Building a Leading Oncology Franchise

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1 ASDAQ: MEIP Building a Leading Oncology Franchise 17th Annual eedham Healthcare Conference March 27, 2018

2 Forward-Looking Statements This presentation contains, and our officers and representatives may from time to time make, statements that are forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of Examples of forward-looking statements include, among others, statements regarding our development strategy; potential advantages of our product candidates; the initiation and completion of preclinical and clinical studies and the reporting of the results thereof; the timing of regulatory submissions and actions; the sufficiency of our existing cash; and all other statements relating to our plans, objectives, expectations and beliefs regarding future performance, operations, financial condition and other future events (including assumptions underlying or relating to any of the foregoing). These forward-looking statements rely on a number of assumptions concerning future events and are subject to a number of risks, uncertainties, and other factors, many of which are outside of our control. Important factors that could cause our actual results and financial condition to differ materially from those indicated in forward-looking statements include, among others: uncertainties relating to the initiation and completion of preclinical and clinical studies; whether preclinical and clinical study results will validate and support the safety and efficacy of our product candidates; the outcome of regulatory reviews of our product candidates; varying interpretation of research and development and market data; risks and uncertainties relating to intellectual property and the other factors discussed under the caption Item 1A. Risk Factors in our most recent annual report on Form 10-K and our most recent quarterly report on Form 10-Q. Any forward-looking statement made by us in this presentation is based only on information currently available to us and speaks only as of the date on which it is made. In addition, we operate in a highly competitive and rapidly changing environment, and new risks may arise. Accordingly, you should not place any reliance on forward-looking statements as a prediction of actual results. We disclaim any intention to, and undertake no obligation to, update or revise any forward-looking statement. You are urged to carefully review and consider the various disclosures in our most recent annual report on Form 10-K, our most recent Form 10-Q and our other public filings with the SEC since the filing of our most recent annual report. 2

3 Building a Leading Oncology Franchise 4 clinical oncology programs leveraging development and oncology expertise 1 Program in Phase 3 registration Second program planned to enter registration study 2H 2018 Multiple near term milestones 3 data readouts 1H study initiation 1H 2018 Efficient business strategy MEI building value in wholly-owned programs Strategic partnerships support late-stage asset development Strong financial position $42M at YE

4 Diverse Clinical Pipeline DRUG CADIDATE IDICATIO / COMBIATIO PRE-CLIICAL CLIICAL PROOF-OF-COCEPT PIVOTAL Pracinostat HDAC Inhibitor ME-401 PI3K Delta Inhibitor Voruciclib Selective CDK Inhibitor ME-344 Mitochondrial Inhibitor Acute Myeloid Leukemia Unfit for intensive chemotherapy Vidaza (azacitidine) Myelodysplastic Syndrome High & very high risk Vidaza (azacitidine) Follicular Lymphoma & CLL Relapsed/refractory Single agent Indolent Lymphoma & DLBCL Relapsed/refractory Rituxan (rituximab) B-Cell Malignancies Relapsed/refractory Single agent HER2- Breast Cancer* Treatment-naïve, early stage Avastin (bevacizumab) REGISTRATIO STUDY * Investigator-sponsored study 4

5 Multiple ear-term Milestones Significant value creation potential in 2018 Pracinostat Data from stage 1 of Phase 2 dose-optimization study in MDS (Q2 2018) ME-401 Data from Phase Ib study in follicular lymphoma & CLL (Q2 2018) Initiate single-agent registration study in R/R follicular lymphoma (2H 2018) Voruciclib Initiate Phase 1 study in R/R B cell malignancies (Q2 2018) ME-344 Data from investigator-sponsored study with Avastin in HER2-negative breast cancer (Q2 2018) Pracinostat, ME-401, voruciclib and ME-344 are investigational agents and have not been approved for commercial use in the U.S. 5

6 ME-401: Differentiated PI3K-d Inhibitor with Potential Best In Class Pharmaceutical Properties

7 ME-401: Differentiated PI3K-d Inhibitor to Meet Unmet Medical eed in R/R Follicular Lymphoma High unmet need in R/R FL 1 Fractured R/R FL treatments denote inadequate options 1,2 Differentiated efficacy valued by physicians Registration Study Initiation in R/R FL ~15,000 will be diagnosed with Follicular Lymphoma in 2018 (U.S.) 3 - Significant number advance to R/R disease 1. MEI Pharma Proprietary Primary Market Research; n=25 U.S. and EU Physicians; 2. MEI Pharma Proprietary Primary Market Research; n=60 U.S. Physicians; 3. (accessed 3/22/18) 7

8 Will Physicians Value a 2 nd Generation PI3K-d Inhibitor? R/R Follicular Lymphoma Still an Unmet Medical eed 4% 4% 28% 48% 16% Low Low Current unmet need in relapsed/refractory FL Low to Moderate Low to Moderate Moderate Moderate Moderate to High Moderate to High High Overall satisfaction with drugs currently available to treat R/R FL 0% 16% 68% 16% 0% High ~2/3: Physicians report high to moderate unmet need >3/4: Physicians report low to moderate satisfaction MEI Pharma Primary Market Research; n=25 U.S. and EU Physicians 8

