What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-08-06 compared with 2025-05-07 · 85 added, 79 removed, 248 unchanged (40% of the section changed)

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You should not place undue reliance on these forward-looking statements, which apply only as of the date of this Report.

Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including the risks faced by us and described in Part II, Item 1A, entitled “Risk Factors,” and in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part I, Item 2 of this Report.

Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including the risks faced by us and described in Part II, Item 1A, titled “Risk Factors,” and in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Part I, Item 2 of this Report.

The following discussion should be read in conjunction with the unaudited condensed consolidated financial statements and notes thereto included in Part I, Item 1 of this Report;

and the sections entitled “Business” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” contained in our Form 10-K for the year ended December 31, 2024 as filed with the SEC on February 27, 2025, or the 2024 Form 10-K.

and the sections titled “Business” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” contained in our Form 10-K for the year ended December 31, 2024 as filed with the SEC on February 27, 2025, or the 2024 Form 10-K.

Company Overview

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high-risk myelofibrosis, or MF, who have relapsed after or are refractory to treatment with a janus associate kinase inhibitor, or JAK inhibitor, or relapsed/refractory MF with overall survival, or OS, as the primary end point.

As of May 2025, the trial has reached approximately 85% enrollment.

As of the end of July 2025, the trial has reached over 95% enrollment and we expect to complete enrollment by the end of 2025.

Based on our current planning assumptions for enrollment and event (death) rates in the trial, we expect the interim analysis for OS in IMpactMF may occur in the second half of 2026 and the final analysis may occur in the second half of 2028.

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Since our inception, we have primarily financed our operations through the sale of equity securities, draw downs on our debt facilities, interest income on our marketable securities, and payments we received under the Royalty Pharma Agreement and our prior collaborative and licensing arrangements.

As of March 31, 2025, we had approximately $457.5 million in cash, cash equivalents, restricted cash and marketable securities.

As of June 30, 2025, we had approximately $432.6 million in cash, cash equivalents, restricted cash and marketable securities.

We began commercializing RYTELO in June 2024, and the commercial potential of and our ability to successfully commercialize RYTELO remains unproven.

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We have incurred significant net losses since our inception in 1990, resulting principally from costs incurred in connection with our research and development activities and from general and administrative costs associated with our operations.

As of March 31, 2025, we had an accumulated deficit of approximately $1.8 billion.

As of June 30, 2025, we had an accumulated deficit of approximately $1.8 billion.

On November 1, 2024, we entered into a loan agreement, or the Pharmakon Loan Agreement, with BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, each, a Lender, which are investment funds managed by Pharmakon Advisors, LP, and BioPharma Credit PLC, as collateral agent, that provides for a 5-year senior secured term loan facility of up to $250.0 million, divided into three committed tranches.

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for lower-risk MDS or to expand its indications of use.

As previously disclosed, weekly sales growth of RYTELO has been relatively flat since the end of the fourth quarter of 2024, which has continued in the first quarter of 2025, resulting in lower net product revenue in the first quarter of 2025 of approximately $39.4 million compared to approximately $47.5 million in the fourth quarter of 2024.

Our priority is to drive new patient starts

across the breadth of the eligible patient population in RYTELO’s approved indication in the U.S.

We have seen and may continue to see variability in RYTELO sales trends.

Net product revenue was approximately $49.0 million in the second quarter of 2025, $39.4 million in the first quarter of 2025, and $47.5 million in the fourth quarter of 2024.

Our priority is to drive new patient starts across the breadth of the eligible patient population in RYTELO’s approved indication in the U.S.

Our strategy to drive sales growth in the U.S.

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However, this strategy and our ongoing commercialization efforts may not achieve meaningful sales growth, which may require us to, among others, further adjust or amend our commercialization strategy and plans and incur significant expenses, and there can be no assurance that we will be able to grow RYTELO net product revenues in future periods.

In particular, our strategy may be unable to drive new patient starts across the breadth of the eligible patient population in RYTELO's approved indication in a timely manner or at all, or the duration of therapy could be shorter than we expect, each of which would limit RYTELO’s growth potential and could preclude or delay our ability to generate meaningful revenue from product sales and to achieve profitability.

