What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-11-07 compared with 2025-08-06 · 77 added, 78 removed, 255 unchanged (38% of the section changed)

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We are preparing for the planned commercialization of RYTELO in select EU markets in 2026.

At this time, we do not plan to commercialize RYTELO independently in the EU (or in any other regions outside of the United States where RYTELO may be approved for marketing in the future).

At this time, we do not plan to commercialize RYTELO independently in the EU (or in any other regions outside of the U.S.

where RYTELO may be approved for marketing in the future).

Accordingly, we plan to work with experienced third parties for the commercialization and marketing of RYTELO in the EU, including on critical path activities for the planned launch of RYTELO in the EU, such as reimbursement, Health Technology Assessment, or HTA, submissions, market access and distribution.

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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 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.

As of the end of September 2025, the trial has reached full enrollment.

Based on our current assumptions for 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.

We believe that telomerase inhibition with imetelstat represents a novel mechanism of action with unique benefits in hematologic malignancies and potentially in other tumor types.

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

As of September 30, 2025, we had approximately $421.5 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 June 30, 2025, we had an accumulated deficit of approximately $1.8 billion.

As of September 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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The Royalty Payments to Royalty Pharma are capped, such that they will cease upon reaching a multiple of 1.65 times the Purchase Price if Royalty Pharma receives Royalty Payments in that amount in respect of net sales occurring on or before June 30, 2031, or upon reaching a multiple of 2.0 times the Purchase Price thereafter.

Based on our current operating plans and assumptions, we expect that our Royalty Payments to Royalty Pharma will reach a multiple of 1.65 times the Purchase Price in respect of net sales occurring on or before June 30, 2031.

Our Royalty Payment obligations under the Royalty Pharma Agreement may be discharged in connection with a change of control of Geron in an amount equal to 1.65 times the Purchase Price minus the aggregate Royalty Payments received by Royalty Pharma as of the date of the closing of the change of control, if the closing of the change of control occurs on or prior to December 31, 2027, or in an amount equal to 2.0 times the Purchase Price minus the aggregate Royalty Payments received by Royalty Pharma as of the date of the closing of the change of control, if the closing of the change of control occurs after December 31, 2027.

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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.

Net product revenue was approximately $47.2 million in the third quarter of 2025, $49.0 million in the second quarter of 2025, and $39.4 million in the first quarter of 2025.

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.

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is currently focused on three key areas:

investing additional resources to increase brand awareness, including increasing the size of our commercial and medical field forces;

investing additional resources to increase brand awareness;

refining our marketing and medical efforts to enhance prescribing clarity and confidence with the approved use of RYTELO;

and implementing programs to expand key opinion leader support and advocacy.

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 the second quarter of 2025, we increased the size of our sales force by 20% and doubled the size of our medical affairs team.

However, our strategy to drive sales growth and our ongoing commercialization efforts has not to date achieved and may not in the future 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 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.

We also expect to continue to seek regulatory approvals of RYTELO in jurisdictions outside of the United States, such as our recent marketing authorization in the EU, and to establish arrangements with third parties to assist us in the commercialization of RYTELO in such jurisdictions.

We also expect to continue to seek regulatory approvals of RYTELO in jurisdictions outside of the U.S., such as our recent marketing authorization in the EU, and to establish arrangements with third parties to assist us in the commercialization of RYTELO in such jurisdictions.

As a result, we expect research and development expenses and selling, general and administrative expenses to increase in future periods as we continue to support the commercialization of RYTELO in the U.S.

and further development of RYTELO, including the conduct and completion of our IMpactMF Phase 3 clinical trial, as well as our ongoing Phase 1 IMproveMF combination clinical trial in frontline MF and our Phase 2 investigator-led IMpress clinical trial in higher-risk MDS and acute myeloid leukemia, and as we prepare for commercialization of RYTELO in the EU in lower-risk MDS.

In addition, we expect our interest expense to increase 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, if available, as well as the non-cash interest expense related to the Royalty Pharma Agreement.

In addition, we expect our interest expense to increase due to the draw down of the Tranche A Loan and potential future dra w downs of the other Term Loans under the Pharmakon Loan Agreement, if available, as well as the non-cash interest expense related to the Royalty Pharma Agreement.