9 Preference Share What is Most Important to Physicians? Prescribing Decisions Primarily Driven by Efficacy in 3L FL 1 55% 50% 45% 40% 35% 30% 25% 20% 50% 60% 70% 80% 90% Overall Response Rate (ORR) 1. MEI Pharma Primary Market Research; n= 60 Physicians 9

10 What is Different About ME-401? Distinct Chemical Structure as Basis for Differentiated Pharmaceutical Properties ME-401 MEI Pharma ICB50465 Incyte Umbralisib TG Therapeutics Duvelisib Verastem Idelalisib Gilead F O R 3 O Cl O F O R 3 R 4 R 2 F C R 5 O C Z X R 1 H H H Y R 6 H H R 4 H 2 F H 2 R 2 O 10

11 Pharmacodynamic Properties Suggest Potential for Improved Outcomes Distinct Structural Attributes Superior on target residence time t 1/2 ~ 28 hours supports daily dosing Large Volume of Distribution Preferential cellular accumulation Intended Improved Outcome Superior PK/PD attributes results in high potency at low plasma concentrations Potential for significant improvement in therapeutic window Versatility for combination approaches 11

12 Phase Ib Study in Follicular Lymphoma and CLL Key objectives: Efficacious Dose Maximum Tolerated Dose (MTD) Minimum Biologically Effective Dose Dose escalation complete All 3 dose levels achieved >50% efficacy Efficacious dose determined Dose Escalation Start at 60 mg Cohort Expansion If no DLTs and 2 responses in 6 patients, then expand cohort to 12 Dose escalate to MTD Efficacious Dose Single-Agent Registration Study To Initiate: H mbed Combination Studies Initiated 12

13 Phase 1b Study in Follicular Lymphoma and CLL Emerging Safety and Efficacy Data 41 patients enrolled 30 patients received monotherapy 12 at 60 mg, 12 at 120 mg, 6 at 180mg 11 patients received 60 mg MEI-401 in combination with rituximab Median follow-up = 4.8 mo. (range = mo.) 30 patients receiving monotherapy evaluable for efficacy Response rate well in excess of 50% at all dose levels o dose limiting toxicities noted (DLT assessed at d56) Safety and efficacy data submitted for presentation at scientific meeting in Q

14 Voruciclib: A Selective CDK Inhibitor

15 Clinical-Stage Oral CDK Inhibitor Differentiated by MCL1 Suppression Oral CDK inhibitor differentiated by potent inhibition of CDK9 CDK9 suppresses MCL1, a BCL2 inhibitor resistance mechanism Tested in > 70 patients with solid tumors Phase 1/2 study in B cell malignancies and then + venetoclax (Q start) Potential utility in multiple indications and combinations 15

16 Voruciclib: Potential to Overcome Venetoclax Resistance Increased MCL1 is an established venetoclax 1 resistance mechanism Venetoclax inhibits BCL2 but not MCL1 Voruciclib inhibits MCL1 via CDK 9 inhibition Voruciclib inhibits CDK9, 4,6 &1 at low nm concentrations 1 Blood Jun 23;127(25):

17 Voruciclib Sensitivity of CLL Patient Samples at Clinically Achievable Drug Levels Voruciclib was dosed from mg QD in Phase 1 solid tumor studies; MTD 350mg Induces apoptosis at μm in >50 patient-derived CLL samples 1 Phase 1 PK results suggest steady state levels >1.5 μm achievable with 150mg daily dosing Voruciclib, μm 1 PLoS One ov 25;10(11):e

18 Voruciclib/Venetoclax Synergy Confirmed in Preclinical in vivo Studies* Voruciclib and venetoclax synergy supports opportunities across multiple indications * U2932 diffuse large B-cell lymphoma (DLBCL) model 18

19 Voruciclib + Venetoclax ot Only a CLL Story: Opportunities in Double-Hit Lymphoma Subtype of diffuse large B-cell lymphoma (DLBCL) characterized by rearrangements of MYC and BCL2 (most common) or BCL6 Poor prognosis, significant unmet need Voruciclib inhibits MYC protein expression (right) Low response rate to BCL2 inhibitor venetoclax as monotherapy in DLBCL (18% ORR; n = 34) 1 Voruciclib synergistic with venetoclax in multiple DLBCL models 2 1 J Clin Oncol Mar 10;35(8): ; 2 Sci Rep :7(1):

20 Phase 1 Study Initiation Q Relapsed/Refractory B cell Malignancies Standard 3-6 patients/group (3+3) Endpoints Safety and tolerability Pharmacokinetics Biologic correlative studies Objective response rates/mrd negativity Voruciclib single agent Dose escalation 50 mg 100 mg 150 mg 200 mg 250 mg Venetoclax + Voruciclib dose escalation 50 mg 100 mg 150 mg 200 mg 250 mg 20

