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Item 2. Management’s Discussion and Analysis

2026-05-06 compared with 2025-11-07 · 126 added, 141 removed, 191 unchanged (58% of the section changed)

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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.

These statements appear throughout this 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.

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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.

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, 2025 as filed with the SEC on March 2, 2026, or the 2025 Form 10-K.

Company Overview

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Lower-risk MDS is a progressive blood cancer with high unmet need, where many patients with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and decreased quality of life.

We believe that the high unmet need in lower-risk MDS and significant product differentiation, including observed benefit, of RYTELO in difficult-to-treat sub-populations such as patients with high transfusion burden and ring sideroblast negative, or RS- patients, as well as the favorable FDA label and the National Comprehensive Cancer Network, or NCCN ® , Clinical Practice Guidelines in Oncology, or NCCN Guidelines ® , position RYTELO to potentially compete for significant market segments in lower-risk MDS.

We believe that the high unmet need in lower-risk MDS and significant product differentiation, including observed benefit, of RYTELO in difficult-to-treat sub-populations such as patients with high transfusion burden and ring sideroblast negative, or RS- patients, as well as the favorable FDA label and the National Comprehensive Cancer Network, or NCCN ® , Clinical Practice Guidelines in Oncology, or NCCN Guidelines®, position RYTELO to potentially compete for significant market segments in lower-risk MDS, including second-line ESA ineligible patients regardless of prior treatment or RS status and first-line ESA ineligible patients.

In March 2025, we were granted marketing authorization by the European Commission, or EC, for RYTELO as a monotherapy for the treatment of adult patients with TD anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic, or non-del 5q, abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy .

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 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.

At this time, we are working 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.

To enable paid access to patients outside the U.S.

through approved Named Patient Programs, or NPPs, in 2025, we partnered with Tanner Pharma, a distributor with broad global reach to support patient access.

To date, product revenue pursuant to NPPs have been minimal.

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 end point.

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.

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 end point.

As of September 2025, the trial was fully enrolled.

Based on our current planning 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.

Financial Overview

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 September 30, 2025, we had approximately $421.5 million in cash, cash equivalents, restricted cash and marketable securities.

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

As of March 31, 2026, we had approximately $341.0 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 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.

As of March 31, 2026, we had an accumulated deficit of approximately $1.9 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, 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:

(i) a $125.0 million Tranche A Loan, which was funded on November 1, 2024;

(ii) a $75.0 million Tranche B Loan, which is available subject to certain limited conditions, at our option;

and (iii) a $50.0 million Tranche C Loan, which is available to us upon reaching a specified trailing twelve-month RYTELO revenue milestone, and together with the Tranche A Loan and the Tranche B Loan, collectively, the Term Loans.

On January 5, 2026, the Pharmakon Loan Agreement was amended to extend the date for requesting the Tranche B Loan and Tranche C Loan, once available, from December 31, 2025 to July 30, 2026.

The amendment also extended the date before which any Term Loan prepayment is subject to a make‑whole premium, or the Makewhole Date, from November 1, 2026 to May 1, 2027.

See Note 7 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

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There are no other royalties payable on RYTELO, which was developed internally and is exclusively owned by Geron.

In December 2025, we implemented a workforce reduction, representing approximately one-third of our workforce prior to the reduction in headcount.

We recorded approximately $17.0 million in restructuring and restructuring-related charges in the fourth quarter of 2025, primarily consisting of one-time employee severance payments, healthcare and related benefits, and other employee-related costs.

The workforce reduction was substantially completed in the first quarter of 2026.

If we are unable to realize the expected operational efficiencies and cost savings from the restructuring, our operating results and financial condition could be adversely affected.

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.

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

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.

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

is currently focused on three key areas:

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.

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.

Our commercial strategy is designed to ensure that RYTELO reaches eligible patients when they are most likely to benefit.

Our commercial execution is focused on targeted engagement with high-volume accounts that treat earlier-line patients, investment in non-personal promotion and third-party education to further consistent, high-quality messaging across multiple touchpoints, and cross-functional execution of effective account management.

In particular, our strategy may not drive new patient starts across the breadth of the eligible patient population in RYTELO's approved indication, particularly across appropriate second-line patients 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 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 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.

