Item 1A of this Report. Our request for NCE exclusivity for RYTELO is pending review by the FDA. If the FDA were to
grant NCE exclusivity for RYTELO, NCE exclusivity would continue until June 2029.
The Hatch-Waxman Act also provides three years of marketing exclusivity for a product that is not an NCE
but that incorporates a change(such as a new indication, dosage form or strength) from the approved product with the same
moiety. Such product may qualify for a three-year period of exclusivity if the NDA contains reports of new clinical
investigations (other than bioavailability studies) conducted or sponsored by the sponsor that were necessary for approval
of the NDA. In that instance, the exclusivity period does not preclude filing or review of an ANDA or 505(b)(2)
application; rather, the FDA is precluded from granting final approval to the ANDA or 505(b)(2) application until three
years after approval of the Reference Listed Drug, or RLD. Additionally, the exclusivity applies only to the extent that any
subsequent ANDA or Section 505(b)(2) product that shares the conditions of approval that required submission of the
clinical data.
In the US, the exclusivity periods and patent-related protections described above also may be eligible for a
six-month extension of regulatory exclusivity, or pediatric exclusivity, pursuant to section 505A of the Federal Food, Drug,
and Cosmetic Act, if the sponsor submits pediatric data that “fairly respond” to a written request from FDA for such data;
however, we do not expect to receive pediatric exclusivity for RYTELO in the U.S.
Europe
In the EEA, a medicinal product containing a new active substance generally receives eight years of data
exclusivity, which prevents competitors from relying on the results of pre-clinical tests and of clinical trials when applying
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for a marketing authorization for a generic or biosimilar product, and an additional two years of market exclusivity, during
which competitors can apply for a marketing authorization by referencing the marketing authorization holder's data but are
not allowed to place their product on the market until the end of this period, conferring a total of ten years of exclusivity for
the medicinal product. The ten years of exclusivity can be extended to a maximum of eleven years if, during the first eight
years of those ten years, the marketing authorization holder obtains an authorization for one or more new therapeutic
indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit
in comparison with existing therapies. When RYTELO was granted marketing authorization by the EC for the treatment of
certain patients with lower-risk MDS on March 7, 2025, it was confirmed to contain a new active substance and so
RYTELO is entitled to ten years of data exclusivity in the EEA from the time of approval.
In addition to data exclusivity, orphan drug designation by the EC provides regulatory and financial
incentives for companies to develop and market therapies that treat a life‐threatening or chronically debilitating condition
affecting no more than five in 10,000 persons in the EU (or if it is unlikely that marketing of the medicinal product would
generate sufficient returns to justify the investment needed for its development), and where no satisfactory treatment is
available, or, if such method exists, the medicine must be of significant benefit to those affected by the condition in
accordance with Regulation (EC) No 141/2000 (the EU Orphan Regulation). Orphan drug designation also entitles a party
to financial incentives such as reduction of fees or fee waivers, as well as protocol assistance from the EMA during the
product development phase, and direct access to the centralized authorization procedure. In addition, ten years of market
exclusivity is granted following receipt of marketing authorization as an orphan medicinal product, meaning that a
competing similar medicinal product for the same therapeutic indication in principle may not enter the market for a period
of 10 years following marketing authorization, subject to specific conditions. To benefit from market exclusivity, a
medicine must maintain its orphan designation at the time of marketing authorization. Each indication with an orphan
designation confers ten years' market exclusivity for the particular indication. This period may be reduced to six years if the
orphan drug designation criteria are no longer met, including where it is shown that the product is sufficiently profitable to
not justify maintenance of market exclusivity.
In December 2015 and July 2020, the EC granted orphan drug designation to imetelstat for the treatment of
MF and MDS, respectively. On March 7, 2025, the EC granted marketing authorization for RYTELO (imetelstat sodium)
as an orphan medicinal product and considered that imetelstat is a new active substance. RYTELO is indicated as
monotherapy for the treatment of adult patients with TD anemia due to very low, low or intermediate risk myelodysplastic
syndromes (MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory
response to or are ineligible for erythropoietin-based therapy. With the grant of the marketing authorization, RYTELO in
principle is currently entitled to maintain orphan market exclusivity in the EEA in the approved indication for ten years
post-approval.
In addition, under the European Pediatric Regulation, if we fulfill our pediatric investigation plan agreed
upon with the EMA, we would be eligible to receive an additional two years of market exclusivity
under the European Pediatrics Regulation (Regulation (EC) No 1901/2006).
Prior Collaboration with Janssen Biotech, Inc.
Upon the effective date of termination of the license and collaboration agreement, or the Prior
Collaboration Agreement, with Janssen Biotech, Inc., or Janssen, on September 28, 2018, we regained global rights to
imetelstat and are continuing the development, commercialization and marketing of imetelstat on our own. In accordance
with the termination provisions of the Prior Collaboration Agreement, we have an exclusive worldwide license for
intellectual property developed under the Prior Collaboration Agreement for the further development, commercialization
and marketing of imetelstat, without any economic obligations to Janssen with respect to such license. Janssen has assigned
to us certain intellectual property developed by it under the Prior Collaboration Agreement. We now are responsible for the
costs of maintaining, prosecuting and litigating all imetelstat intellectual property that we own.
Licensing
We have no material license agreements. We have global rights to imetelstat, which was discovered and
developed at Geron.
Manufacturing
A typical sequence of steps in the manufacture of imetelstat drug product includes the following key
components:
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•starting materials, which are well‐defined raw materials that are used to make bulk drug
substance;
•bulk drug substance, which is the active pharmaceutical ingredient in a drug product that
provides pharmacological activity or other direct effect in the treatment of disease; and
•final drug product, which is the finished dosage form that contains the drug substance and is
supplied for patient treatment.
Since September 2018, we have engaged third‐party contract manufacturers and have established a supply
chain to manufacture and supply imetelstat that meets applicable regulatory standards for current and potential commercial
uses and current and potential future clinical trials.
