GERN 10-K & 10-Q changes, risk factors and insider trading

Our near-term prospects are wholly dependent on RYTELO. We have limited experience with the commercialization of RYTELO, and if we are unable to successfully commercialize RYTELO in the U.S. for lower-risk MDS, or to expand its indication of use, our ability to generate meaningful revenue or achieve profitability will be materially and adversely affected.

In June 2024, we received FDA approval to commercialize RYTELO in the U.S. for certain patients with lower-risk MDS, and we initiated a commercial launch of RYTELO in the U.S. in that indication. While we have generated revenue from U.S. sales of RYTELO issince mid-2024, the commercialization of RYTELO marks our onlyfirst effort to commercially launch a product approved for marketing by the FDA,candidate, and our ability to generategrow revenue from product sales and achieve profitability is wholly dependent on oura abilitysustained tobasis successfully commercialize RYTELO in the U.S. for lower-risk MDS or to expand its indications of use. We mayhas not beyet ablebeen to successfully commercialize RYTELO for a number of reasons, including:proven.

•we may not be able to establish or demonstrate in the medical community the safety and efficacy of RYTELO and its potential advantages overover, and side effects compared toto, existing treatments;

•physicians may be reluctant to prescribe RYTELO until longer-term efficacy and safety data exists;

•our limited historical experience in marketing, selling and distributing RYTELO;

•potential future changes in the reimbursement and coverage policies of government and private payors such as Medicare, Medicaid, insurance companies, health maintenance organizations and other plan administrators;

•the relative price of RYTELO as compared to alternative treatment options;

•the relatively low incidence and prevalence of patients in RYTELO’s approved indication, including the reliability of our market and sales estimates;

•future competitive or other market factors may adversely affect the commercial potential of RYTELO;

•we may not be able to obtain and maintain regulatory approvals for RYTELO in any other jurisdictions for lower-risk MDS or for any other indications, including in the EU for lower-risk MDS or in any other jurisdiction for relapsed/refractory MF;

•changed or increased regulatory restrictions;

•changes to the label for RYTELO that further restrict how we market and sell RYTELO, including adverse events observed in ongoing and future studies of imetelstatimetelstat, such as our Phase 3 IMpactMF clinical trial;

•the capabilities of third party manufacturers may adversely affect the success of our commercialization of RYTELO;

•we may need additional financial or other resources that might not be available to us to successfully commercialize RYTELO; and we may not be able to maintain adequate commercial supplies of RYTELO to meet demand or at an acceptable cost or at all.

Moreover, successful commercialization of RYTELO may not generate sufficient revenue from product sales, and we may not become profitable in the near term, or at all. In any event, if we are unable to successfully commercialize RYTELO in the U.S. for lower-risk MDS, or to expand its indications of use, our ability to generate meaningful revenue from product sales and achieve profitability will be materially and adversely affected, which in turn would severely and adversely affect our financial results, business and business prospects, and might cause us to cease operations.

WeAlthough we have limited experience as aestablished commercial companyoperations and our sales, marketing,marketing and distribution ofinfrastructure for RYTELO in the U.S., our commercialization strategy may ultimately be unsuccessful or less successful than anticipated.anticipated

ToIn successfullyDecember commercialize2025, RYTELOwe announced a strategic restructuring plan designed to position us for long-term value creation and improve our financial discipline. However, there can be no assurance that our recent restructuring will result in long-term value creation or that our commercialization strategy will result in meaningful improvement in our sales performance. Additionally, we may determine that we need further changes in the U.S.,future that require additional hiring or other organizational changes to adequately support our strategy, which could further increase our costs. As such, we need to continue to execute on our sales, marketing and distribution plans. The ongoing execution of our sales, marketing and distribution plans requires investment of capital and time, and we cannot be certain that we will be able to continue to execute on our plans successfully. In addition, we compete with many companies that currently have extensive, experienced and well-funded sales, distribution and marketing operations to recruit, hire, train and retain marketing and sales personnel. If we are unable to recruit as needed, and torecruit, retain and effectively train marketingmarketing, sales and salesmedical personnel as needed and equip them with compliant and effective materials, our efforts to successfully commercialize RYTELO could be adversely affected.

WeFurther, although we received marketing authorization for RYTELO in the EU for the treatment of certain adult patients with TD anemia due to lower-risk MDS in March 2025, we currently have no marketing or sales organization outside of the U.S., and as a company, we have no experience selling and marketing approved drugs outside of the U.S. To successfully commercialize RYTELO outside ofin the U.S,EU or in any other regions outside the U.S. where we might seek marketing authorization in the future, we will need to develop these capabilities, eitherwhich onwe ourplan ownto do at this time by working with experienced third-party contractors or withcommercialization others, including third party contractors.partners. Doing so will require additional investment of capital and time. We maycurrently intend to seek strategic partnerships, collaborations, alliances or licensingcontractual arrangements, at an appropriate time, to assist us in the potential development and commercialization of RYTELO in the EU, or we may seek to self-commercialize and need to establish business operations in such regions. If we receive regulatory approval to commercialize RYTELO in any other regions, such as the EU, we may be unsuccessful in our efforts to recruit, hire, train and retain personnel to support such business operations; or we may be unable to enter into and conduct successful strategic partnerships, collaborations, alliances or licensing arrangements with third parties to commercializeassist us in the commercialization of RYTELO in suchthe regions,EU shouldand wein seekany toother doregions so.outside of the U.S. Any failure or delay in theentering execution of our sales, marketinginto and distributionconducting planssuch contractual arrangements, strategic partnerships, collaborations, alliances or licensing arrangements with third parties would adversely impact the commercialization of RYTELO in the EU or in any other regions outside the U.S. where RYTELO may be approved for marketing in the future.

If we do not obtainmaintain acceptable prices or adequate reimbursement for RYTELO, the use of RYTELO could be severely limited.