In particular, our strategy may not drive new patient starts across the breadth of the eligible patient population in RYTELO's approved indication in a timely manner or at all, or the duration of therapy could be shorter than we expect, each of which would limit RYTELO’s growth potential and could preclude or delay our ability to generate meaningful revenue from product sales and to achieve profitability.

In addition, in an effort to expand its indications of use, we are also developing RYTELO for the treatment of several myeloid hematologic malignancies that will continue to require additional time and significant investment in clinical trials to complete.

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A critical accounting policy is one that is both important to the portrayal of our financial condition and results of operations and requires management’s most difficult, subjective or complex judgments, often as a result of the need to make estimates about the effect of matters that are inherently uncertain.

Our critical accounting policies are described in Item 7, “Critical Accounting Policies and Estimates” in our Annual Report on Form 10-K.

There have been no significant changes in our critical accounting policies and estimates during the three months ended March 31, 2025, as compared to the critical accounting policies and estimates disclosed in our 2024 Form 10-K.

Our critical accounting policies are described in Item 7, “Critical Accounting Policies and Estimates” in our 2024 Form 10-K.

There have been no significant changes in our critical accounting policies and estimates during the three months and six months ended June 30, 2025, as compared to the critical accounting policies and estimates disclosed in our 2024 Form 10-K.

RESULTS OF OPERATIONS

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In this regard, although we have begun to recognize revenue from RYTELO product sales in the U.S., we are early in our commercialization efforts.

We expect that our sales revenue may vary significantly from period to period as our commercialization efforts progress.

We expect that our sales revenue may vary from period to period as our commercialization efforts progress.

RYTELO is our only product approved for marketing in the U.S.

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our ability to obtain and maintain contractual arrangements, strategic partnerships, collaborations, alliances or licensing arrangements with third parties to assist us with the commercialization of RYTELO in jurisdictions outside of the U.S.;

overcoming disruptions and/or delays due to macroeconomic or other global conditions, such as further changes in tariffs and other trade restrictions, renegotiation of existing international trade agreements or further escalation of trade tensions, inflation, rising interest rates, prospects of a recession, government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues;

overcoming disruptions and/or delays due to macroeconomic or other global conditions, such as further changes in tariffs and other trade restrictions, renegotiation of existing international trade agreements or further escalation of trade tensions, inflation, fluctuations in interest rates, prospects of a recession, government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues;

our ability to obtain additional capital if and when needed;

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and potential competition.

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

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

Three Months Ended June 30, Six Months Ended June 30,

Change $ Change % 2025 2024

Change $ Change %

(in thousands, except for percentage data)

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Total revenues 49,036 882 48,154 * 88,639 1,186 87,453 *

Operating expenses:

Costs and operating expenses:

Cost of goods sold 1,190 17 1,173 * 2,396 17 2,379 *

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Product Revenues, Net

On June 6, 2024, we announced that the FDA approved RYTELO for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes, or lower-risk MDS, with transfusion-dependent, or TD, anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents, or ESA.

On June 6, 2024, we announced that the FDA approved RYTELO for the treatment of adult patients with lower-risk MDS, with TD anemia requiring four or more red blood cell units over eight weeks who have not responded to, or have lost response to, or are ineligible for ESAs.

To date, our only source of product revenue has been from the U.S.

sales of RYTELO, which we began shipping to our customers in June 2024.

We did not generate any revenue from product sales prior to the three months ended June 30, 2024 .

Total product revenue, net for the three months ended March 31, 2025 was approximately $39.4 million.

Total gross-to-net adjustments for the three months ended March 31, 2025 was 13.0% of gross product revenue.

The reconciliation of gross product sales to net product sales by each significant category of gross-to-net adjustments was as set forth below for the three months ended March 31, 2025.

We expect total gross-to-net adjustments to be in the mid- to high-teens percentage of gross product revenue in the remaining quarters of 2025.

We did not generate any revenue from product sales prior to June 30, 2024 .

Total product revenues, net for the three and six months ended June 30, 2025 were approximately $49.0 million and $88.4 million, respectively.

