What changed in this filing

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Item 1A. Risk Factors

2026-08-05 compared with 2026-05-06 · 91 added, 81 removed, 1287 unchanged (12% of the section changed)

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Our business faces significant risks and uncertainties, and those described below may not be the only risks and uncertainties we face.

Additional risks and uncertainties not presently known to us or that we currently believe are immaterial may also significantly impair our business, financial condition or results of operations.

If any of these risks or uncertainties occur,

our business, financial condition or results of operations could suffer, the market price of our common stock could decline and you could lose all or part of your investment in our common stock.

Additional risks and uncertainties not presently known to us or that we currently believe are immaterial may also

significantly impair our business, financial condition or results of operations.

If any of these risks or uncertainties occur, our business, financial condition or results of operations could suffer, the market price of our common stock could decline and you could lose all or part of your investment in our common stock.

RISKS RELATED TO THE COMMERCIALIZATION OF RYTELO

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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.

Our RYTELO sales trends and resulting revenue have been and may continue to be variable.

Net product revenue was approximately $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.

Our commercialization efforts in the U.S.

have not to date achieved meaningful sales growth and 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 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.

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 RYTELO net product revenue in future periods.

In particular, our strategy may not drive new patient starts in lower-risk MDS U.S.

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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.

The 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.

The 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 are critical to market acceptance of RYTELO.

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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.

However, the Medicare Drug Pricing Negotiation Program provisions of the law currently contain an orphan 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.

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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, o n 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.

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In the EU, orphan drug designation for any additional indication we may pursue for RYTELO does confer certain regulatory advantages—such as protocol assistance—but it does not shorten development timelines or accelerate the EMA’s overall review process.

In April 2023, the EC published a proposal to reform the current pharmaceutical framework, including revision of orphan market exclusivity.

The new legislation, if adopted, is expected to start to apply from mid-2028.

The proposal to reform the current pharmaceutical framework intends to revise the orphan drug designation and exclusivity regime.

In the latest version of the proposal, orphan market exclusivity will be reduced from the current 10 years to 9 years.

Extension by another two years will be possible for so-called “breakthrough orphan medicinal products”.

Previous drafts also included the concept of “global orphan marketing authorization”, which would no longer grant additional 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, authorization of future indications may be affected by the new legislation.

In December 2025, the European Commission, Parliament and Council reached a political agreement on the Pharma Package, which includes proposed changes to the EU orphan medicinal product framework that may modify the length of orphan market exclusivity and change the way in which market exclusivity is awarded to drugs with more than one approved orphan indication.

The Pharma Package is subject to formal adoption by the European Parliament and Council and, if adopted, could alter the exclusivity protections currently available for our orphan designated products in the EU, including by reducing orphan market exclusivity from the current 10 years to 9 years, with a 2-year extension for so-called “breakthrough orphan medicinal products”, and introducing the concept of “global orphan marketing authorization”, which would prevent 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 if adopted.

Even though we reported positive top-line results from IMerge Phase 3 in January 2023 and received regulatory approval from the FDA in June 2024 to commercialize RYTELO in the U.S.

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• the failure of the quality or stability of RYTELO to meet acceptable regulatory standards;

• 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;

• 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 or any third-party service providers may be unable to demonstrate compliance with GMP, good clinical practices, or GCP, or other applicable regulatory and other requirements to the satisfaction of the FDA, the EMA, the competent authorities of the individual EU Member States or similar international regulatory authorities;

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We are incurring and have incurred net losses every year since our operations began in 1990, except for one.

As of March 31, 2026, our accumulated deficit was approximately $1.9 billion.

As of June 30, 2026, our accumulated deficit was approximat ely $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.

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Successful drug development and commercialization requires significant amounts of capital.

As of March 31, 2026, we had approximately $341.0 million in cash, cash equivalents, restricted cash and marketable securities.

As of June 30, 2026, we had approximately $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), collaborations, strategic alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible.

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, 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 approval of RYTELO in the U.S.

in June 2024 and 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 for lower-risk MDS in the U.S., the EU or other potential jurisdictions or indications, if at all.

