Slide 1: Enhancing the Lives of Patients with Hematologic Malignancies
Enhancing the Lives of Patients with Hematologic Malignancies Corporate Presentation January 2024
Slide 2: Forward-Looking Statements and Safe Harbor
Forward-Looking Statements and Safe Harbor Except for the historical information contained herein, this presentation contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements
Slide 3: Geron is On Target for a Successful Transition to Commercial Company in 2024
Geron is On Target for a Successful Transition to Commercial Company in 2024 PDUFA date of June 16, 2024 for imetelstat in transfusion-dependent (TD) LR-MDS* MAA review completion expected by end-2024, with potential EU approval and launch in 2025 Imetelstat Ph3 data showed unprecedented durability
Slide 4: Exploring the Broad Potential of Imetelstat and Telomerase Inhibition
Exploring the Broad Potential of Imetelstat and Telomerase Inhibition Indications Discovery Preclinical Phase 1 Phase 2 Phase 3 TD LR-MDS U.S. PDUFA: June 16, 2024 IMerge EU MAA under review Single Agent R/R MF Interim Analysis est.: 1H 2025 IMpactMF Final Analysis est.: 1H 2026 Single Agent Frontli
Slide 5: Imetelstat is a First-in-Class Telomerase Inhibitor
Imetelstat is a First-in-Class Telomerase Inhibitor Evidence for Based on Nobel-Prize Winning Science potential disease Potentially powerful mechanism for treating hematologic malignancies modification • Clinical efficacy: durable TI (TD LR- Imetelstat binds to Apoptosis of malignant MDS Ph3); Telom
Slide 6: Transfusion-Dependent Lower-Risk MDS
Transfusion-Dependent Lower-Risk MDS PDUFA date of June 16, 2024* *Imetelstat is currently under regulatory review in the U.S. and EU for the treatment of transfusion-dependent anemia in adult patients with low- to intermediate- 1 risk myelodysplastic syndromes (MDS) who have failed to respond or ha
Slide 7: Lower-Risk MDS Patient Experience
Lower-Risk MDS Patient Experience Diagnosis Median age Symptomatic Increasing ~70 years Anemia Transfusion Lengthy Dependence Office Visits High-Cost Burden Poor Quality of Life Symptomatic anemia and transfusion dependence are Higher Risk key drivers of patient burden and poor quality of life of Pr
Slide 8: Treatment Landscape for LR-MDS
Treatment Landscape for LR-MDS Continuing unmet need presents significant opportunity for imetelstat; ~$3.5B TAM in 2031 (U.S./EU)^ HR MDS Lower Risk MDS (~70%) Significant Complex potential Symptomatic Anemia (~90%) presentations opportunity ESAs and for imetelstat: sEPO (>500 sEPO (>500 luspaterce
Slide 9: Differentiated Imetelstat Profile in IMerge Phase 3
Differentiated Imetelstat Profile in IMerge Phase 3 Robust & Durable Response Additional Attributes 40% ≥ 8-wk RBC-TI response rate; 1-year median 3.6 g/dL median Hgb rise TI duration 50% of imetelstat-treated patients reported less fatigue (PRO data) 18% ≥ 1-yr RBC-TI response rate; 2.4 years media
Slide 10: Durable Transfusion Independence and Significant Response Rates Observed with Imetelstat
Durable Transfusion Independence and Significant Response Rates Observed with Imetelstat 52 weeks 50.0 median duration 45.0 80 weeks 39.8% median duration Percentage of patients (%) 40.0 35.0 123 weeks P<0.001 30.0 median duration 28.0% 25.0 20.0 17.8% P<0.001 15.0% 15.0 P=0.002 10.0 5.0 1.7% 3.3% 0
Slide 11: Significant Hemoglobin Rises and Reduction in Transfusions Observed with Imetelstat
Significant Hemoglobin Rises and Reduction in Transfusions Observed with Imetelstat 3.6 g/dL median Hgb rise ≥4U/8 wks transfusion reduction in 8wk RBC-TI responders in ~60% of imetelstat-treated patients Number of patients Number of patients The mean changes from the minimum Hgb of the values that