CRITICAL ACCOUNTING POLICIES AND ESTIMATES

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These changes historically have been minor and have been included in the condensed consolidated financial statements as soon as they became known.

Based on a critical assessment of our accounting policies and the underlying judgments and uncertainties affecting the application of those policies, management believes that our condensed consolidated financial statements are fairly stated in accordance with accounting principles generally accepted in the United States, and present a meaningful presentation of our financial condition and results of operations.

Based on a critical assessment of our accounting policies and the underlying judgments and uncertainties affecting the application of those policies, management believes that our condensed consolidated financial statements are fairly stated in accordance with accounting principles generally accepted in the U.S., and present a meaningful presentation of our financial condition and results of operations.

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 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.

There have been no significant changes in our critical accounting policies and estimates during the three and nine months ended September 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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Thus, historical results should not be viewed as indicative of future operating results.

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 from period to period as our commercialization efforts progress.

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 and our related strategy to drive sales growth in the U.S.

We expect that our sales revenue may continue to vary from period to period as a result of the evolving effects of our commercialization strategy and as our commercialization efforts otherwise progress.

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

and the EU for certain patients with lower-risk MDS.

Revenue based on sales of RYTELO is dependent on our ability to successfully commercialize RYTELO in the U.S.

Revenue based on sales of RYTELO is dependent on our ability to successfully commercialize RYTELO

and the EU and to obtain regulatory approvals to commercialize RYTELO in other jurisdictions and in other indications.

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and potential competition.

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,

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

Three Months Ended September 30, Nine Months Ended September 30,

Change $ Change % 2025 2024

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Selling, general and administrative expenses 39,001 35,877 3,124 9 % 117,588 102,361 15,227 15 %

Total operating expenses 61,490 70,215 (8,725) (12) % 117,797 126,653 (8,856) (7) %

Total costs and operating expenses

61,114 56,486 4,628 8 % 178,911 183,139 (4,228) (2) %

Loss from operations (13,887) (28,215) 14,328 (51) % (43,045) (153,681) 110,636 (72) %

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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.

Total product revenues, net for the three and nine months ended September 30, 2025 were approximately $47.2 million and $135.6 million, respectively.

Total gross-to-net adjustments for the three and nine months ended September 30, 2025 was 21.6% and 17.0% of gross product revenue, respectively.

The reconciliation of gross product sales to net product sales by each significant category of gross-to-net adjustments is set forth below for the three and nine months ended September 30, 2025.

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

Three Months Ended September 30, Nine Months Ended September 30,

2025 2024 2025 2024

(in thousands)

Three Months Ended

June 30, 2025 Three Months Ended

June 30, 2024 Six Months Ended

June 30, 2025 Six Months Ended

June 30, 2024

Gross product revenue $ 60,188 $ 32,657 $ 163,374 $ 33,547

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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.

We recognized royalty revenues of $60,000 and $256,000 in the three and nine months ended September 30, 2025, respectively, compared to $62,000 and $468,000 for the same periods in 2024.

Royalty revenues in the three and nine months ended September 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:

Costs and Operating Expenses:

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 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 .

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 health technology assessment, or HTA, evaluation process.

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,

We expect our operating expenses in the remaining quarter of 2025 to increase over 2024 levels, 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 health technology assessment, or HTA, evaluation process.

The following table summarizes our costs and operating expenses, including as a percentage of total expenses, for the three and nine months ended September 30 :

Three Months Ended September 30, Nine Months Ended September 30,

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

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Selling, general and administrative 39,001 35,877 9 % 117,588 102,361 15 %

Total operating cost and expenses $ 61,490 $ 70,215 (12) % $ 117,797 126,653 (7) %

*Percentage not meaningful

Total costs and operating expenses

$ 61,114 $ 56,486 8 % $ 178,911 183,139 (2) %

Cost of Goods Sold

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 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.

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

Cost of goods sold was approximately $1.0 million and $3.4 million for the three and nine months ended September 30, 2025,

respectively, which consisted of costs to manufacture and distribute our market product, RYTELO.

We began capitalizing inventory upon FDA approval of RYTELO.

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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 and six months ended June 30, 2025.

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.

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 9 to 12 months as we sell through certain inventory that was partially expensed prior to FDA approval.

Research and Development Expenses

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.

