Healthcare Conference John Scarlett, M.D. President and CEO, Geron - - PowerPoint PPT Presentation

Stifel Healthcare Conference

John Scarlett, M.D.

President and CEO, Geron Corporation November 2018

Forward-Looking Statements

2 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 include, without limitation, statements regarding the expectations, plans, timelines and prospects for imetelstat and Geron, including without limitation: (i) that the imetelstat IND transfers from Janssen to Geron in the second quarter of 2019; (ii) that IMbark and IMerge will continue; (iii) Geron’s plans to initiate the Phase 3 portion of IMerge in mid-2019; (iv) that Geron will

• utline a decision for myelofibrosis development by the end of the third quarter 2019; (v) that imetelstat for MDS would be prescribed before or in lieu
• f lenalidomide and/or hypomethylating agents; (vi) financial or operating projections or requirements of Geron; and (vii) other statements that are not

historical facts, constitute forward-looking statements. These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation: (i) whether contingencies delay or prevent the start of the Phase 3 portion of IMerge by mid-year 2019; (ii) whether regulatory authorities permit the further development of imetelstat on a timely basis, or at all; (iii) whether Geron decides for any reason not to develop imetelstat for myelofibrosis; (iv) whether any circumstances arise that prevent a timely transition of the imetelstat program from Janssen; (v) whether Geron’s patents protect the commercial value of imetelstat; (vi) whether imetelstat’s benefit-risk profile for MDS is better than lenalidomide and/or hypomethylating agents; and (vii) whether Geron can obtain sufficient funding to support further development of imetelstat. Additional information and factors that could cause actual results to differ materially from those in the forward-looking statements are contained in Geron's periodic reports filed with the Securities and Exchange Commission under the heading “Risk Factors,” including Geron's quarterly report on Form 10-Q for the quarter ending September 30, 2018. Undue reliance should not be placed on forward- looking statements, which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may

• change. Except as required by law, Geron disclaims any obligation to update these forward-looking statements to reflect future information, events or

circumstances.

Geron Overview

3

Imetelstat

• A first-in-class telomerase inhibitor, focused on hematologic myeloid malignancies
• Clinical evidence of potential disease-modifying activity in three myeloid malignancies: essential thrombocythemia (ET),

myelofibrosis (MF) and myelodysplastic syndromes (MDS)

• Granted Fast Track designation by FDA for treatment of lower risk MDS patients

Current Clinical Trials

• IMerge: Phase 2/3 trial in lower risk MDS
• IMbark: Phase 2 trial in Intermediate-2 or High-risk MF
• Oral presentations of updated data for both trials scheduled at American Society of Hematology (ASH) Annual Meeting

Future Plans

• Screening and enrollment for Phase 3 portion of IMerge planned to begin by mid-year 2019
• Exploring potential for late-stage development in MF, expect decision by end of third quarter 2019

Company

• A late-stage clinical development biopharmaceutical company with 100% ownership of imetelstat, a Phase 3-ready asset
• Sufficient cash ($185M as of 9/30/18) to move imetelstat forward into Phase 3

Telomerase

A novel molecular target in oncology

4

Telomerase enzyme

• Comprised of an RNA template component

(hTR) and a reverse transcriptase catalytic protein subunit (hTERT)

• In 2009, three Geron collaborators won the

Nobel Prize for Medicine for discovery of telomerase and its relationship with telomeres

telomeric DNA RNA template (hTR) catalytic subunit (hTERT)

Somatic Cells

Telomerase inactive

Normal Progenitor Cells

Telomerase transiently upregulated to support controlled proliferation

Malignant Progenitor Cells

Telomerase highly upregulated, in over 90% of cancers, enabling continued and uncontrolled proliferation

Imetelstat

A first-in-class telomerase inhibitor

• Target: malignant progenitor cell clonal

proliferation

• Structure: 13-mer thio-phosphoramidate

(NPS) oligonucleotide complementary to hTR, with covalently-bound lipid tail to increase cell permeability/tissue distribution

