Geron Corporation · NASDAQ:GERN
Geron — Investor Presentation (January 2020)
25 slides from Geron's investor presentation, Jan 13, 2020
Slide 1: Corporate Presentation
geron Corporate Presentati
Slide 2: Forward-Looking Statements
ooking Statements jeron 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 forward-looking statements include statements rega
Slide 3: Imetelstat, a Novel Drug with a Unique Target
Imetelstat, a Novel Drug with a Unique Target + Geron proprietary drug targeting telomerase to inhibit uncontrolled progenitor cell proliferation in hematologic malignancies * Clinical and non-clinical evidence of potential disease-modifying activity + Development focused on hematologic myeloid mali
Slide 4: Telomerase drives malignant proliferation in myeloid malignancies
gic Myeloid Malignancies telomerase drives malignant proliferation in these ‘malignant ‘malignant malignant hematopoietic progenitor progenitor stem cells cell cell clones YY e—> —> @ > eo Telomerase Upregulated @ @ platelets = —> (abnormal) red blood cells. > (abnormal) collagen & reticulin fibers
Slide 5: Imetelstat Mechanism of Action
apoptosis of malignant clones malignant ‘malignant hematopoietic progenitor stem cells cell e— — @ Telomeraspfipregulated “® recovery of normal Imetelstat RBCs, WECS; ot Platelets enabled inhibits telomerase activity RBCs, red blood cells; WBCs, white blood cells, telomerase inhibitor with disease-m
Slide 6: Imetelstat overview
Qeron + Structure: Proprietary 13-mer thio-phosphoramidate (NPS) oligonucleotide, with covalently-bound lipid tail to increase cell permeability/tissue distribution * Clinical experience: more than 600 patients treated in Phase 1and 2 trials * Phase 3 | trial in lower risk MDS currently enrolling *
Slide 7: Myelodysplastic Syndromes
geron Myelodysplastic Syndromes
Slide 8: Myelodysplastic Syndromes: Unmet medical need
Myelodysplastic Syndromes Inmet medical need malignant malignant malignant immature blood hematopoietic progenitor progenitor cells stem cells cell cell clones © o—&, ——> ——> Se — oh ——> Myelodysplastic syndromes (MDS) F Telomerase Upregulated @) @ N Chronic anemia is the hallmark of low and interme
Slide 9: MDS Patient Population in the U.S.
MDS Patient Population in th addressable market opportunity > 40,000 Lower risk MDS patients in the U.S. > 10,000 Lower risk MDS cases diagnosed annually in the U.S. Imetelstat target patient population* ~85% of lower risk MDS Cogle, Curr Hematol Malig Rep; 2015, 10272-10281 * Lower risk MDS patient
Slide 10: IMerge: 2-Part Phase 2/3 Trial
|Merge: 2-Part Phase 2/3 Trial Part 1 — Phase 2 portion ‘Transfusion Dependent, Low or Intermediate-1 Risk MDS, non- del(5q), R/R to ESAs, no prior treatment with lenalidomide or IMerge Part 1: Phase 2 Portion single arm, open label Imetelstat 7.5mag/kg every 4 weeks Clinical gov (cro2ssacea) 1° Eff
Slide 11: Phase 2 Results and durable transfusion independence
Phase 2 Results 1 and durable transfusion independence Data Reported at European Hematology Association (EHA) Annual Congress in June 2019 No. of patients 38 Median baseline transfusion burden, units/8 weeks (range) 8 (4-14) Rate of 8-week RBC-TI, % (n) 42% (16/38) Rate of 24-week RBC-TI, % (n) 29%
Slide 12: Phase 2 Results across different patient subgroups
ase 2 Results across different patient subgroups All subjects WHO 2008 category RARS / RCMD-RS OTHER RBC transfusion burden 4-6 units >6 units IPSS risk status Intermediate-1 Low Serum erythropoietin level <= 500 mU/mL > 500 mU/m! N__8Week Ti 95% Cl Rea 38 16(42.1) (26.3-59.2) He 27° 12(44.4) (25.5
