Geron Corporation · NASDAQ:GERN
Geron — Investor Presentation (January 2019)
34 slides from Geron's investor presentation, Jan 7, 2019
Slide 1: Corporate Presentation
Corporate a
Slide 2: Forward-Looking Statements
geron ooking Statements Except for statements of historical fact, the statements contained in this presentation are forward-looking statements made pursuant to the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. These include, without limitation, statements regarding
Slide 3: Company Overview: About Geron
y Overview About Geron * Focused on the development and commercialization of innovative therapeutics for hematologic myeloid malignancies + 100% global rights to imetelstat, a first-in-class telomerase inhibitor discovered and developed by Geron + Planning to enter Phase 3 by mid-year 2019 in lead i
Slide 4: Imetelstat Development Highlights
Sequencing ahead of hypomethylating agents (HMAs) rat Development Highlights Target Patient Population Int-2 or High-risk MF Relapsed/refractory to JAKi No new drug approved since 2006 Approved Products No approved drugs in relapsed/refractory 60,000 patients with MDS U.S. Market 13,000 patients wit
Slide 5: Imetelstat in Myeloid Malignancies
ESSENTIAL ‘XN THROMBOCYTHEMIA(ET) platelets Baerlocher etal, NEM 2015; 373920928 POLYCYTHEMIA VERA, (Pv) tedbood al ef? a MYELOFIBROSIS (MF) collagen and reticulin by fibers (fibrosis) Teller etal, NEM 2035; 373:508-919, Pe @ myevopyspiastic SYNDROMES (MDS) Immature blood cells . ‘Steensma eal, 2018
Slide 6: Myelodysplastic Syndromes
geron Myelodysplastic Syndromes _
Slide 7: Myelodysplastic Syndromes (MDS)
plastic Syndromes (MDS) ~ geron acteristics a diverse group of clonal hematologic malignancies + Comprised of numerous subtypes, including refractory -sideroblasts (RARS) and refractory cytopenia with multi-lineage dysplasia-RS (RCMD-RS) + RS+ MDS associated with lower risk of AML SHOU aEsTione) tra
Slide 8: Lower Risk MDS Patient Population in the U.S.
ent Population in the U.S. | ron sa large unmet need 60,000 MDS patients in the U.S. 16,000 Cases diagnosed annually in the U.S. ‘Cogle, Curr Hematol Malig Rep; 2015, 10272-10281 Greenberg etal, Blood 1997; 89:2079-2088, Geron Proprietary Market Research 8 ~10% () of MDS patients have Lower Risk
Slide 9: Treatment Landscape for Lower Risk MDS
Erythropoiesis Stimulating Agents (ESAs) Patients refractory to + Improvement in anemia in ~50% of > ESAs become patients dependent on * Median treatment red blood cell duration: ~2 years transfusions Fenaux and Adés, Blood 2013; 121:4280-4286 Santini etal, J Clin Oncol 2016; 34:2988-2996 Tobiasson
Slide 10: Treatment Landscape for Lower Risk MDS
2nt Landscape for Lower Risk metelstat ahead of available therapies Erythropoiesis Stimulating Agents (ESAs) Lenalidomide Not approved in U.S. or Europe for Patients non-del(5q) patients refractory to + 28-week RBC-TI: 27% + Improvement in anemia in“50% of > ESAs become patients dependent on * Media
Slide 11: IMerge: 2-Part Phase 2/3 Trial
|Merge: 2-Part Phase 2/3 Trial Part 1 — Phase 2 portion Merge Part 1: Phase 2 Portion single arm, open label Transfusion Y Efficacy: Dependent, Low Red Blood or Intermediate-1 Imetelstat 7.5mg/kg 2 Efficacy: every 4 RBC-TI224 weeks; weeks to and duration of HMA smatologic improvement (HI); reduction
Slide 12: IMerge Part 1 Baseline Characteristics