• Long tissue residence time in bone marrow,

spleen, liver

• Potent competitive inhibitor of telomerase
• Clinical experience: more than 600 patients

treated in Phase 1 and 2 trials

5

imetelstat lipid tail telomere Prevents binding by and maintenance of telomeres Imetelstat binds to RNA template

• f telomerase

X

NPS oligonucleotide

Imetelstat

Key differentiating features

Targeting telomerase to tackle the underlying disease

• Telomerase activity is high in patients with myeloproliferative neoplasms
• Inhibiting telomerase expression limits the proliferation of malignant progenitor cell clones

that drive the disease

6

Unique Mechanism

• f Action

Broad Clinical Activity Challenging Patient Populations Tested Trial eligibility criteria emphasizes difficult-to-treat patients

• IMerge – open to all MDS subtypes, minimum transfusion burden of 4.0 units/8 weeks, pre-

transfusion hemoglobin ≤ 9.0 g/dL

• IMbark – must show objective evidence of disease progression during or after treatment with

a JAK inhibitor (JAKi) or never responded to JAKi treatment plus active symptoms of MF

Activity within multiple outcome measures suggest clinical benefit

• IMerge – ≥8-week red blood cell (RBC) transfusion independence (TI) achieved across MDS

subtypes (ring-sideroblast, RS +/-) and erythropoietin (EPO) levels, reductions in transfusion burden in all patients

• IMbark – range of reductions in Total Symptom Score, potential improvement in overall

survival for 9.4 mg/kg arm, including patients with at least one high-risk mutation and triple negative mutation patients

Hematologic Myeloid Malignancies

Clinical evidence of potential disease modifying activity

7 telomerase

All arise from malignant progenitor cell clones in the bone marrow

Steensma et al, 2018 ASH Abstract #463 Baerlocher et al, NEJM 2015; 373:920-928 Tefferi et al, NEJM 2015; 373:908-919 Tefferi Pilot Study, unpublished

8 telomerase

• MDS is a diverse group of clonal hematologic

malignancies

• Comprised of numerous subtypes, including

refractory anemia with ring-sideroblasts (RARS) and refractory cytopenia with multi-lineage dysplasia-RS (RCMD-RS)

• RS+ MDS associated with lower risk of AML

transformation and better survival than RS- patients

• Median age at diagnosis is 70
• Up to 30% of patients progress to acute leukemia

(AML) Lower Risk MDS

• Median overall-survival is 3.5-5.7 years
• Chronic anemia is the predominant clinical problem,

many patients are dependent on RBC transfusions

• Transfusion dependency is associated with iron
• verload, and shorter survival - 2 units of RBC

monthly may reduce life expectancy by 50% and increase risk of progression to AML

Myelodysplastic Syndromes (MDS)

Disease characteristics

Sekeres, Natl Compr Canc Netw 2011; 9:57-63 Bejar & Steensma, Blood 2014; 124:2793-2803 Greenberg et al, Blood 1997; 89:2079-2088 Malcovati et al, JCO 2007; 25:3503-3510 www.cancer.org/cancer/myelodysplastic-syndromes

Cogle, Curr Hematol Malig Rep; 2015, 10272-10281 Greenberg et al, Blood 1997; 89:2079-2088

~70%

• f MDS patients have

Lower Risk MDS

60,000

MDS patients in the U.S.

16,000

Cases diagnosed annually in the U.S.

9

MDS Patient Population in the U.S.

Addressing a large unmet need

10

Fenaux and Adès, Blood 2013; 121:4280-4286 Santini et al, J Clin Oncol 2016; 34:2988-2996 Tobiasson et al, BCJ 2014; 4: e189

Patients refractory to ESAs become dependent on red blood cell transfusions

Erythropoiesis Stimulating Agents (ESAs)

• Improvement in

anemia in ~50% of patients

• Median treatment

duration: ~2 years

Hypomethylating Agents (HMAs)

Approved in U.S. for all patients, including del(5q) and non-del(5q) Not approved in Europe