Total gross-to-net adjustments for the three and six months ended June 30, 2025 was 15.3% and 14.3% of gross product revenue, respectively.

The reconciliation of gross product sales to net product sales by each significant category of gross-to-net adjustments was as set forth below for the three and six months ended June 30, 2025.

total gross-to-net adjustments to be in the mid- to high-teens percentage of gross product revenue in the remaining quarters of 2025.

(in thousands)

Three Months Ended

March 31, 2025

June 30, 2025 Three Months Ended

June 30, 2024 Six Months Ended

June 30, 2025 Six Months Ended

June 30, 2024

Gross product revenue $ 57,874 $ 890 $ 103,186 $ 890

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Chargebacks and customer credits

(7,890) (97) (13,587) (97)

Government rebates (459) (11) (525) (11)

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Net product revenue $ 49,007 $ 780 $ 88,443 $ 780

which acquired Asterias Biotherapeutics, Inc.) in 2013, we are entitled to receive royalties on sales from certain research or commercial products utilizing our divested intellectual property.

We recognized royalty revenues of $167,000 in the three months ended March 31, 2025, compared to $304,000 for the same period in 2024.

Royalty revenues in the three months ended March 31, 2025 and 2024 primarily reflect estimated royalties from sales of cell-based research products from our divested stem cell assets.

In connection with the divestiture of our human embryonic stem cell assets, including intellectual property and proprietary technology, to Lineage Cell Therapeutics, Inc., or Lineage, (formerly BioTime, Inc., which acquired Asterias Biotherapeutics, Inc.) in 2013, we are entitled to receive royalties on sales from certain research or commercial products utilizing our divested intellectual property.

We recognized royalty revenues of $29,000 and $196,000 in the three and six months ended June 30, 2025, respectively, compared to $102,000 and $406,000 for the same periods in 2024.

Royalty revenues in the three and six months ended June 30, 2025 and 2024 primarily reflect estimated royalties from sales of cell-based research products from our divested stem cell assets.

Future license fee and royalty revenues are dependent on additional agreements being signed, if any, our current license agreement with Lineage being maintained and the underlying patent rights for the license remaining active.

Operating Expenses:

In connection with the FDA approval of RYTELO on June 6, 2024, we subsequently begin capitalizing inventory manufactured or purchased after this date.

In connection with the FDA approval of RYTELO on June 6, 2024, we began capitalizing inventory manufactured or purchased after this date.

As a result, we expensed certain manufacturing costs of RYTELO as research and development expense prior to FDA approval and, therefore, these costs are not included in cost of goods sold.

We expect our operating expenses to be in the range of approximately $270 million to $285 million for 2025, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

We expect our operating expenses in 2025 to increase compared to 2024, primarily due to continued investment in our RYTELO commercialization strategy, investment in commercial supply redundancy, and post marketing commitments, as well as initial preparations to launch RYTELO in selected EU countries in 2026, including the HTA evaluation process.

The following table summarizes our expenses, including as a percentage of total expenses, for the three months ended March 31, 2025:

Three Months Ended March 31,

(In thousands) 2025 2024 Change %

We expect our operating expenses to be in the range of approximately $270 million to $285 m illion for 2025, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization .

The following table summarizes our expenses, including as a percentage of total expenses, for the three and six months ended June 30 :

Three Months Ended June 30, Six Months Ended June 30,

(In thousands) 2025 2024 Change % 2025 2024 Change %

Cost of goods sold $ 1,190 $ 17 * $ 2,396 $ 17 *

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Our cost of goods sold consist of raw materials, third-party manufacturing costs to manufacture the raw materials into finished product, freight, and indirect overhead costs associated with the sale of RYTELO in the U.S.

Cost of goods sold was approximately $1.2 million for the three months ended March 31, 2025, which consisted of costs to manufacture, and distribute our market product, RYTELO.

We began capitalizing inventory upon FDA approval of

Cost of goods sold was approximately $1.2 million and $2.4 million for the three and six months ended June 30, 2025, respectively, which consisted of costs to manufacture and distribute our market product, RYTELO.