As a result, we expect research and development expenses and selling, general and administrative expenses to continue to be substantial 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, our Phase 2 investigator-led IMpress clinical trial in higher-risk MDS and acute myeloid leukemia, and our Phase 1/2 investigator-led IMAGINE clinical trial in relapsed/refractory acute myeloid leukemia, and as we pursue paths to make RYTELO available to eligible LR-MDS patients outside of the U.S., including in the EU.

CRITICAL ACCOUNTING POLICIES AND ESTIMATES

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Our critical accounting policies are described in Item 7, “Critical Accounting Policies and Estimates” in our 2025 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.

There have been no significant changes in our critical accounting policies and estimates during the three months ended March 31, 2026, as compared to the critical accounting policies and estimates disclosed in our 2025 Form 10-K.

RESULTS OF OPERATIONS

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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

and in 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.

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and potential competition.

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

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

Three Months Ended March 31,

Change $ Change %

(in thousands, except for percentage data)

Product revenues, net $ 47,167 $ 28,209 $ 18,958 67 % $ 135,610 $ 28,989 $ 106,621 368 %

Product revenue, net $ 51,771 $ 39,436 $ 12,335 31 %

Royalties 66 167 (101) (60) %

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Selling, general and administrative expenses 35,425 40,023 (4,598) (11) %

Restructuring charges (394) — (394) (100) %

Total costs and operating expenses 51,679 56,307 (4,628) (8) %

Income (loss) from operations

158 (16,704) 16,862 (101) %

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

Interest income 3,421 5,152 (1,731) (34) %

Interest expense (7,147) (8,200) 1,053 (13) %

Other income and (expense), net (161) (63) (98) 156 % (305) (188) (117) 62 %

Net income (loss) $ (18,428) $ (26,447) $ 8,019 (30) % $ (54,638) $ (149,220) $ 94,582 (63) %

*Percentage not meaningful

Product Revenues, Net

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 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,

Other income (expense), net

(74) (83) 9 (11) %

Net income (loss) $ (3,642) $ (19,835) $ 16,193 (82) %

Product Revenue, Net

To date, our only source of product revenue has been from the sales of RYTELO, which we began shipping to our customers in the U.S.

in June 2024.

Total product revenue, net for the three months ended March 31, 2026 and 2025 were approximately $51.8 million and $39.4 million, respectively.

Product revenue, net, increased by $12.4 million during the three months ended March 31, 2026, compared to the same period in 2025, primarily due to increased sales volumes and, to a lesser extent, a higher selling price, partially offset by higher gross-to-net adjustments.

The increase in sales volume was primarily driven by a higher number of patients on therapy.

Total gross-to-net adjustments for the three months ended March 31, 2026 and 2025 were 20.8% and 13.0% of gross product revenue, respectively.

The increase in gross-to-net adjustments was driven by a greater proportion of sales made under governmental managed-care pricing programs and group purchasing organizations in addition to greater volume of product returns.

We expect total gross-to-net adjustments to be in the percentage range of low to mid-twenties of gross product revenue in the remaining quarters of 2026.

The reconciliation of gross product revenue to product revenue, net by each significant category of gross-to-net adjustments is set forth below:

Three Months Ended March 31,

(in thousands) 2026 2025

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Gross-to-net adjustments:

Chargebacks and customer credits

(8,164) (4,171)

Distributor service fees

(2,169) (1,526)

Government rebates (1,658) (66)

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Total gross-to-net adjustments (13,582) (5,876)

Net product revenue $ 47,167 $ 28,209 $ 135,610 $ 28,989

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 $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.

Product revenue, net

$ 51,771 $ 39,436

In connection with the divestiture of our human embryonic stem cell assets, including intellectual property and proprietary technology, to Lineage Cell Therapeutics, Inc.

(formerly BioTime, Inc., which acquired Asterias Biotherapeutics, Inc.), or Lineage, 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 less than $0.1 million in the three months ended March 31, 2026, compared to $0.2 million for the same period in 2025.

Royalty revenues in the three months ended March 31, 2026 and 2025 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.

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 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 %

The following table summarizes our costs and operating expenses:

Three Months Ended March 31,

(In thousands) 2026 2025 Change %

Cost of goods sold $ 1,692 $ 1,206 40 %

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Selling, general and administrative 35,425 40,023 (11) %

Restructuring charges (394) — (100) %

Total costs and operating expenses $ 51,679 $ 56,307 (8) %

$ 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 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.