• ≥8-week RBC-TI: ~17%

Treatment Landscape for Lower Risk MDS

Chronic anemia remains an unmet need

Lenalidomide

Not approved in U.S. or Europe for non-del(5q) patients

• ≥8-week RBC-TI: 27%

Treatment Landscape for Lower Risk MDS

Opportunity to sequence imetelstat ahead of available therapies

11

Fenaux and Adès, Blood 2013; 121:4280-4286 Santini et al, J Clin Oncol 2016; 34:2988-2996 Tobiasson et al, BCJ 2014; 4: e189

Patients refractory to ESAs become dependent on red blood cell transfusions

Erythropoiesis Stimulating Agents (ESAs)

• Improvement in

anemia in ~50% of patients

• Median treatment

duration: ~2 years

Hypomethylating Agents (HMAs)

Approved in U.S. for all patients, including del(5q) and non-del(5q) Not approved in Europe

• ≥8-week RBC-TI: ~17%

Imetelstat

≥8-week RBC-TI: 37%

potential to sequence ahead of lenalidomide and HMAs

ASH 2018 Abstract #463: Imetelstat Treatment Leads to Durable Transfusion Independence (TI) in RBC Transfusion-Dependent (TD), Non-Del(5q) Lower Risk MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Who Are Lenalidomide (LEN) and HMA Naïve; Steensma D, et.al.

Lenalidomide

Not approved in U.S. or Europe for non-del(5q) patients

• ≥8-week RBC-TI: 27%

12

Clinical Trial Conduct and Status

• Initial enrollment: transfusion dependent patients

with IPSS Low or Intermediate-1 Risk MDS that is relapsed or refractory (R/R) to erythropoiesis stimulating agent (ESA) treatment

• Results from initial enrollment presented at ASH in

December 2017 and the European Hematology Association (EHA) in June 2018

• Starting dose confirmed
• Target patient population defined: no del(5q)

chromosomal abnormality or non-del(5q), naïve to lenalidomide or HMA treatment

• Fast Track designation by FDA for treatment of lower

risk MDS patients

• More mature data for target patient population

(n=38) to be presented at ASH in December

• Treatment and follow-up continuing as per protocol

ClinicalTrials.gov (NCT02598661)

IMerge: 2-Part Phase 2/3 Trial

Part 1 – Phase 2 portion

Imetelstat 7.5mg/kg every 4 weeks

• Transfusion dependent is defined as an RBC transfusion

requirement of ≥4 units over 8 weeks prior to trial entry

• ESA R/R following ≥8 weeks of weekly epoetin alfa 40,000 U or

darbepoetin alfa 150 mcg (or equivalent) or serum erythropoietin (sEPO) >500 mU/mL

• Supportive care permitted in both arms: RBC transfusions, myeloid

growth factors per investigator discretion as clinically needed and local guidelines single arm,

• pen label

(n = 57); target patient population (n=38) 1° Efficacy: Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 weeks 2° Efficacy: RBC TI ≥24 weeks; time to and duration of RBC TI; hematologic improvement (HI); reduction in RBC burden

IMerge Part 1: Phase 2 Portion

Lower Risk MDS Trials

Targeting different patient populations

IMerge MEDALIST

Geron Company Acceleron/Celgene Imetelstat Drug name Luspatercept Telomerase inhibitor Mechanism of action TGF-b inhibitor Phase 2 (n=38) Phase of development Phase 3 (n=229) active (n=153), placebo (n=76) RS+ or RS- MDS subtype Ring-sideroblasts (RS) RS+ only ≥4 units/8 weeks Minimum transfusion burden for enrollment “Required RBC transfusions” 8 (4-14) Median baseline transfusion burden

# units/8 weeks (range)

5 (1-20) 29% < 4 units

13

ASH 2018 Abstract #463: Imetelstat Treatment Leads to Durable Transfusion Independence (TI) in RBC Transfusion-Dependent (TD), Non-Del(5q) Lower Risk MDS Relapsed/Refractory to Erythropoiesis- Stimulating Agent (ESA) Who Are Lenalidomide (LEN) and HMA Naïve; Steensma D, et.al. ASH 2018 Abstract #1: The Medalist Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept to Treat Anemia in Patients with Very Low-, Low-, or Intermediate- Risk Myelodysplastic Syndromes (MDS) with Ring Sideroblasts (RS) Who Require Red Blood Cell (RBC) Transfusions