During the three and nine months ended September 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 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.

For the three and nine months ended September 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,

We expect our research and development expenses to increase in the remaining quarter of 2025 over 2024 levels, primarily due to ongoing investments to support our chemistry, manufacturing and controls, or CMC, strategy.

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

Three Months Ended September 30, Nine Months Ended September 30,

(In thousands) 2025 2024 2025 2024

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Clinical program:

Imetelstat $ 13,527 $ 20,032 $ 19,755 $ 40,082

$ 12,605 $ 13,502 $ 32,361 $ 53,584

Personnel-related expenses 7,943 6,207 23,584 25,206

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Total research and development expenses $ 21,070 $ 20,153 $ 57,884 $ 80,305

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.

The increase in research and development expenses for the three months ended September 30, 2025, compared to the same period in 2024, was primarily due to increased CMC and personnel-related expenses.

The decrease in research and development expenses for the nine months ended September 30, 2025, compared to the same period 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.

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Selling, General and Administrative Expenses

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.

Selling, general and administrative expenses were $39.0 million and $117.6 million for the three and nine months ended September 30, 2025, respectively, compared to $35.9 million and $102.4 million for the same periods in 2024.

The increase in selling, general and administrative expenses for the three and nine months ended September 30, 2025, compared to the same periods in 2024, is primarily due to an increase in sales and marketing full-time employees and additional investment in marketing programs.

We expect our selling, general and administrative expenses to increase over the remainder of the year, primarily due to continued investment in our RYTELO commercialization strategy.

Interest Income

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 income was $4.3 million and $14.1 million for the three and nine months ended September 30, 2025, respectively, compared to $4.9 million and $14.4 million for the same periods in 2024.

The decrease in interest income for the three and nine months ended September 30, 2025, compared to the same periods in 2024, primarily reflects a decrease in interest rates between the periods.

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

Interest Expense

Interest expense was $8.5 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.

Interest expense was $8.6 million and $25.4 million for the three and nine months ended September 30, 2025, respectively, compared to $3.0 million and $9.8 million for the same periods in 2024.

The increase in interest expense for the three and nine months ended September 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 $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.

Other income and (expense), net was an expense of $161,000 and $305,000 for the three and nine months ended September 30, 2025, respectively, compared to an expense of $63,000 and $188,000 for the three and nine months ended September 30, 2024.

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

LIQUIDITY AND CAPITAL RESOURCES

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.

As of September 30, 2025, we had cash, restricted cash, cash equivalents, and marketable securities of $421.5 million, compared to $502.9 million at December 31, 2024.

The decrease in cash, restricted cash, cash equivalents and marketable securities during the nine months ended September 30, 2025 was primarily the net result of cash used in operations partially offset by the receipt of net cash proceeds 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.

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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 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.

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 September 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 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.

From January 1, 2025 through September 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 September 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 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.

As of September 30, 2025, we had approximately $421.5 million in cash, cash equivalents, restricted cash and marketable securit ies.

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.

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.

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Cash Flows from Operating Activities

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.

Net cash used in operations for the nine months ended September 30, 2025 and 2024 was $89.0 million and $174.7 million, respectively.

The decrease in net cash used in operations for the nine months ended September 30, 2025, compared to the same period in 2024, primarily reflects a decrease in net loss, 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 $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.

Net cash provided by investing activities w as $86.1 m illion for the nine months ended September 30, 2025 and net cash used by investing activities was $2.5 million for the nine months ended September 30, 2024 .

The increase in net cash provided by investing activities for the nine months ended September 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 six months ended June 30, 2025 and 2024 was $1.3 million and $164.6 million, respectively.

Net cash provided by financing activities for the nine months ended September 30, 2025 and 2024 was $1.9 million and $168.2 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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Therefore, we make substantial and often long-term investments in our supply chain in order to ensure we have enough drug product to meet potential future commercialization requirements, as well as clinical trial needs.

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 approximatel y $124.7 million in the aggregate as of June 30, 2025.

We have engaged third‑party contract manufacturers and have 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 approximatel y $97.6 million in the aggregate as of September 30, 2025.

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

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.

As of September 30, 2025, we had a long-term principal debt balance of $119.3 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

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QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

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.

During the nine months ended September 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