offset by a $31.6 million decrease in purchases of investments.

The $39.6 million decrease in net cash provided by investing activities for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was driven by a $76.3 million decrease in proceeds from the maturities of investments, partially offset by a $36.5 million decrease in purchases of investments.

Financing activities .

The $40.6 million decrease in net cash provided by financing activities for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was driven primarily by the Refinancing Transactions which occurred during the six months ended June 30, 2024 and consisted of $83.3 million of proceeds from the term loan, partially offset by a $40.5 million payment of our deferred royalty obligation and a $2.6 million payment of debt issuance costs.

Sources of Liquidity

On September 14, 2019, we and certain of our subsidiaries entered into the Revenue Interest Financing Agreement with certain entities managed by HealthCare Royalty Management, LLC (“HCRx”), which was subsequently amended on June 23, 2021, August 1, 2023 and May 8, 2024 (the “Revenue Interest Agreement” and, as amended, the “Amended Revenue Interest Agreement”), pursuant to which, HCRx paid us a total of $135.0 million, less certain transaction expenses.

On September 14, 2019, we and certain of our subsidiaries entered into the Revenue Interest Financing Agreement with certain entities managed by HCRx, which was subsequently amended on June 23, 2021, August 1, 2023 and May 8, 2024, and which was subsequently assigned by HCRx to KKR in connection with its acquisition of a majority ownership stake in HCRx in July 2025 (the “Revenue Interest Agreement” and, as amended, the “Amended Revenue Interest Agreement”), pursuant to which, HCRx paid us a total of $135.0 million, less certain transaction expenses.

For additional information on the Amended Revenue Interest Agreement, see Note 10, “ Long-Term Obligations ”, to the condensed consolidated financial statements included under Part I, Item I of this Quarterly Report on Form 10-Q.

On May 8, 2024, we entered into a credit and guaranty agreement (the “Credit Agreement”) with certain existing lenders and HCRx, which provides for a senior secured term loan facility of $100.0 million.

On May 8, 2024, we entered into a credit and guaranty agreement (the “Credit Agreement”) with certain existing lenders and HCRx, which was subsequently assigned by HCRx to KKR in connection with its acquisition of a majority ownership stake in HCRx in July 2025, which provides for a senior secured term loan facility of $100.0 million.

For additional information, see Note 10, “ Long-Term Obligations ”, to the condensed consolidated financial statements included under Part I, Item I of this Quarterly Report on Form 10-Q.

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Under the 2023 Open Market Sale Agreement, we may issue and sell shares of our common stock having an aggregate offering price of up to $100.0 million (the “Shares”) from time to time through Jefferies.

We did not sell any Shares under the 2023 Open Market Sales Agreement during the three months ended March 31, 2025 and 2024.

As of March 31, 2025, $100.0 million of Shares was available for issuance and sale under the 2023 Open Market Sale Agreement.

We did not sell any Shares under the 2023 Open Market Sales Agreement during the three and six months ended June 30, 2025 and 2024.

As of June 30, 2025, $100.0 million of Shares was available for issuance and sale under the 2023 Open Market Sale Agreement.

Under the license agreement we entered into with Menarini in December 2021 (the “Menarini Agreement”), Menarini is required to reimburse us for 25% of all documented expenses we incur for the global development of selinexor from 2022 through 2025, provided that such reimbursements shall not exceed $15.0 million per calendar year.

We received $7.0 million of reimbursements under the Menarini Agreement during the six months ended June 30, 2025.

Commitments, Contingencies and Contractual Obligations

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We are party to an operating lease of office and research space in Newton, Massachusetts, which was amended in November 2024 and under which we currently lease a total of 98,502 square feet of research and office space through September 30, 2025, which will be reduced to 52,224 square feet of solely office space from October 1, 2025 through September 30, 2030.

As of March 31, 2025, we expect to incur total lease costs of $10.9 million from March 31, 2025 to September 30, 2030.

As of June 30, 2025, we expect to incur total lease costs of $10.8 million from June 30, 2025 to September 30, 2030.

Contractual Obligations

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Our ability to become and remain profitable depends on our ability to generate revenue.

There can be no assurance as to the amount or timing of any such revenue, and we may not achieve profitability for several years, if at all, as described more fully in the risk factor entitled “ We have incurred significant losses since inception, expect to continue to incur significant losses, and may never achieve or maintain profitability ,” under the heading “ Risk Factors ” in this Quarterly Report on Form 10-Q.

and may never achieve or maintain profitability ,” under the heading “ Risk Factors ” in this Quarterly Report on Form 10-Q.

Accordingly, we will need to continue to rely on additional financing to achieve our business objectives.

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We may seek additional capital due to favorable market conditions or strategic considerations, even if we believe we have sufficient funds for our current or future operating plans.

If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, reduce or eliminate our research and development programs or commercialization efforts.

See Note 1 “ Nature of Business ” to the condensed consolidated financial statements included under Part I, Item I of this Quarterly Report on Form 10-Q for a further discussion of the conditions that raise substantial doubt regarding our ability to continue as a going concern.