IMerge focused on high transfusion burden patients and open to all MDS subtypes

IMerge Part 1: Target Patient Population

Results from ASH abstract

14

ASH 2018 Abstract #463: Imetelstat Treatment Leads to Durable Transfusion Independence (TI) in RBC Transfusion-Dependent (TD), Non-Del(5q) Lower Risk MDS Relapsed/Refractory to Erythropoiesis- Stimulating Agent (ESA) Who Are Lenalidomide (LEN) and HMA Naïve; Steensma D, et.al.

More mature data expected in oral presentation at ASH on December 2, 2018

IMerge Target Patient Population* (n=38)

Baseline median RBC transfusion burden 8.0 units/8 weeks; range 4-14 ≥8-week RBC-TI 37% (14/38) ≥8-week RBC-TI observed across MDS subtypes RARS/RCMD-RS (RS+) 33% Other Patients (RS-) 27% ≥8-week RBC-TI consistent across baseline EPO levels EPO >500 mU/mL 33% EPO ≤500 mU/mL 32%

*Transfusion dependent patients with IPSS Low or Intermediate-1 Risk MDS, who are non-del(5q), R/R to ESAs and naïve to lenalidomide and HMA treatment

ClinicalTrials.gov (NCT02598661)

Safety

Safety profile consistent with previous imetelstat clinical trials in hematologic myeloid malignancies

• Adverse events such as cytopenias,

gastrointestinal or constitutional symptoms and hepatic biochemistry abnormalities

• No new safety signals reported
• Myelosuppression is dose-limiting toxicity
• bserved (managed through dose holds and

modification rules)

• Most cytopenias resolved within 4 weeks

IMerge: 2-Part Phase 2/3 Trial

Part 2 – Phase 3 portion

15 Imetelstat (n = ~115) 7.5mg/kg every 4 weeks Placebo (n = ~55)

• Transfusion dependent is defined as an RBC transfusion requirement of ≥4 units over 8 weeks prior to clinical trial entry
• ESA R/R following ≥8 weeks of weekly epoetin alfa 40,000 U or darbepoetin alfa 150 mcg (or equivalent) or serum

erythropoietin (sEPO) >500 mU/mL

• Supportive care permitted in both arms: RBC transfusions, myeloid growth factors per investigator discretion as

clinically needed and local guidelines Randomize (2:1) Double-blind Transfusion Dependent, Low or Intermediate-1 Risk MDS, Non-del(5q), R/R ESAs, Naïve to Lenalidomide and HMA (n = ~170) 1° Efficacy: Red Blood Cell (RBC) Transfusion Independence (TI) ≥8 weeks 2° Efficacy: RBC TI ≥24 weeks; time to and duration of RBC TI; hematologic improvement (HI); reduction in RBC burden

Current Clinical Trial Design for IMerge Part 2: Phase 3 Portion

Data from Part 1 for target patient population support moving forward with Part 2 Patient screening and enrollment planned to begin mid-year 2019*

ClinicalTrials.gov (NCT02598661)

*Timing dependent upon transfer of imetelstat investigational new drug (IND) sponsorship from Janssen to Geron.

Myelofibrosis (MF)

Disease characteristics

16

• 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

• riginating from the malignant progenitor cell

clone

– Constitutional symptoms (e.g., fever, weight

loss, night sweats, pruritus) present in approximately 35% of patients also thought to be due to cytokines produced by malignant megakaryocytes

– Impaired bone marrow hematopoiesis shifts

blood production to spleen and liver (palpable splenomegaly in approximately 80% of patients)

• Serious and life-threatening illness

− Leukemic transformation to AML (blast-phase

MF)

− Thrombohemorrhagic complications associated

with dysfunctional hematopoiesis

− Median survival: ~1-3 years for intermediate-2 or

high-risk disease

telomerase

Tefferi, JCO 2005; 23:8520-8530 Tefferi, Mayo Clin Proc 2012; 87:25-33 Gangat et al, JCO 2011; 29:392-397

MF Patient Population in the U.S.

