GERON CORP

Previous wording

This Report contains forward-looking statements that involve risks and uncertainties, as well as assumptions that, if they never materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. In some cases, forward-looking statements can be identified by the use of terminology such as “may,” “expects,” “plans,” “intends,” “will,” “should,” “projects,” “believes,” “predicts,” “anticipates,” “estimates,” “potential” or “continue,” or the negative thereof or other comparable terminology. These statements are within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. These statements appear throughout the Report and are statements regarding our intent, belief, or current expectations, primarily with respect to our business and related industry developments. 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.

Current wording

This Report contains forward-looking statements that involve risks and uncertainties, as well as assumptions that, if they never materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. In some cases, forward-looking statements can be identified by the use of terminology such as “may,” “expects,” “plans,” “intends,” “will,” “should,” “projects,” “believes,” “predicts,” “anticipates,” “estimates,” “potential” or “continue,” or the negative thereof or other comparable terminology. These statements are within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. These statements appear throughout the Report and are statements regarding our intent, belief, or current expectations, primarily with respect to our business and related industry developments. 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, 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.

Previous wording

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.

Current wording

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 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.

Previous wording

In addition to lower-risk MDS, we are developing imetelstat for the treatment of other myeloid hematologic malignancies. Our Phase 3 IMpactMF clinical trial is evaluating imetelstat in patients with intermediate-2 or 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 endpoint. As of May 2025, the trial has reached approximately 85% enrollment. 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.

Current wording

In addition to lower-risk MDS, we are developing imetelstat for the treatment of other myeloid hematologic malignancies. Our Phase 3 IMpactMF clinical trial is evaluating imetelstat in patients with intermediate-2 or 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 endpoint. 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.

Previous wording

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.

Current wording

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.

Previous wording

The significance of future losses, future revenues and any potential future profitability will depend primarily on the clinical and commercial success of RYTELO, our sole product. In this regard, our ability to generate meaningful revenue from product sales and achieve profitability is wholly dependent on our ability to successfully commercialize RYTELO in the U.S. 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. Our strategy to drive sales growth in the U.S. 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; 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 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 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. 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…

Excerpt shortened here; the source contains the full paragraph.

Current wording

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. 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; 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 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. 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.

Previous wording

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.

Current wording

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.

Previous wording

Our results of operations have fluctuated from period to period and may continue to fluctuate in the future. Results of operations for any period may be unrelated to results of operations for any other period. 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 inour commercialization efforts. We expect that our sales revenue may vary significantly from period to period as our commercialization efforts progress.

Current wording

Our results of operations have fluctuated from period to period and may continue to fluctuate in the future. Results of operations for any period may be unrelated to results of operations for any other period. 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 inour commercialization efforts. We expect that our sales revenue may vary from period to period as our commercialization efforts progress.

Previous wording

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. and the EU and to obtain regulatory approvals to commercialize RYTELO in other jurisdictions and in other indications. We are subject to risks common to companies in our industry and at our stage of development, including, but not limited to, risks inherent in the development, manufacture, regulatory approval for and commercialization of RYTELO; uncertainty of non-clinical and clinical trial results or regulatory approvals or clearances; the future development of imetelstat by us and its use by patients generally, including any future efficacy or safety results from clinical or commercial use that may cause the benefit-risk profile of imetelstat to become unacceptable; the uncertain and unpredictable drug research and development process; 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; our ability to obtain additional capital if and when needed; enforcement of our patent and proprietary rights; reliance upon our CROs, contract manufacturing organizations, or CMOs, consultants, licensees, investigators and other third parties; and potential competition.

Current wording

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. and the EU and to obtain regulatory approvals to commercialize RYTELO in other jurisdictions and in other indications. We are subject to risks common to companies in our industry and at our stage of development, including, but not limited to, risks inherent in the development, manufacture, regulatory approval for and commercialization of RYTELO; uncertainty of non-clinical and clinical trial results or regulatory approvals or clearances; the future development of imetelstat by us and its use by patients generally, including any future efficacy or safety results from clinical or commercial use that may cause the benefit-risk profile of imetelstat to become unacceptable; the uncertain and unpredictable drug research and development process; 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, 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; enforcement of our patent and proprietary rights; reliance upon our CROs, contract manufacturing organizations, or CMOs, consultants, licensees, investigators and other third parties; and potential competition.

Previous wording

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

Current wording

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

Previous wording

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. 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.

Current wording

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 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.

Previous wording

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.

Current wording

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. 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.

Previous wording

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.

Current wording

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.

Previous wording

In connection with the FDA approval of RYTELO on June 6, 2024, we subsequently begin 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.