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.

Cost of goods sold was approximately $1.7 million and $1.2 million for the three months ended March 31, 2026 and 2025, respectively, which consists 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.

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

As a result, a significant portion of the manufacturing costs related to the inventory manufactured prior to receiving FDA approval was partially expensed in a prior period and is therefore excluded from the cost of goods sold for the three months ended March 31, 2026 and 2025.

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

Research and Development Expenses

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.

During the three months ended March 31, 2026 and 2025, 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 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.

For the three months ended March 31, 2026 and 2025, 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 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,

Our research and development expenses were as follows:

Three Months Ended March 31,

(In thousands) 2026 2025

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Clinical program:

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

imetelstat $ 8,748 $ 6,228

Personnel-related expenses 5,282 8,225

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Total research and development expenses $ 14,956 $ 15,078

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.

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.

The decrease in research and development expenses for the three months ended March 31, 2026, compared to the same period in 2025, was primarily due a decrease in personnel-related costs due to lower labor costs driven by a decrease in headcount as a result of the workforce reduction in December 2025, partially offset by higher clinical trial costs associated with an increase of activity in our IMpactMF study.

Selling, General and Administrative Expenses

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.

Selling, general and administrative expenses were $35.4 million for the three months ended March 31, 2026, compared to $40.0 million for the same period in 2025.

The decrease in selling, general and administrative expenses for the three months ended March 31, 2026, compared to the same period in 2025, was primarily due to lower general and administrative personnel-related expenses from decreased headcount, partially offset by additional investment in marketing programs of approximately $1.8 million and approximately $1.0 million higher legal expenses.

Restructuring Charges

In December 2025, we implemented a workforce reduction, representing approximately one-third of our workforce prior to the reduction in headcount and recorded restructuring charges of $17.0 million.

Restructuring charges consisted of termination benefits such as one-time employee severance payments, healthcare and related benefits, and other employee-related costs.

For the three months ended March 31, 2026, a credit of $0.4 million in restructuring charges was recognized, primarily due to lower‑than‑expected utilization of previously accrued amounts related to healthcare and related benefits.

The credit was recorded in the condensed consolidated statement of operations as restructuring charges.

The workforce reduction was substantially completed in the first quarter of 2026.

There were no restructuring charges during the three months ended March 31, 2025.

Interest Income

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 income was $3.4 million for the three months ended March 31, 2026, compared to $5.2 million for the same period in 2025.

The decrease in interest income for the three months ended March 31, 2026, compared to the same period in 2025, primarily reflects a decrease in interest rates between the periods and a smaller marketable securities

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

Interest Expense

Interest expense was $8.6 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.

The liability related to Revenue Participation Right and the related non-cash interest expense are measured based on our current estimate of the timing and amount of expected future Royalty Payments expected to be paid over the estimated term of the Royalty Pharma Agreement using a discounted cash flow model.

The liability is amortized using the effective interest rate method, resulting in recognition of non-cash interest expense over the estimated term of the agreement.

Each reporting period, we assess the estimated timing and amount of future expected Royalty Payments over the estimated term.

If there are changes to the estimate, we recognize the impact to the liability’s amortization schedule and the related non-cash interest expense prospectively.

Additionally, the transaction costs associated with the liability will be amortized to non-cash interest expense over the estimated term of the Royalty Pharma Agreement.

Interest expense was $7.1 million for the three months ended March 31, 2026, compared to $8.2 million for the same period in 2025.

The decrease in interest expense for the three months ended March 31, 2026, compared to the same period in 2025, was primarily due to lower average interest rates related to the Royalty Pharma Agreement and the Pharmakon Loan Agreement, which were entered into in November 2024.

See Note 7 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information.

Other Income and (Expense), Net

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.

Other Income (Expense), Net

Other income (expense), net was an expense of $0.1 million for each of the three months ended March 31, 2026 and 2025.

Other income (expense), net primarily reflects realized losses on marketable securities and foreign currency transaction adjustments.

LIQUIDITY AND CAPITAL RESOURCES

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.

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 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 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.

