Geron Corporation · NASDAQ:GERN
Geron — J.P. Morgan Global Healthcare Conference 2012
28 slides from Geron's conference presentation, Jan 11, 2012
Slide 1: J.P. Morgan Global Healthcare Conference 2012
geron J.P. Morgan Global Healthcare Conference 2012 John A. Scarlett, MD President & CEO
Slide 2: Forward-Looking Statement
Forward-Looking Statement Except forthe 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 without limitation, the following
Slide 3: Building a World-Class Oncology Development Business
geron Building a World-Class Oncology Development Business
Slide 4: Geron: Poised for Value Creation
Geron: Poised for Value Creation = Two first-in-class oncology drug candidates: imetelstat and GRN1005 = Large unmet oncology needs addressed by novel mechanisms = Phase 2 top-line results expected Q4 2012 (imetelstat) and Q2 2013 (GRN1005) = Broad patent protection through at least 2025 for both pr
Slide 5: Multiple Opportunities for Success
Multiple Opportunities for Success Expected Product Candidate Results Imetelstat (telomerase inhibitor) ‘Randomized, controlled trials Metastatic Breast Cancer Lung Cancer (NSCLC) ‘Single-arm trials Essential Thrombocythemia Multiple Myeloma GRN1005 (LRP directed peptide-drug conjugate) Single-arm t
Slide 6: Imetelstat: First-in-Class Telomerase Inhibitor
Imetelstat: First-in-Class Telomerase Inhibitor geron
Slide 7: Telomerase is a Promising Oncology Target
Telomerase is a Promising Oncology Target = Telomeres are caps at the end of the chromosome = Shorten with each cell division, determining the life span of the cell = Telomerase, a reverse transcriptase enzyme, extends telomere length = Telomerase is a key molecular target in oncology - Required for
Slide 8: Focus on Preventing / Delaying Tumor Regrowth after Debulking
Focus on Preventing / Delaying Tumor Regrowth after Debulking Debulking with chemotherapy leaves residual progenitor cells Regrowth of tumor from residual progenitors — proliferative capacity requires telomerase upregulation = Telomerase inhibition inhibits growth of tumors from cancer progenitor ce
Slide 9: Imetelstat: First-in-Class Telomerase Inhibitor, Built with Geron's Proprietary Nucleic Acid Chemistry
Imetelstat: First-in-Class Telomerase Inhibitor, Built with Geron’s Proprietary Nucleic Acid Chemistry = Direct, competitive, high affinity inhibitor of telomerase = Modified lipid-oligonucleotide conjugate = Designed to address liabilities inherent in historical oligonucleotide drugs = Lipid tail t
Slide 10: Imetelstat: Phase 1 Program Summary
Imetelstat: Phase 1 Program Summary erview Pharmacokinetics Pharmacodynamics folerability 183 subjects with relapsed or progressing solid tumors or hematologic malignancies including breast, lung, multiple myeloma Targeted exposures achieved at doses lower than MTD Telomerase inhibition >50% in tiss
Slide 11: Imetelstat: Multiple Opportunities for Success
Imetelstat: Multiple Opportunities for Success Product Candidate ‘tot Imetelstat (telomerase inhibito Randomized, controlled trials Metastatic Breast Cancer Lung Cancer (NSCLC) a4 2012 a4 2012 Single-arm trials Essential Thrombocythemia Multiple Myeloma a4 2012 a4 2012 7 geron
Slide 12: Imetelstat Metastatic Breast Cancer Phase 2 Trial (B014)
Imetelstat Metastatic Breast Cancer Phase 2 Trial (B014) Rationale Study Design Success Criteria Cancer progenitor cells drive breast cancer progression and relapse Imetelstat inhibits breast cancer: = Progenitor cells in vitro and in vivo - Tumor growth and metastases in vitro and in vivo Synergist
Slide 13: Imetelstat Lung Cancer (NSCLC) Phase 2 Trial (B012)
Imetelstat Lung Cancer (NSCLC) Phase 2 Trial (BO12) Rationale Success Criteria Cancer progenitor cells drive lung cancer progression and relapse Imetelstat inhibits lung cancer: = Progenitor cells in vitro and in vivo - Tumor growth and metastases in vitro and in vivo Support advancement to Phase 3
Slide 14: Imetelstat in Essential Thrombocythemia (B015)
Imetelstat in Essential Thrombocythemia (BO15) Myeloproliferative Disorder Resulting in Overproduction of Platelets Imetelstat inhibits malignant hematopoietic progenitor cells Significant decrease in platelet counts in Phase 1 studies 50% of patients have a specific clonal marker (JAK2 or MPL) Demo
Slide 15: Imetelstat in Multiple Myeloma (B013)
