Corporate Presentation March 2020
Forward-Looking Statements Except for the historical information contained herein, this presentation contains forward-looking statements made pursuant to t he “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i ) Geron’s plan to . complete enrollment for IMerge by the end of 2020; (ii) that Geron expects top-line results from IMerge by mid-year 2022; (iii) Geron’s plan to meet with the FDA in the second quarter of 2020 to discuss a potential regulatory approval path in MF and subsequently provide a decision by mid-year 2020 regarding potential late-stage development of imetelstat in MF; (iv) Geron’s plan to commence a proof of concept study in 2020 in higher risk MDS and AML; ( v) that in 2020 Geron expects to present: (a) more mature data from the Phase 2 IMerge clinical trial, including durability of transfusion independence and (b) new analyses of Phase 2 IMbark data that provide supporting the observed improvement in overall survival as indicator of the potential disease-modifying activity with imetelstat treatment in MF; (vi) that imetelstat may have disease-modifying activity; and (vii) other statements that are not historical facts, constitute forward looking statements. These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation, risks and uncertainties related to: (i) whether the Company overcomes all the clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing and regulatory challenges to enable complete enrollment of IMerge in 2020, the availability of top-line results from IMerge by mid-year 2022 and commencement of the proof of concept study; (ii) whether regulatory authorities permit the further development of imetelstat on a timely basis, or at all, without any clinical holds; (iii) whether imetelstat is demonstrated to be safe and efficacious in clinical trials; (iv) whether any future efficacy or safety results may cause the benefit-risk profile of imetelstat to become unacceptable; (v) whether the Company decides not to pursue late-stage development of imetelstat in MF or early stage development in higher risk MDS and AML; (vi) whether the MDS and MF data the Company plans to present at strengthens the rationale for the Company to complete IMerge or pursue a Phase 3 clinical trial in MF; (vii) whether imetelstat actually demonstrates disease-modifying activity in patients; (viii) that Geron may not be able to prepare for discussions with the FDA in the second quarter of 2020, or at all, and its decision regarding potential late-stage development of imetelstat in MF, if any, may be delayed beyond mid-2020; and (ix) whether imetelstat has adequate patent protection and freedom to operate. Additional information on the above risks and uncertainties and additional risks, uncertainties and factors that could cause actual results to differ materially from those in the forward-looking statements are contained in Geron’s periodic reports filed with the Securities and Exchange Commission under the heading “Risk Factors,” inc lud ing Geron’s annual report on Form 10-K for the year ended December 31, 2019. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may change. Except as required by law, Geron disclaims any obligation to update these forward- looking statements to reflect future information, events or circumstances. 2
Company Snapshot Imetelstat, a Novel Drug with a Unique Target • Geron proprietary drug targeting telomerase to inhibit uncontrolled progenitor cell proliferation in hematologic malignancies • Clinical and non-clinical evidence of potential disease-modifying activity • Development focused on hematologic myeloid malignancies with significant unmet medical need and market opportunity • Fast Track and Orphan Drug designations for both lower risk MDS and Int-2/High-risk MF • Issued patents plus patent term extensions expected to provide coverage in U.S. until 2033 Late-Stage Clinical Development • Plan to complete enrollment for Phase 3 IMerge clinical trial in lower risk myelodysplastic syndromes (MDS) by year-end 2020 and top-line results expected mid-year 2022 • Plan to discuss with FDA potential regulatory approval path for imetelstat in myelofibrosis (MF), including registration-enabling Phase 3 trial design, in second quarter and decide on potential late-stage development in MF by mid-year 2020 In-House Leadership and Expertise Establishes a Foundation for Potential Future Growth • Highly-experienced, multi-functional, in-house hematology-oncology and late-stage drug development team • Capable of advancing current imetelstat development as well as potentially pursuing additional indications and broader asset development opportunities Cash Resources • As of 12/31/19, $159.2M in cash and marketable securities 3
Hematologic Myeloid Malignancies Upregulation of telomerase drives malignant proliferation in these diseases Essential Thrombocythemia (ET) platelets (abnormal) Polycythemia Vera (PV) red blood cells (abnormal) malignant malignant malignant hematopoietic progenitor progenitor stem cells cell cell clones Myelofibrosis (MF) collagen & reticulin fibers (fibrosis) Telomerase Upregulated Myelodysplastic Syndromes (MDS) immature blood cells Acute Myeloid Leukemia (AML) white blood cells (abnormal) 4
Imetelstat A first-in-class telomerase inhibitor with disease-modifying potential apoptosis of malignant clones Imetelstat Mechanism of Action • Potent competitive inhibitor of malignant malignant hematopoietic progenitor telomerase activity stem cells cell • Disease-modifying potential: X ➢ Via apoptosis of malignant Telomerase Upregulated progenitor cell clones, which reduces dysfunctional cell production and enables recovery of normal blood recovery of normal cell production RBCs, WBCs, Imetelstat Platelets enabled inhibits telomerase activity RBCs, red blood cells; WBCs, white blood cells 5
Imetelstat A first-in-class telomerase inhibitor with disease-modifying potential Imetelstat Mechanism of Action Apoptosis of • Potent competitive inhibitor of telomerase activity malignant cells • Structure: Proprietary 13-mer thio-phosphoramidate (NPS) oligonucleotide, with covalently-bound lipid tail to increase cell permeability/tissue distribution Malignant Malignant progenitor • Disease-modifying potential: selective killing of malignant hematopoietic stem cells cell stem and progenitor cells enabling normal blood cell production X Imetelstat Patent/Market Exclusivity Telomerase Upregulation • Composition of matter patent coverage through 2024 in EU and 2025 in U.S. • Patent term extension includes potential five-years in EU, through 2029 and through 2030 in U.S. Imetelstat Recovery of normal inhibits telomerase • Methods of use patents until 2033 for MF in EU and until RBCs, WBCs, activity platelets enabled 2033 for both MDS and MF in U.S. • Expected market exclusivity extension related to orphan drug designation in EU for up to ten years and in U.S. for up to seven years RBCs, red blood cells; WBCs, white blood cells Confidential and Proprietary 6
Imetelstat A novel drug with extensive clinical experience • Structure: Proprietary 13-mer thio-phosphoramidate (NPS) oligonucleotide, with covalently-bound lipid tail to increase cell permeability/tissue distribution • Clinical experience: more than 600 patients treated in Phase 1 and 2 trials • Phase 3 clinical trial in lower risk MDS currently enrolling • Patent/Market exclusivity: ➢ C omposition of matter patent coverage through 2024 in EU and 2025 in U.S. ➢ Potential five-year patent term extension in EU through 2029 and through 2030 in U.S. ➢ Methods of use patents until 2033 for MF in EU and until 2033 for both MDS and MF in U.S. ➢ Expected market exclusivity extension related to orphan drug designation in EU for up to ten years and in U.S. for up to seven years 7
Myelodysplastic Syndromes
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