We began capitalizing inventory upon FDA approval of RYTELO.

All product costs incurred prior to FDA approval of RYTELO in June 2024 were expensed as research and development expenses.

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Prior to receiving FDA approval for RYTELO in June 2024, we manufactured inventory to be sold upon commercialization and recorded the costs as research and development expense.

As a result, a significant portion of the manufacturing costs related to the inventory manufactured prior to receiving FDA approval were expensed in a prior period and are therefore excluded from the cost of goods sold for the three months ended March 31, 2025.

We estimate our cost of sales related to product revenue as a percentage of net product revenue will continue to be positively affected for the next 15 to 21 months as we sell through certain inventory that was previously expensed prior to FDA approval.

We estimate our cost of sales related to product revenue as a percentage of net product revenue will continue to be positively impacted for the next 12 to 15 months as we sell through certain inventory that was previously expensed prior to FDA approval.

Research and Development Expenses

During the three months ended March 31, 2025 and 2024, our RYTELO (imetelstat) program and our research discovery program related to potential next generation telomerase inhibitors were the only research and development programs we supported.

During the three and six months ended June 30, 2025 and 2024, our RYTELO (imetelstat) program and our research discovery program related to potential next generation telomerase inhibitors were the only research and development programs we supported.

For these research and development programs, we incur direct external, personnel-related and other research and development costs.

For the three months ended March 31, 2025 and 2024, research and development expenses consist of expenses incurred in developing and testing imetelstat and research related to potential next generation telomerase inhibitors.

These expenses include, but are not limited to, payroll and personnel expense, lab supplies, non-clinical studies, clinical trials, including support for investigator-led clinical trials, raw materials to manufacture clinical trial supply, manufacturing costs for research and clinical trial materials, sponsored research at other labs, consulting, costs to maintain technology licenses and research-related overhead.

We expect our research and development expenses to increase over the remainder of the year, primarily due to post-marketing commitments.

Research and development expenses for the three months ended March 31, 2025 and 2024 were as follows:

Three Months Ended March 31,

For the three and six months ended June 30, 2025 and 2024, research and development expenses consist of expenses incurred in developing and testing imetelstat and research related to potential next generation telomerase inhibitors.

These expenses include, but are not limited to, payroll and personnel expense, lab supplies, non-clinical studies, clinical trials, including support for investigator-led clinical trials, raw materials to manufacture clinical trial supply, manufacturing costs for research and clinical trial materials, sponsored research at other labs, consulting expenses, costs to maintain technology licenses and research-related overhead.

We expect our research and development expenses to increase over the remainder of the year, primarily due to our Phase 3 IMpactMF clinical trial and post-marketing commitments.

Research and development expenses for the three and six months ended June 30, 2025 and 2024 were as follows:

Three Months Ended June 30, Six Months Ended June 30,

(In thousands) 2025 2024 2025 2024

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Total research and development expenses $ 21,736 $ 30,779 $ 36,814 $ 60,152

The decrease in research and development expenses for the three months ended March 31, 2025, compared to the same period in 2024, was primarily due to decreased clinical trial costs associated with a decrease of activity in our IMerge MDS study after FDA approval of RYTELO in 2024, as well as manufacturing and quality costs that were capitalized in the current period now that RYTELO is approved, versus being expensed in the prior period.

A discussion of the risks and uncertainties associated with the development of imetelstat can be found in the sub‑sections entitled “ Risks Related to the Further Development of Imetelstat ” and “ Risks Related to the Commercialization of RYTELO ® (Imetelstat)” and “Risks Related to Regulatory Approval of RYTELO ” in Part II, Item 1A entitled “Risk Factors” and elsewhere in this Report.

The decrease in research and development expenses for the three and six months ended June 30, 2025, compared to the same periods in 2024, was primarily due to decreased clinical trial costs associated with a decrease of activity in our Phase 3 IMerge MDS study after FDA approval of RYTELO in 2024, as well as manufacturing and quality costs that were capitalized in the current period now that RYTELO is approved, versus being expensed in the prior period.

