Needham Virtual Healthcare Conference John Scarlett, M.D. Chairman and Chief Executive Officer, Geron Corporation April 15, 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, tho se regarding: (i) Geron’s scheduled meeting with the FDA in the second quarter of 2020 to discuss a potential regulatory approval path in MF, including a Phase 3 trial design, and plan to make a decision by mid-year 2020 regarding any potential late-stage development of imetelstat in MF; (ii) 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 supporting observed potential improvement in overall survival as an indicator of disease-modifying activity; (iii) that imetelstat may have disease-modifying potential; (iv) that for imetelstat Geron has issued patents plus patent term extensions expected to provide coverage in the U.S. until 2033; (v) that Geron’s 2020 operating expenses wil l be lower than previously announced; (vi) that the U.S. revenue potential for imetelstat in lower risk MDS could exceed $500 million; 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 delays caused by COVID-19 and overcomes the clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing and regulatory challenges to enable complete enrollment of the IMerge Phase 3 trial, if at all; (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; (vi) whether the MDS and MF data that the Company expects to present strengthens or supports 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; (ix) that Geron may not successfully market or that competition could cause imetelstat not to achieve at least $500 million, or greater than $500 million, in U.S. revenue for MDS; and (x) 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,” including 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
Business Update Related to COVID-19 Operating Remotely • Implemented work-from-home policy in alignment with public health directives to slow the spread of COVID-19 • Employees working from home effectively using remote working tools Clinical Development • Patient treatment and follow-up continuing for IMerge Phase 2 clinical trial • Enrollment and patient treatment in IMerge Phase 3 clinical trial ongoing • Sufficient drug supply for clinical trial needs • Based on COVID-19 challenges, Geron no longer expects to: – Complete enrollment in the IMerge Phase 3 trial by the end of 2020 – Commence a proof of concept study in high risk MDS and acute myeloid leukemia (AML) by the end of 2020 • Communicating frequently with sites and investigators regarding patient and trial status Managing 2020 Operating Expenses • Implemented measures to manage cash burn – Suspended travel and in-person meetings – Re-evaluating near-term hiring plan • Clinical development expenses expected to be lower due to impact of COVID-19 on clinical activities • Expect overall company operating expenses will be lower than previously disclosed guidance Next business update related to COVID-19 to be provided on Q1 2020 quarterly conference call 3
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 lower risk myelodysplastic syndromes (MDS) and Int-2/High-risk myelofibrosis (MF) • Issued patents plus patent term extensions expected to provide coverage in U.S. until 2033 Late-Stage Clinical Development • Phase 3 IMerge clinical trial in lower risk MDS was initiated in October 2019. As of the end of March 2020, 66% of total sites were opened for enrollment. • Scheduled meeting with FDA in second quarter to discuss potential regulatory approval path for imetelstat in MF, including registration- enabling Phase 3 trial design. Geron’s decision on any potential late -stage development in MF planned by mid-year 2020. In-House Leadership and Expertise Establishes a Foundation for Potential Future Growth • Highly-experienced, multi-functional, in-house hematology-oncology drug development team • Deep expertise supports current and future development plans in hematologic malignancies Cash Resources • As of 12/31/19, $159.2M in cash and marketable securities 4
Hematologic Myeloid Malignancies Upregulated telomerase is a key feature of these malignancies malignant stem and progenitor cells Telomerase and Heme Malignancies malignant malignant • Telomerase is an enzyme present in most hematopoietic progenitor cells allowing for normal and controlled stem cell cells cell turnover • Telomerase is continually upregulated in malignant stem and progenitor cells, resulting in uncontrolled proliferation associated with disease progression in Telomerase Upregulated hematologic myeloid malignancies , such as MDS and MF proliferation of abnormal RBCs, red blood cells; WBCs, white blood cells RBCs, WBCs or Platelets drive clinical manifestations of various hematologic myeloid malignancies 5
Imetelstat – A First-In-Class Telomerase Inhibitor Targeting telomerase to inhibit malignant cells driving hematologic myeloid malignancies Imetelstat induces apoptosis of malignant stem and progenitor cells Imetelstat Disease-Modifying Potential malignant malignant hematopoietic progenitor • Imetelstat inhibits telomerase and stem cell cells thereby: ➢ Induces apoptosis (death) of malignant stem and progenitor cells X ➢ Enables potential recovery of Telomerase Upregulated normal RBC, WBC and platelet counts and function Imetelstat Enables potential RBCs, red blood cells; WBCs, white blood cells recovery of normal inhibits telomerase RBCs, WBCs, Platelets activity 6
Imetelstat A novel telomerase inhibitor discovered and developed by Geron • Structure: Proprietary 13-mer thio-phosphoramidate (NPS) oligonucleotide, with covalently-bound lipid tail to increase cell permeability/tissue distribution • Potent competitive inhibitor of telomerase activity • 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: ➢ 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. ➢ 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
Hematologic Myeloid Malignancies Potential applications of telomerase inhibition across multiple indications Evidence of clinical activity in multiple Essential Thrombocythemia (ET) myeloid malignancies after treatment with imetelstat Excess platelets with abnormal functionality Polycythemia Vera (PV) Excess red blood cells with abnormal functionality malignant malignant hematopoietic progenitor stem cell cells Myelofibrosis (MF) Fibrosis of marrow and disruption of blood production Myelodysplastic Syndromes (MDS) Telomerase Upregulated Abnormal production and maturation of blood cells Acute Myeloid Leukemia (AML) Abnormal production and maturation of blood cells; High proportion of immature white blood cells (blasts) 8
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