Slide 12: Consistent Responses Across MDS Subgroups (≥ 8-wk RBC-TI)
Consistent Responses Observed Across MDS Subgroups with Imetelstat (≥ 8-wk RBC-TI Responses) Imetelstat, Placebo, % Difference n/N (%) n/N (%) (95% CI) p-value Overall 47/118 (39⋅8) 9/60 (15⋅0) 24⋅8 (9⋅9–36⋅9) <0⋅001 WHO category^ RS+ 33/73 (45⋅2) 7/37 (18⋅9) 26⋅3 (5⋅9–42⋅2) 0⋅016 RS- 14/44 (31⋅8) 2
Slide 13: Consistent Responses Across MDS Subgroups (≥ 24-wk RBC-TI)
Consistent Responses Observed Across MDS Subgroups with Imetelstat (≥ 24-wk RBC-TI Responses) Imetelstat, Placebo, % Difference n/N (%) n/N (%) (95% CI) p-value Overall 33/118 (28⋅0) 2/60 (3⋅3) 24⋅6 (12⋅64–34⋅18) <0⋅001 WHO category^ RS+ 24/73 (32⋅9) 2/37 (5⋅4) 27⋅5 (10⋅0–40⋅37) 0⋅003 RS- 9/44 (20⋅5
Slide 14: Improvement in Patient-Reported Fatigue Associated with Clinical Responses with Imetelstat
Improvement in Patient-Reported Fatigue Associated with Clinical Responses with Imetelstat Significant patient-reported fatigue improvements Sustained meaningful improvement in fatigue in 8 and 24-wk RBC-TI responders reported in imetelstat-treated patients Platzbecker, Santini et al. The Lancet 202
Slide 15: Significant Reductions in Variant Allele Frequency of Genes Frequently Mutated in MDS
Significant Reductions in Variant Allele Frequency (VAF) of Genes Frequently Mutated in MDS with Imetelstat • Among patients with evaluable mutation data, the maximum 75 Imetelstat reductions in VAF of P=0.032 Placebo the SF3B1, TET2, DNMT3A and ASXL1 Maximum % Change in VAF in Blood 50 P=0.146 P<0.
Slide 16: The Most Common AEs were Manageable, Short-Lived Thrombocytopenia and Neutropenia
The Most Common AEs were Manageable, Short-Lived Thrombocytopenia and Neutropenia Consistent with prior clinical experience, the most Grade 3-4 cytopenias were generally of short duration common imetelstat AEs were hematologic and reversible • Grade 3-4 thrombocytopenia and neutropenia most often re
Slide 17: Grade 3-4 Cytopenias were Manageable with Low Incidence of Clinical Consequences
Grade 3-4 Cytopenias were Manageable with Low Incidence of Clinical Consequences Clinical consequences of grade 3–4 infection and bleeding Infections were not common in the setting of grade 3-4 were low and similar for imetelstat and placebo neutropenia and were similar for imetelstat and placebo Im
Slide 18: IMerge Phase 3 Data Received Favorably by Surveyed Practicing Hematologists
IMerge Phase 3 Data Received Favorably by Surveyed Practicing Hematologists Across U.S./EU Key Markets Key attributes of imetelstat resonated strongly with community and academic hematologists Totality of Meaningful Durability Clinical Benefit of Response EFFICACY Compelling TI rates across RS 16- a
Slide 19: Hematologists' Feedback Confirms Imetelstat Opportunity Across RS Subtypes
Hematologists’ Feedback Confirms Imetelstat Opportunity Across RS Subtypes and High Transfusion Burden Patients Imetelstat’s novel mechanism of action Physicians view imetelstat’s novel/first-in-class MOA together with its (MOA) is seen as a key driver for future durability of response as highly com
Slide 20: Imetelstat Expected to Become New Standard-of-Care in Second-Line TD LR-MDS
Imetelstat Expected to Become New Standard-of-Care in Second-Line TD LR-MDS, ~32k patients by 2031 (U.S./EU) Expected Second Expected Front Line Usage Line Usage • Imetelstat is expected to be used in the ESA-experienced luspatercept -experienced ESA-ineligible patients second-line regardless of fro
Slide 21: Imetelstat Expected to Become Important New Option in RS- Subgroup
Imetelstat Expected to Become Important New Option in RS- Subgroup, Comprising 75% of LR-MDS Patients Expected Second Expected Front Line Usage Line Usage • Imetelstat is expected to be used second line ESA-experienced luspatercept -experienced ESA-ineligible patients following ESAs in the majority