A discussion of the risks and uncertainties associated with the development of imetelstat can be found in the sub‑sections titled “ Risks Related to the Further Development of Imetelstat ”, “ Risks Related to the Commercialization of RYTELO ® (Imetelstat)” and “Risks Related to Regulatory Approval of RYTELO ” in Part II, Item 1A titled “Risk Factors” and elsewhere in this Report.

As a result of these risks and uncertainties, we are unable to determine with any degree of certainty the duration and completion costs of ongoing and potential future imetelstat research and development projects, anticipated completion dates, or when and to what extent we will receive cash inflows from the commercialization and sale of RYTELO in any other jurisdictions or indications we are pursuing or may in the future pursue, if at all.

Selling, General and Administrative Expenses

Selling, general and administrative expenses were $40.0 million for the three months ended March 31, 2025, compared to $27.1 million for the same period in 2024.

The increase in selling, general and administrative expenses for the three months ended March 31, 2025, compared to the same period in 2024, primarily due to higher personnel-related expenses from increased headcount to support the commercial launch of RYTELO.

Selling, general and administrative expenses were $38.6 million and $78.6 million for the three and six months ended June 30, 2025, respectively, compared to $39.4 million and $66.5 million for the same periods in 2024.

The decrease in selling, general and administrative expenses for the three months ended June 30, 2025, compared to the same period in 2024, is attributed to initial RYTELO launch costs in 2024.

The increase in the six months ended June 30,2025 is primarily due to higher personnel-related expenses from increased headcount to support the commercialization of RYTELO.

We expect our selling, general and administrative expenses to increase over the remainder of the year in support of our commercialization efforts.

Interest Income

Interest income was $5.2 million for the three months ended March 31, 2025, compared to $4.2 million for the same period in 2024.

The increase in interest income for the three months ended March 31, 2025, compared to the same period in 2024, primarily reflects a larger marketable securities portfolio with the receipt of net cash proceeds from the Pharmakon Loan Agreement and the Royalty Pharma Agreement with Royalty Pharma entered into in November of 2024.

The increase in interest income for the three months ended March 31, 2025, compared to the same period in 2024 was partially offset by a decrease in interest rates.

Interest income was $4.7 million and $9.8 million for the three and six months ended June 30, 2025, respectively, compared to $5.3 million and $9.6 million for the same periods in 2024.

The decrease in interest income for the three months ended June 30, 2025, compared to the same period in 2024, primarily reflects a smaller marketable securities portfolio with the receipt of net cash proceeds from the underwritten public offering completed in March 2024.

The increase in interest income for the six months ended June 30, 2025, compared to the same period in 2024, was partially offset by an increase in interest rates.

Interest earned in future periods will depend on the size of our marketable securities portfolio and prevailing interest rates.

Interest Expense

Interest expense was $8.2 million for the three months ended March 31, 2025, compared to $3.4 million for the same period in 2024.

The increase in interest expense for the three months ended March 31, 2025, compared to the same period in 2024, primarily reflects $4.8 million in non-cash interest expense related to the Royalty Pharma Agreement and $3.3 million in interest expense related to the Pharmakon Loan Agreement.

Interest expense was $8.5 million and $16.7 million for the three and six months ended June 30, 2025, respectively, compared to $3.3 million and $6.8 million for the same periods in 2024.

The increase in interest expense for the three and six months ended June 30, 2025, compared to the same periods in 2024, primarily reflects interest expense related to the Royalty Pharma Agreement and the Pharmakon Loan Agreement which were entered into in November 2024.

We accounted for the Royalty Pharma Agreement as a liability financing, primarily because it has significant continuing involvement in generating the future revenue on which the Royalty Payments are based.

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Other Income and (Expense), Net

Other income and expense, net was an expense of $83,000 for the three months ended March 31, 2025, compared to an expense of $62,000 for the three months ended March 31, 2024.

Other income and expense, net was an expense of $61,000 and $144,000 for the three and six months ended June 30, 2025, respectively, compared to an expense of $63,000 and $125,000 for the three and six months ended June 30, 2024.

Other income and (expense), net, primarily reflects bank charges related to our cash operating accounts and marketable securities portfolio and foreign currency transaction adjustments.