Our ability to successfully commercialize RYTELO will depend significantly on obtainingmaintaining acceptable prices and the availability of coverage and adequate reimbursement to patients from third-party payors. Government payors, such as the Medicare and Medicaid programs, and other third-party payors, such as private health insurers and health maintenance organizations, determine which medications they will cover and the reimbursement levels. Although CMS assigned a permanent and product specific J-Code (J0870) for RYTELO, which became effective on January 1, 2025, until CMS and commercial payor systems are updated, physicians may continue to use the non-specific miscellaneous J-Code to bill third-party payors for RYTELO. Because miscellaneous J-Codes may be used for a wide variety of products, health plans may have more difficulty determining the actual product used and billed for the patient. These claims increase the provider administrative burden and must often be submitted with additional information and manually processed, which can delay claims processing times as well as increase the likelihood for claim denials and claim errors. Further, theThe resulting reimbursement payment rates may not be adequate or may require significant restrictions on use or increased co-payments from commercially insured patients that patients may find unacceptably high. Patients are unlikely to use RYTELO unless coverage is provided, and reimbursement is adequate to cover all or a significant portion of its cost. Therefore, coverage and adequate reimbursement will be critical to market acceptance of RYTELO.

In addition, government authorities and other third-party payors in the U.S. and other jurisdictions are developing increasingly sophisticated methods of controlling healthcare costs, such as by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payors are requiring that drug companies provide them with predetermined discounts from list prices as a condition of coverage, are using restrictive formularies and preferred drug lists to leverage greater discounts in competitive classes, and are challenging the prices charged for medical products. The Inflation Reduction Act of 2022 includes several provisions to lower prescription drug costs for people with Medicare and reduce drug spending by the federal government, including the Medicare Drug Price Negotiation Program, which may ultimately have a negative effect on the pricing for RYTELO. However, the Medicare Drug Pricing Negotiation Program provisions of the law are currently subject to legal challenges. Further, nothe uniformfinal policyCY requirement2026 Medicare Physician Fee Schedule rule, issued by the Centers for coverageMedicare & Medicaid Services, among other things, increases bona fide service fee documentation requirements, defines “bundled arrangement,” to require “unbundling” of both contingent and non-contingent discounts and includes sales of Part B units at the Maximum Fair Price in average sales price calculations. These changes could lower reimbursement for drugMedicare productsPart existsB among third-party payors in the U.S. As a result, the coverage determination process is often a time-consumingutilization and costly process that will require usmanufacturers to providecomply scientificwith new, uncertain or complex reporting obligations and clinicaldrug supportpricing fordocumentation the use of RYTELO to each payor separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance.practices.

We cannot be sure that coverage and reimbursement will be available for RYTELO, and, if reimbursement is available, what the level of reimbursement will be. Although we have received a permanent and product-specific J-Code (J0870) for RYTELO which became effective on January 1, 2025, therecoverage may alsosignificantly bechange significant delays in obtaining coverage and reimbursement for newly approved drugs, and coverageor may be more limited than the purposesindications for which the drug is approved by the FDA or similar international regulatory authorities. Coverage and reimbursement may impact the demand for, or the price of RYTELO, and reimbursement policies in the U.S.U.S., the EU, and other jurisdictions may evolve which may adversely impact our ability to successfully commercialize RYTELO. Even if favorable coverage and reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future. If coverage and reimbursement are not available or the reimbursement amount is available only to limited levels,inadequate, we may not be able to successfully commercialize RYTELO, which would negatively impact our business and business prospects.

•the clinical indications for which RYTELO is or may in the future be approved;

•the establishment and demonstration to the medical community of the clinical efficacy and safety of RYTELO;

•the ability to demonstrate that RYTELO is superior to alternatives on the market at the time, including with respect to efficacy, safety, cost or route of administration;

•the willingness of medical professionals to prescribe, and patients to use, RYTELO, or to continue to use RYTELO;

•the publication of unfavorablefavorable safety or efficacy data concerning RYTELO by third parties or us;

•restrictions on use of RYTELO alone or in combination with other products;

•the label and promotional claims allowed by the FDA for RYTELO, as well as any such claims allowed by similar international regulatory authorities for RYTELO, if any, including usage for only certain indications and any limitations or warnings about the prevalence or severity of any side effects;

•the timing of market introduction of RYTELO asfor wellnew as competitive products, including sequencing of available productsindications;

•the effectiveness of sales, marketing and distribution supportinfrastructure for RYTELO;

•the ability of the third partythird-party distributors and specialty pharmacies we contract with to process prescriptions and dispense RYTELO and the processes required to place orders with such distributors and specialty pharmacies;

•the extent to which RYTELO is approved for inclusion on formularies in hospitals and managed care organizations;

•the pricing of RYTELO, both in absolute terms and relative to alternative treatments;

•the availability of coverage and adequate reimbursement by government and third-party payors; and the willingness of patients to pay out-of-pocket in the absence of coverage by third-party payors, including governmental authorities.

We may be unable to demonstrate any therapeutic or economic advantage for RYTELO compared to established or standard-of-care therapies, or newly developed therapies, for myeloid hematologic malignancies. National health insurance and/or third-party payors may decide that any potential benefit that RYTELO may provide to clinical outcomes in myeloid hematologic malignancies is not adequate to justify the potential adverse effects or the costs of treatment with RYTELO. If the healthcare community does not accept RYTELO for any of the foregoing reasons, or for any other reasons, our ability to commercialize RYTELO in the U.S. or the EU for lower-risk MDS or for any other indications for which RYTELO may be approved, may be negatively impacted or precluded altogether, which would seriously and adversely affect our business and business prospects.

We are commercializing RYTELO in lower-risk MDS, and the addressable patient population in lower-risk MDS is based on our estimates. These estimates, which have been derived from a variety of sources, including scientific literature, surveys of clinics, patient foundations and market research, may prove to be incorrect. Further, new information from us or others may change the estimated incidence or prevalence of patients with lower-risk MDS in the U.S. or the EU. Additionally, the potentially addressable patient population for RYTELO may not ultimately be amenable to treatment with RYTELO, or we may be unable to successfully identify patients and achieve a significant market share in all eligible patient segments in RYTELO’s approved indication, or initialthe salesduration of RYTELOtherapy may deplete the prevalence pool offor patients inreceiving theRYTELO RYTELO’scould approvedbe indication more quicklyshorter than expected,we expect, each of which would have a negative impact on sales of RYTELO in the future.future and may limit its growth potential. Our commercialization of RYTELO in the U.S. and our planned commercialization in the EU is limited to certain patients with lower-risk MDS, and any future potential commercialization will be limited to the therapeutic indications examined in our clinical trials and as determinedapproved by the FDA and similar international regulatory authorities, which would not permitpreclude us tofrom marketmarketing RYTELO for any other indications not expressly approved by those regulatory authorities. Future regulatory approvals for RYTELO, if any, could be conditioned upon label restrictions that materially limit the addressable patient population.