We do not have direct control over third‐party personnel or operations. These third‐party contract
manufacturers, and/or any other third parties that we may rely upon for the manufacture and/or supply of imetelstat,
typically complete their services on a proposal by proposal basis under master supply agreements and may need to make
substantial investments to enable sufficient capacity increases and cost reductions, and to implement those regulatory and
compliance standards necessary for commercial production and successful Phase 3 clinical trials. These third‐party contract
manufacturers, and/or any other third parties that we may rely upon for the manufacture and/or supply of imetelstat, may
not be able to achieve such capacity increases, cost reductions, or regulatory and compliance standards, and even if they do,
such achievements may not be at a commercially reasonable cost. We are responsible for establishing any long‐term
commitments or commercial supply agreements with any of the third‐party contract manufacturers for imetelstat. The
information provided in this section should be reviewed in the context of the section entitled “Risks Related to
Manufacturing RYTELO (Imetelstat)” under Part I, Item 1A, “Risk Factors” of this Report.
Competition
The pharmaceutical and biotechnology industries are characterized by intense and dynamic competition
with rapidly advancing technologies and a strong emphasis on proprietary products. While we believe our proprietary
oligonucleotide chemistry; experience with the biological mechanisms related to RYTELO, telomeres and telomerase;
clinical data to date indicating potential disease-modifying activity with RYTELO treatment; and knowledge and expertise
around the development of potential treatments for myeloid hematologic malignancies provide us with competitive
advantages, we face competition from many different sources, including major pharmaceutical, specialty pharmaceutical
and biotechnology companies, academic institutions, governmental agencies, and public and private research institutions.
RYTELO is competing with other products and therapies that currently exist, are being developed or will in the future be
developed, some of which we may not currently be aware of.
Competition in Lower-Risk MDS
The current standard of care for the treatment of lower-risk MDS is the use of ESAs as supportive care, and more
recently luspatercept to improve upon disease-associated chronic anemia. Historically, ESAs have been used as a mainstay
of treatment; once no longer effective, serial blood transfusions are often administered, which can cause organ damage due
to iron overload and result in poor outcomes and shorter survival. In recent years, considerable advancements have been
made in the treatment of LR-MDS with the approval of new therapies, expanding indications, and ongoing clinical
investigation of novel agents.
In lower-risk MDS, data from the IMerge Phase 3 clinical trial resulted in FDA approval of RYTELO in June
2024 and EC approval in March 2025 for the treatment of certain patients with lower-risk MDS. IMerge showed
meaningful and durable transfusion independence, activity across MDS patient subtypes, and potential disease-modifying
activity achievable with RYTELO treatment. We believe that these key features are differentiators compared to currently
approved products as well as investigational drugs currently in clinical development.
In lower-risk MDS, RYTELO competes against a number of currently existing therapies, including ESAs (Epoetin
alfa, Procrit; Darbepoetin alfa, Aranesp-Amgen) that are indicated for anemia; immunomodulators, such as Revlimid
(lenalidomide) by Celgene Corporation, or Celgene, a Bristol Myers Squibb, or BMS, company; hypomethylating agents,
such as Vidaza (azacitidine) by BMS and Dacogen (decitabine) by Otsuka America Pharmaceutical, Inc. and other
manufacturers in the U.S. and Janssen in the EU; Inqovi (oral combination of decitabine and cedazuridine) by Astex
Pharmaceuticals, Inc., or Astex, and Taiho Oncology; Tibsovo (ivosidenib), an IDH1 inhibitor, by Servier Pharmaceuticals,
LLC; and Reblozyl (luspatercept), a TGF-beta ligand trap, by BMS. In August 2023, luspatercept was approved for the
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treatment of anemia in ESA-naive adult patients with very low-to intermediate-risk MDS who may require regular RBC
transfusions.
Other therapies currently in Phase 3 development in lower-risk MDS include elritercept (KER-050), a
TGF-beta inhibitor, by Keros Therapeutics, Inc.; and Reblozyl (luspatercept) in non-transfusion-dependent lower-risk MDS
patients, by BMS.
In addition, there are multiple Phase 1 and Phase 2 clinical trials of other agents being developed for lower-
risk MDS, including but not limited to: LB‐100, a PP2A inhibitor by Lixte Biotechnology Holdings, Inc., and bemcentinib,
an AXL inhibitor by BerGenBio. Also, a lower dose of ASTX727, an oral formulation of decitabine and cedazuridine,
referred to as ASTX727 LD, by Astex; ASTX030, an oral formulation of azacitidine and cedazuridine, by Astex; R289, a
dual oral inhibitor of interleukin receptor-associated kinases 1 and 4, or IRAK1/4, by Rigel Pharmaceuticals, Inc.; and a
combination regimen of luspatercept and ESA.
Competition in Relapsed/Refractory MF
The current standard of care for the treatment of Intermediate-2 or High-risk MF is the use of JAK
inhibitors to address the patient’s symptoms. Once JAK inhibitors fail or are no longer effective, a variety of best available
therapies are used since there are no approved treatments for this patient population and median OS is limited.
In Intermediate-2 or High-risk relapsed/refractory MF, data from IMbark suggest potential disease-
modifying activity with RYTELO treatment and a potential meaningful improvement in OS, which is supported in a
comparison to real-world data.
If approved for commercial sale for the treatment of relapsed/refractory MF, RYTELO would compete
against currently approved JAK inhibitors: Jakafi (ruxolitinib) by Incyte Corporation, or Incyte, Inrebic (fedratinib) by
BMS, and OJJAARA (momelotinib) by GlaxoSmithKline plc, or GSK, which was approved in September 2023 for the
treatment of intermediate or high-risk MF, including primary MF or secondary MF (postpolycythemia vera and post-
essential thrombocytopenia), in adults with anemia; and Vonjo (pacritinib), by Sobi, Inc., which was approved in February
2022 for the treatment of adults with intermediate or high-risk primary or secondary MF with a platelet count below 50 ×
109/L. Other treatment modalities for MF include hydroxyurea for the management of splenomegaly, leukocytosis,
thrombocytosis and constitutional symptoms; splenectomy and splenic irradiation for the management of splenomegaly and
co-existing cytopenias; chemotherapy; and pegylated interferon. Drugs for the treatment of MF-associated anemia include
ESAs, androgens, danazol, corticosteroids, thalidomide and lenalidomide. In addition, luspatercept has been used to treat
MF-associated anemia, although the Phase 3 trial did not meet its primary endpoint for RBC-TI.