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, pandemics or other health crises and supply chain and resource issues.

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

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

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• the costs of manufacturing, developing, commercializing and marketing RYTELO, including with respect to third-party vendors and service providers and our ability to achieve any meaningful reduction in manufacturing costs;

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

• the sales price for RYTELO;

• the availability of coverage and adequate third-party reimbursement for RYTELO;

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

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

In the event we need to raise additional capital to fund our business, including pursuant to the 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.

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), 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.

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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.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 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.

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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 are as follows:

(a) a Tranche B Loan of $75.0 million, which is 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 is 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.

If we do not achieve such revenue milestone within the required timeline, we will not be eligible to draw down the Tranche C Loan.

In addition, before we would consider drawing down any of the remaining tranches under the Pharmakon Loan Agreement, 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 liquidity and ability to fund our operations may be substantially impaired.

The Pharmakon Loan Agreement had provided for two additional committed term loan tranches:

(a) a Tranche B Loan of $75.0 million, which was 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 was 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.

As of the date of this Report, neither the Tranche B Loan nor the Tranche C Loan was drawn, and both are no longer available.

All obligations under the Pharmakon Loan Agreement are secured by substantially all of our assets, including our intellectual property.

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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.

Our indebtedness could also have important negative consequences, including:

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We may be required to delay, limit, reduce or terminate our RYTELO development or commercialization efforts or grant to others rights to develop and market RYTELO.

The lenders could also exercise their rights to take possession and dispose of the collateral securing the Pharmakon Loan Agreement, which collateral includes substantially all of our property including, without limitation, our intellectual property, subject to certain exceptions.

The lenders could also exercise their rights to take possession and dispose of the collateral securing the

Pharmakon Loan Agreement, which collateral includes substantially all of our property including, without limitation, our intellectual property, subject to certain exceptions.

Our business, financial condition and results of operations could be materially adversely affected as a result of any of these events.

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and in other countries.

Such challenges may result in loss of exclusivity or in patent claims being narrowed, invalidated, or held unenforceable, which could limit our ability to

stop others from using or commercializing RYTELO or our technology and/or limit the duration of the patent protection for RYTELO and our technology.

Such challenges may result in loss of exclusivity or in patent claims being narrowed, invalidated, or held unenforceable, which could limit our ability to stop others from using or commercializing RYTELO or our technology and/or limit the duration of the patent protection for RYTELO and our technology.

While we have method-of-use patents that protect the use of RYTELO for the treatment of certain diseases, this type of patent does not prevent a generic competitor from making and marketing a product that is identical to RYTELO for an indication that is outside the scope of our approved use after our composition-of-matter patents or their patent term extensions, and any regulatory exclusivities have expired.

Moreover, even if competitors do not actively promote their product for our approved indications, physicians may prescribe or use these generic products “off-label,” which would result in decreased sales for us.

Moreover, even if competitors do not actively promote their

product for our approved indications, physicians may prescribe or use these generic products “off-label,” which would result in decreased sales for us.

In addition to our patents covering RYTELO, we also expect to rely on regulatory exclusivity, including orphan drug exclusivity of up to seven years in the U.S.

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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 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 an NDA and the approval of that application less any time the sponsor did not act with due diligence during the period.

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 the 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.

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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.

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.

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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.

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.

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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 NDA filed with the FDA to obtain permission to sell a generic version of RYTELO.

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 ANDA 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.

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As of June 1, 2023, all European patents, including those issued prior to June 1, 2023 in principle fall under the jurisdiction of the UPC and allow for the possibility of obtaining injunctions for the UPC Member States and are at risk of central revocation at the UPC in participating UPC Member States.

Under the EU Patent Package, patent holders are permitted to “opt out” their European patents (but not their Unitary Patents) from the UPC on a patent-by-patent basis during an initial seven year

transitional period after June 1, 2023.

Under the EU Patent Package, patent holders are permitted to “opt out” their European patents (but not their Unitary Patents) from the UPC on a patent-by-patent basis during an initial seven year transitional period after June 1, 2023.

Owners of European patent applications who receive notice of grant after the EU Patent Package came into effect could, for the UPC Member States, either obtain a Unitary Patent or validate the European patent nationally and optionally file an opt-out demand.

