Geron Corporation · NASDAQ:GERN
Geron — 32nd Annual J.P. Morgan Healthcare Conference 2014
31 slides from Geron's conference presentation, Jan 13, 2014
Slide 1: 32nd Annual J.P. Morgan Healthcare Conference
32"4 Annual J.P. Morgan Healthcare Conference jeron John Scarlett, M.D, Chief Executive Officer January
Slide 2: Forward-looking statements
geron forward-looking statements Except for statements of historical fact, this presentation contains forward-looking statements made pursuant tothe “safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including without limitation, statements regarding: anticipated timel
Slide 3: Advancing clinical development of imetelstat in hematologic myeloid malignancies
geron advancing clinical development of imetelstat in hematologic myeloid malignancies © Imetelstat: differentiated and proprietary oncology product ~ first telomerase inhibitor in clinical development ~ discovered and developed by Geron; IP through 2025 with patent extension expected beyond ~ may s
Slide 4: Imetelstat: a telomerase inhibitor
geron imetelstat a telomerase inhibitor Telomerase enzyme: © Adds nucleotide repeats to offset the loss of telomeric DNA occurring with each replication cycle © Not active in somatic cells; transiently upregulated in normal hematopoietic progenitor cells to maintain telomeres and support controlled
Slide 5: Hematologic myeloid malignancies
geron hematologic myeloid malignancies _arise from malignant progenitors in the bone marrow > essential thrombocythemia platelets (ET) (abnormal BRS — soircynemiaveraov) malignant ted blood cells re L (abnormal) progenitor OC PR myelofibrosis (MF) SE thane reticulin fibers malignant (fibrosis) clone
Slide 6: Essential thrombocythemia
geron essential thrombocythemia ‘S_ —> essential thrombocythemia platelets (eT) (abnormal) malignant myeloid x& —> polycythemia vera (Pv) progenitor acute myelogenous leukemia (amu) malignant clone myelodysplastic syndromes (ms) [earnest Cowes ncn Gan towtoow proof-of-concept for selective effect on
Slide 7: Broader imetelstat opportunities
geron broader imetelstat opportunities —> essential thrombocythemia (er) malignant myeloid progenitor ~ —> myelofibrosis (Me) j2® malignant © \ reticulin fibers clone Y» (fibrosis) acute myelogenous leukemia (aml) myelodysplastic syndromes (mps)
Slide 8: Unmet medical needs in hematologic myeloid malignancies
unmet medical needs in hematologic myeloid malignancies myelofibrosis (MF) acute myelogenous leukemia myelodysplastic syndromes (amy) (mps) © ~3,000 cases diagnosed (0 ~13,000 cases diagnosed (© 712,000 cases diagnosed per year in the US per year in the US. per year in the US © ~13,000 people in the
Slide 9: Focus on myelofibrosis (MF)
focus on myelofibrosis (MF) disease process and characteristics © Malignant clonal proliferation and atypical megakaryocytic hyperplasia leads to bone marrow fibrosis and impaired hematopo ~ Fibrosis thought to be induced by cytokines produced by the malignant progenitor cell clone? ~ Abnormal cytok
Slide 10: The unmet medical need in MF
geron the unmet medical need in MF addressing the underlying malignant process 2013 IWG criteria for response in myelofibrosis? . JAK inhibitors. CR: ‘© normal cellularity and reversal of bone marrow fibrosis and CaTIESS) © normal peripheral blood counts and smears and Pamieciei) © complete resoluti
Slide 11: Intermediate-2 and high risk MF patients have a poor prognosis
geron intermediate-2 and high risk MF patients have a poor prognosis Dynamic International Prognostic Scoring System (DIPSS) Plus* (© Eight risk factors for inferior survival: ~ Red blood cell transfusion need ~ Anemia = Thrombocytopenia 100. = leukocytosis ~ Circulating blasts 21% s” = Constitution
Slide 12: Investigator-sponsored single center study of imetelstat in myelofibrosis
SD investigator-sponsored single center study of imetelstat in myelofibrosis Principal Investigator & Study Sponsor: Ayalew Tefferi, MD ~ Mayo Clinic, Rochester Single Agent Imetelstat High risk or 2hr Lv. infusion intermediate-2 (DIPSS Plus) rate (CR, PR or Cl) per IWG criteria Primary or secondary
Slide 13: Preliminary efficacy data suggest differentiated activity in MF
preliminary efficacy data suggest differentiated activity in MF PF ama inest) Parma (n=) | Totl(n=22) | Best Response by 2013 ING criteria N(%) N(%) N (6) Overall Response (CR+PR+Cl) 3 (27.3%) 6 (54.5%) 9 (40.9%) Remission (CR+PR) 2 (18.2%) 3 (27.3%) 5 (22.7%) Complete Remission (CR) 2 (18.2%) 1 (9.