Other therapies currently in Phase 3 development in MF, some of which may obtain regulatory approval earlier
than RYTELO for MF, include pelabresib (CPI-0610), a BET inhibitor, by Novartis AG; and navtemadlin, an MDM2-
inhibitor, by Kartos Therapeutics, Inc. Other approaches for MF currently under investigation that could compete with
RYTELO in the future include luspatercept; zinpentraxin alfa (RG6354, formerly PRM-151), an anti-fibrosis antibody, by
F. Hoffmann-La Roche, Ltd.; INCB160058, a JAK2 inhibitor, by Incyte; AJ1-11095, a JAK2 inhibitor, by Ajax
Therapeutics, Inc.; SLT-5505, a pan-LOX inhibitor, by Syntara Limited; tasquinimod, an S100A9 inhibitor, by Active
Biotech AB; XPOVIO (selinexor), a nuclear export inhibitor, by Karyopharm Therapeutics, Inc.; TL-895, an oral tyrosine
kinase inhibitor, by Telios Pharma, Inc.; pelcitoclax (APG-1252), a dual BCL-2/BCL-XL inhibitor, by Ascentage Pharma;
DISC-0974, a monoclonal antibody against hemojuvelin (HJV) by DISC Management Inc.; elritercept (KER-050) in
combination with ruxolitinib, by Keros Therapeutics; CK0804, an allogeneic T-regulatory cell agent, by Cellenkos, Inc. in
collaboration with Incyte; nuvisertib (TP-3654), an oral PIM kinase inhibitor by Sumitomo Pharma Co., Ltd.; and a
mutated-CALR peptide vaccine, from the Icahn School of Medicine at Mount Sinai.
Government Regulation
Regulation by governmental authorities in the U.S. and other countries is a significant factor in the
development, manufacture, distribution and marketing of RYTELO (imetelstat). Imetelstat will require regulatory approval
by regulatory authorities prior to commercialization in any jurisdictions where it is not yet approved. In particular, potential
human therapeutic products, such as imetelstat, are subject to rigorous preclinical testing, clinical testing and quality
standards by the FDA and similar regulatory authorities in European and other countries. Various statutes and regulations
at the federal and state level—as well as regulations and guidance at the international level—also govern or influence the
testing, manufacturing, safety reporting, labeling, storage, import, export, distribution, sale and recordkeeping related to
such products and their marketing. The process of obtaining these approvals and the subsequent compliance with
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appropriate statutes and regulations require the expenditure of substantial time and money, and there can be no guarantee
that approvals will be granted. Moreover, compliance with government regulations governing personal data and
information security requires the expenditure of substantial time and financial resources. The information provided in this
section should be reviewed in the context of the sections entitled “Risks Related to the Further Development of RYTELO
(Imetelstat)” and “Risks Related to Regulatory Approval of RYTELO” under Part I, Item 1A, “Risk Factors” of this
Report.
United States Food and Drug Administration Regulatory Approval Process
Prior to commencement of clinical trials of new pharmaceutical products in humans, government
authorities generally require the completion of laboratory studies as well as certain preclinical testing in animals to evaluate
the potential efficacy and safety of a product candidate, though, under the 2022 FDA Modernization Act 2.0, FDA has
begun to phase out the requirement for animal testing by incorporating new approval methodologies such as AI-based
computational models, organ-on-a-chip systems, and advanced in vitro assays. The results of animal testing or alternative
models are submitted to the FDA as part of an Investigational New Drug Application, or IND, which must become
effective before clinical testing in humans can begin. The FDA can place an IND on clinical hold at any time, which can
prevent the conduct of a clinical trial or all trials under an IND or it may be a partial hold (e.g., a hold on further
enrollment) until safety concerns or questions are addressed by the IND sponsor to the FDA’s satisfaction.
Typically, a clinical development program is lengthy and involves three phases. In Phase 1, clinical trials
are conducted with a small number of healthy volunteers or patients afflicted with a specific disease to assess safety and to
evaluate the metabolism, pharmacokinetics, and pharmacologic action of the drug in humans, and, if possible, to gain early
evidence on effectiveness. In Phase 2, clinical trials are typically well controlled, closely monitored, and conducted in a
relatively small number of patients, usually involving no more than several hundred subjects to evaluate the effectiveness
of the drug in patients to determine the common short-term side effects and risks associated with the drug. Phase 2 trials
can be conducted comparing the investigational treatment to a comparator arm, or not. If used, a comparator arm often
includes standard of care therapy. Safety and efficacy data from Phase 2 clinical trials, even if favorable, may not provide
sufficient rationale for proceeding to a Phase 3 clinical trial. In Phase 3, clinical trials are typically large scale, multi‐center,
comparative trials conducted with patients with the disease or condition under study and are intended to gather sufficient
data to demonstrate the efficacy and safety required by the FDA. The FDA closely monitors the progress of each of the
three phases of clinical testing and may, at its discretion, re‐evaluate, alter, suspend, or terminate the trials. Human clinical
trials must be conducted in compliance with Good Clinical Practice, or GCP, regulations and applicable laws, with
informed consent from patients and the oversight of Institutional Review Boards for the protection of human subjects.
Drugs used in clinical trials must be manufactured, packaged and labeled in conformity with current Good Manufacturing
Practices, or cGMP, and applicable laws.
The results of the preclinical and clinical testing of drugs and complete manufacturing information are
submitted to the FDA in the form of an NDA for review and approval prior to commencement of commercial sales.