Current wording

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 health technology assessment, or HTA, evaluation process.

Previous wording

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

Current wording

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

Previous wording

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

Current wording

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

Previous wording

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.

Current wording

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.

Previous wording

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. 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.

Current wording

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 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.

Previous wording

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

Current wording

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

Previous wording

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. 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.

Current wording

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. Adiscussion 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.

Previous wording

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

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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, primarily reflects bank charges related to our cash operating accounts and marketable securities portfolio and foreign currency transaction adjustments.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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. All of the securities were issued separately. The offering price of the common stock was $3.00 per share. The offering price of the 2024 pre-funded warrant was $2.99 per share. 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. 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.

Current wording

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. The offering price of the common stock was $3.00 per share. The offering price of the 2024 pre-funded warrant was $2.99 per share. 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. 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.

Previous wording

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. 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.

Current wording

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. 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. 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.

Previous wording

Despite receiving FDA approval of RYTELO in the U.S. in June 2024 and marketing authorization in the EU in March 2025, the outcome of any clinical activities and/or regulatory approval process is highly uncertain, and we cannot reasonably estimate whether our future development activities may succeed, whether we will obtain regulatory approval for RYTELO in any other jurisdictions or indications we are pursuing or may in the future pursue, or whether we will be able to effectively commercialize RYTELO in the U.S. or in the EU for lower-risk MDS or other potential indications, if at all. 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. Further, our future capital requirements are difficult to forecast and will depend on many factors, including:

Current wording

Despite receiving FDA approval of RYTELO in the U.S. in June 2024 and marketing authorization in the EU in March 2025, the outcome of any clinical activities and/or regulatory approval process is highly uncertain, and we cannot reasonably estimate whether our future development activities may succeed, whether we will obtain regulatory approval for RYTELO in any other jurisdictions or indications we are pursuing or may in the future pursue, or whether we will be able to effectively commercialize RYTELO in the U.S. or in the EU for lower-risk MDS or other potential indications, if at all. 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, 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 are difficult to forecast and will depend on many factors, including:

Previous wording

In the event we need to raise additional capital to fund our business, including pursuant to the 2023 Sales Agreement with B. Riley Securities, Inc., the Tranche B Loan and the Tranche C Loan under the Pharmakon Loan Agreement, which are subject to certain funding conditions, capital lease transactions or other financing sources, such additional capital may not be available on acceptable terms, or at all. 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. These effects have increased market volatility and could result in a significant long-term disruption of global financial markets, 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. Similarly, these macroeconomic conditions have created extreme volatility and disruption in the capital markets and is expected to have further global economic consequences. If the equity and credit markets deteriorate, including as a result of macroeconomic or other global conditions, such as inflation, changes in interest rates, prospects of a recession, government shutdowns, further changes in tariffs and other trade restrictions, 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, it may make any necessary debt or equity financing more difficult to obtain in a timely manner or on favorable terms, more costly or more dilutive. If we are unable to effectively commercialize RYTELO, or raise additional capital, if needed, or establish alternative collaborative arrangements with third-party collaborative partners for RYTELO, when needed, the development and commercialization of RYTELO may be further delayed, altered or abandoned, which might cause us to cease operations.

Current wording

In the event we need to raise additional capital to fund our business, including pursuant to the 2023 Sales Agreement with B. Riley Securities, Inc., the Tranche B Loan and the Tranche C Loan under the Pharmakon Loan Agreement, which are subject to certain funding conditions, capital lease transactions or other financing sources, such additional capital may not be available on acceptable terms, or at all. 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, 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 markets, 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. Similarly, these macroeconomic conditions have created extreme volatility and disruption in the capital markets and is expected to have further global economic consequences. If the equity and credit markets deteriorate, including as a result of macroeconomic or other global conditions, such as inflation, changes in interest rates, prospects of a recession, government shutdowns, further changes in tariffs and other trade restrictions, 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, it may make any necessary debt or equity financing more difficult to obtain in a timely manner or on favorable terms, more costly or more dilutive. If we are unable to effectively commercialize RYTELO, or raise additional capital, if needed, or establish alternative collaborative arrangements with third-party collaborative partners for RYTELO, when needed, the development and commercialization of RYTELO may be further delayed, altered or abandoned, which might cause us to cease operations.

Previous wording

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.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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. 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.

Current wording

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.

Previous wording

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 for approximately $110.9 million in the aggregate as of March 31, 2025. These purchase commitments can vary based on the commercial demand of RYTELO and are binding based on future manufacturing needs.

Current wording

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 for approximately $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.

Previous wording

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.

Current wording

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.

Previous wording

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

Current wording

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