Median age (range), years 715 (46-83) Male, n (%) 25 (66) ECOG PS 0-1, n (%) 34 (89) IPSS risk, n (9%) Low 24 (63) Intermediate-1 14 (37) Baseline median (range) RBC transfusion burden, units/8 weeks 8 (4-14) WHO 2001 category, n (%) RARS or RCMD-RS. 27(71) All others 11 (23) Prior ESA use, n (%) 34
Slide 13: IMerge Part 1 Efficacy Results Presented at ASH
Part 1 results presented at ASH Key Efficacy Outcomes 8 Rate of 8-week RBC-TI, n (%) 14 (37) Rate of 24-week RBC-TI, n (%) 10 (26) Median time to onset of RBC-TI (range), weeks 8.1 (0.1-33.1) Median duration of RBC-TI (range), weeks NE (17.0-NE) Rate of transfusion reduction (HI-E), n (%) 27 (71) Me
Slide 14: IMerge Part 1 Safety: Treatment Emergent Adverse Events
ee Aeron Most Common Treatmer mergent Patients > 1 TEAE Recovered in Occurrence and All events* & PURSE OSIGUBI Ma Grade 1-2 _Grade>3 Reversiality ofCytonentast manasa noe oce beeen Neutropenia 1 21 with an event Thrombocytopenia 2 23 Neutrophils, n (%) ‘Anemia 2 7 Grade 3 10 (26) 8 (80) Leukopenia
Slide 15: Lower Risk MDS Trials Comparison
‘isk MDS Trials different disease burdened patient populati IMerge Part Phase 2 MEDALIST — Phase 3 Imetelstat Luspatercept Placebo 38 No. of patients 153 76 Non-del(5q), lenalidomide/HMA naive ‘Target patient population Non-del(5q), lenalidomide/HMA naive Rovand RS: itheelderobiee (RS) Rerenly 0m Me
Slide 16: Planned Phase 3 Trial in MDS
Planned Phase 3 Trial in MDS Data from Part 1 for target patient population support moving forward with Part 2 Patient screening and enrollment planned to begin mid-year 2019* Current Clinical Trial Design for IMerge Part 2: Phase 3 Portion Imetelstat (n=~115) Red Blood Celll (RBC) Transfusion Indep
Slide 17: Myelofibrosis
geron Myelofibrosis 4
Slide 18: Myelofibrosis (MF)
‘osis (MF) acteristics + Malignant clonal proliferation and atypical megakaryocytic hyperplasia leads to bone marrow fibrosis and impaired hematopoiesis ~ Fibrosis thought to be induced by inflammatory cytokines produced by megakaryocytes originating from the malignant progenitor cell clone ~ Consti
Slide 19: MF Patient Population in the U.S.
Population in the U.S. geron | drug for patients relapsed/refractory to ruxoli 13,000 MF patients in the U.S. 3,000 Cases diagnosed annually in the U.S. ‘Mehta et al, Leuk Lymphoma 2014; 55:595-600 Gangat et al, JCO 2011; 29:392-397 Geron Proprietary Market Research 19 ~10% () of patients have Int-
Slide 20: Treatment Landscape for Int-2/High-Risk MF
Landscape for Int-2/High-Ris' ‘meaningful survival in poor-prognosis pi Ruxolitinib + Primarily for symptoms or splenomegaly 0 * Oral JAK1/JAK2 inhibitor > 7 5 % * Only approved product 5-year ruxolitinib for MF in U.S./Europe + Stay on drug as long as tolerated * Conventional drugs viewed as ineffe
Slide 21: IMbark Phase 2 Trial
IMbark Phase 2 Trial Rigorously defined relapsed/refractory MF patients teases) Trial Population: * Patients with Intermediate-2 or High-risk MF IM Cliniateals. gov + Relapsed or refractory to JAKi defined as documented progressive disease during or after JAKi: © Patients must have worsening of sple
Slide 22: Intent-to-treat patient population
‘-to-treat patient population Selected Patient Baseline Characteristics (N= 107) Median age (range), years 68.5 (44-84) 67 (31-86) 68.0 (31-86) ‘Myelofibrosis subtype, n (%) Primary 27 (56) 36 (61) 63 (59) Post-ET 9 (19) 10 (17) 19 (18) Post-PV 12.25) 13 (22) 25 (23) DIPSS risk status, n (%) Interme