Submission of an NDA requires the payment of a substantial user fee to the FDA, which may be waived in certain cases. In
responding to an NDA submission, the FDA may approve the drug for commercialization, impose limitations on its
indications for use and labeling, including in the form of Risk Evaluation and Mitigation Strategies or may issue a complete
response letter explaining the reason the NDA cannot be approved in its present form. Even if an NDA is approved, its
sponsor and drug will continue to be subject to ongoing and pervasive regulatory compliance requirements.
European Union and Other Regulatory Approval Process
Prior to initiating clinical trials in a region outside of the U.S., a clinical trial application must be submitted
and reviewed by the appropriate regulatory authority governing clinical trials in the country in which the trial will be
conducted. Whether or not FDA clearance or approval has been obtained, approval or authorization of a product by
comparable regulatory authorities in the EU and other countries is necessary prior to marketing the product in such
countries. The competent regulatory authorities may impose their own requirements and may refuse to grant an approval,
or may require additional data before granting it, even though the relevant product has been cleared or approved by the
FDA or another authority. As with the FDA, the regulatory authorities in the EU and other developed countries have
lengthy approval processes for pharmaceutical products. The process for gaining approval in particular countries varies, but
it generally follows a similar sequence to that described for FDA approval. In Europe, the EMA and the CHMP provide a
mechanism for EU member states to exchange information on all aspects of product licensing. The EU has established the
EMA for the evaluation of medical products, with a centralized procedure which is mandatory for orphan and oncology
products and which grants a single marketing authorization valid in all EU member states.
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Fraud and Abuse, and Transparency Laws and Regulations
We may also be subject to additional regulation and enforcement by the federal government and by
authorities in the states and foreign jurisdictions in which we conduct our business. These additional regulations could
affect our current and future arrangements with healthcare professionals, principal investigators, consultants, customers and
third‐party payors. Such laws include, without limitation, state and federal bribery/anti‐kickback, the False Claims Act,
privacy and data security laws, and healthcare professionals payment transparency laws.
The federal Anti‐Kickback Statute makes it illegal for any person or entity, including a prescription drug
manufacturer (or a party acting on its behalf) to knowingly and willfully, directly or indirectly, solicit, receive, offer, or pay
any remuneration that is intended to induce the referral of business, including the purchase, order, or lease of any good,
facility, item or service for which payment may be made under a federal healthcare program, such as Medicare, Medicaid
TRICARE, and the Veterans Health Care Program. The term “remuneration” has been broadly interpreted to include
anything of value. Several courts have interpreted the statute’s intent requirement to mean that if any one purpose of an
arrangement involving remuneration is to induce referrals, the Anti‐Kickback Statute has been violated. In addition, a
person or entity does not need to have actual knowledge of the statute or specific intent to violate, in order to commit a
violation.
Federal civil and criminal false claims and false statement laws, including the federal civil False Claims Act
and its whistleblower or qui tam provisions (which permit private individuals to bring an action on behalf of the
government to enforce the civil False Claims Act), prohibit, among other things, any person or entity from knowingly
presenting, or causing to be presented, for payment to, or approval by, federal programs, including Medicare and Medicaid,
claims for items or services, including drugs, that are false or fraudulent or not provided as claimed. Entities can be held
liable under these laws if they are deemed to “cause” the submission of false or fraudulent claims by, for example,
providing inaccurate billing or coding information to customers, promoting a product off‐label, or for providing medically
unnecessary services or items. In addition, a claim including items or services resulting from a violation of the federal Anti-
Kickback Statute constitutes a false or fraudulent claim for purposes of the federal civil False Claims Act. Criminal
prosecution is also possible for making or presenting a false, fictitious or fraudulent claim to the federal government.
The federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, created criminal and
civil liability for, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any
healthcare benefit program, including private third‐party payors, knowingly and willfully embezzling or stealing from a
healthcare benefit program, willfully obstructing a criminal investigation of a healthcare offense, and knowingly and
willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent
statement in connection with the delivery of or payment for healthcare benefits, items or services.
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or
HITECH, and their implementing regulations, impose obligations, including mandatory contractual terms, with respect to
safeguarding the privacy, security, transmission and breach reporting of individually identifiable health information, upon
entities subject to the law, such as health plans, healthcare clearinghouses and certain healthcare providers and their
respective business associates and their subcontractors that perform services for them that involve individually identifiable
health information.
The federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics
and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance
Program to report annually to the Centers for Medicare & Medicaid Services, or CMS, information related to payments or
other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists, and
chiropractors), other healthcare professionals (such as physicians assistants and nurse practitioners), and teaching hospitals,
and information related to ownership and investment interests held by physicians and their immediate family members.
Analogous state and foreign laws and regulations, such as state anti‐kickback and false claims laws, may
apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by
non‐governmental third-party payors, including private insurers. Additionally, we may be subject to state and foreign laws
that require pharmaceutical companies to comply with the pharmaceutical industry’s otherwise voluntary compliance
guidelines and certain industry compliance guidance documents. Further, we may be subject to state and foreign laws that
require drug manufacturers or other pharmaceutical companies to report information related to payments and other
transfers of value to physicians, other healthcare providers and healthcare entities, or marketing expenditures, as well as
state, foreign and local laws that require the registration of pharmaceutical sales representatives; state and foreign laws that
require the reporting of information related to drug pricing; and state, federal and foreign laws governing the privacy and
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security of personal data (including key-coded data and health information), including the European Union’s General Data
Protection Regulation, or EU GDPR, many of which differ from each other in significant ways, thus complicating
compliance efforts.