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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.

Further, even if we prevail, the infringer may file an appeal and the court judgment may be overturned and/or 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.

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We may not be able to protect our intellectual property rights in the U.S or worldwide and challenges to our owned or licensed patent rights would result in costly and time-consuming legal proceedings that could prevent or limit development or commercialization of RYTELO.

Our patents or those patent rights we have licensed, including patent rights that we may seek with respect to inventions made by past or future collaborators, may be challenged through administrative or judicial proceedings, which could result in the loss of important patent rights.

which could result in the loss of important patent rights.

For example, where more than one party seeks U.S.

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The commercial success of RYTELO will depend upon our ability to research, develop, manufacture, market and sell RYTELO without infringing or otherwise violating the intellectual property and other proprietary rights of third parties.

There is considerable intellectual property litigation in the biotechnology and pharmaceutical industries, and

many pharmaceutical companies, including potential competitors, have substantial patent portfolios.

There is considerable intellectual property litigation in the biotechnology and pharmaceutical industries, and many pharmaceutical companies, including potential competitors, have substantial patent portfolios.

Since we cannot be aware of all intellectual property rights potentially relating to RYTELO and its uses, we do not know with certainty that RYTELO, or the commercialization thereof, does not and will not infringe or otherwise violate any third party’s intellectual property.

For example, we are aware that certain third parties have or may be prosecuting patents and patent estates that may relate to RYTELO, and while these patents have expired, or we believe that a reasonable court should find they are invalid and/or would not be infringed by the manufacture, use or sale of RYTELO, it is possible that the owner(s) of these patents will assert claims against us in the future.

they are invalid and/or would not be infringed by the manufacture, use or sale of RYTELO, it is possible that the owner(s) of these patents will assert claims against us in the future.

In the event our technologies infringe the rights of others or require the use of discoveries and technologies controlled by third parties, we may be prevented from pursuing research, development, manufacturing or commercialization of RYTELO, or may be required to obtain unblocking licenses from such third parties, develop alternative non-infringing technologies, which we may not be able to do at an acceptable cost or on acceptable terms, or at all, or cease the commercialization and continued development of RYTELO.

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Under some circumstances, it may be difficult to determine who invents and owns a particular invention, or whether it is jointly owned, and disputes can arise regarding inventorship, ownership and use of those inventions.

disputes could be costly and time-consuming, and an unfavorable outcome could have a significant adverse effect on our business if we are not able to protect or license rights to these inventions.

These disputes could be costly and time-consuming, and an unfavorable outcome could have a significant adverse effect on our business if we are not able to protect or license rights to these inventions.

In addition, clinical trial investigators, scientific consultants and research collaborators generally have contractual rights to publish data and other proprietary information, subject to review by the trial sponsor.

Publications by us, or by investigators, scientific consultants, previous employees, research collaborators or others, either with permission or in contravention of the terms of their agreements with us or with our past or future collaborators, may impair our ability to obtain patent protection or protect proprietary information, which could have a material adverse effect on our business and might cause us to cease operations.

Publications by us, or by investigators, scientific consultants, previous employees,

research collaborators or others, either with permission or in contravention of the terms of their agreements with us or with our past or future collaborators, may impair our ability to obtain patent protection or protect proprietary information, which could have a material adverse effect on our business and might cause us to cease operations.

Much of the information and know-how that is critical to our business is not patentable, and we may not be able to prevent others from obtaining this information and establishing competitive enterprises.

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and in the EU for lower-risk MDS, and to continue to develop RYTELO in other myeloid hematologic malignancies depends to a significant extent on the skills, experience and efforts of our executive officers and key members of our staff.

In addition, we need to recruit, maintain, motivate and integrate additional personnel with expertise and experience in sales, marketing, market access, commercial

operations, pricing, clinical science, biostatistics, clinical operations, pharmacovigilance, quality, manufacturing, regulatory affairs, medical affairs, legal affairs, and compliance to enable us to further commercialize and further develop RYTELO.

