Slide 1: Corporate Presentation
Our mission is to change lives by changing the course of blood cancer Corporate Presentation August 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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, include, witho
Slide 3: Harout Semerjian, President and Chief Executive Officer
Harout Semerjian President and Chief Executive Officer Seasoned Commercial Leader in Hematology and Oncology INCOMING PRESIDENT AND CHIEF EXECUTIVE OFFICER Over 30 years of deep Proven success leading Leadership roles as CEO, experience in hematology U.S. and global commercial CCO and across biophar
Slide 4: A first-in-class telomerase inhibitor with a unique mechanism of action
A first-in-class telomerase inhibitor with a unique mechanism of action representing a highly differentiated treatment for eligible U.S. patients with lower-risk myelodysplastic syndromes (LR-MDS)* *RYTELO (imetelstat) is approved by FDA for adults with low- to intermediate-1 risk MDS with transfusi
Slide 5: Focused on generating long-term U.S. RYTELO commercial success
U.S. RYTELO commercial success Focused on is #1 corporate priority generating long-term value EU commercialization plans MF interim data analysis anticipated 2H 2026* * Based on expectations about event rates (deaths) and enrollment, which can change over time and may differ from our current expecta
Slide 6: Telomerase Inhibition Represents a Novel MOA with Unique Benefits
Telomerase Inhibition Represents a Novel MOA with Unique Benefits Based on Nobel Prize-winning science, imetelstat is wholly owned by Geron Apoptosis of malignant cells Telomerase is increased in Imetelstat binds to telomerase, and recovery of effective malignant cells inhibiting its activity hemato
Slide 7: Significant Commercial Opportunity in LR-MDS
Significant Commercial Opportunity in LR-MDS 7
Slide 8: RYTELO LR-MDS U.S. Market Opportunity with Significant Potential
RYTELO LR-MDS U.S. Market Opportunity with Significant Potential Estimated 2025 U.S. RYTELO Total Addressable LR-MDS Patient Population ∼ 16,800 ∼45% 1L patients expected to progress to 2L treatment in 2025 ESA Eligible: ~13,400 ~15,400 ∼59% ∼ 7,600 2L patients expected U.S. RYTELO total to progress
Slide 9: Accounts Treating Eligible LR-MDS Patients Identified
Accounts Treating Eligible LR-MDS Patients Identified Refined Commercial Targeting to Reach Accounts Treating the Majority of Diagnosed Patients >10,000 HCPs treat diagnosed MDS patients in the U.S. ~6,300 HCPs treat ~80% of the diagnosed MDS patients ~1,300 HCPs treat ~50% of the diagnosed MDS pati
Slide 10: Commercial Strategy and Plan of Action
Commercial Strategy and Plan of Action #1 Increase HCP RYTELO #2 Improve HCP Prescribing #3 Generate KOL Brand Awareness Confidence & Clarity Support & Advocacy Refined prescriber targeting Social & digital brand campaigns New regional marketing roles Increased field sales headcount Region
Slide 11: Compelling Opportunity in JAKi R/R Myelofibrosis
Compelling Opportunity in JAKi R/R Myelofibrosis 11
Slide 12: ~10,000 U.S. JAKi R/R MF patients
Phase 3 Trial in JAKi R/R MF Could Potentially Double the RYTELO Commercial Opportunity, if Successful and Approved ~12,000 U.S. JAKi naïve / well-controlled MF patients (currently only 3 approved treatments – all JAK inhibitors) 75% of those JAKi treated patients fail or discontinue treatment Poten
Slide 13: Median OS in Phase 2 IMbark Compared to Historical Controls