If our operations are found to be in violation of any of these or any other healthcare regulatory laws that
may apply to us, we may be subject to significant penalties, including the imposition of significant civil, criminal and
administrative penalties, damages, monetary fines, disgorgement, imprisonment, possible exclusion from participation in
Medicare, Medicaid and other federal healthcare programs, reputational harm, diminished profits and future earnings,
additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar
agreement to resolve allegations of non-compliance with these laws, and curtailment of our operations, any of which could
adversely affect our ability to operate our business and our results of operations. Defending against any such actions can be
costly, time-consuming and may require significant financial and personnel resources. Therefore, even if we are successful
in defending against any such actions that may be brought against us, our business may be impaired.
Data Privacy and Security
In the ordinary course of our business, we process personal or sensitive data. Accordingly, we are, or may
become, subject to numerous data privacy and security obligations, including federal, state, local, and foreign laws,
regulations, guidance, and industry standards related to data privacy and security. Efforts to ensure that our current and
future business arrangements will comply with applicable data privacy and data security laws and regulations will involve
substantial costs. For example, foreign data privacy and security laws (including but not limited to the EU GDPR and UK
GDPR) impose strict significant and complex compliance obligations on entities that are subject to those laws. As one
example, the EU GDPR applies to any company established in the EEA and to companies established outside the EEA that
process personal data in connection with the offering of goods or services to data subjects in the EEA or the monitoring of
the behavior of data subjects in the EEA. These obligations in the EU and UK include limiting the collection and
processing of personal data to only what is necessary for specified, explicit, and legitimate purposes; requiring a legal
basis for personal data processing; requiring the appointment of a data protection officer in certain circumstances;
increasing transparency obligations to data subjects; requiring data protection impact assessments in certain circumstances;
limiting the duration for which personal data may be retained; increasing rights for data subjects formalizing a heightened
and codified standard for data subject consent, requiring the implementation and maintenance of technical and
organizational safeguards for personal data, mandating data breach notifications to relevant supervisory authority(ies), and
mandating the appointment of representatives in the UK and/or the EU in certain circumstances. Moreover, there have
recently been, and we expect that there will continue to be, new data privacy and security laws, regulations and industry
standards in the U.S. As one example, the California Consumer Privacy Act of 2018, or CCPA, imposes numerous
obligations on covered business. Although the CCPA exempts certain data (such as some data processed in the context of
clinical trials), the CCPA, to the extent applicable to our business and operations, may increase our compliance costs and
potential liability with respect to the personal data we maintain about California residents. The CCPA provides for civil
penalties and a private right of action for data breaches which may include an award of statutory damages. Failure, or
perceived failure, to comply with all applicable obligations could result in enforcement actions, fines, litigation, and other
consequences. See the section titled “We and third parties with whom we work are subject to stringent and changing U.S.
and foreign laws, regulations, rules, contractual obligations, industry standards, policies and other obligations related to
data privacy and security. Our (or the third parties with whom we work) actual or perceived failure to comply with such
obligations could lead to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business
operations; reputational harm; loss of revenue and profits; and other adverse business impacts,” under “Risk Factors” in
Part I, Item 1A of this Report for additional information about the laws and regulations to which we may become subject
and about the risks to our business associated with such laws and regulations.
Coverage and Reimbursement
Significant uncertainty exists as to the coverage and reimbursement status of any product candidate that
receives regulatory approval. In the U.S. and markets in other countries, sales of RYTELO will depend, in part, on the
extent to which third‐party payors provide coverage and establish adequate reimbursement levels.
In the U.S., third‐party payors include federal and state healthcare programs, government authorities,
private managed care providers, private health insurers, employer health plans, and other organizations. Decisions
regarding the extent of coverage and amount of reimbursement to be provided are made on a plan-by-plan basis.
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Reimbursement by a third-party payor may depend upon a number of factors, including the third-party payor’s
determination that a product is safe, effective and medically necessary; appropriate for the specific patient; cost-effective;
supported by peer-reviewed medical journals; included in clinical practice guidelines; and neither cosmetic, experimental,
nor investigational. A third-party payor could also require that certain lines of therapy be completed or failed prior to
reimbursing our therapy. The principal decisions about reimbursement for new medicines are typically made by CMS.
CMS decides whether and to what extent products will be covered and reimbursed under Medicare and private payors tend
to follow CMS to a substantial degree. Third-party payors determine which products and procedures they will cover and
establish reimbursement levels. Third‐party payors are increasingly challenging the price, examining the medical necessity
and reviewing the cost‐effectiveness of medical drug products and medical services, in addition to questioning their safety
and efficacy. Such payors may limit coverage to specific drug products on an approved list, also known as a formulary,
which might not include all of the FDA‐approved drugs for a particular indication. We may need to conduct expensive
pharmacoeconomic studies in order to demonstrate the medical necessity and cost‐effectiveness of RYTELO, in addition to
the costs required to obtain the FDA approvals. Nonetheless, RYTELO may not be considered medically necessary or
cost‐effective. Moreover, the process for determining whether a third‐party payor will provide coverage for a drug product
may be separate from the process for setting the price of a drug product or for establishing the reimbursement rate that such
a payor will pay for the drug product. A payor’s decision to provide coverage for a drug product does not imply that an
adequate reimbursement rate will be approved. Further, one payor’s determination to provide coverage for a drug product
does not assure that other payors will also provide coverage for the drug product, as there is no uniform coverage and
reimbursement policy among third-party payors in the U.S. Adequate third‐party reimbursement may not be available to
enable us to maintain price levels sufficient to realize an appropriate return on our investment in RYTELO. Even if a third-
party payor covers a particular product or procedure, the resulting reimbursement payment rates may not be adequate.
Coverage policies and third-party payor reimbursement rates may change. Thus, even if favorable coverage and
reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the
future. These third-party payors are increasingly reducing coverage and reimbursement for medical products, drugs and
services. In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-
containment programs, including price controls, restrictions on coverage and reimbursement and requirements for
substitution of generic products. Adoption of price controls and cost-containment measures, and adoption of more
restrictive policies in jurisdictions with existing controls and measures, could further limit sales of any product. Decreases
in third-party reimbursement for any product or a decision by a third-party payor not to cover a product could reduce
demand for the product and also have a material adverse effect on future sales.