Addressing an underserved market

~70%

• f MF patients have

Intermediate-2 or High-risk MF

13,000

MF patients in the U.S.

3,000

Cases diagnosed annually in the U.S.

17

Mehta et al, Leuk Lymphoma 2014; 55:595-600 Gangat et al, JCO 2011; 29:392-397

75%

5-year ruxolitinib discontinuation rate

18

Ruxolitinib

• Primarily for symptoms
• r splenomegaly
• Oral JAK1/JAK2 inhibitor
• Only approved product

for MF in U.S./Europe

• Stay on drug as long as

tolerated

• Conventional drugs

viewed as ineffective, especially in advanced disease

Harrison et al, ASH 2015; Gupta et al, ASCO 2016 Newberry et al, Blood 2017; 130:1125-1131 Kuykendall et al, Ann Hematol 2018; 97:435-441 Spiegel et al, Blood Advances 2017; 1:1729-1738

Current Treatments for Int-2/High-Risk MF

No approved drug for patients relapsed/refractory to ruxolitinib

Primary reasons:

• Suboptimal response
• Loss of therapeutic effect

Median Overall Survival is ~14-16 months

After discontinuation of ruxolitinib

Investigational Agents (e.g., imetelstat)

19

Trial Population:

• Patients with Intermediate-2 or High-risk MF
• Relapsed or refractory (R/R) to JAKi treatment

Key Goals:

• Define appropriate dosing (9.4 or 4.7 mg/kg every 3 weeks)
• Confirm safety and clinical benefit in this high unmet need population

ClincialTrials.gov (NCT02426086)

Intermediate-2 or High-risk MF R/R to JAKi treatment

Randomize (1:1)

Imetelstat 9.4 mg/kg every 3 weeks Imetelstat 4.7 mg/kg every 3 weeks Co-1° Efficacy: Spleen response rate and symptom response rate 2° Efficacy: CR, PR and CI, anemia response per 2013 IWG-MRT criteria, duration of responses,

• verall survival (OS)

Exploratory: Cytogenetic and molecular responses, leukemia free survival

IMbark Phase 2 Trial

Rigorously defined relapsed/refractory MF patients

First trial requiring patients to meet rigorous definition of relapsed/refractory to JAKi

• Objective evidence of disease progression during or after treatment with a JAKi through worsening splenomegaly
• r never responded to JAKi treatment through no reduction in spleen size after 12 weeks of JAKi therapy
• Active symptoms of MF with minimum symptom score of 5 out of 10

Patient demographic highlights: 1) Considerable proportion of DIPSS high-risk MF

• 41% (44/107)
• These patients have a high risk for leukemic transformation

2) Sizeable triple negative patient population

• 25% (26/105)
• Triple negative means an absence of JAK2, MPL or CALR mutations
• Triple negative patients have poor overall survival, an increased risk of leukemic transformation and do not

respond well to existing therapies

3) Significant number of high molecular risk patients

• 68% (71/105)
• Presence of at least 1 mutation in high-molecular risk genes, ASXL1, EZH2, IDH1/2 and SRSF2
• These genes have been associated with inferior prognosis

ClincialTrials.gov (NCT02426086)

IMbark Results from ASH Abstract

Eligibility criteria yields poor-prognosis MF patient population

20

ASH 2018 Abstract #685: Imetelstat is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels; Mascarhenas, J., et al.

IMbark Results from ASH Abstract

Efficacy results – primary endpoints

21

4.7 mg/kg (n=48) 9.4 mg/kg (n59) Spleen Response 0% 10% (6/59)

ASH 2018 Abstract #685: Imetelstat is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels; Mascarhenas, J., et al.