IfWe face competition from existing products, product candidates and technologies, and competitors may develop new products and technologies. If these products, product candidates or technologies thatare aredeemed by the healthcare community to be superior to or more cost-effective than RYTELO, it would significantly impact the development and commercial viability of RYTELO, which would severely and adversely affect our financial results, business and business prospects, and the future of RYTELO, and might cause us to cease operations.

We commercially launched RYTELO in the U.S. in June 2024 following its approval by the U.S. Food and Drug Administration, or FDA on June 6, 2024 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 ESAs. 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 uptake of RYTELO since launch is supported by 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.patients, Weas believewell thatas the favorable FDA label and the National Comprehensive Cancer Network, or NCCN®, Clinical Practice Guidelines in Oncology, or NCCN Guidelines®, position RYTELO asto a potential blockbuster treatment that canpotentially compete for significant market segments in lower-risk MDS, including first-line ESA ineligible patients and second-line patients regardless of prior treatment or RS status.MDS.

In SeptemberMarch 2023,2025, we submittedwere agranted marketing authorization application, or MAA, in the European Union, or EU, that was validated for review by the European Medicines Agency,Commission, or EMA,EC, for RYTELO for the same proposed indication as in the U.S., and in December 2024, the Committee for Medicinal Products for Human Use, or CHMP, of the EMA adopted a positive opinion recommending the approval of RYTELOmonotherapy 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. The European Commission, or EC, is reviewing the CHMP's recommendation, and we expect a potential approval decision by the EC in the first half of 2025. We are preparing for the potentialplanned commercialization of RYTELO in select EU countriesmarkets in 2026,2026. subjectAt this time, we do not plan to regulatorycommercialize approval,RYTELO whichindependently couldin includethe workingEU (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 whofor canthe providecommercialization contractedand services,marketing of RYTELO in the EU, including essentialon 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, or R/R MF, with overall survival, or OS, as the primary endpoint. As of FebruarySeptember 2025, the trial reachedwas approximatelyfully 80% enrollment.enrolled. 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.

Since our inception, we have financed our operations primarily through the sale of equity securities, draw downs on our debt facilities, cash generated from sales of RYTELO, interest income on our marketable securities andsecurities, payments we received under the Royalty Pharma Agreement and our prior collaborative and licensing arrangements. As of December 31, 2024,2025, we had approximately $502.9$401.1 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 isremains unproven. Our success in commercializing RYTELO will require, among other things, effective sales, marketing, manufacturing, distribution, information systems and pricing strategies, as well as compliance with applicable laws and regulations. InPrior addition,to althoughour wecommercialization recently began commercializingof RYTELO, substantially all of our revenues towere dategenerated have beenfrom payments under prior collaboration agreements, and milestones, royalties and other revenues from our licensing arrangements. We reported a small profit for the year ended December 31, 2015, and we have not reported any profit since. 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 December 31, 2024,2025, we had an accumulated deficit of approximately $1.8$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, 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: (i) a Tranche A Loan in an aggregate principal amount of $125.0 million, or the Tranche A Loan, which was funded on November 1, 2024, or the Tranche A Closing Date; (ii) a Tranche B Loan in an aggregate principal amount of $75.0 million, or the Tranche B Loan, which is available, subject to certain limited conditions, at our option; and (iii) a Tranche C Loan in an aggregate principal amount of $50.0 million, or the Tranche C Loan, and together with the Tranche A Loan and the Tranche B Loan, collectively, the Term Loans, which is available to us upon reaching a specified trailing twelve-month RYTELO revenue milestone. The Tranche B Loan and the Tranche C Loan, once available, maycould behave been requested on or prior to December 31, 2025. A portion of the proceeds from the Tranche A Loan were used to repay, in full, all amounts owed ($86.5 million) under the Hercules Loan Agreement, which was terminated effective November 1, 2024. The Term Loans mature on November 1, 2029. The Term Loans bear interest at a variable rate per annum equal to 5.75% plus the three-month Secured Overnight Financing Rate, or SOFR, with a SOFR floor of 3.00%. See Note 10 on Debt in Notes to Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

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 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,U.S., such as our MAArecent submissionmarketing forauthorization in the EU, and to establish arrangements with third parties to assist us in the commercialization of RYTELO in thesuch EU.jurisdictions. As a result, we expect research and development expenses and selling, general and administrative expenses to increasecontinue into futurebe periodssubstantial as we continue to support the commercialization of RYTELO in the U.S. and further development of RYTELO, including the conduct and completion of IMpactMF,our IMproveMFIMpactMF andPhase IMpress,3 clinical trial, as well as theour potentialongoing commercializationPhase 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 RYTELOthe U.S., including in the EU, if approved, in lower-risk MDS.EU. 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.

Product Sales, NetRevenue

Royalty Pharma Agreement Interest Expense

The liability related to the Royalty Payments under the Royalty Pharma Agreement and the related revenue 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 beis amortized to non-cash interest expense over the estimated term of the Royalty Pharma Agreement.

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 in our commercialization efforts and our related strategy to drive sales growth in the product launch.U.S.. We expect that our sales revenue may continue to vary significantly from period to period as a result of the launchevolving progresses.effects of our commercialization strategy and as our commercialization efforts otherwise progress.

RYTELO is our only product approved for marketing and is approved solely in the U.S. 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 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 research and development efforts, including 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 discoverydevelopment 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, risingfluctuations 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; our needability forto substantialobtain 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.

Product Revenues,Revenue, netNet

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,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 June 2024. Total product revenue, net for the twelve months ended December 31, 2025 and 2024 was approximately $183.6 million and $76.5 million.million, We expect product revenues, net to increase in 2025.respectively.

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. Total gross-to-net adjustments for the twelve months ended December 31, 2025 and 2024 waswere 14.4%17.7% and 14.5% of gross product revenue.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 twelve months ended December 31, 2024.respectively. We expect total gross-to-net adjustments to be in the percentage range of mid-high-teen to high-teen percentageslow-twenties of gross product revenue in 2025.2026.