Imetelstat in Multiple Myeloma (B013) Exploratory Phase 2 Study Cancer progenitors drive progression and relapse of myeloma Imetelstat inhibits myeloma progenitor cells in vitro and in vivo In myeloma, malignant progenitor cells accessible in bone marrow Rationale = Demonstrate imetelstat directly i
Slide 16: Enrollment and Timing of Top-Line Data Readouts
Enrollment and Timing of Top-Line Data Readouts Randomized, controlled trials (metastatic breast cancer, NSCLC) = Enrollment ahead of schedule; continue to expect to report in Q4 2012 = Asis customary, these are event driven analyses (PFS) - Actual occurrence of events will determine timing for read
Slide 17: Highlights of Imetelstat Program
Highlights of Imetelstat Program Transformative approach to treating range of tumor types - Inhibit indefinite replication of cancer cells - Selectively inhibit tumor progenitor cells Strong scientific rationale and pre-clinical support Only clinical-stage telomerase inhibitor Phase 2 in four indica
Slide 18: GRN1005: LRP-Directed Peptide-Drug Conjugate to Treat Metastatic and Primary Brain Tumors
GRN1005: LRP-Directed Peptide-Drug Conjugate to Treat Metastatic and Primary Brain Tumors geron
Slide 19: GRN1005 LRP-Directed Peptide-Drug Conjugate
GRN1005 LRP-Directed Peptide-Drug Conjugate LRP-1 receptors transport large molecules across the BBB - LRP-1 is highly expressed on BBB and on various tumors Conjugate of three molecules of paclitaxel covalently linked to Angiopep-2, a proprietary peptide that binds to LRP-1 receptor Mechanism of ac
Slide 20: GRN1005: Summary of Phase 1 Results
GRN1005: Summary of Phase 1 Results armacokinetics 119 patients in two single agent Phase 1 studies - Solid Tumor Metastases (n = 56) - Primary Brain Tumors (n = 63) Clinically significant GRN1005 drug concentrations detected in primary brain tumor samples within a few hours of dosing GRN1005 remain
Slide 21: GRN1005: Summary of Phase 1 Results (cont'd)
GRN1005: Summary of Phase 1 Results (cont’d) = Responses observed at MTD in patients with solid tumor and extra-cranial disease) - 4/17 (24%) patients with brain metastases had >30% shrinkage in brain lesions - 5/10 (50%) patients with lung lesions had >30% shrinkage in those lesions = Dose-limiting
Slide 22: GRN1005 Phase 2 Program Initiated
GRN1005 Phase 2 Program Initiated Product Candidate Phase 1 pany GRN1005 (LRP directed peptide-drug conjugate) si ils Brain Metastases (Breast Cancer) Brain Metastases (Lung Cancer) 2 2013 2 2013 7 geron
Slide 23: GRN1005 Phase 2 Program: Breast Cancer with Brain Metastases
GRN1005 Phase 2 Program Breast Cancer with Brain Metastases Trial (GRABM-B) 100 patient (50 HER2+ and 50 HER2-)' Phase 2 study } Study Design | estimating the effect of GRN1005 (as defined by intra-cranial response rate) in breast cancer patients with brain metastases — Intra-cranial response rate s
Slide 24: GRN1005 Phase 2 Program: Lung Cancer with Brain Metastases
GRN1005 Phase 2 Program Lung Cancer with Brain Metastases Trial (GRABM.: ‘Success Criteria 50 patient Phase 2 study estimating the effect of GRN1005 (as defined by ORR) in NSCLC patients with brain metastases Overall response rate significantly higher than historical control (-8% in NSCLC patients r
Slide 25: Highlights of GRN1005 Program
Highlights of GRN1005 Program No approved drug therapies for metastatic brain cancer As treatments for breast and lung cancer improve, brain metastasis becomes more pressing area of unmet need Intra-cranial response rates to single agent anti-cancer drugs approximately 5% First-in-class peptide-drug
Slide 26: Strong Balance Sheet
Strong Balance Sheet = Over $150 million in cash and investments as of December 31, 2011 = Burn rate guidance of $65 million for 2012 = Expect to have sufficient resources to report top-line data on all current Phase 2 trials without additional financing ” geron
Slide 27: Geron: Poised for Value Creation
Geron: Poised for Value Creation = Two first-in-class oncology drug candidates: imetelstat and GRN1005 = Large unmet oncology needs addressed by novel mechanisms = Phase 2 top-line results Q4 2012 (imetelstat) and Q2 2013 (GRN1005) = Broad patent protection through at least 2025 for both programs =
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Geron — J.P. Morgan Global Healthcare Conference 2012 on 6ix