We face intense competition for qualified individuals from numerous pharmaceutical, biopharmaceutical and biotechnology companies, as well as academic and other research institutions, and competition in our geographic regions is particularly intense.

In addition, we need to recruit, maintain, motivate and integrate additional personnel with expertise and experience in sales, marketing, market access, commercial operations, pricing, clinical science, biostatistics, clinical operations, pharmacovigilance, quality, manufacturing, regulatory affairs, medical affairs, legal affairs, and compliance to enable us to further commercialize and further develop RYTELO.

regions is particularly intense.

The substantial risks and uncertainties related to our commercialization and further development of RYTELO, and the risks and uncertainties regarding our future business viability could have an adverse impact on our ability to retain and recruit qualified personnel.

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In addition, the terms of our Pharmakon Loan Agreement may limit our ability to enter into certain collaborative arrangements and any future debt agreements may continue or further limit our ability to enter into such agreements.

We may not be able to establish collaborative

arrangements on acceptable terms, or at all.

We may not be able to establish collaborative arrangements on acceptable terms, or at all.

In this regard, collaborative arrangements with third parties may require us to relinquish material rights, including revenue from commercialization, or assume material ongoing development obligations that we would have to fund or otherwise support.

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has called for substantial changes to foreign trade policy and has recently imposed significant increases in tariffs on international trade and the renegotiation of international trade agreements.

We cannot predict what effects these and potential additional tariffs or renegotiation of existing

international trade agreements, scope and nature of tariffs in the future, including as a result of litigation or other challenges, will have on our business, including in the context of escalating global trade and political tensions.

We cannot predict what effects these and potential additional tariffs or renegotiation of existing international trade agreements, scope and nature of tariffs in the future, including as a result of litigation or other challenges, will have on our business, including in the context of escalating global trade and political tensions.

However, such tariffs and other trade restrictions could increase our cost of doing business, reduce our gross margins or otherwise negatively impact our financial results.

Likewise, the ongoing Russia-Ukraine military conflict and the ongoing military conflict involving the U.S., Israel and Iran have had and may continue to have further global economic consequences, including continued disruptions of the global supply chain and energy markets, which could disrupt our existing supply chain and increase our cost of doing business, reduce our gross margins or otherwise negatively impact our financial results.

Likewise, the ongoing Russia-Ukraine military conflict and the ongoing military conflict involving the U.S., Israel and Iran have had and may continue to have further global economic consequences,

including continued disruptions of the global supply chain and energy markets, which could disrupt our existing supply chain and increase our cost of doing business, reduce our gross margins or otherwise negatively impact our financial results.

See the risk factors titled, “ Global trade issues and changes in and uncertainties with respect to trade policies and export regulations, including import and export license requirements, trade sanctions, tariffs and international trade disputes, could increase our costs and negatively impact net revenues from sales of RYTELO ” and “ Failure by us to maintain a manufacturing supply chain to appropriately and adequately supply RYTELO for commercial and future clinical uses would adversely affect our ability to commercialize RYTELO and result in a further delay in or cessation of clinical trials, and our business and business prospects could be severely harmed, and we could cease operations.

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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.

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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.

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.

Further, the continued threats of new or increased tariffs, sanctions, trade restrictions and trade barriers as well as ongoing changes in U.S.

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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 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 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.

We intend to move to dismiss the second amended complaint.

On May 18, 2026, 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.

In addition, on April 15, 2025 and April 16, 2025, three purported stockholders filed derivative complaints, each filed in the United States District Court for the Northern District of California, captioned Bishop v.

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Most of our employees work remotely, resulting in increased risks of loss or theft of company devices as well as increased risks to our information technology systems and data, as employees utilize network connections, computers, and devices outside our premises and networks, including working at home and while in transit and in public locations.

Future or past business transactions, such as acquisitions or integrations, could expose us to additional cybersecurity risks and vulnerabilities, as our systems could be negatively affected by vulnerabilities present in acquired or integrated entities’ systems and technologies.

integrated entities’ systems and technologies.

Furthermore, we may discover security issues that were not found during due diligence of such acquired or integrated entities, and it may be difficult to integrate companies into our information technology environment and security program.