We recognized royalty revenues of $499,000,$0.3 $237,000million, $0.5 million and $596,000$0.2 million during the years ended December 31, 2024,2025, 20232024 and 2022,2023, respectively. Royalty revenues 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. We expect royalty revenues in 2025 to be lower than 2024 as a result of reduced royalties from sales of cell-based research products from our divested stem cell assets.

Costs and Operating Expenses

The following table summarizes our costs and operating expenses, including as a percentage of expenses,expenses for the years ended December 31:

Our costCost of goods sold consistwas approximately $4.7 million for the year ended December 31, 2025, compared to $1.3 million for the year ended December 31, 2024, 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 in the U.S.RYTELO.

During the year ended December 31, 2024,2025, 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 years ended December 31, 2024,2025, 20232024 and 2022,2023, 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,consulting expenses, costs to maintain technology licenses and research-related overhead.

The decrease in research and development expenses in 20242024, as compared to 2023, was primarily due to manufacturing and quality costs that were capitalized in the current period, beginning with the third quarter of 2024, due to FDA approval of RYTELO in June 2024, versus being expensed as research and development expenses in 2023. The decrease is partially offset by an increase in labor costs due to higher headcount and incentive and stock-based compensation expense recognized due to the vesting of performance-based stock options upon FDA approval. We expect research and development expenses to increase in the future as we support IMpactMF, IMproveMF and IMpress, and continue the long-term treatment and follow-up of remaining patients in IMerge Phase 3.

Selling, general and administrative expenses were $145.7$159.3 million, $69.1$145.7 million, and $43.6$69.1 million for the years ended December 31, 2024,2025, 2024 and 2023 and 2022,, respectively.

Interest income was $18.1 million, $19.6 million, and $18.2 million for the years ended December 31, 2025, 2024 and 2023, respectively. The increasedecrease in interest income in 20242025 compared to 20232024 primarily reflects a largersmaller marketable securities portfolio due to the receipt of net cash proceeds from the underwritten offering completed in March 2024, as well as higher yields from marketable securities purchases.portfolio. Interest earned in future periods will depend on the size of our marketable securities portfolio and prevailing interest rates.

The increase in interest income in 20232024 compared to 20222023 primarily reflects a larger marketable securities portfolio,portfolio withdue to the receipt of net cash proceeds from the underwritten public offering completed in JanuaryMarch 2023 and cash proceeds from warrant exercises in 2023,2024, as well as higher yields from marketable securities purchases. Interest earned in future periods will depend on the size of our marketable securities portfolio and prevailing interest rates.

As a company, we have limited prior experience in selling and marketing or commercializing an approved drug product in the U.S., and we have no experience selling, marketing or commercializing an approved drug outside of the U.S. The success of our commercialization efforts is subject to, among other things, managing our internal sales, marketing, and distribution capabilities and our ability to navigate the significant expenses and risks involved with the management of such capabilities. Our initial commercialization efforts in the U.S. have not to date achieved meaningful sales growth.growth Ourand our RYTELO sales trends and resulting revenue have been and may continue to be variable. Net product revenue was approximately $57.5 million in the second quarter of 2026, $51.8 million in the first quarter of 2026, $48.0 million in the fourth quarter of 2025, $47.2 million in the third quarter of 2025, and $49.0 million in the second quarter of 2025. In an effort to better execute on our plans to commercialize RYTELO and generate more meaningful product revenue, we decided to focus our commercial execution on targeted engagement with high-volume accounts that treat earlier-line patients, and invest in non-personal promotion and third-party education to reach the full breadth of the eligible patient population, while cross-functionally collaborating for effective account management. However, our revised strategy to drive RYTELO commercial growth and our ongoing commercialization efforts may still not achieve meaningful sales growth, which may require us to, among other things, 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 meet or grow projected RYTELO net product revenue in future periods. In particular, our strategy may not drive new patient starts in lower-risk MDS U.S. patients, particularly in second-line lower-risk MDS, 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. If we are unsuccessful in accomplishing our objectives from our commercialization efforts and strategy, we may not be able to generate meaningful revenue from the commercialization of RYTELO in lower-risk MDS, or may require significant additional capital and financial resources to do make further investment in our commercial operations. Any of these outcomes would severely and adversely affect our financial results, business and business prospects, and might cause us to cease operations.

In addition, government authorities and other third-party payors in the U.S. and other jurisdictions are developing increasingly sophisticated methods of controlling healthcare costs, such as by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payors are requiring that drug companies provide them with predetermined discounts from list prices as a condition of coverage, are using restrictive formularies and preferred drug lists to leverage greater discounts in competitive classes, and are challenging the prices charged for medical products. The Inflation Reduction Act of 2022 includes several provisions to lower prescription drug costs for people with Medicare and reduce drug spending by the federal government, including the Medicare Drug Price Negotiation Program, which may ultimately have a negative effect on the pricing for RYTELO. However, the Medicare Drug Pricing Negotiation Program provisions of the law are currently subjectcontain toan legalorphan challenges.drug exception. Further, the final CY 2026 Medicare Physician Fee Schedule rule, issued by the Centers for Medicare & Medicaid Services, among other things, increases bona fide service fee documentation requirements, defines “bundled arrangement,” to require “unbundling” of both contingent and non-contingent discounts and includes sales of Part B units at the Maximum Fair Price in average sales price calculations. These changes could lower reimbursement for Medicare Part B utilization and require manufacturers to comply with new, uncertain or complex reporting obligations and drug pricing documentation practices.

