Geron Corporation · NASDAQ:GERN
Geron — Jefferies 2014 Global Healthcare Conference
24 slides from Geron's conference presentation, Jun 2, 2014
Slide 1: Jefferies 2014 Global Healthcare Conference
Jefferies 2014 Global Healthcare Conference yeron John Scarlett, M.D. Chief Executive Offi
Slide 2: Forward-looking statements
forward-looking STATEMENTS _ Except for statements of historical fact, this presentation and question and answer session contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including without limitation, statements
Slide 3: Developing imetelstat to treat hematologic myeloid malignancies
developing imetelstat to treat . . 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 selectively inhibit
Slide 4: Imetelstat: a telomerase inhibitor
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 prolif
Slide 5: Hematologic myeloid malignancies arise from malignant progenitors in the bone marrow
geron hematologic myeloid malignancies _arise from malignant progenitors in the bone marrow Sk —> essential thrombocythemia platelets (ET) (abnormal) BY — soryerineniaver ow matinant redboot es myeloid (abnormal) progenitoy © —> myelofibrosis (MF) Rw cactheare malignars reticulin fibers tiene (fibro
Slide 6: Essential thrombocythemia: proof-of-concept for selective effect on malignant clone
geron essential thrombocythemia proof-of-concept for selective effect on malignant clone 5 —> essential thrombocythemia © Hematologic response: platelets, (ET) to assess effect of imetelstat (abnormal) on platelet count © Molecular response: BR — soeynemio vers ov (eduton in n2V67 lee , ———~— burden
Slide 7: Essential thrombocythemia data: responses suggest disease-modifying activity
essential thrombocythemia data responses suggest disease-modifying activity Primary analysis (per clinical protocol) Hematologic response rate 18/18 (100%) 18/18 (100%) ‘Complete response (CR) rate 16/18 (89%) 16/18 (89%) Partial response (PR) rate 2/18 (11%) 2/18 (11%) Median time on therapy (range
Slide 8: Essential thrombocythemia data: most frequently reported adverse events
essential thrombocythemia data most frequently reported adverse events 20 patients (18 ET & 2 PV) were enrolled inthe study prswretvenceroon | Micra abene | tastes | Infections: 20 (100%) 4 (20%) 4 (20%) 1 (8%)" ee. Fatigue 18 (90%) 2(10%) 17 (85%) 2 (10%) RL eee Cytopenias 14 (70%) 10 (50%) 13 (65%
Slide 9: Essential thrombocythemia data: laboratory abnormalities
geron essential thrombocythemia data laboratory abnormalities 20 patients (18 ET & 2 PV) were enrolled inthe study ‘Alanine transaminase (ALT) 18 (90%) 2(10%)"* Aspartate transaminase (AST) 18 (90%) 1 (6%)"* 0 Alkaline phosphatase (ALP) 15 (75%) 0 0 Bilirubin, total 8 (40%) 0 ° Neutropenia 16 (80%)
Slide 10: Recent FDA action related to Geron's imetelstat IND
geron recent FDA action related to Geron’s imetelstat IND © Geron’s IND for imetelstat placed on full clinical hold: ~ patients in Phase 2 ET and MM trials cannot receive any further treatment with imetelstat ~ Geron cannot initiate a new clinical trial under its IND until hold is lifted or partiall
Slide 11: Addressing the clinical hold
geron addressing the clinical hold related to Geron’s imetelstat IND © Clinical activities: ~ amend Geron-sponsored clinical protocols, seek IRB approvals and obtain patient informed consent to allow for longer follow-up after imetelstat was discontinued to collect LFT and other relevant information
Slide 12: Focus on myelofibrosis: disease with greater unmet medical need
focus on myelofibrosis disease with greater unmet medical need & — potyerthemia vera (Pv) —> essential thrombocythemia (ET) malignant (0 ~3,000 cases diagnosed my per year in the US, Progenitor, <2 (© ~13,000 people in the US a myelofibrosis (MF) living with ME Se othr © drug therapies include: mali