ClinicalTrials.gov Identifier: NCT02426086 Median OS in Phase 2 IMbark Compares Median OS More than Double Favorably to Historical Controls (11-16 months) Compared to BAT in RWD Study RWD BAT vs. Imetelstat 9.4 mg/kg 29.9 months median OS 29.9 mos Survival Probability BAT Moffitt Imetelstat 9.4 mg/k
Slide 14: Phase 3 IMpactMF Trial Designed to Confirm Strong OS Signal
Phase 3 IMpactMF Trial Designed to Confirm Strong ClinicalTrials.gov Identifier: NCT04576156 OS Signal Observed in Phase 2 Study Full enrollment expected by YE 2025 >95% Enrolled at the end of July 2025 Interim Analysis expected 2H 2026* Final Analysis expected 2H 2028* Imetelstat 9.4mg/kg IV# every
Slide 15: Development Pipeline
Development Pipeline —————————
Slide 16: Exploring the Potential of Telomerase Inhibition Across Multiple Hematologic Malignancies
Exploring the Potential of Telomerase Inhibition Across Multiple Hematologic Malignancies Indications Discovery Preclinical Phase 1 Phase 2 Phase 3 Approved LR-MDS EU commercialization Single Agent expected 2026** Approved in the U.S. and the EU* R/R MF Interim Analysis est. 2H 2026^ IMpactMF Single
Slide 17: Financial Overview
Financial Overview 17
Slide 18: Financial Overview
Financial Overview Operating Expenses Cash Balance Net Revenue $61.5M Q2 2025 total OpEx $432.6M $49.0M Cash and marketable Q2 2025 net securities as of 6/30/25 product revenue $270M to $285M 2025 expected OpEx range 18
Slide 19: Appendix
_ Appendix ——————
Slide 20: Multiple Opportunities to Support Long-Term Growth
Multiple Opportunities to Support Long-Term Growth 2026 2026 June 2024 Nov 2024 Q4 2024 Initial Results from Launch in select FDA approval & Secured up to $375M in $47.5M product EU countries in Part 2 of Ph1 U.S. launch of non-equity financings revenue LR-MDS IMproveMF RYTELO in LR-MDS Dec 2024 Mar
Slide 21: LR-MDS Represents a Significant Commercial Opportunity for RYTELO
LR-MDS Represents a Significant Commercial Opportunity for RYTELO Phase 3 IMerge data, FDA label and NCCN Guidelines position RYTELO as highly differentiated treatment 21
Slide 22: Strong IP Position for RYTELO Supports Commercial Opportunity
Strong IP Position for RYTELO Supports Commercial Opportunity Exclusivity for LR-MDS Expected into 2037 in the U.S. and 2038 in the EU Expected Regulatory Exclusivities and Patent Terms Dec 2025 Dec 2030 Mar 2033 ~Aug 2037 • PTE Applications filed in U.S. for RYTELO patents Jun Jun 2024 U.S. Data/Ma
Slide 23: Favorable U.S. Prescribing Information for RYTELO
Favorable U.S. Prescribing Information for RYTELO Supports Significant Market Opportunity No boxed warning No REMS program No contraindications Indication and Usage Recommended Dosage RYTELO is indicated for the treatment of adult patients with 7.1 mg/kg* administered as an intravenous low- to inter
Slide 24: NCCN Guidelines Guide Clinical, Formulary and Treatment Pathway Decision-Making
NCCN Guidelines®# Guide Clinical, Formulary and Treatment Pathway Decision-Making and Position Imetelstat as 2nd Line Therapy of Choice MDS NCCN Guidelines include imetelstat for use in both RS+ and RS- 1st-line ESA ineligible patients and in both RS+ and RS- 2nd-line patients, regardless of prior 1
Slide 25: Durable Red Blood Cell Transfusion Independence and Response Rates
Durable Red Blood Cell Transfusion Independence and Response Rates Differentiate Imetelstat 52 weeks 50.0 median duration 45.0 80 weeks 39.8% median duration Percentage of Patients (%) 40.0 35.0 P<0.001 123 weeks 30.0 28.0% median duration 25.0 20.0 P<0.001 17.8% 15.0% 15.0 Nominal P=0.002 10.0 5.0