The requirements governing drug pricing and reimbursement vary widely from country to country. For example, within the EU member states may restrict the range of medicinal products for which their national health insurance systems provide reimbursement and may control the prices of medicinal products for human use. Reference pricing used by various EU Member States and parallel distribution, or arbitrage between low-priced and high-priced EU Member States, can further reduce prices. An EU Member State may approve a specific price for the medicinal product or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market. In some countries in the EU, we may be required to conduct a clinical study or other studies that compare the cost-effectiveness of RYTELO to other available therapies in order to obtain or maintain reimbursement or pricing approval. This Health Technology Assessment (HTA) process is the procedure according to which the assessment of the public health impact, therapeutic impact and the economic and societal impact of use of a given medicinal product in the national healthcare systems of the individual country is conducted. The outcome of HTA regarding specific medicinal products will often influence the pricing and reimbursement status granted to these medicinal products by the competent authorities of individual EU Member States. At the EU level, on January 12, 2025, Regulation No 2021/2282 on Health Technology Assessment (HTA Regulation), entered into application through a phased implementation. The Regulation initially applies to new active substances for oncology and ATMPs. It will be expanded to orphan medicinal products in January 2028, and to all centrally authorized medicinal products as of 2030. Select high-risk medical devices also came into scope in 2026. The HTA Regulation is intended to boost cooperation among Member States in assessing health technologies, including new medicinal products. The Regulation establishes a framework for EU‑level joint clinical assessments, joint scientific consultations, and the early identification of emerging health technologies. It permits EU Member States to use common tools, methodologies, and procedures and requires them to rely on EU‑level joint clinical assessment reports for the clinical components of their national HTA evaluations. Individual EU Member States will continue to be responsible for assessing non-clinical (e.g., economic, social, ethical) aspects of health technologies, and making decisions on pricing and reimbursement. There can be no assurance that any country that has price controls or reimbursement limitations for biopharmaceutical products will allow favorable reimbursement and pricing arrangements for RYTELO. Historically, products launched in the EU do not follow price structures of the U.S. and generally prices tend to be significantly lower. Publication of discounts by third-party payors or authorities may lead to further pressure on the prices or reimbursement levels within the country of publication and other countries. If pricing is set at unsatisfactory levels or if reimbursement of RYTELO is unavailable or limited in scope or amount, our revenues from sales and the potential profitability of RYTELO in those countries would be negatively affected.

In AprilDecember 2023,2025, the ECEuropean publishedCommission, Parliament and Council reached a proposalpolitical agreement on the Pharma Package, which includes proposed changes to reformthe EU orphan medicinal product framework that may modify the current pharmaceutical framework, including revisionlength of orphan market exclusivity.exclusivity and change the way in which market exclusivity is awarded to drugs with more than one approved orphan indication. The newPharma legislation,Package is subject to formal adoption by the European Parliament and Council and, if adopted, iscould expected to start to apply from mid-2028. The proposal to reformalter the currentexclusivity pharmaceuticalprotections frameworkcurrently intendsavailable tofor reviseour orphan designated products in the orphanEU, drugincluding designationby and exclusivity regime. In the latest version of the proposal,reducing orphan market exclusivity will be reduced from the current 10 years to 9 years.years, Extensionwith bya another2-year two years will be possibleextension for so-called “breakthrough orphan medicinal products”., Previousand drafts also includedintroducing the concept of “global orphan marketing authorization”, which would no longer grant additionalprevent separate orphan market exclusivity for second or further orphan therapeutic indications. Although the final text has not yet been published and RYTELO for the treatment of MDS is and remains regulated under the current regulatory framework, our activities, including authorization of future indications may be affected by the new legislation.legislation if adopted.

•the EMA or the competent authorities of the individual EU Member States or similar international regulatory authorities may lack resources or be delayed in conducting pre-approval inspections due to lack of resources or other reasons;

We are incurring and have incurred net losses every year since our operations began in 1990, except for one. As of MarchJune 31,30, 2026, our accumulated deficit was approximately $1.9 billion. Losses have resulted principally from costs incurred in connection with our research and development activities and from general and administrative costs associated with our operations. Although we began commercializing RYTELO in June 2024, the commercial potential of and our ability to successfully commercialize RYTELO remains unproven, and our limited operating history as a commercial company makes our future operating results difficult to predict. We expect that our sales revenue may continue to vary from period to period as a result of the evolving effects of our commercialization strategy and as our commercialization efforts otherwise progress. If we do not generate sufficient revenue from commercial sales of RYTELO, or if we experience unforeseen events or choose to make other investments in our business, we may continue to experience negative cash flow as we fund our operations and imetelstat clinical development activities and research programs, and continue with the commercialization of RYTELO, including as a result of our obligation to pay royalty payments under the Royalty Pharma Agreement and service our debt obligations. We will need to generate significant revenues to achieve consistent future profitability, and we may never achieve consistent future profitability. Even if we do become profitable in the future, we may not be able to sustain or increase profitability on a quarterly or annual basis. Our failure to achieve consistent future profitability could negatively impact the market price of our common stock and our ability to sustain operations.

Successful drug development and commercialization requires significant amounts of capital. As of MarchJune 31,30, 2026, we had approximately $341.0$326.9 million in cash, cash equivalents, restricted cash and marketable securities. While we believe that, based on our current operating plans and assumptions, 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, 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, if available, under the Pharmakon Loan Agreement (as defined below),financings, 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.

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 in the U.S., the EU or other potential jurisdictions or 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,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 are difficult to forecast and will depend on many factors, including:

•the sales price for RYTELO, including the availability of coverage and adequate third-party reimbursement for RYTELO;

In the event we need to raise additional capital to fund our business, including pursuant to the 2026 Sales Agreement with TD Cowen (as defined below), 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 inflation, fluctuations 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, 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 equity and credit markets and are 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.Ifrisk. If the equity and credit markets deteriorate, 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.

On November 1, 2024, we entered into the Pharmakon Loan Agreement, which we amended on January 5, 2026. We drew the Tranche A Loan of $125.0 million on November 1, 2024 and as of November 1, 2024, the total outstanding principal amount under the Pharmakon Loan Agreement was $125.0 million. The tranches for the remaining $125.0 million available to us under the Pharmakon Loan Agreement arehad asprovided followsfor two additional committed term loan tranches: (a) a Tranche B Loan of $75.0 million, which iswas available for us to request at our option until July 30, 2026, subject to certain customary and limited conditions; and (b) a Tranche C Loan of $50.0 million, which iswas available for us to request until July 30, 2026, subject to certain conditions, including us reaching a specified trailing twelve-month RYTELO revenue milestone on or prior to June 30, 2026. IfAs we do not achieve such revenue milestone withinof the requireddate timeline,of wethis willReport, notneither bethe eligibleTranche toB drawLoan downnor the Tranche C Loan. In addition, before we would consider drawing down any of the remaining tranches under the Pharmakon Loan Agreement,was if available, we must first satisfy ourselves that we will have access to future alternate sources of capital, such as from commercial revenues or the equity capital markets or debt capital markets, in order to repay any additional principal borrowed, which we may be unable to do, in which case, our liquiditydrawn, and abilityboth toare fundno ourlonger operations may be substantially impaired.available.