21 ME-344: A ovel Tumor Selective Mitochondrial Inhibitor

22 ME-344: A ovel Mitochondrial Inhibitor with the Potential to Exploit Antiangiogenic Escape Antiangiogenics resistance is a major challenge in cancer therapeutics VEGF inhibition can shift tumor glycolytic reliance towards mitochondrial dependence 1,2 Single agent activity in Phase 1 dose-escalation study in refractory solid tumors 4 Targeting the metabolic escape path would open an important therapeutic opportunity ME-344, as a mitochondrial inhibitor, induces rapid loss of cellular energy (ATP) Proof-of-Concept may support application to additional tumor types where VEGF inhibition applicable Investigator-sponsored study of ME-344 with Avastin in HER2-negative breast cancer 1 J PharmacolExpTher Aug;358(2): ; 2 Cell Rep Jun 21;15(12): ; 3 Am J Cancer Res Jan 15;5(2): ; 4 Cancer Apr 1;121(7): ; 22

23 Clinical Study in HER2-egative Breast Cancer * Interim data review supported study continuation Group A (=20) ME Avastin Biopsy / Analysis Group B (=20) Placebo + Avastin Interim data expected Q Biopsy on Day 28 / Analysis Prospective, randomized study being conducted at 8 sites in Madrid, Spain Study Objectives: Assess ability of Avastin to shift tumor reliance from glycolysis to mitochondrial metabolism Access ability of ME Avastin to inhibit tumor proliferation compared to Avastin + placebo * Sponsored by Spanish ational Cancer Research Centre 23

24 Pracinostat: Potential Best-in- Class Phase 3 HDAC Inhibitor

25 Pracinostat: Orally Available HDAC Inhibitor with Best-in-Class Potential Ongoing Phase 3 AML registration study FDA breakthrough therapy designation Ongoing Phase 2 MDS study Improved pharmacokinetics compared to approved drugs Increased bio-availability Increased half-life Highly active primary metabolite Evaluated in >300 patients Helsinn global development and commercialization partnership Unmet eeds in AML & MDS AML: Treating older AML patients challenged by difficulty in predicting risk and benefit MDS: Limited reliable options to treat high and very high risk MDS 25

26 Potential Class Leading Survival Benefit in AML Breakthrough Therapy Designation by FDA Phase 2 Study: Pracinostat + azacitidine in elderly patients with newly diagnosed AML, not candidates for induction chemotherapy Endpoint Pracinostat + Azacitindine (=50) CR rate 42% 60-day mortality rate 10% Duration of Response (CR/CRi) 17.2 months (95%CI: ) 1-year survival rate 62% Median overall survival 19.1 months (95%CI: ) Pracinostat + azacitidine was generally well tolerated: Most common grade 3/4 treatment-emergent adverse events in 25% of patients included febrile neutropenia, thrombocytopenia, anemia and fatigue 26

27 Pivotal Phase 3 Study in AML: First Patient Dosed in July 2017 ewly Diagnosed AML Patients Unfit to Receive Induction Therapy Group A (=~250) Pracinostat + Azacitidine Group B (=~250) Placebo + Azacitidine Randomized, double-blind, placebo-controlled study to enroll up to 500 patients Primary endpoint: Overall survival Secondary endpoints: Morphologic CR rate, event free survival & duration of CR Inclusion criteria: ewly diagnosed AML patients 18 years who are unfit to receive intensive remission induction chemotherapy due to age ( 75 years) or comorbidities 27

28 Phase 2 Dose-Optimization Study in MDS Potential Expansion to a second indication Patients with High and Very High Risk MDS Previously Untreated with Hypomethylating Agents Stage 1 (~32) Pracinostat (45 mg) + Azacitidine If rate of discontinuation (for reasons other than progressive disease) in first 3 cycles 20%, proceed to Stage 2 Stage 2: Group A (=40) Pracinostat + Azacitidine Stage 2: Group B (=40) Placebo + Azacitidine Two-stage study: sites in stage 1; approximately 25 sites in stage 2 Primary objectives: Safety and tolerability; overall response rate (ORR) 28

29 Multiple ear-term Milestones Significant value creation potential in 2018 Pracinostat Data from stage 1 of Phase 2 dose-optimization study in MDS (Q2 2018) ME-401 Data from Phase Ib study in follicular lymphoma & CLL (Q2 2018) Initiate single-agent registration study in R/R follicular lymphoma (2H 2018) Voruciclib Initiate Phase 1 study in R/R B cell malignancies (Q2 2018) ME-344 Data from investigator-sponsored study with Avastin in HER2-negative breast cancer (Q2 2018) Pracinostat, ME-401, voruciclib and ME-344 are investigational agents and have not been approved for commercial use in the U.S. 29

30 ASDAQ: MEIP Building a Leading Oncology Franchise 17th Annual eedham Healthcare Conference March 27, 2018

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