Slide 14: Remissions observed in preliminary data suggest disease-modifying activity in MF
geron remissions observed in preliminary data suggest disease-modifying activity in MF ir Nani ea SS Sea|SeS mmesyr | Sriptams | reponse oe Includes Requirement, Palpation per WG ama Response’ independence Post ET MF = post-essential thrombocythemia myelofibrosis ymunomodulatory agents; JAKi= JAK in
Slide 15: Patient case report: histology shows bone marrow complete remission
PCL SS ED patient 1 case report: histology shows bone marrow complete remission ine (12/17/12) ‘7-months post-imetelstat therapy (7/15/13) [rye ation eng nbsvac a retreat »
Slide 16: Remissions observed in preliminary data suggest disease-modifying activity in MF
geron remissions observed in preliminary data suggest disease-modifying activity in MF Pron fnemiaor pavent| 220. | rherpy | 228, | trantsion | Sethe mmeglyby | Symptoms oe Includes Requirement, Palpation ‘ArmA Response: PM, HU, 7 ay Spleen ti 2 SO iree2 ka [Mansy [necro Pesce oa |e Z CR resolved oa
Slide 17: Patient case report: histology shows bone marrow complete remission
Mi patient 2 case report: histology shows bone marrow complete remission 5.5-months post imetelstat therapy
Slide 18: Remissions observed in preliminary data suggest disease-modifying activity in MF
geron remissions observed in preliminary data suggest disease-modifying activity in MF Pron fnemiaor pavent| 220. | rherpy | 228, | trantsion | Sethe mmeglyby | Symptoms oe Includes Requirement, Palpation ArmA Response PM, HU, ob Spleen ti 2 3 int-2risk iwids CR transfusion ‘t # CR “resolved oa cs i
Slide 19: Patient case report: histology shows bone marrow complete remission
Pe SEE patient 3 case report: histology shows bone marrow complete remission 3-months post-imetelstat t [iisryasa ernie te 2015 15 pnt etn absvact 662 Teter tat) 5
Slide 20: Patient CR: 6-months post-imetelstat therapy
a SS Patient 3 - CR: 6-months post-imetelstat therapy: “Normocellular bone marrow with normal trilineage hematopoiesis. No bone marrow fibrosis” (isnot anraos strat necting act ese (eterna) 7
Slide 21: Remissions observed in preliminary data suggest disease-modifying activity in MF
remissions observed in preliminary data suggest disease-modifying activity in MF fnemiaor Pest patent | Demo shone | transtuson | Lethe | thrombo| rive. | megayby | Symptoms | Response oe Requirement, blastosis_|_Palpation per WG ‘ArmA Response PME, HU, oh = Spleen tip: , A | caer | mors | | feet |
Slide 22: Preliminary efficacy data suggest potential for broad clinical benefit
preliminary efficacy data suggest potential for broad clinical benefit. ase characteristics affected Secondary Endpoints [ama | ame | Total | Spleen Response (by palpation) 2/7 (28.6%) 3/6 (50%) 5/13 (38.5%) ‘Anemia Response 3/5 (60%) 1/7(14.3%) 4/12 (33.3%) - {Exploratory Endpoints | arma | armB |
Slide 23: Majority of the first MF patients enrolled remain on imetelstat treatment
geron majority of the first 22 MF patients enrolled remain on imetelstat treatment Pe it Status ArmA Total (n=11) (n= 22) Median months of follow-up (range) 7.6 (13-104) 4.8 (1.6-5.6) 5.3 (1.3-10.3) On treatment 8 (73%) 9 (82%) 17 (77%) Discontinued treatment 3 (27%) 2 (18%) 5 (23%) Reason for Treat
Slide 24: Preliminary safety data in MF: non-hematologic adverse events
geron preliminary safety data in MF non-hematologic adverse events not dose-limiting ‘Treatment-related non-hematologic toxicity of imetelstat among the first 33 patients enrolled with high or intermediate-2 risk myelofibrosis Grade 1 nausea 5 (15%) Grade 1 vomiting 1(3%) Grade 1/2 fatigue 4 (12%) G
Slide 25: Preliminary safety data in MF: myelosuppression is dose-limiting
PL La “DD preliminary safety data in MF myelosuppression is dose-limiting toxicity Treatment-related hematologic toxicity of imetelstat among the enrolled with high or intermediate-2 risk myelofibrosis t 33 patients Grade 3/4 neutropenia 7 (21%) 2(11%) 5 (36%) Grade 3/4 thrombocytopenia 10 (80%) 5 (
Slide 26: Planned Geron-sponsored phase 2 study
geron planned Geron-sponsored phase 2 study preliminary design based on current input from FDA, investigators, experts © Key goal for the planned Phase 2 study is to identify parameters that are appropriate for randomized Phase 3 trials designed to support potential full approval: = identify a dosin
Slide 27: Planned Geron-sponsored phase 2 study design
planned Geron-sponsored phase 2 study preliminary design based on current input from FDA, investigators, experts High risk or intermediate-2 (DIPSS Plus) ‘Multiple 2° and Exploratory Primary or rere Endpoints secondary : ave “ane (post-PV or post-ET) myelofibrosis (© Projected duration: "3 years (©
Slide 28: Potential for broader development in AML & MDS
geron potential for broader development - in hematologic myeloid malignancies: AML & MDS © Mayo Clinic IST enrolling additional cohorts: H blast-phase MF metelstat patients weekly MDS subpopulat (including RARS*) imetelstat a4 week © Data to inform company-sponsored pilot studies in MDS and/or AML ©
Slide 29: Company statistics as of December 31, 2013
as of december 31, 2013 Company Statistics © employees: 46 © cash and investments: ~$66 million © shares outstanding: ~131 million © fully diluted shares (with options and warrants outstanding): ~148 million > ee 2
Slide 30: Potential clinical milestones
geron potential Clinical milestones I 201 20s 2016 HL 2 HL #2 HL #2 Current focus ‘Mayo Clinic IST in MF —_______ ------------------ {ongoing) | Phase 2 study in MF ihlomed) nn * * Broader opportunities Potential pilot study = = 9 in AML *To be informed, in part, by the “Mayo Clini ISTin MF, includi
Slide 31: Geron - For further information, please contact
Veron For further information, please contact: Anna Krassowska, Ph.D., Investor Relations +1.650.566.7127 or investor@geron.com
Geron — 32nd Annual J.P. Morgan Healthcare Conference 2014 on 6ix