Spleen Response: proportion of patients who achieve a ≥35% reduction in spleen volume assessed by imaging at 24 weeks in comparison to baseline Symptom Response: proportion of patients who achieve a ≥50% reduction in Total Symptom Score at 24 weeks in comparison to baseline

ClincialTrials.gov (NCT02426086)

SVR Per IRC at Week 24 Symptom Response based on TSS at Week 24

4.7 mg/kg (n=48) 9.4 mg/kg (n59) Symptom Response 6% (3/48) 32% (19/59)

IMbark Results from ASH Abstract

Efficacy results – overall survival

Median follow-up: 22.6 mos (range: 0.2 – 27.4)

22

4.7 mg/kg (n=48) 9.4 mg/kg (n59) Median Overall Survival 20 mos not reached

ASH 2018 Abstract #685: Imetelstat is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels; Mascarhenas, J., et al.

Median Overall Survival: length of time from trial enrollment where half of the assessable patients in a group are still alive

Additional Overall Survival Analyses:

1) Improvement in OS for the 9.4 mg/kg arm does not appear to be due to post-imetelstat

intervention with either JAKi or allogeneic stem cell transplant

• A sensitivity analysis censoring patients at the time of subsequent JAKi therapy or stem cell

transplant resulted in a median OS that had still not been reached

2) Triple negative patients in the 9.4 mg/kg dosing arm showed promising overall survival despite

this subpopulation being difficult to treat using current available therapy

ClincialTrials.gov (NCT02426086)

Most Common Adverse Event on Treatment (all grades) 4.7 mg/kg (n=48) 9.4 mg/kg (n=59)

Thrombocytopenia 23% 49% Anemia 31% 44% Neutropenia NR 36% Diarrhea 38% NR Nausea 31% 34%

IMbark Results from ASH Abstract

Safety results

23

ASH 2018 Abstract #685: Imetelstat is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels; Mascarhenas, J., et al.

Grade 3/4 4.7 mg/kg (n=48) 9.4 mg/kg (n=59)

Thrombocytopenia 29% 42% Neutropenia 13% 34%

NR: not reported in abstract ClincialTrials.gov (NCT02426086)

Safety

Safety profile consistent with previous imetelstat clinical trials in hematologic myeloid malignancies

• Adverse events such as cytopenias,

gastrointestinal or constitutional symptoms and hepatic biochemistry abnormalities

• No new safety signals reported
• Myelosuppression is dose-limiting toxicity
• bserved (managed through dose holds and

modification rules)

• Most cytopenias resolved within 4 weeks

IMbark Results

Key conclusions

Clinical activity demonstrated in 9.4 mg/kg dosing arm

• No formal clinical trial conducted previously for patients R/R to JAKi
• Potential improvement in survival observed
• Median OS for previously treated JAKi patients reported to be 12 – 14 mos (Kuykendall Ann Hematol

2018; Newberry Blood 2017)

Safety profile considered acceptable for this poor-prognosis population Data warrant further exploration in future studies Geron’s plan in MF

• Discuss IMbark results with MF experts and regulatory authorities to explore potential for late-stage

development in MF

• Expect to outline a decision by end of third quarter of 2019

24

ASH 2018 Abstract #685: Imetelstat is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels; Mascarhenas, J., et al.

More mature data from extension phase of IMbark, including updated median

• verall survival for the 9.4 mg/kg arm, expected in oral presentation at ASH on

December 3, 2018

ClincialTrials.gov (NCT02426086)

Upcoming Milestones

25

Clinical Development Plans

Discussions with MF experts and regulatory authorities to determine next steps Start of first quarter 2019 Imetelstat IND transfers from Janssen to Geron Second quarter 2019 Initiate screening and enrollment for Phase 3 portion of IMerge By mid-year 2019 Outline decision regarding potential late- stage development in MF End of third quarter 2019

✓

Data Presentations

ASH abstracts published online November 1, 2018 IMerge Phase 2 data presentation at ASH December 2, 2018 IMbark Phase 2 data presentation at ASH December 3, 2018 Analyst and investor event December 10, 2018

Thank you

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