Healthcare Reform
There has been increasing legislative and enforcement interest in the U.S. with respect to specialty drug
pricing practices. Specifically, there have been several recent U.S. Congressional inquiries, Presidential executive orders,
and federal and state legislative activity designed to, among other things, bring more transparency to drug pricing, review
the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform
government program reimbursement methodologies for drugs. For example, the Inflation Reduction Act of 2022, or IRA,
which, among other things, (i) directs the Department of Health and Human Services, or HHS, to negotiate the price of
certain high-expenditure, single-source drugs and biologics covered under Medicare that have been on the market for at
least seven years (the “Medicare Drug Price Negotiation Program”), and subject drug manufacturers to civil monetary
penalties and a potential excise tax by offering a price that is not equal to or less than the negotiated “maximum fair price”
for such drugs and biologics under the law, and (ii) imposes rebates with respect to certain drugs and biologics covered
under Medicare Part B or Medicare Part D to penalize price increases that outpace inflation. The IRA permits HHS to
implement many of these provisions through guidance, as opposed to regulation, for the initial years. These provisions
began to take effect progressively in fiscal year 2023. The agreed upon prices of the first ten drugs that were subject to
price negotiations took effect on January 1, 2026, although the Medicare Drug Price Negotiation Program is currently
subject to legal challenges. In2025, HHS selected fifteen additional products covered under Part D for price negotiation and
announced “maximum fair prices” that will go into effect on January 1, 2027. Each subsequent year, more Part B and Part
D products will become subject to the Medicare Drug Price Negotiation Program. Further, on December 7, 2023, an
initiative was announced to evaluate whether the use of march-in authorities under the Bayh-Dole Act could impact the
price of or promote equitable access to prescription drugs. On December 8, 2023, the National Institute of Standards and
Technology published for comment a Draft Interagency Guidance Framework for Considering the Exercise of March-In
Rights, or the Draft Framework, which contemplated the price of a product as one of several factors an agency could use
when deciding to exercise march-in rights. The Bayh-Dole Act does not include product pricing as an express basis for
exercising march-in rights, and prior rulemaking initiatives have not adopted proposed regulatory revisions that would have
permitted the exercise of march-in rights on the basis of product pricing. While march-in rights have not previously been
exercised, it is uncertain if that will continue if the Draft Framework ultimately is adopted. Additionally, at the state level,
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legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical and
biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product
access and marketing cost disclosure and transparency measures, and, in some cases, to encourage importation from other
countries and bulk purchasing.
The U.S. and some foreign jurisdictions are considering or have enacted legislative and regulatory
proposals to contain healthcare costs, as well as to improve quality and expand access. For example, the IRA also
eliminated the “donut hole” under the Medicare Part D program by significantly lowering the beneficiary maximum out-of-
pocket cost and creating a new manufacturer discount program. It is possible that the IRA will be subject to additional
judicial or Congressional challenges in the future. We expect that other healthcare reform measures that may be adopted in
the future may result in more rigorous coverage criteria and lower reimbursement, and additional downward pressure on
the price that may be charged for RYTELO. It is unclear how any such healthcare reform measures will impact the
pharmaceutical industry.
In addition, aggregate reductions to Medicare payments to providers of up to 2% per fiscal year, which
went into effect beginning on April 1, 2013 under provisions of the Budget Control Act of 2011 will stay in effect through
2032 unless additional Congressional action is taken.
Information About Our Executive Officers
The following table sets forth certain information with respect to our executive officers and other members
of management as of January 31, 2026:
Name Age Position
Executive Officers
Harout Semerjian 55 President, Chief Executive Officer and Board member
Joseph Eid, M.D. 58 Executive Vice President, Research and Development
Ahmed ElNawawi 45 Executive Vice President, Chief Commercial Officer
Harout Semerjian has served as our President and Chief Executive Officer and member of our Board
since August, 2025. Prior to joining the Company, Mr. Semerjian served as President and Chief Executive Officer of
GlycoMimetics, Inc., a late-stage clinical biotechnology company subsequently combined with Crescent Biopharma, Inc.,
from August 2021 until February 2025. He also previously served on the board of directors of GlycoMimetics until
February 2025. Since October 2023, Mr. Semerjian has also served as a member of the board of directors at the
Biotechnology Innovation Organization. From June 2020 to July 2021, Mr. Semerjian served as an independent healthcare
consultant at Emerge Bio Consulting, advising private equity firms on healthcare investment projects. Mr. Semerjian
served as President and Chief Executive Officer of Immunomedics Inc., a biotechnology company specializing in antibody-
drug conjugates for cancer treatment, from April 2020 to May 2020, prior to its acquisition by Gilead Sciences, Inc. From
March 2018 to April 2020, Mr. Semerjian served as Executive Vice President, Chief Commercial Officer of Ipsen Pharma,
a global, pharmaceutical company focusing on areas of high unmet medical need, leading and executing Ipsen’s global
commercial strategy and functions across oncology, neurosciences and rare diseases. From February 2017 to February
2018, he served as President and Head of Ipsen’s Specialty Care International Region & Global Franchises. From 1994 to
January 2017, Mr. Semerjian held several commercial, marketing and sales positions of increasing responsibility within
Novartis Pharmaceuticals, a global pharmaceutical company, including serving as Senior Vice President and Global
Launch Leader for KISQALI®, in regional vice president hematology and oncology roles in the U.S., MENA and the
Nordics, and as global brand director for Gleevec®, as well as for Merck, a global pharmaceutical company, and Solvay, a
multinational chemical and materials company. Mr. Semerjian holds an MBA from Cornell University and Queen's
University in Canada, and a B.S. in Biology from Lebanese American University.