This indebtedness may create additional financing risk for us, particularly if our business or prevailing financial market conditions are not conducive to paying off or refinancing the outstanding debt obligations at maturity. If we draw down any of the remaining tranches under the Pharmakon Loan Agreement, our indebtedness will increase, which would further increase our risk of being unable to pay off or refinance our outstanding debt obligations at maturity.

In the U.S., the Hatch-Waxman Act permits one patent per approved product to receive one patent term extension of up to five years beyond its normal expiration. The USPTO, in consultation with the FDA, reviews and approves the application for any patent term extension or restoration. The length of the patent term extension is typically calculated as one half of the period between the effective date of an IND and the submission date of an NDA less any time the sponsor did not act with due diligence during the period, plus the time between the submission date of anthe NDA and the approval of that application less any time the sponsor did not act with due diligence during the period. There is also a limit on the patent term extension to a term that is no greater than fourteen years from drug approval. We have applied to the USPTO for patent term extension of some of our patents. Currently, communication of patent term extension approval and the length of the granted extension period by the USPTO may occur up to several years from filing of an application for patent term extension. If the USPTO and the FDA determine the extension period for each proposed eligible patent, we will select the one patent to be extended. We expect to apply any patent term extension that is granted in the U.S. to our method of treatment patent for MDS and MF that expires on March 15, 2033. If such patent term extension is granted, we expect the term of the patent to extend through August 2037, although such timing is subject to approval by the USPTO and could differ from our calculation.

If a third party files an application under Section 505(b)(2) of the FDCA or an abbreviated new drug application, or ANDA, under Section 505(j) to obtain permission to sell a generic or follow-on version of RYTELO, and relies in whole or in part on studies conducted by or for us, the third-party will be required to certify to the FDA that either: (1) there is no patent information listed in the Orange Book with respect to our NDA for RYTELO; (2) the patents listed in the Orange Book have expired; (3) the listed patents have not expired, but will expire on a particular date and approval is sought after patent expiration; or (4) the listed patents are invalid or will not be infringed by the manufacture, use or sale of the third-party’s generic product. A certification that the new product will not infringe the Orange Book-listed patents for RYTELO, or that such patents are invalid, is called a paragraph IV certification. If the third-party submits a paragraph IV certification to the FDA, a notice of the paragraph IV certification must also be sent to us within 20 days after the third-party’s ANDA is accepted for filing by the FDA. We may then initiate a lawsuit to defend the patents identified in the notice. The filing of a patent infringement lawsuit within 45 days of receipt of the notice automatically prevents the FDA from approving the third-party’s ANDA until the earliest of 30 months after the NDA holder’s receipt of the notice of Paragraph IV Certification, the expiration of the patent, certain settlements of the lawsuit, or the court reaches a decision in the infringement lawsuit in favor of the third-party. If the product has NCE exclusivity and the notice is given and the suit filed in the fifth year of exclusivity, the regulatory stay extends until 7.5 years after approval of the reference product. If we do not file a patent infringement lawsuit within the required 45-day period, the third-party’s ANDA will not be subject to the 30-month stay of FDA approval. However, if an appropriate patent covering RYTELO is not listed in the Orange Book or is subsequently removed from the Orange Book, a manufacturer of generic drugs would not be required to provide advance notice to us of any abbreviated NDAANDA filed with the FDA to obtain permission to sell a generic version of RYTELO. Any of the foregoing could harm our competitive position, business, financial condition, results of operations and prospects.

In an infringement proceeding, a court may decide that a patent of ours is not valid or is unenforceable, or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. The standards that courts use to interpret patents are not always applied predictably or uniformly and can change, particularly as new technologies develop. As a result, we cannot predict with certainty how much protection, if any, will be given to our patents if we attempt to enforce them and they are challenged in court and if any such lawsuits will ultimately be resolved successfully. Further, even if we prevail, the infringer may file an appeal and the court judgment may be overturned and/or that an adverse decision may be issued by an appeals court relating to the validity or enforceability of our patents. An adverse result in any litigation or defense proceedings could put one or more of our patents at risk of being invalidated or interpreted narrowly in a manner insufficient to achieve our business objectives. Even if we establish infringement, we may not seek, or the court may decide not to grant, an injunction against further infringing activity and instead award only monetary damages, which may or may not be an adequate remedy.

Uncertainty in the regulatory framework and future legislation could lead to disruption in the execution of international multi-center clinical trials, the monitoring of adverse events through pharmacovigilance programs, the evaluation of the benefit-risk profiles of new medicinal products, and determination of marketing authorization across different jurisdictions. Changes to existing regulations may add considerably to the time from clinical development to marketing authorization and commercialization of products in foreign jurisdictions and increase our costs. Ongoing legislative initiatives in the EU include the proposed SPC Regulation revision, the proposed Critical Medicines Act (for which a provisional political agreement was reached in May 2026), and the proposed Biotech Act. We cannot predict the impact of such changes and future regulation on our business or the results of our operations. These and other risks associated with international operations may materially adversely affect our ability to attain or maintain profitable operations.

The ongoing trade tensions between the U.S. and other jurisdictions have resulted in multiple rounds of tariffs and potential tariffs affecting pharmaceuticals and pharmaceutical ingredients, including finished drug products, manufacturing equipment, and related supplies. In April 2025, the U.S. government imposed a 10% baseline global tariff and in August 2025, the U.S. imposed higher “reciprocal” tariffs on numerous other territories, including EU Member States and South Korea. While the U.S. Supreme Court recently issued a ruling invalidating tariffs imposed by the Trump administration under the International Emergency Economic Powers Act, other tariffs imposed by the U.S. government remain in place, including the 10% global tariff imposed by the Trump administration under Section 122 of the Trade Act of 1974 following the U.S. Supreme Court decision. Moreover, the Bureau of Industry and Security, U.S. Department of Commerce, has initiated an investigation to determine whether pharmaceutical ingredients, including finished drug product, manufactured outside the U.S. pose a national security risk and should be subject to additional tariffs. Unlike consumer goods, pharmaceuticals face unique regulatory constraints that make rapid supply chain adjustments particularly difficult and costly. Although a significant portion of our supply chain for imetelstat is currently based in the U.S., the active pharmaceutical ingredient, or API, for imetelstat is manufactured in South Korea and our 47mg vial of RYTELO drug product is currently manufactured in Italy. In addition, in the future we may choose to utilize other contract manufacturers for different presentations of imetelstat and source materials for our supply chain from other international jurisdictions. Accordingly, such global or industry-specific tariffs, or the renegotiation of current international trade agreements, or other trade restrictions could result in additional costs on our business, including generally increasing our manufacturing costs, and may decrease our gross margins and increase our supply chain complexity. Moreover, other governments have imposed and may continue to impose retaliatory tariffs, trade restrictions or trade barriers impacting RYTELO, which could impose additional costs and complexity on our business, including with respect to our planned commercialization of RYTELO in select EU markets in 2026 or restrict our ability to sell RYTELO in the EU or in other international markets where we may obtain approval of RYTELO.