As of March 31, 2026, we had cash, restricted cash, cash equivalents, and marketable securities of $341.0 million, compared to $401.1 million at December 31, 2025.

The decrease in cash, restricted cash, cash equivalents and marketable securities during the three months ended March 31, 2026 was primarily the net result of cash used in operations partially offset by the receipt of net cash proceeds from accounts receivable.

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

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The Pharmakon Loan Agreement provides two additional committed term loan tranches, the Tranche B Loan and the Tranche C Loan, in principal amounts of $75.0 million and $50.0 million, respectively, subject to customary conditions to fund and, in the case of the Tranche C Loan, achieving certain minimum net sales milestone.

The Tranche B Loan and the Tranche C Loan may be requested on or prior to December 31, 2025.

The Tranche B Loan and the Tranche C Loan could have been requested on or prior to December 31, 2025.

The Term Loans mature on November 1, 2029.

The Term Loans bear interest at a variable rate per annum equal to 5.75% plus three-month SOFR with a SOFR floor of 3.00%.

On January 5, 2026, the Pharmakon Loan Agreement was amended to extend the date for requesting the Tranche B Loan and Tranche C Loan, once available, from December 31, 2025 to July 30, 2026.

The amendment also extended the Makewhole Date from November 1, 2026 to May 1, 2027.

See Note 7 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

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On November 1, 2023, we entered into an At Market Issuance Sales Agreement, or the 2023 Sales Agreement, with B.

Riley Securities, pursuant to which we may elect to issue and sell shares of our common stock having an aggregate offering price of up to $100.0 million in such quantities and on such minimum price terms as we set from time to time through B.

Riley Securities, pursuant to which we could have elected to issue and sell shares of our common stock having an aggregate offering price of up to $100.0 million in such quantities and on such minimum price terms as we set from time to time through B.

Riley Securities as our sales agent.

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Riley Securities an aggregate commission equal to up to 3.0% of the gross proceeds of the sales under the agreement.

No sales of common stock occurred under the 2023 Sales Agreement, which was terminated in January 2026.

On February 27, 2026, we entered into a sales agreement, or the 2026 Sales Agreement, with TD Securities (USA) LLC, or TD Cowen, pursuant to which we may issue and sell shares of our common stock having an aggregate offering price of up to $150 million from time to time through TD Cowen as the sales agent.

We will pay TD Cowen an aggregate commission rate equal to up to 2.5% of the gross proceeds of the sales price per share for common stock sold through TD Cowen under the 2026 Sales Agreement.

We are not obligated to make any sales of common stock under the 2026 Sales Agreement.

To date, no sales of common stock have occurred under the 2026 Sales Agreement.

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Treasury securities, municipal securities, government and agency securities, commercial paper and corporate notes.

Our investment portfolio does not contain securities with exposure to sub-prime mortgages, collateralized debt obligations, asset-backed securities or auction rate securities and, to date, we have not recognized any impairment charges on our marketable securities or any significant changes in aggregate fair value that would impact our cash resources or liquidity.

Our investment portfolio does not contain securities with exposure to sub-prime mortgages, collateralized debt obligations, asset-backed securities or auction rate securities and, to date, we have not recognized any other-than-temporary impairment charges on our marketable securities or any significant changes in aggregate fair value that would impact our cash resources or liquidity.

To date, we have not experienced lack of access to our invested cash and cash equivalents;

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Successful drug development and commercialization requires significant amounts of capital.

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.

As of March 31, 2026, we had approxim ately $341.0 mi llion 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 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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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.

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 such as the ongoing Russia and Ukraine military conflict and the ongoing military conflict involving the U.S., Israel and Iran, pandemics or other health crises and supply chain and resource issues.