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In addition to experiencing a security incident, third parties may gather, collect, or infer sensitive information about us from public sources, data brokers, or other means that reveals competitively sensitive details about our organization and could be used to undermine our competitive advantage or market position.

Additionally, sensitive

information of the Company could be leaked, disclosed, or revealed as a result of or in connection with our employees’, personnel’s, or vendors’ use of generative AI technologies.

Additionally, sensitive information of the Company could be leaked, disclosed, or revealed as a result of or in connection with our employees’, personnel’s, or vendors’ use of generative AI technologies.

Many of our contracts with relevant stakeholders include obligations relating to the safeguard of sensitive information, and a breach could lead to claims against us by such stakeholders.

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Likewise, we expect that there will continue to be new proposed laws, regulations and industry standards relating to data privacy and security in the U.S.

For example, HIPAA, as amended by HITECH, imposes specific requirements relating to the privacy, security, and transmission of individually identifiable health data.

For example, HIPAA, as amended by HITECH, imposes specific

requirements relating to the privacy, security, and transmission of individually identifiable health data.

Additionally, the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act of 2020, or CPRA, collectively CCPA, imposes obligations on businesses to which it applies.

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Historically, our stock price has been extremely volatile.

Between January 1, 2016 and March 31, 2026, our stock has traded as high as $6.38 per share and as low as $0.89 per share.

Between April 1, 2025 and March 31, 2026,

the price has ranged between a high of $1.95 per share and a low of $1.04 per share.

Between July 1, 2016 and June 30, 2026, our stock has traded as high as $6.38 per share and as low as $0.89 per share.

Between July 1, 2025 and June 30, 2026, the price has ranged between a high of $1.95 per share and a low of $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:

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The pending lawsuits and any lawsuits brought against us in the future could result in substantial costs and divert our management’s attention and resources, which could have a material adverse effect on our financial condition and business operations and lead to increased volatility in our stock price and a decrease in the value of our stockholders’ investment in our securities.

price of our common stock, liquidity, our ability to access the capital markets and our stockholders’ ability to sell their shares.

Our failure to maintain compliance with the continued listing requirements of the Nasdaq Global Select Market may result in our common stock being delisted from the Nasdaq Global Select Market, which could negatively impact the price of our common stock, liquidity, our ability to access the capital markets and our stockholders’ ability to sell their shares.

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 per share through May 1, 2026.

Historically, our stock price has been extremely volatile and recently, our

stock has traded as low as $1.07 per share through July 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.

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or laws that require the true, complete and accurate reporting of financial information or data.

Specifically, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, kickbacks, self-dealing and other abusive practices.

Specifically, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud,

kickbacks, self-dealing and other abusive practices.

These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements.

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If material weaknesses or other significant deficiencies continue to occur, such weaknesses or deficiencies could result in misstatements of our results of operations, restatements of our financial statements, a decline in our stock price, or other material adverse effects on our business, reputation, results of operations, financial condition or liquidity.

may result in material misstatements of our consolidated financial statements or cause us to fail to meet our periodic reporting obligations.

We have previously identified a material weakness in our internal control over financial reporting and may identify additional material weaknesses in the future or otherwise fail to maintain an effective system of internal controls, which may result in material misstatements of our consolidated financial statements or cause us to fail to meet our periodic reporting obligations.

A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial reporting, such that there is a reasonable possibility that a material misstatement of a company’s annual or interim financial statements will not be prevented or detected on a timely basis.

We previously identified a material weakness in internal control related to the design and operation of certain Information Technology General Controls, or ITGCs, related to user access and program change management for our Enterprise Resource Planning and payment processing systems.

Consequently, IT application controls and IT dependent manual business process controls that rely upon information from these systems, were also deemed ineffective.

Consequently, IT application controls and IT dependent manual business process controls that rely upon information from

these systems, were also deemed ineffective.

Although the material weakness identified did not result in any material misstatements in our consolidated financial statements for the periods presented in this Report and there were otherwise no changes to our previously issued financial statements, our management concluded that these control deficiencies existed as of September 30, 2025 and constitute a material weakness.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1A. Risk Factors