Securities class action lawsuits and/or derivative lawsuits have often been brought against companies, including biotechnology and biopharmaceutical companies, that experience volatility in the market price of their securities. This risk is especially relevant for us because we often experience significant stock price volatility in connection with our activities. On March 13 and March 14, 2025, we and certain of our current and former officers were named as defendants in two putative securities class action lawsuits filed in the United States District Court for the Northern District of California captioned Dabestani v. Geron Corporation, et al., No. 3:25-cv-02507 and Potvin v. Geron Corporation, et al., No. 3:25-cv-02563, respectively. Both lawsuits allege violation of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934, as amended, and Rule 10b-5 promulgated thereunder in connection with allegedly false and misleading statements concerning the commercial potential of RYTELO. The plaintiffs allege, among other things, that we overstated RYTELO's commercial potential by making materially false and misleading statements and/or concealing material adverse facts concerning RYTELO's commercial potential, including the lack of awareness among healthcare providers for RYTELO, the burden of monitoring requirements in administering the drug, and the impacts of seasonality and existing competition on RYTELO's sales, and that our stock price dropped when we disclosed in our earnings call on February 26, 2025 that we had observed flat revenue trends over the prior few months. The plaintiffs seek damages and interest, and an award of reasonable costs, including attorneys' and experts' fees. On May 29, 2025, the Court consolidated the Dabestani and Potvin cases into one consolidated action captioned In re Geron Corporation Securities Litigation, or the Securities Class Action, and appointed lead plaintiffs and counsel for lead plaintiffs. On August 8, 2025, lead plaintiffs filed a consolidated amended complaint. On October 7, 2025, we filed our motion to dismiss the consolidated amended complaint. A hearing on the motion to dismiss was held on March 19, 2026, and on March 30, 2026, the Court entered an order granting our motion to dismiss without prejudice. On April 13, 2026, lead plaintiffs filed a second amended complaint. WeOn intendMay to18, move2026, we filed our motion to dismiss the second amended complaint. On June 22, 2026, lead plaintiffs filed their opposition to our motion to dismiss the second amended complaint, and on July 10, 2026, we filed our reply in support of our motion to dismiss the second amended complaint.

Historically, our stock price has been extremely volatile. Between JanuaryJuly 1, 2016 and MarchJune 31,30, 2026, our stock has traded as high as $6.38 per share and as low as $0.89 per share. Between AprilJuly 1, 2025 and MarchJune 31,30, 2026, the price has ranged between a high of $1.95 per share and a low of $1.04$1.07 per share. The significant market price fluctuations of our common stock have been due to and may in the future be influenced by a variety of factors, including:

Our common stock is currently listed on the Nasdaq Global Select Market, or Nasdaq, under the symbol “GERN.” The listing standards of Nasdaq provide that a company, in order to qualify for continued listing, must maintain a minimum stock price of $1.00 and satisfy standards relative to minimum stockholders’ equity, minimum market value of publicly held shares and various additional requirements. Historically, our stock price has been extremely volatile and recently, our stock has traded as low as $1.04$1.07 per share through MayJuly 1,31, 2026. While our common stock is currently listed on Nasdaq, we can give no assurance that we will be able to maintain compliance with the continued listing requirements for Nasdaq. If we fail to maintain compliance with any such continued listing requirement, there can also be no assurance that we will be able to regain compliance with any such continued listing requirement in the future or that our common stock will not be delisted in the future. If Nasdaq delists our securities from trading on its exchange for failure to meet the listing standards, we and our stockholders could face significant negative consequences including:

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 MarchJune 31,30, 2026, we had approximately $341.0$326.9 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. Our success in commercializing RYTELO will require, among other things, effective sales, marketing, manufacturing, distribution, information systems and pricing strategies, as well as compliance with applicable laws and regulations. Prior to our commercialization of RYTELO, substantially all of our revenues were generated from payments under prior collaboration agreements, and milestones, royalties and other revenues from our licensing arrangements. We reported a small profit for the year ended December 31, 2015, and we have not reported any profit since. 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 MarchJune 31,30, 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,agent. thatWe 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 withdrew the Tranche A Loan andof $125.0 million on November 1, 2024, a portion of which was utilized to repay all outstanding indebtedness associated with the TrancheHercules BLoan Loan,Agreement. collectively, the Term Loans. On January 5, 2026, theThe Pharmakon Loan Agreement was amended to extend the dateprovided for requestingtwo theadditional Tranchecommitted Bterm Loanloan andtranches, Tranchewhich Cwere Loan,no oncelonger available,available fromas Decemberof 31,August 2025 to July 30,1, 2026. The amendment also extended the date before which any Term LoanLoans prepaymentmature is subject to a make‑whole premium, or the Makewhole Date, fromon November 1, 20262029. The Term Loans bear interest at a variable rate per annum equal to May5.75% 1,plus 2027.three-month SOFR with a SOFR floor of 3.00%. See Note 7 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

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

Our commercial strategy is designed to ensuresuch 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. However, our strategy to drive sales growth and our ongoing commercialization efforts hashave 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 revenue 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, 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.

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 six months ended MarchJune 31,30, 2026, as compared to the critical accounting policies and estimates disclosed in our 2025 Form 10-K.