Slide 22: Well-Positioned for U.S. Launch Upon Potential Approval
Well-Positioned for U.S. Launch Upon Potential Approval Experienced commercial and medical affairs teams fully-onboarded; field reimbursement and sales onboarding expected Q1-Q2 2024 Commercial and medical affairs leadership hired Prepare Geron Sales force hiring on track for Q1-Q2 2024 Infras
Slide 23: Imetelstat in Relapsed/Refractory MF
Imetelstat in Relapsed/Refractory MF Evaluating potential improved survival 23
Slide 24: Expected MF Market Evolution and Imetelstat Opportunity
Expected MF Market Evolution and Imetelstat Opportunity Continuing unmet need in JAKi-treated patients presents significant opportunity for imetelstat; ~$3.5B TAM in 2031 (U.S./EU)^ We expect the future MF market to expand significantly with potential approvals of JAKi-based combination regimens and
Slide 25: First and Only Phase 3 Trial in MF with OS as Primary Endpoint
First and Only Phase 3 Trial in MF with OS as Primary Endpoint 50% enrolled as of Nov 2023 Actively enrolling global trial • Sites across United States, South America, Europe, ClincialTrials.gov Australia and Asia (NCT0457615) Planned analyses Primary Endpoint: • Interim Analysis expected in 1H 2025
Slide 26: Median OS in IMbark Phase 2 Compares Favorably to Historical Controls
Median OS in IMbark Phase 2 Compares Favorably to Historical Controls Imetelstat 9.4 mg/kg Improvement in overall survival (OS) observed for JAKi relapsed/refractory MF patients in IMbark Phase 2 29.9 mos • 14 – 16 mos median OS for historical controls for JAKi relapsed/refractory MF patients • 29.9
Slide 27: Median OS More than Double vs BAT Treatment in RWD Study
Median OS More than Double vs BAT Treatment in RWD Study Evaluating imetelstat vs BAT in IMbark Phase 2 data compared to real world data (RWD) from a closely-matched JAKi relapsed/refractory MF cohort of patients at the Moffitt Cancer Center who had discontinued ruxolitinib and were subsequently tre
Slide 28: Manageable Imetelstat Safety Results in IMbark Phase 2
Manageable Imetelstat Safety Results in IMbark Phase 2 Limited clinical consequences of 9.4 mg/kg (n=59) reversible, on target cytopenias n (%) All Grades Grade ≥ 3 • Thrombocytopenia and neutropenia Hematologic (≥10% in either arm)§ characterization: Thrombocytopenia 29 (49) 24 (41) ◦ Short time to
Slide 29: Financials & Summary
Financials & Summary 29
Slide 30: Financial Resources to Support Potential US Commercial Launch of Imetelstat
Financial Resources to Support Potential US Commercial Launch of Imetelstat Financial resources ~$48M ~$377M $200M Up to to $210M $210M expected to support Q3 2023 Est. cash and 2023 expected projected level of operating marketable range of GAAP expenses securities as operating operations into the o
Slide 31: Geron Positioned for Successful Imetelstat Launch, If Approved
Geron Positioned for Successful Imetelstat Launch, If Approved Imetelstat TD LR-MDS PDUFA Date of June 16, 2024*; MAA under review in same indication Underserved TD LR-MDS and R/R MF markets with potential combined TAM >$7B in 2031 (U.S./EU)^ Differentiated first-in-class investigational telomerase
Slide 32: Thank you!
Thank you! Contact: Investor Relations ir@geron.com 32
Slide 33: Appendix: TAM (U.S./EU)
Appendix: TAM (U.S./EU) Potential LR MDS Patient Population Potential R/R MF Patient Population ESA Ineligible (RS+/RS-): ~4,000 JAKi treated: ~29,000 ESA R/R RS+: ~8,000 ESA R/R RS-: ~24,000 Become part of SOC in LR-MDS Expand as SOC in R/R MF >$7B (~$3.5B TAM 2031) (~$3.5B TAM 2031) by 2031 LR-MDS