Michelle Robertson has served as our Executive Vice President, Chief Financial Officer and Treasurer
since September 2023. Prior to joining Geron, she served as the Chief Financial Officer and Treasurer of Editas Medicine,
Inc., a CRISPR genome editing company, from January 2020 to May 2023. Before that, she served as Chief Financial
Officer of Momenta Pharmaceuticals, Inc. from 2018 until 2020, when Momenta was acquired by Johnson & Johnson.
Prior to joining Momenta, Ms. Robertson held multiple commercial finance roles of increasing responsibility, including
Vice President, Oncology Finance for Baxalta Incorporated following its spin-off from Baxter International Inc., from 2015
to 2016; Head of Financial Planning and Analysis and Operations Excellence at Ironwood Pharmaceuticals, Inc. from 2012
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to 2015; and various finance and commercial operations roles at Genzyme Corporation (acquired by Sanofi). She also
currently serves as a member of the board of directors and as the chair of the audit committee for Verastem, Inc., a
publicly-traded biopharmaceutical company. Ms. Robertson received her B.S. in Finance and A.S. in Accounting and
Management from Bentley University.
Joseph Eid, M.D., has served as our Executive Vice President Research and Development since November
2024. Prior to joining Geron, Dr. Eid served as President, Research and Development for Dragonfly Therapeutics, Inc., a
clinical stage biopharmaceutical company, with overall responsibilities for Dragonfly's discovery and clinical research
strategy and execution, from February 2023 to March 2024. Before joining Dragonfly Dr. Eid served as Executive Vice
President, Chief Medical Officer at Luzsana Biotechnology, Inc., a pharmaceutical company and a subsidiary of Hengrui
Pharmaceuticals, from October 2021 to September 2022. Prior to his biotechnology leadership roles, Dr. Eid served as
Senior Vice President and Head, Global Medical Affairs for Bristol Myers Squibb, or BMS, from 2017 to 2021, where he
led global medical affairs across four therapeutic franchises. Prior to BMS, Dr. Eid spent nine years at Merck, first at
Merck Research Labs, where he led the first-in-human strategy of their global KEYTRUDA® program, and then at Merck
Global Human Health, where he built Merck's global oncology medical affairs team. Dr. Eid started his pharmaceutical
career at Hoffmann La Roche, where he was responsible for both early- and late-stage assets and led several clinical teams.
Prior to entering the biopharmaceutical industry, Dr. Eid was an Assistant Professor in the hematology department of
Robert Wood Johnson Medical School in New Jersey from 1999 to 2004 and as a volunteer, through 2019. Dr. Eid
received his M.D. from Saint Joseph University, Faculty of Medicine, and serves on ALSAC/St Jude Children’s Research
Hospital board, and on the board of Angle PLC, a liquid biopsy company.
Ahmed ElNawawi has served as our Executive Vice President, Chief Commercial Officer since October
2025. Prior to joining the Company, Mr. ElNawawi most recently served as Senior Vice President and U.S. Commercial
Head at Stemline Therapeutics, a wholly-owned subsidiary of the Menarini Group, from April 2022 until October 2025,
where he led the U.S. commercial organization spanning the sales, marketing, market access, commercial excellence, and
data analytics functions. Before joining Stemline, Mr. ElNawawi spent nearly two decades at Novartis Oncology, from
April 2004 until April 2022, in roles of increasing responsibility, including Oncology General Manager for Romania and
the Gulf region, Executive Director for U.S. Melanoma, and Global Indication Lead for both breast and lung cancer.
Earlier in his career, he held commercial and marketing positions in Egypt, the UAE, and Saudi Arabia with Merck and
Schering-Plough. He received an MBA from the University of Leicester and a B.S. in Clinical Pharmacy from Ain Shams
University in Cairo, Egypt.
Human Capital
Corporate Values
Fostering and maintaining a strong, healthy culture is a key strategic focus. We recognize and value the
unique strengths of each of our team members, and the impact and contributions of every employee.
Our core values are the foundational principles of our organization. These values reflect who we are, how
we work and the way our employees interact with one another, our partners, our communities, and our shareholders. They
are the essential tenets that guide our business decisions, govern our relationships, both internally and externally, and
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articulate what we stand for and who we are. These values dictate the ways in which we interact, work and communicate,
how we resolve conflicts and ultimately, how we strive to make Geron successful.
Our team of talented professionals is the foundation of our company and fuels our historical and
prospective achievements for patients. We consider the intellectual capital of our employees to be an essential driver of our
business and key to our future opportunities. As of December 31, 2025, we had 258 full‐time employees. However, in
December 2025, we announced a strategic restructuring plan that is intended to position us for long-term value creation and
improve our financial discipline. The restructuring plan will result in a reduction in headcount of approximately one-third
of our workforce. We anticipate that the restructuring plan will be substantially completed in the first quarter of 2026. See
Note 16 on Restructuring in Notes to Consolidated Financial Statements of this Report for additional information. Every
employee plays a vital role in furthering our business goals and advancing the development and delivery of our novel
medicine to patients.
In addition to our employee base, we have established, and expect to continue to establish, consulting
agreements with drug development professionals, clinicians, attorneys and regulatory experts with experience in numerous
fields, including clinical science, biostatistics, clinical operations, pharmacovigilance, quality, manufacturing and
regulatory affairs.
To succeed in our mission, we must attract, recruit, retain, develop and motivate qualified clinical,
nonclinical, commercial, scientific, manufacturing, regulatory, management and other personnel needed to support our
business and operations. As a biotechnology company with office locations in the San Francisco Bay Area and northern
New Jersey, and with remote employees throughout the U.S., we operate in a highly competitive industry and geographies
for employee talent. We maintain a comprehensive dashboard of measurements, including recruitment productivity,
employee engagement scores, total rewards benchmarking, participation rates and satisfaction scores for internal training,
turnover rates and exit interview results, to guide our human capital management efforts.
We believe that our ability to attract highly skilled and talented employees in a competitive labor market is
enhanced by nurturing our workplace culture, providing competitive compensation and benefits programs and supporting
employee career development and related management training. To that end, we continue to invest resources and energy
into being an employer of choice – attracting and engaging individuals who are innovative, curious, driven, diligent,
collaborative and of the highest integrity and ethics. Some of our key efforts in this area and management of our human
capital assets generally are described here.