Slide 13: Investigator-sponsored single center initial study of imetelstat in myelofibrosis
investigator-sponsored single center initial study of imetelstat in myelofibrosis Principal Investigator & Study/IND Sponsor: Ayalew Tefferi, MD — Mayo Clinic, Rochester Single Agent Imetelstat 9 int: High risk or Be . 1 ‘ential 2 ; oy + Overall response rate intermediate 2 (Groner pei ( us) criteri
Slide 14: Potential for differentiation: addressing the underlying malignant process
potential for differentiation addressing the underlying malignant process 2013 IWG criteria for response in myelofibrosis* JAK inhibitors. CR: © normal cellularity and reversal of bone marrow fibrosis and Cn sec-y) © normal peripheral blood counts and smears and Tetsu © complete resolution of sympto
Slide 15: Preliminary efficacy data suggest differentiated activity in MF
preliminary efficacy data suggest differentiated activity in MF PF aemainess) [ama init) | rotl(n= 22) Best Response by 2013 IWG criteria N(%) N(%) N (2%) 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(9.1
Slide 16: Remissions observed in preliminary data suggest disease-modifying activity in MF
remissions observed in preliminary data suggest disease-modifying activity in MF all manifestations of disease must be addressed in patients to achieve a remission [eexresponse pes wGemale oR | | Pm | Normal cellularity and reversal of bone marrow fibrosis ¥ ¥ Y VY x 5 Normal peripheral blood counts
Slide 17: Patient bone marrow: complete remission on imetelstat
a iron patient 2 (prior Jak inhibitor): bone marrow complete remission on imetelstat SP FaieSFVaSalFFEPEHREA ELAS 01S ASH Anal eeting Abstract nEG2 (Tet
Slide 18: Preliminary efficacy data suggest potential for broad clinical benefit
preliminary efficacy data suggest . potential for broad clinical benefit multiple disease manifestations affected Secondary Endpoints [ama | ame | Total | Spleen Response (by palpation) 2/7 (28.6%) 3/6 (50%) 5/13 (38.5%) ‘Anemia Response 2/5 (40%) 1/7 (14.3%) 3/12 (25%) - {Exploratory Endpoints | ar
Slide 19: Preliminary safety data in MF: myelosuppression is dose-limiting toxicity
ee preliminary safety data in MF myelosuppression is dose-limiting toxicity Treatment-related hematologic toxicity of imetelstat among the first 33 patients enrolled with high or intermediate-2 risk myelofibrosis. Myelosuppression Arm A Arm B All patients (n=19) (n=14) (n=33) Grade 3/4 neutropenia 2
Slide 20: 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%) Gr
Slide 21: Recent FDA action related to Dr. Tefferi's myelofibrosis IND
geron recent FDA action related to Dr. Tefferi’s myelofibrosis IND © Dr. Tefferi’s myelofibrosis IND for imetelstat placed on partial clinical hold: ~ previously enrolled patients (with MF, MDS RARS and blast-phase MF) deriving clinical benefit may continue treatment with imetelstat © Safety issues
Slide 22: Candidate indications among hematologic myeloid malignancies
geron candidate indications among hematologic myeloid malignancies © If the full clinical hold on Geron’s IND is lifted, or partially lifted: expect to pursue development of imetelstat in MF and potenti ly one or more indications where there is a greater unmet medical need than ET myelodysplastic sy
Slide 23: Supporting imetelstat development in hematologic myeloid malignancies
supporting imetelstat development in hematologic myeloid malignancies Activities to seek release of full clinical hold: © compile and submit preclinical and clinical data and information to FDA © maintain focus on development plan in diseases with high unmet need Updated Myelofibrosis IST efficacy a
Slide 24: Thank you
thank you Veron For further information, please contact: Anna Krassowska, Ph.D., Investor Relations +1.650.566.7127 or investor@geron.com