LIQUIDITY AND CAPITAL RESOURCES

As of March 31, 2025, we had cash, restricted cash, cash equivalents, and marketable securities of $457.5 million, compared to $502.9 million at December 31, 2024.

The decrease in cash, restricted cash, cash equivalents and marketable securities during the three months ended March 31, 2025 was primarily the net result of cash used in operations partially offset by the receipt of net cash proceeds of $ 45.9 million from accounts receivable.

On March 21, 2024, we completed an underwritten public offering consisting of 41,999,998 shares of our common stock and a pre-funded warrant to purchase 8,002,668 shares of our common stock.

As of June 30, 2025, we had cash, restricted cash, cash equivalents, and marketable securities of $432.6 million, compared to $502.9 million at December 31, 2024.

The decrease in cash, restricted cash, cash equivalents and marketable securities during the six months ended June 30, 2025 was primarily the net result of cash used in operations partially offset by the receipt of net cash proceeds of $0.7 million from accounts receivable.

On March 21, 2024, we completed an underwritten public offering consisting of 41,999,998 shares of our common stock and a pre-funded warrant, or 2024 pre-funded warrant, to purchase 8,002,668 shares of our common stock.

All of the securities were issued separately.

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The 2024 pre-funded warrant has an exercise price of $0.001 per share and may be exercised at any time until it is exercised in full.

The net cash proceeds from this offering were approximately $141.0 million, after deducting the underwriting discount and other offering expenses paid by us, and excluding any future proceeds from the exercise of the pre-funded warrant.

As of March 31, 2025, 5,380,000 pre-funded warrants issued in connection with our 2024 underwritten public offering have been net exercised, which resulted in the issuance of 5,378,199 shares of our common stock.

The net cash proceeds from this offering were approximately $141.0 million, after deducting the underwriting discount and other offering expenses paid by us, and excluding any future

proceeds from the exercise of the pre-funded warrant.

As of June 30, 2025, 5,380,000 pre-funded warrants issued in connection with our 2024 underwritten public offering have been net exercised, which resulted in the issuance of 5,378,199 shares of our common stock.

See Note 8 on Stockholders’ Equity in Notes to Condensed Consolidated Financial Statements of this Report for additional information about the underwritten offering completed in March 2024.

From January 1, 2025 through March 31, 2025, there were no exercises of purchase warrants that we issued in connection with an underwritten public offering of our securities in 2020.

As of March 31, 2025, we had purchase warrants with an exercise price of $1.30 per share exercisable for 1,402,520 shares of our common stock remaining, which if exercised in full for cash, would provide $1.8 million in cash proceeds.

From January 1, 2025 through June 30, 2025, there were no exercises of purchase warrants that we issued in connection with an underwritten public offering of our securities in 2020.

As of June 30, 2025, we had purchase warrants with an exercise price of $1.30 per share exercisable for 1,402,520 shares of our common stock remaining, which if exercised in full for cash, would provide $1.8 million in cash proceeds.

On November 1, 2024, we entered into the Pharmakon Loan Agreement.

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Successful drug development and commercialization requires significant amounts of capital.

As of March 31, 2025, we had approximately $457.5 million in cash, cash equivalents, restricted cash and marketable securities.

Based on our current operating plans and assumptions, we believe that our existing cash, cash equivalents, and marketable securities, together with anticipated net revenues from U.S.

As of June 30, 2025, we had approximately $432.6 million in cash, cash equivalents, restricted cash and marketable securities.

our current operating plans and assumptions, we believe that our existing cash, cash equivalents, and marketable securities, together with anticipated net revenues from U.S.

sales of RYTELO, will be sufficient to fund our projected operating requirements for the foreseeable future.

However, if we do not generate net revenues from commercial sales of RYTELO at the levels we anticipate, if we experience unforeseen events or choose to make other investments in our business, or our assumptions regarding our projected operating expenses are otherwise incorrect, we may require additional funding, which could include a combination of public or private equity offerings, debt financings (including additional tranches under the Pharmakon Loan Agreement, if available), collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible.