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Compensation and Benefits
Our compensation philosophy is to provide pay and benefits that are competitive in the biotechnology and
pharmaceutical industry where we compete for talent. We monitor our compensation programs closely and review them
annually to provide what we consider a competitive mix of compensation and health, welfare and retirement benefits for all
our employees. Our compensation package for all employees includes market-competitive base salaries, eligibility for
annual performance bonuses or incentive compensation for field- based roles, and equity grants. Annual cash bonus
opportunity and equity compensation increase as a percentage of total compensation based on level of responsibility.
Actual bonus payouts for annual performance bonus are generally based on a combination of achievement of our annual
corporate goals and individual performance, with our CEO’s annual performance bonus being based entirely on the level of
achievement of our annual corporate goals. All regular-status, full-time employees are eligible to participate in our
comprehensive benefit program, pursuant to plan terms and conditions. Plan choices include medical, dental, vision, life
insurance, flexible spending accounts, short and long-term disability insurance, a 401(k) retirement savings plan with a
discretionary matching employer contribution, and an employee stock purchase plan. We also provide regular-status, full-
time employees with a generous time off program that includes vacation, sick, holiday, and paid leave for certain life
events.
Every year, we undertake a detailed review of our compensation by position and level and make
adjustments necessary to ensure that we continue to provide competitive compensation. We publish pay ranges in all job
postings for jobs as required by various states’ pay disclosure requirements.
Corporate Culture
We pride ourselves on an open culture that respects co-workers, values employees’ health and well-being
and fosters professional development. We support employee growth and development in a variety of ways, including with
group training, individual mentoring and coaching, conference attendance and tuition reimbursement. Our management
conducts annual employee engagement surveys and reports to our board of directors on human capital management topics,
including corporate culture, employee development and retention, and compensation and benefits. Similarly, our board of
directors regularly provides input on important decisions relating to these matters, including with respect to employee
compensation and benefits, talent retention and development.
During 2025, we furthered the development of our hybrid workforce program that provides a variety of
virtual and in-person collaboration opportunities, such as leadership training and coaching resources. Since 2021, we have
utilized a peer-centric employee recognition program to empower employees to champion our workplace culture and
values, and promote direct praise to peers. In addition, we have implemented a reward program that enables managers to
recognize employees who have demonstrated exceptional performance.
Corporate Responsibility Efforts
Our commitment to corporate responsibility is integrated throughout our business and informed by our
values and ambition to change lives by changing the course of blood cancer. To support lower-risk MDS patients eligible
for RYTELO, we have a patient support program called REACH4RYTELO that can help patients navigate access and
coverage. Our corporate responsibility initiatives reflect our commitment to making a difference for blood cancer patients
and health care providers who care for them through RYTELO. Our corporate responsibility priorities also reflect our
commitment to fostering a strong culture for employees and governing with integrity to advance our mission and create
value for stockholders. We review our corporate responsibility practices and disclosures on an ongoing basis.
Communication and Engagement
We believe that part of what sets us apart from other companies is our culture and, in particular, our focus
on providing timely and transparent communications and creating a strong sense of belonging and inclusiveness. We
engage in periodic in-office and in-person meetings and interactions, as well as in-office and in-person training and
development opportunities, to encourage cross-functional team-building and collaboration, in conjunction with which
many of our teams engage in group lunches and dinners. We held a summer contest that encouraged our employees to
share summer travel experiences and special events, building rapport and strengthening employee relationships, and we
conduct organizational and team-specific holiday events to promote connectivity among our employees. We share
information and news with employees through quarterly all-hands meetings, monthly newsletters to employees, social
media posts on our intranet and outward facing social media sites, such as LinkedIn, and regular employee chats with our
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Chief Executive Officer and other members of senior management. We survey our employees each year to measure their
level of engagement at the Company. Our employee engagement scores have remained relatively steady over the past three
years. These surveys provide rich feedback each year that helps us to continue to grow our culture and make Geron a great
place to work.
Health, Wellness and Safety
We offer benefits that promote our employees’ whole health and wellness, including reimbursement for
certain wellness costs, external support from our employee assistance programs and mental wellness services, which covers
therapy and/or coaching for our employees and their dependents, including high school and college-aged children.
None of our employees is subject to a collective bargaining agreement or represented by a trade or labor
union. We consider our relations with our employees to be good.
Corporate and Available Information
Geron Corporation was incorporated in the State of Delaware on November 28, 1990. Geron UK Limited
was incorporated in the United Kingdom on September 29, 2021. Geron Netherlands B.V. was incorporated in the
Netherlands on February 17, 2023. Our principal executive offices are located at 919 E. Hillsdale Blvd., Suite 250, Foster
City, CA 94404, and our telephone number is 650-473-7700. Our website address is http://www.geron.com.
We file or furnish electronically with the U.S. Securities and Exchange Commission, or the SEC, annual
reports on Form 10-K, quarterly reports on Form 10-Q, current reports on Form 8-K and amendments to those reports filed
or furnished pursuant to Section 13(a) or 15(d) of the Securities Exchange Act of 1934, as amended, or the Exchange Act.
We make copies of these reports available free of charge through the “SEC Filings” tab on the “Investors &
Media” page of our website as soon as reasonably practicable after we file or furnish them with the SEC.
Information contained on or accessible through our website is not incorporated into, and does not form a
part of, this Report or any other report or document we file with the SEC, and any references to our website are intended to
be inactive textual references only.
ITEM 1A. RISK FACTORS
We operate in a dynamic and rapidly changing environment involving numerous risks and uncertainties
that may have a material adverse effect on our business, financial condition or results of operations. You should carefully
consider the risks and uncertainties described below, together with all of the other information included in this Report. 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.
RISKS RELATED TO THE COMMERCIALIZATION OF RYTELO
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.