Geron Corporation · NASDAQ:GERN
Geron — Investor Presentation (January 2018)
21 slides from Geron's investor presentation, Jan 8, 2018
Slide 1: Corporate Presentation January 2018
geron, Corporate Presentati January 2018
Slide 2: Forward-Looking Statements
ooking Statements geron 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 statements regarding: (i) continue
Slide 3: Overview
verview geron Inhibiting telomerase is a novel, potentially transformative therapeutic approach to the treatment of hematologic myeloid malignancies + Telomerase highly upregulated in malignant progenitor cell clones, conferring replicative immortality and unrestrained proliferation + Imetelstat, th
Slide 4: Telomerase as a target in oncology
target in oncology Telomerase enzyme * Comprised of an RNA template component (hTR) and a reverse transcriptase catalytic protein subunit (hTERT) RNA template * Binds to the 3’ strand of DNA and adds TTAGGG SS 0 (hTR) nucleotide repeats to offset the loss of telomeric DNA at the end of chromosomes t
Slide 5: Imetelstat: first-in-class telomerase inhibitor
ss telomerase inhibitor imetelstat \\c_ sas NPS oligonucleotide lipid tail | Imetelstat binds to RNA template of telomerase telomere Prevents binding by and maintenance of telomeres geron Proprietary: 13-mer thio-phosphoramidate (NPS) oligonucleotide complementary to hTR, with covalently-bound lipid
Slide 6: Hematologic myeloid malignancies
+ ~3,000 cases diagnosed per year in the US + ~13,000 people in the US ing with MF + ~70% of patients have high/intermediate-2 risk disease gic Myeloid Malignancies alignant progenitor cells in the bone ESSENTIAL ‘THROMBOCYTHEMIA (ET) oar 2, * 4 MYELODYSPLASTIC 16,000 cases diagnosed xen ‘SYNDROMES
Slide 7: Myelofibrosis
yrosis iracteristics + 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 - Constitu
Slide 8: Current treatments for High/Int-2 Risk MF
atments for High/Int-2 Risk MI pproved for ruxolitinib relapsed or refractory Symptoms and/or splenomegaly + treatment with ruxolitinib - oral JAK1/JAK2 inhibitor - only approved product for myelofibrosis in U.S. and EU om | * stay on drug as long as tolerated - conventional drugs (e.g., 5-year disc
Slide 9: Focusing imetelstat on relapsed or refractory MF: IMbark trial design
Focusing imetelstat on relapsed or refractory MF IMbark: Phase 2 trial design Study Population: * Patients with Intermediate-2 or High Risk myelofibrosis who are relapsed or refractory to JAK inhibitor treatment Key Goals: * Define appropriate dosing (9.4 or 4.7 mg/kg every 3 weeks) * Confirm safety
Slide 10: IMbark data review and expected data readouts
geron Date and Expected Data Readouts September 2016 internal data review findings from 40 patients who had completed 12 wks on protocol: * — Safety profile consistent with previous clinical trials in heme malignancies; no new safety signals identified + Activity within multiple outcome measures was
Slide 11: Myelodysplastic Syndromes characteristics
cteristics * Diverse group of clonal hematologic malignancies with disordered and ineffective production of the myeloid lineage in the bone marrow characterized by abnormal cell morphology and counts (anemia and other cytopenias) * Median age at diagnosis is ~70 years + Up to 30% of patients progres
Slide 12: Current treatments for Lower Risk MDS
reatments for Lower Risk MD ‘mia remains an unmet need Del(5q)* patients Erythropoiesis stimulating agents . Lenalidomide (ESAs) Patients dependent + 28-week RBC-Tl: ~67% + improvement in anemia mmmp on red blood cell in “50% of patients transfusions + median duration: ~2 years Non-Del(5q) patients
Slide 13: IMerge Phase 2/3 trial design in lower risk MDS
Imetelstat in transfusion dependent patients with IPSS Low or Intermediate-1 Risk MDS that is relapsed or refractory to erythropoiesis stimulating agent (ESA) treatment * Part 1 (Phase 2) to evaluate safety and efficacy of imetelstat to advance to Part 2 based on positive assessment of benefit-risk
Slide 14: IMerge Part 1: preliminary efficacy data
_ IMerge Part 1 Preliminary efficacy data promising All Treated Lenalidomide and HMA naive (N=32) and Non-del(5q) (n=13) >8-week RBC-TI 12* (38%) 7 (54%) mm ; i F Increased RBC-TI rate and lean relative reduction from . . durability in 13-patient baseline transfusion burden cae are subset who were n
Slide 15: Example: Non-del(5q) patient naive to HMA and lenalidomide
1 Example: Non-del(5q) patient naive to | * Suggests suppression of the underlying malignant clone by imetelstat Pelt cto" 1) E 2 Example of a 24-week RBC-TI responder Hematology and imetelstat Administration Over Time ik + 21.3.5 7 911131517 1921 23 25 27 29 31 33 3537 39 41 43 45 47 49 51 53 55 $7
Slide 16: IMerge safety profile
geron Safety profile consistent with previous imetelstat clinical trials in hematologic myeloid malignancies * No new safety signals were identified ¢ Adverse events with imetelstat (mostly cytopenias) were predictable, manageable, and reversible for most patients * 50% of patients had dose reductio
Slide 17: Current status of IMerge
Current status of IMerge Part 1 enrollment expanded in target patient population Part 1 overall population (n=32) RBC-TI (28-wks): 38% Enroll * Increase experience and confirm ee poccitions ay benefit-risk of imetelstat dosed naz Olpavent., at 7.5 mg/kg every four weeks in subset (n=13) : ’ els Naiv
Slide 18: Partnership with Janssen
artnership with Janssen lusive worldwide collaboration for imetelstat inuation Stage (after positive Continuation Decisio: Phase 3: MF, MDS Phase 2: Additional exploratory indications Phase 2,3: AML IMerge (Phase 2/3 MDS trial) + Janssen conducting IMbark and IMerge + Geron has Opt-In right to share
Slide 19: Timing for imetelstat key milestones
ming for tat Key Milestones 2018 2019 o IMbark o IMbark primary analysis: o Latest time for Janssen internal data begins upon the earlier of the Continuation Decision expected review expected occurrence of a pre-specified by Q4 2018 or Q1 2019 by end of Q1 2018 number of deaths or the end of Q3 2018
Slide 20: Pipeline Diversification
ipeline Diversification General profile of candidates: * Oncology focused + Ideally late preclinical or early clinical stage * Credible target and/or demonstration of preclinical or clinical proof-of-concept * Target relevance in multiple indications * Attractive technology platform or early pipelin
Slide 21: Thank you