Slide 26: Meaningful Hemoglobin Rises and Reduction in Transfusions
Meaningful Hemoglobin Rises and Reduction in Transfusions Observed with Imetelstat ≥4U/8 weeks transfusion reduction 3.6 g/dL median Hgb rise in 8-week RBC-TI responders in ~60% of imetelstat-treated patients 5 1 Nominal P<0.001 Nominal P=0.042 Mean Change in RBC Transfusion 4 0 Mean Change in HGB 3
Slide 27: Consistent Responses Observed Across MDS Subgroups
Consistent Responses Observed Across MDS Subgroups with Imetelstat (≥ 8-week RBC-TI Responses) Imetelstat, Placebo, % Difference n/N (%) n/N (%) (95% CI) Overall 47/118 (39⋅8) 9/60 (15⋅0) 24⋅8 (9⋅9–36⋅9) WHO category^ RS+ 33/73 (45⋅2) 7/37 (18⋅9) 26⋅3 (5⋅9–42⋅2) RS- 14/44 (31⋅8) 2/23 (8⋅7) 23⋅1 (-1⋅
Slide 28: Sustained Improvement in FACIT-Fatigue
Improvement in Patient-Reported Fatigue Associated with Clinical Responses with Imetelstat Per Exploratory Analysis Meaningful patient-reported fatigue improvements Sustained meaningful improvement in fatigue in 8 and 24-week RBC-TI responders reported in imetelstat-treated patients Imetelstat Respo
Slide 29: ESA R/R/Ineligible LR-MDS Patients With Prior Luspatercept Treatment
ESA R/R/Ineligible LR-MDS Patients With Prior Luspatercept Treatment Experienced Clinical Benefit from Imetelstat Treatment Clinical activity of imetelstat was evident across lines of prior therapy (including luspatercept, lenalidomide and HMA) regardless of prior response status, suggesting that im
Slide 30: Well-Characterized Safety Profile with Manageable Thrombocytopenia and Neutropenia
Well-Characterized Safety Profile with Generally Manageable and Short-Lived Thrombocytopenia and Neutropenia These are familiar adverse reactions for hematologists who are experienced with managing cytopenias Consistent with prior clinical experience, the most AEs were generally manageable with supp
Slide 31: Imetelstat Represents a Potential First-in-Class Treatment in MF
Imetelstat Represents a Potential First-in-Class Treatment with a New Mechanism of Action in MF 31
Slide 32: First Myelofibrosis Trial with Overall Survival Primary Endpoint
First Myelofibrosis Trial with Overall Survival Primary Endpoint Based on Significant Imetelstat Clinical Experience 2015 2021 ONGOING Single Institution Pilot Study Ph 2 Dose Finding Study Pivotal Ph 3 Trial Median OS in 9.4 mg/kg arm more than First and only Phase 3 trial in MF with Basis for Ph2
Slide 33: Part 1 Findings Suggest Tolerability of Imetelstat Combined with Ruxolitinib
Part 1 Findings Suggest Tolerability of Imetelstat ClinicalTrials.gov Identifier: NCT05371964 Combined with Ruxolitinib in Patients with MF Actively enrolling patients for dose confirmation with imetelstat 9.4mg/kg PART 2: Dose Confirmation & PART 1: Dose Finding Results Presented at ASH 2024 Expans
Slide 34: Clinical Benefits Observed in LR-MDS and MF with Unique Mechanism of Action
Clinical Benefits Observed in LR-MDS and MF with Unique Mechanism of Action 34
Slide 35: Association of Clinical Benefit and Reduction of Disease Markers
Association of Clinical Benefit and Reduction of Disease Markers Observed in Imetelstat-Treated MF and MDS Patients A B C D A. Disappearance of bone marrow fibrosis in imetelstat treated HR MF patient B. Association of survival improvement and reduction in VAF for HR R/R imetelstat treated MF patien