Slide 23: IMbark Results Presented at ASH: Primary Endpoints
-k Results Presented at ASH data — primary endpoints Spleen Response Per IRC at Week 24 Symptom Response Based on TSS at Week 24 Tan go ERT ‘Treen re METELSTA MGS OE) Te eR SRE an “rer eo: METELTAT 84 A 88) + rong ate 4 + cng suo 4.7 mg/kg 9.4 mg/kg 4.7 mg/kg 9.4. mg/kg. Ce) (n=59) (n=48) (n=59) Spl
Slide 24: IMbark Results Presented at ASH: Overall Survival
Results Presented at ASH meaningful improvement in survival 100: 90: , @ Clinical cut-off (10/22/2018): 23 + Median follow-up: 27.4 (0.2-33.0) mos i 2 Median survival: é 30. + 19.9 months (95% Cl, 17.1, NE) in 4.7 mg/kg 2 a it bs Imeteistat 4.7 mglkg 29.9 months (95% Cl, 22.8, NE) in 9.4 mg/kg 0 nan
Slide 25: IMbark Results: Most Frequent Adverse Events
me/kg (n = 48) < Results Presented at ASH lata — most frequent adverse events 4 mg/kg ( 9) All Grades Grade 23 All Grades Grade Thrombocytopenia 11 (23) 11 (23) 29 (49) 24 (41) ‘Anemia 15 (31) 15 (31) 26 (44) 23 (39) Neutropenia 5 (10) 5 (10) 21 (36) 19 (32) Leukopenia 3 (6) 36) 8 (14) 8 (14) Non-he
Slide 26: IMbark Results: Incidence and Reversibility of Cytopenias
rk Results Presented at ASH snce and reversibility of cytopenias Most grade 3/4 cytopenias were reversible within 4 weeks Clinical consequences of cytopenias: Grade 3 febrile neutropenia Grade 2 3 hemorrhagic events Grade 2 3 infections 10 (21) Grade 3/4 LFT elevations were observed in 7 patients on
Slide 27: Clinical activity demonstrated in 9.4 mg/kg dosing arm
Clinical activity demonstrated in 9.4 mg/kg dosing arm * No formal clinical trial conducted previously for patients R/R to JAKi * Potential improvement in survival observed + Median OS for previously treated JAKi patients reported to be 12-14 mos (Kuykendall Ann Hematol 2018; Newberry Blood 2017) Sa
Slide 28: Milestones
geron Milestones 4
Slide 29: Data Presentations and Clinical Development Plans
Data Presentations Clinical Development Plans IMerge Phase 2 oral presentation at ASH December 2, 2018 IMbark Phase 2 oral presentation at ASH December 3, 2018 Analyst and investor event December 10, 2018 Discussions with MF experts and regulatory authorities to determine next steps Initiate first q
Slide 30: Thank you
Slide 31: Addendum
geron Addendum 4
Slide 32: Telomerase enzyme
Telomerase enzyme Comprised of an RNA template component (hTR) and a reverse transcriptase catalytic protein subunit (hTERT) In 2009, three Geron collaborators won the Nobel Prize for Medicine for discovery of telomerase and its relationship with telomeres telomeric DNA catalytic subunit (hTERT) Mal
Slide 33: Imetelstat
geron * Target: malignant progenitor cell clonal VN proliferation NPS oligonucleotide * Structure: 13-mer thio-phosphoramidate (NPS) oligonucleotide complementary to hTR, with covalently-bound lipid tail to increase cell permeability/tissue distribution Imetelstat binds é . ‘ eee to RNA template Lon
Slide 34: IMerge Part 1 Efficacy Results from ASH Presentation
_ IMerge Part 1 Efficacy Results from ASH Presentation 3 Pree | gg 4 g q Rate of 8-week Ti, n (%) 14(37) Among the patients achieving durable TI, all 2 S10 4 Rate of 24-week TI, n (%) 10 (26) showed a Hb rise of 2 3.0 g/dl compared to ea Median time to onset of T (range), weeks | 8.1 (0.1-33.1) ‘bas