Geron Corporation · NASDAQ:GERN
Geron — Investor Presentation (January 2025)
35 slides from Geron's investor presentation, Jan 13, 2025
Slide 1: Our mission is to change lives by changing the course of blood cancer
Our mission is to change lives by changing the course of blood cancer Corporate Presentation January 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: 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 with blockbuster potential for eligible U.S. patients with lower-risk myelodysplastic syndromes (LR-MDS)* *RYTELO (imetelstat) is approved by FDA for adults with low- to intermediat
Slide 4: Geron's Value Drivers
Geron’s Value Drivers LR-MDS represents a potential blockbuster market opportunity for RYTELO based on high unmet need and significant product differentiation U.S. RYTELO commercial launch off to a very strong start: • $28.2M Q3 2024 net revenues • $45M-$46M Q4 2024 estimated net revenues* Phase 3 t
Slide 5: 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 increased in Imetelstat binds to telomerase, and recovery of effective malignant cells inhibiting its activity hematopoi
Slide 6: Strong U.S. Launch in LR-MDS with Significant Commercial Opportunity
Strong U.S. Launch in LR-MDS with Significant Commercial Opportunity 6
Slide 7: Favorable U.S. Label and NCCN Guidelines Position RYTELO to Compete
Favorable U.S. Label and NCCN Guidelines Position RYTELO to Compete for Significant Market Segments in LR-MDS Market Segments Consistent With the FDA Label* MDS NCCN Guidelines^ Category 2A treatment 1st line ESA Ineligible (~20%) for 1st line ESA-ineligible RS+/RS- patients Category 1 treatment in
Slide 8: Large U.S. Market Opportunity with Blockbuster Potential for RYTELO in LR-MDS
Large U.S. Market Opportunity with Blockbuster Potential for RYTELO in LR-MDS 2025 U.S. RYTELO Treatment-Eligible Population Consistent with U.S. Label ∼ 16,800 ~15,400 ∼45% Treatment-eligible 1L patients expected to progress to 2L LR-MDS patients treatment in 2025 consistent with FDA ESA Eligible:
Slide 9: RYTELO U.S. Launch Off to a Strong Start
RYTELO U.S. Launch Off to a Strong Start Strong Early Launch Breadth of Prescriptions Broad Payor Coverage Trajectory $28.2M Q3 2024 ~590 ~80% net revenue ordering centers payor coverage from launch through Q4 2024, as of the end of Q4 2024^ $45-$46M Q4 2024 representing ~70% of our key targeted acc
Slide 10: Compelling Opportunity in JAKi R/R Myelofibrosis
Compelling Opportunity in JAKi R/R Myelofibrosis 10
Slide 11: Phase 3 Trial in JAKi R/R MF Would Potentially Double the RYTELO Commercial Opportunity
Phase 3 Trial in JAKi R/R MF Would Potentially Double the RYTELO Commercial Opportunity, if Positive ~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 Potential to treat ~
Slide 12: Median OS in Phase 2 IMbark Compares Favorably to Historical Controls
ClinicalTrials.gov Identifier: NCT02426086 Median OS in Phase 2 IMbark Compares Median OS More than Double Favorably Historical Controls (11-16 months) Compared to BAT in RWD Study RWD BAT vs. Imetelstat 9.4 mg/kg 29.9 months median OS Survival Probability BAT Moffitt Imetelstat 9.4 mg/kg Survival P
Slide 13: Phase 3 IMpactMF Trial Designed to Confirm Strong OS Signal Observed in Phase 2
Phase 3 IMpactMF Trial Designed to Confirm Strong ClinicalTrials.gov Identifier: NCT04576156 OS Signal Observed in Phase 2 Study 75% Enrolled as of Interim Analysis expected early 2026* December 2024 Final Analysis expected early 2027* Imetelstat 9.4mg/kg IV# every 3 weeks (n ~214) Primary Endpoint:
Slide 14: Development Pipeline
Development Pipeline == )geron’ “
Slide 15: 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 approval expected 1H Single Agent 2025 Approved in the U.S.* R/R MF Interim Analysis est. early 2026^ Single Agent IMpactMF Final Ana
Slide 16: Positioned for Profitability Without Additional Financing
Positioned for Profitability Without Additional Financing 16
Slide 17: Financial Overview
Financial Overview Net Revenue Cash Balance Operating Expenses $28.2M ~$500M $250M to $260M Q3 2024 net Cash and marketable 2024 expected product revenue securities as of 12/31/24* OpEx range* $45-46M $270M to $285M Q4 2024 estimated net 2025 expected product revenue* OpEx range Expect to Reach Prof
Slide 18: Thank you!
Thank you! Contact: Investor Relations investor@geron.com 18
Slide 19: Appendix
Appendix 19
Slide 20: Multiple Opportunities to Fuel Growth in 2025 & Beyond
Multiple Opportunities to Fuel Growth in 2025 & Beyond June 2024 Nov 2024 2026 2026 FDA approval & Secured up to $375M in Launch in select Primary analysis U.S. launch of non-equity financings EU countries in from Ph1 IMproveMF RYTELO in LR-MDS LR-MDS Q3 2024 Dec 2024 1H 2025 Early 2026 Early 2027 $
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/Mar
Slide 23: Favorable U.S. Prescribing Information for RYTELO Supports Significant Market Opportunity
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 Differentiate Imetelstat
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 Observed with Imetelstat
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 with Imetelstat
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: Improvement in Patient-Reported Fatigue Associated with Clinical Responses
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 Experienced Clinical Benefit
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 First-in-Class Treatment with a New Mechanism of Action in MF
Imetelstat Represents a First-in- Class Treatment with a New Mechanism of Action in MF 31
Slide 32: First MF Trial with OS Primary Endpoint Based on Significant Imetelstat Clinical Experience
First MF Trial with OS Primary Endpoint Based on Significant Imetelstat Clinical Experience 2015 2021 ongoing Single Institution Pilot Study Ph 2 Dose Finding Study Pivotal Ph 3 Trial Pilot Study in HR/Int-2 MF Phase 2 Dose Finding Phase 3 Study in JAKi (~50% of patients received prior JAKi therapy)
Slide 33: Part 1 Findings Suggest Tolerability of Imetelstat Combined with Ruxolitinib in Patients with MF
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 Observed in Imetelstat-Treated MF and MDS Patients
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