For example, changes in our operations, such as increased development, manufacturing and clinical trial expenses, or our undertaking of additional programs, business activities, or entry into strategic transactions, including potential future acquisitions of products, technologies or businesses,

may cause our operating expenses to increase, perhaps significantly, which could require us to raise additional funding.

If adequate funds are not available to us when we need them, our RYTELO commercialization efforts may be adversely affected and we may be unable to pursue further development of imetelstat, which would severely harm our business and we might cease operations.

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We may never recoup our investment in any RYTELO development, which would adversely affect our financial condition and our business and business prospects, and might cause us to cease operations.

In addition, our plans and timing expectations could be further delayed or interrupted by the effects of macroeconomic or other global conditions, including those resulting from further changes in tariffs and other trade restrictions, renegotiation of existing international trade agreements or further escalation of trade tensions, inflation, rising interest rates, prospects of a recession, government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues.

In addition, our plans and timing expectations could be further delayed or interrupted by the effects of macroeconomic or other global conditions, including those resulting from further changes in tariffs and other trade restrictions, renegotiation of existing international trade agreements or further escalation of trade tensions, inflation, fluctuations in interest rates, prospects of a recession, government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues.

Further, our future capital requirements ar e difficult to forecast and will depend on many factors, including:

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We may be unable to raise equity capital, or may be forced to do so at a stock price or on other terms that could result in substantial dilution of ownership for our stockholders.

The receptivity of the public and private debt and equity markets to proposed financings has been substantially affected by uncertainty in the general economic, market and political climate due to the effects of macroeconomic or other global conditions, such as further changes in tariffs and other trade restrictions and uncertainty around further escalation of trade tensions and renegotiation of existing international trade agreements, inflation, rising interest rates, prospects of a recession, government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues, and may in the future be affected by other factors which are unpredictable and over which we have no control.

The receptivity of the public and private debt and equity markets to proposed financings has been substantially affected by uncertainty in the general economic, market and political climate due to the effects of macroeconomic or other global conditions, such as further changes in tariffs and other trade restrictions and uncertainty around further escalation of trade tensions and renegotiation of existing international trade agreements, inflation, fluctuations in interest rates, prospects of a recession, government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues, and may in the future be affected by other factors which are unpredictable and over which we have no control.

These effects have increased market volatility and could result in a significant long-term disruption of global financial mar kets, which could reduce or eliminate our ability to raise additional funds through financings, and could negatively impact the terms upon which we may raise those funds.

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In addition, we have borrowed, and in the future may borrow, additional capital from institutional and commercial banking sources to fund development and our future growth, including pursuant to our Pharmakon Loan Agreement or potentially pursuant to new arrangements with different lenders.

We may borrow funds on terms under agreements, such as our Pharmakon Loan Agreement, that include restrictive covenants, including covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

We may borrow funds on terms under agreements, such as our Pharmakon Loan Agreement, that include restrictive covenants, including covenants limiting or

restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

Moreover, if we raise additional funds through alliance, collaborative or licensing arrangements with third parties, we may have to relinquish valuable rights to RYTELO or our technologies or grant licenses on terms that are not favorable to us.

Cash Flows from Operating Activities

Net cash used in operations for the three months ended March 31, 2025 and 2024 was $48.0 million, and $62.3 million respectively.

The decrease in net cash used in operations for the three months ended March 31, 2025,

compared to the same period in 2024, primarily reflects an decrease in net loss to $19.8 million, adjusted for non-cash items including stock-based compensation expense related for employees and directors.

Net cash used in operations for the six months ended June 30, 2025 and 2024 was $75.4 million and $115.7 million, respectively.

The decrease in net cash used in operations for the six months ended June 30, 2025, compared to the same period in 2024 , primarily reflects a decrease in net loss of $36.2 million, adjusted for non-cash items including stock-based compensation expense for employees and directors.

Cash Flows from Investing Activities

Net cash provided by investing activities was $52.4 million for the three months ended March 31, 2025 and $34.2 million for the three months ended March 31, 2024.