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

Total productProduct revenue, net for the three and six months ended MarchJune 31,30, 20262026, and 2025 werewas approximately $51.8$57.5 million and $39.4$109.2 million, respectively, compared to $49.0 million and $88.4 million for the three and six months ended June 30, 2025, respectively. Product revenue, net,net increased by $12.4$8.5 million and $20.8 million during the three and six months ended MarchJune 31,30, 2026, respectively, compared to the same periodperiods 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 in both periods was primarily driven by a higher number of patients on therapy.

Total gross-to-net adjustments for both the three and six months ended MarchJune 31,30, 2026 andwas 2025 were 20.8% and 13.0%20.7% of gross product revenue, compared to 15.3% and 14.3% for the three and six months ended June 30, 2025, respectively. The increase in gross-to-net adjustments in both periods was driven by a greater proportion of sales made under governmental managed-care pricing programs and group purchasing organizations in addition to a 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.

We recognized royalty revenues of less than $0.1 million and $0.1 million in the three and six months ended MarchJune 31,30, 2026, respectively, compared to less than $0.1 million and $0.2 million for the same periodperiods in 2025.2025, respectively. Royalty revenues in the three and six months ended MarchJune 31,30, 2026 and 2025 primarily reflect estimated royalties from sales of cell-based research products from our divested stem cell assets.

Cost of goods sold was approximately $1.7$9.2 million and $1.2$10.9 million for the three and six months ended MarchJune 31,30, 20262026, respectively, compared to $1.2 million and $2.4 million for the three and six months ended June 30, 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.RYTELO as well as inventory provisions and non-cash inventory write-offs for the applicable period. Cost of product sales increased in the three and six months ended June 30, 2026, compared to the same periods in 2025, primarily due to non-cash inventory write-offs of $6.3 million during the second quarter of 2026.

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 was partially expensed in a prior period and is therefore excluded from the cost of goods sold for the three and six months ended MarchJune 31,30, 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.

During the three and six months ended MarchJune 31,30, 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 six months ended MarchJune 31,30, 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.

The decreaseincrease in research and development expenses for the three and six months ended MarchJune 31,30, 2026, compared to the same periodperiods in 2025, was primarily due a decreaseresult of investments in personnel-relatedsupply costschain duedevelopment, topartially offset by lower laborpersonnel-related costsexpenses 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.2025.

Selling, general and administrative expenses were $35.4$38.9 million and $74.3 million for the three and six months ended MarchJune 31,30, 2026, respectively, compared to $40.0$38.6 million and $78.6 million for the same periodperiods in 2025. The decreasechange in selling, general and administrative expenses for the three and six months ended MarchJune 31,30, 2026, compared to the same periodperiods in 2025, was primarily due to lower general and administrative personnel-related expenses from decreased headcount,headcount as a result of the workforce reduction in December 2025, partially offset by additional investment in marketing programs of approximately $1.8 million and approximately $1.0 million higher legal expenses.

For the three and six months ended MarchJune 31,30, 2026, a credit of $0.4$0.2 million and $0.6 million, respectively, 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 and six months ended June 30, 2025.

Interest income was $3.4$3.0 million and $6.4 million, respectively, for the three and six months ended MarchJune 31,30, 2026, compared to $5.2$4.7 million and $9.8 million for the same periodperiods in 2025. The decrease in interest income for the three and six months ended MarchJune 31,30, 2026, compared to the same periodperiods in 2025, primarily reflects a decrease in interest rates between the periods and a smaller marketable securities portfolio. Interest earned in future periods will depend on the size of our marketable securities portfolio and prevailing interest rates.

Interest expense was $7.1 million and $14.3 million, respectively, for the three and six months ended MarchJune 31,30, 2026, compared to $8.2$8.5 million and $16.7 million for the same periodperiods in 2025. The decrease in interest expense for the three and six months ended MarchJune 31,30, 2026, compared to the same periodperiods 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 (expense), net was an expense of less than $0.1 million and $0.1 million, respectively, for the three and six months ended June 30, 2026, compared to $0.1 million for each of the three and six months ended MarchJune 31, 2026 and30, 2025. Other income (expense), net primarily reflects realized losses on marketable securities and foreign currency transaction adjustments.

As of MarchJune 31,30, 2026, we had cash, restricted cash, cash equivalents, and marketable securities of $341.0$326.9 million, compared to $401.1 million at December 31, 2025. The decrease in cash, restricted cash, cash equivalents and marketable securities during the threesix months ended MarchJune 31,30, 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. We drew the Tranche A Loan of $125.0 million on November 1, 2024, a portion of which was utilized to repay all outstanding indebtedness associated with the Hercules Loan Agreement. The Pharmakon Loan Agreement providesprovided for two additional committed term loan tranches, the Tranche B Loan and the Tranche C Loan, in principal amountseach of $75.0which 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 could have been requestedexpired on orJuly prior30, to December 31, 2025.2026. 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.

Successful drug development and commercialization requires significant amounts of capital. As of MarchJune 31,30, 2026, we had approximately $341.0$326.9 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 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),financings, 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.

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 jurisdictions or 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 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 are difficult to forecast and will depend on many factors, including:

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, 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, 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 equity and credit markets and isare 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. 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.

Net cash used in operations for the threesix months ended MarchJune 31,30, 2026 and 2025 was $62.9$78.7 million and $48.0$75.4 million, respectively. The increase in net cash used in operations for the threesix months ended MarchJune 31,30, 2026, compared to the same period in 2025, was primarily due to payments related to our workforce reduction and inventory purchases.

Net cash provided by investing activities for the threesix months ended MarchJune 31,30, 2026 and 2025 was $51.9$60.2 million and $52.4$72.8 million, respectively. The decrease in net cash provided by investing activities for the threesix months ended MarchJune 31,30, 2026, compared to the same period in 2025, primarily reflects decreased proceeds from the sale and maturities of marketable securities, partially offset by lower purchases of marketable securities.

Net cash provided by financing activities for the threesix months ended MarchJune 31,30, 2026 and 2025 was $2.4$3.8 million and $0.3$1.3 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.

As of MarchJune 31,30, 2026, we had a long-term principal debt balance of $119.8$120.1 million related to 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. 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. Neither the Tranche B Loan nor the Tranche C Loan was drawn, and both are no longer available. 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 dueto toreflect the drawcontinued downamortization of debt discount and issuance costs associated with the Tranche A Loan and potential future draw downs of the other Term Loans under the Pharmakon Loan Agreement. See Note 7 on Debt in Notes to Condensed Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.