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

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• the overall level of sales and market acceptance of RYTELO;

• the scope, progress, timing, magnitude and costs of non-clinical and clinical development, manufacturing and commercialization of RYTELO, including commercialization in the EU for lower-risk MDS, or in any other jurisdictions or other indication we may pursue, subject to clearances and approvals by the FDA and similar international regulatory authorities;

• the scope, progress, timing, magnitude and costs of non-clinical and clinical development, manufacturing and commercialization of RYTELO, including commercialization in the U.S.

and any commercialization in the EU for lower-risk MDS, or in any other jurisdictions or other indication we may pursue, subject to clearances and approvals by the FDA and similar international regulatory authorities;

• delays or disruptions in opening sites, screening and enrolling patients or treating and following patients, in our current or any potential future clinical trials of RYTELO;

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• the extent to which we acquire or in-license other drugs and technologies, or invest in businesses, products or technologies, although we currently have no commitments or agreements relating to any of these types of transactions, or to which we out-license RYTELO;

• the extent to which we are able to enter into and conduct successful arrangements with third parties, including for the commercialization and marketing of RYTELO in the EU or in any other regions outside of the U.S., if approved for commercialization in such other regions;

• the extent to which we are able to enter into and conduct successful commercial arrangements with third parties, including for the commercialization and marketing of RYTELO in the EU and in any other regions outside of the U.S., if approved for commercialization in such other regions;

• expenses associated with the pending putative securities class action and shareholder derivative lawsuits and potential additional related lawsuits, as well as any other litigation;

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• the costs of enabling our personnel to work remotely, including providing supplies, equipment and technology necessary for them to perform their responsibilities.

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.

In the event we need to raise additional capital to fund our business, including pursuant to the 2026 Sales Agreement with TD Cowen, 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 mar kets, which could reduce or eliminate our ability to raise additional funds through financings, and could negatively impact the terms upon which we may raise those funds.

Similarly, these macroeconomic conditions have created extreme volatility and disruption in the capital markets and is expected to have further global economic consequences.

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 equity and credit markets and is expected to have further global economic consequences.

For example, the ongoing Russia-Ukraine military conflict and the ongoing military conflict involving the U.S., Israel and Iran have created extreme volatility and disruption in the equity and credit markets and have had and may continue to have further global economic consequences, including continued disruptions of the global supply chain and energy markets, which could continue to drive inflationary pressures and increase global recession risk.

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.

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

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

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

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

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

Cash Flows from Operating Activities

Net cash used in operations for the 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 Used in Operating Activities

Net cash used in operations for the three months ended March 31, 2026 and 2025 was $62.9 million and $48.0 million, respectively.

The increase in net cash used in operations for the three months ended March 31, 2026, compared to the same period in 2025, primarily due to payments related to workforce reduction and inventory purchases.

Cash Flows from Investing Activities

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.

Net cash provided by i nvesting activities for the three months ended March 31, 2026 and 2025 was $51.9 million and $52.4 million, respectively.

The decrease in net cash provided by investing activities for the three months ended March 31, 2026, compared to the same period in 2025, primarily reflects decreased proceeds the sale and maturities of marketable securities, partially offset by lower purchases of marketable securities.

Cash Flows from Financing Activities

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.

Material Cash Requirements

Our material cash requirements in the short- and long-term consist of the following operational and manufacturing expenditures, a portion of which contain contractual or other obligations.

We currently plan to fund our material cash requirements with our current financial resources together with net revenues from sales of RYTELO;

however, if we do not generate sufficient funds from commercial sales of RYTELO, 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 to fund our material cash requirements, which could include a combination of additional equity and debt financings, new collaborative arrangements, strategic alliances, or from other sources.

Net cash provided by financing activities for the three months ended March 31, 2026 and 2025 was $2.4 million and $0.3 million, respectively.

The increase in net cash provided by financing activities primarily reflects higher proceeds from issuances of common stock under our equity plans and proceeds from the exercise of warrants.

Contractual Obligations

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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 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 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

which the Hercules Loan Agreement was terminated.

We have engaged third‑party contract manufacturers to manufacture and supply additional 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 binding commercial purchase commitments of approxim ately $15.9 million that can be cancelled, but would incur cancellation penalties, and approximately $37.7 million in agreed upon manufacturing commitments that can be adjusted based on commercial demand of RYTELO.

We expect to utilize all r elated commercial purchase commitments.

As of March 31, 2026, we had a long-term principal debt balance of $119.8 million related to the Pharmakon Loan Agreement.

On January 5, 2026, the Pharmakon Loan Agreement was amended to extend the date for requesting the Tranche B Loan and Tranche C Loan, once available, from December 31, 2025 to July 30, 2026.

The amendment also extended the Makewhole Date from November 1, 2026 to May 1, 2027.

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

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QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

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.

During the three months ended March 31, 2026, 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 2025 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