Slide 13: Phase 2 Results: disease-modifying activity observed
Phase 2 Results ease-modifying activity observed Impact on biomarkers of MDS disease suggest potential effect on malignant progenitor cell clones and disease modification > 75% (12/16) of patients who achieved an 8-week RBC-TI also had a Hgb rise > 3g/dL from the pretreatment level, which suggests r
Slide 14: Lower Risk MDS Trial Comparison
isk MDS Trial Comparison* ifferent disease burdened patient populations IMerge Part 1 — Phase 2 MEDALIST** — Phase Imetelstat Luspatercept Placebo 38 No. of patients 153 76 Non-del(5q), R/R to ESAs, no prior Non-del(5q), R/R to ESAs, no prior treatment with lenalidomide or HMAs treatment with lenali
Slide 15: Phase 3 in Lower Risk MDS
ase 3 in Lower Risk MDS ‘olling Imetelstat 1° Efficacy: (n=~115) Red Blood Cell (RBC) Transfusion vee Independence (I) 2 2 efficacy RBCTI224 weeks; time to and duration of REC-T iatologic improvement (HI); ction in RBC burden Intermediate-1 Risk MDS, non-del(5q), R/R to ESAs, no prior treatment lena
Slide 16: Myelofibrosis
geron Myelofibrosis um -
Slide 17: Myelofibrosis (MF): Unmet medical need
sis (MF) inmet medical need malignant malignant malignant collagen & hematopoietic progenitor progenitor reticulin fibers stem cells cell cell ee (fibrosis) @—&, > o —_> %o —_> Ba — esr Telomerase Upreguated @ @ Estimated Int-2/High-risk MF Patient Population in the U.S. + Prevalence: > 9,000 patien
Slide 18: IMbark Phase 2 Trial
IMbark Phase 2 Trial IM. | Clinciattiale gov Clinical activity in relapsed/refractory MF patients teraz) ‘Co-1" Efficacy: Imetelstat Spleen response rate and symptom response rate 9.4 mg/kg 2° Efficacy every 3 weeks CR, PR and Cl, anemia response per 2013 IWG-MRT criteria, duration of responses, ove
Slide 19: Phase 2 Results: symptom response
hase 2 Results ponse at 24 weeks 400 IMETELSTAT 9.4 MG/KG N=59 ° 8 Percent Change from Baseline & 100 19 (32%) patients in the 9.4 mg/kg arm had 2 50% symptom response using total symptom score (TSS) at Week 24 ‘Asi 2018 Presentation: Mascarenhas et.
Slide 20: Phase 2 Results: potential improvement in survival
hase 2 Results t potential improvement in survival 1.00: ons: a oso z 025 om o é Number at risk Imetektot 94 WGKG 58 53 ry 78 2 Months From First Dose Adminstration 46 a2 23 30 ‘ASH 2018 Presentation: Mascarenhas ab Clinical cut-off (10/22/2018): * Median follow-up: 27.4 (0.2-33.0) mos Median overal
Slide 21: Potential survival benefit: IMbark vs. real-world data
potential surviva IMbark Phase 2 Data vs. Real-World Data (RWD) hase 2 Data vs. Real World D alan Haier Garves (Una ghicd) or Nahe Compara Sina po Numer ato benefit Purpose: Assess potential overall survival benefit with ) imetelstat treatment + IMbark data compared to RWD of a closely-matched cohor
Slide 22: Fast Track designation and next steps
Granted Fast Track designation by the FDA for Int-2/High-risk MF Q4 2019 Next Steps + Submit several Phase 3 trial design proposals to FDA + Discuss potential regulatory approval path with FDA + Decision regarding potential late-stage development EOP2 Meeting with the FDA 22
Slide 23: Development Priorities
geron Development Priorities
Slide 24: Development Priorities
velopment Priorities Phase 3 IMerge Clinical Trial in Lower Risk MDS Q Plan to complete enrollment Myelofibrosis Q Submit several Phase 3 trial design proposals to the FDA Q Discuss potential regulatory approval path with the FDA Q Decision regarding potential late-stage development Additional Indic
Slide 25: Thank you
Qeron Than