The increase in net cash used in investing activities for the three months ended March 31, 2025, compared to the same period in 2024, primarily reflects decreased purchases of marketable securities.

Net cash provided by investing activities was $72.8 million for the six months ended June 30, 2025 and net cash used by investing activities was $1.9 million for the six months ended June 30, 2024.

The increase in net cash provided by investing activities for the six months ended June 30, 2025, compared to the same period in 2024, primarily reflects decreased purchases of marketable securities.

Cash Flows from Financing Activities

Net cash provided by financing activities for the three months ended March 31, 2025 and 2024 was $0.3 million and $147.8 million, respectively.

Net cash provided by financing activities for the six months ended June 30, 2025 and 2024 was $1.3 million and $164.6 million, respectively.

Financing activities in 2024 primarily reflect an underwriting offering of 41,999,998 shares of common stock and a pre-funded warrant to purchase 8,002,668 shares of common stock, resulting in net cash proceeds of $141.0 million completed in March 2024.

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We have engaged third‑party contract manufacturers and have re-established our own manufacturing supply chain to manufacture and supply quantities of RYTELO that meet applicable regulatory standards for current and potential future clinical trials and commercial uses.

Related to those contract manufacturing agreements, we have commercial purchase commitments f or approximately $110.9 million in the aggregate as of March 31, 2025.

Related to those contract manufacturing agreements, we have commercial purchase commitments f or approximatel y $124.7 million in the aggregate as of June 30, 2025.

These purchase commitments can vary based on the commercial demand of RYTELO and are binding based on future manufacturing needs.

As of March 31, 2025, we had a long-term principal debt balance of $118.7 million, consisting of $125.0 million aggregate principal amount of the Tranche A Loan under the Pharmakon Loan Agreement.

On November 1, 2024, we entered into the Pharmakon Loan Agreement, and in connection with this transaction, all obligations outstanding under the Hercules Loan Agreement were repaid in full on November 1, 2024, upon which the Hercules Loan Agreement was terminated.

As of June 30, 2025, we had a long-term principal debt balance of $119.0 million, consisting of $125.0 million aggregate principal amount of the Tranche A Loan under the Pharmakon Loan Agreement.

which the Hercules Loan Agreement was terminated.

We expect our interest expense to increase in future periods due to the draw down of the Tranche A Loan and potential future draw downs of the other Term Loans under the Pharmakon Loan Agreement.

See Note 6 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

On November 1, 2024, we entered into the Royalty Pharma Agreement, pursuant to which we received an upfront payment of $125.0 million, or the Purchase Price, and Royalty Pharma obtained the right to receive Royalty Payments on future U.S.

On November 1, 2024, we entered into the Royalty Pharma Agreement, pursuant to which we received an upfront payment of $125.0 million and Royalty Pharma obtained the right to receive Royalty Payments on future U.S.

net sales of RYTELO for each calendar quarter during the term of the agreement.

We are obligated to make Royalty Payments each quarter based on U.S.

net sales of RYTELO at the royalty rates set forth in the

agreement, which Royalty Payments are not determinable at this time, until the date when the aggregate Royalty Payments equal or exceed 1.65 times the Purchase Price, if this occurs by June 30, 2031, or the date when the aggregate Royalty Payments equal or exceed 2.0 times the Purchase Price.

net sales of RYTELO at the royalty rates set forth in the agreement, which Royalty Payments are not determinable at this time, until the date when the aggregate Royalty Payments equal or exceed 1.65 times the Purchase Price, if this occurs by June 30, 2031, or the date when the aggregate Royalty Payments equal or exceed 2.0 times the Purchase Price.

See Note 6 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information on the Royalty Pharma Agreement.

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QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

During the three months ended March 31, 2025, there were no material changes to our quantitative and qualitative disclosures about market risk as set forth in Part II, Item 7A "Quantitative and Qualitative Disclosures About Market Risk" of our 2024 Form 10-K,

During the six months ended June 30, 2025, there were no material changes to our quantitative and qualitative disclosures about market risk as set forth in Part II, Item 7A "Quantitative and Qualitative Disclosures About Market Risk" of our 2024 Form 10-K.

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