We currently expect that cash, cash equivalents and investments as of March 31, 2025 will fund our current operating plans and debt obligation requirements into early fourth quarter of 2025 while we continue to commercialize XPOVIO in the U.S.

and continue the clinical trials of our product candidates.

Our future long-term capital requirements will depend on many factors, as described more fully in the risk factor entitled “ We will need additional funding to achieve our business objectives.

If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, reduce or eliminate our research and development programs and/or commercialization efforts ,” under the heading “Risk Factors ” in this Quarterly Report on Form 10-Q.

In addition to the expenses required to fund our operations described above, our funding requirements as of March 31, 2025 also include the following:

If we are unable to raise capital or enter into strategic alternatives sufficient to meet our needs or on attractive terms, we would be forced to delay, reduce or eliminate our research and development programs or commercialization efforts.

Based on our current business plan and current capital resources, given the uncertainty regarding the availability of additional funding or other strategic alternatives and considering our debt service obligations, including the October 15, 2025 maturity date of the 2025 Notes with an aggregate principal amount of $24.5 million and a requirement under our Credit Agreement and the indenture governing the 2029 Notes to maintain cash, cash equivalents and investments of at least $25.0 million at all times, we have concluded that there is substantial doubt regarding our ability to continue as a going concern within one year after the date the accompanying consolidated financial statements are issued.

See Note 1 “ Nature of Business, Basis of Presentation and Segment Information ” to the condensed consolidated financial statements included under Part I, Item I of this Quarterly Report on Form 10-Q for a further discussion of the conditions that raise substantial doubt regarding our ability to continue as a going concern.

Excluding repayment of the 2025 Notes and minimum liquidity covenant, we expect that our cash runway would be sufficient to fund planned operations into January 2026.

Our future long-term capital requirements will depend on many factors, as described more fully in the risk factor entitled “ We will need additional funding or to enter into strategic alternatives to achieve our business objectives.

If we are unable to raise sufficient capital or to enter into strategic alternatives on acceptable terms to meet our needs, we may be forced to delay, reduce or eliminate our research and development programs and/or commercialization efforts ,” under the heading “Risk Factors ” in this Quarterly Report on Form 10-Q.

In addition to the expenses required to fund our operations described above, our funding requirements as of June 30, 2025 also include the following:

• Lease costs of our headquarters in Newton, Massachusetts of $10.8 million through September 30, 2030;

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• Future obligations related to the Credit Agreement of $135.0 million through May 2028 in addition to our requirement to maintain cash, cash equivalents and investments of at least $25.0 million at all times;

• Future royalty obligations to HCRx under the Amended Revenue Interest Agreement of $117.8 million by October 1, 2031.

• Future royalty obligations to KKR under the Amended Revenue Interest Agreement of $116.2 million by October 1, 2031.

On July 11, 2025, we announced that our Board is considering potential financing transactions along with strategic alternatives to maximize near and long-term stockholder value, including, but not limited to, a merger or sale of the Company, in or out of court restructurings, repurchases, redemptions, exchanges or other refinancings of our existing debt, and financing transactions, among other potential alternatives.

Our ability to successfully execute on a strategic alternative is dependent on a number of factors and we may not be able to execute upon a transaction or other strategic alternative having favorable terms within an advantageous time frame and/or recognize significant value for our assets, if at all.

Further, our indebtedness, as discussed under the risk factor titled “ Our indebtedness could limit cash flow available for our operations, expose us to risks that could adversely affect our business, financial condition and results of operations and impair our ability to satisfy our obligations under the Term Loan, the 2029 Notes, the 2025 Notes or the Amended Revenue Interest Agreement ,” may be unattractive to potential sources of funding and strategic partners and may decrease our ability to consummate a financing transaction or enter into a strategic alternative.

Additionally, the negotiation and consummation of a financing transaction or strategic alternative may be costly and time-consuming.

If we are not able to successfully consummate a successful financing transaction or strategic alternative, our Board may decide to pursue a dissolution and liquidation of our company.

In such an event, we would be required under Delaware corporate law to pay our outstanding obligations, as well as to make reasonable provision for contingent and unknown obligations, prior to making any distributions in liquidation to our stockholders.

As a result of this requirement, a portion of our assets may need to be reserved pending the resolution of such obligations.

In addition, we may be subject to litigation or other claims related to a dissolution and liquidation of our company.

If our Board determined to pursue a dissolution and liquidation, our Board, in consultation with its advisors, would need to evaluate these matters and make a determination about a reasonable amount to reserve.

Depending on these factors, the amount available for distribution to our common stockholders in such an event could be as low as zero and result in a total loss of investment to our stockholders.

Quantitative and Qualitati ve Disclosures About Market Risk.

We are exposed to market risk related to changes in interest rates.

We had cash, cash equivalents and investments of $69.9 million as of March 31, 2025.

We had cash, cash equivalents and investments of $51.7 million as of June 30, 2025.

Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 2. Management’s Discussion and Analysis