[ NASDAQ: MEIP ] Cowen and Company Health Care Conference March 2015

Transcription

1 [ NASDAQ: MEIP ] Cowen and Company Health Care Conference March 2015

2 Forward-Looking Statements These slides and the accompanying oral presentation contain forward-looking statements. Actual events or results may differ materially from those projected in any of such statements. Additional information concerning factors that may cause actual events or results to differ from those projected is contained in MEI Pharma s most recent annual report on Form 10-K and quarterly reports on Form 10-Q, as well as other subsequent filings with the SEC. 2

3 MEI Pharma (Nasdaq: MEIP) San Diego-based oncology company focused on the clinical development of three wholly owned drug candidates Pracinostat: Oral HDAC inhibitor with clinical activity in MDS and AML o Significant activity in combination with azacitidine in elderly AML 3 o Data from randomized Phase II MDS study expected in March 2015 ME-344: Novel mitochondrial inhibitor with single-agent activity o Data from Phase Ib study expected in 2015 PWT143: PI3K delta inhibitor with compelling pre-clinical activity o On track to enter clinic in 1H 2015 Strong intellectual property protection extending past 2028 in US Management team with proven oncology drug development experience

4 Management Team EXECUTIVE MANAGEMENT Daniel Gold, PhD President & Chief Executive Officer Former Chief Scientific Officer & Founder, Favrille Robert Mass, MD Chief Medical Officer Former Head of Medical Affairs, BioOncology, Genentech Thomas Zech Chief Financial Officer Former Chief Financial Officer, Pacira Pharmaceuticals David Urso, JD SVP, Corporate Development & General Counsel Former Principal, Forward Ventures / COO, Tioga Pharmaceuticals Karen Potts, PhD SVP, Regulatory Affairs Former SVP of Regulatory Affairs, Trius Therapeutics BOARD OF DIRECTORS Christine White, MD (Lead Director) Former Head of Global Medical Affairs, Biogen Idec Charles Baltic, JD Co-Head of Healthcare, Needham & Co. Leah Cann, MBA Two-time Wall Street Journal All-Star Analyst Kevan Clemens, PhD Former Head of Global Oncology, Roche Nick Glover, PhD Former President & CEO, YM BioSciences Daniel Gold, PhD President & CEO, MEI Pharma Thomas Reynolds, MD, PhD Former Chief Medical Officer, Seattle Genetics William Rueckert Former Chairman, Novogen Limited 4

5 SIGNALING PROGRAM CANCER METABOLISM PROGRAM EPIGENETICS PROGRAM Clinical Development Pipeline DRUG CANDIDATE INDICATION / COMBINATION PRE-CLINICAL PHASE I PHASE II PHASE III Myelodysplastic Syndrome Front Line, Int-2 & High-Risk Azacitidine (Vidaza ) Pracinostat HDAC Inhibitor ME-344 Mitochondrial Inhibitor Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza ) Myelodysplastic Syndrome Refractory to HMA Azacitidine (Vidaza ) or Decitabine (Dacogen ) Myelofibrosis Front Line & Relapsed/Refractory Ruxolitinib (Jakafi ) Small Cell Lung Cancer Advanced or Metastatic Topotecan (Hycamtin ) Ovarian Cancer Advanced or Metastatic Topotecan (Hycamtin ) PWT143 PI3K Delta Inhibitor Hematologic Cancers 5

6 Pracinostat: A Differentiated HDAC Inhibitor Potent inhibitor of Class I, II and IV HDAC isoenzymes Well tolerated Tested in 300+ adult and pediatric patients in multiple hematologic and solid tumor Phase I and Phase II clinic trials Manageable side effects consistent with drugs of this class include fatigue and gastrointestinal discomfort Best-in-class pharmacokinetic profile, broadly active Generation of SB991, the major and highly active in vivo metabolite of Pracinostat, yields in vivo combined on target IC 50 activity for HDAC1 predicted to be >24 hours 6

7 Pracinostat: Clinical Activity in MDS Evidence of activity in combination with azacitidine in intermediate-2 or high-risk myelodysplastic syndrome (MDS) 1 90% (9/10) CR/CRp rate in pilot study o CR = 60% (6/10) Rapid complete bone marrow responses observed o o 50% (5/10) achieved complete cytogenetic bone marrow response 50% (5/10) went on to bone marrow transplantation 7 1 Quintás-Cardama et al. Very high rates of clinical and cytogenetic response with the combination of the histone deacetylase inhibitor Pracinostat (sb939) and 5-azacitidine in high-risk myelodysplastic syndrome ASH Annual Meeting

8 MDS Pilot Study Three Years Later 8

9 Phase II Study in Front Line MDS (MEI-003) Intermediate Risk-2 or High Risk MDS Patients Previously Untreated w/ HMA Pracinostat + Azacitidine Placebo + Azacitidine Primary Endpoint: CR 9 Secondary endpoints: overall response rate, hematologic improvement, clinical benefit rate, duration of response, progression-free survival, rate of leukemic transformation, overall survival, safety & tolerability 102 evaluable patients enrolled, one-to-one randomization 24 sites in the U.S. Expect to unblind study and report top-line data in March 2015

10 Phase II Study in HMA Refractory MDS (MEI-005) Reached clinical milestone in December 2014 Three responses (one PR and two marrow CRs) out of first 28 patients who received Pracinostat + HMA (azacitidine or decitabine) after progressing on the same HMA alone Exceeded the pre-specified response rate (two out of 29) for expansion of study enrollment Combination of Pracinostat + azacitidine or decitabine generally welltolerated in the study with no unexpected toxicities Most common treatment-emergent adverse events: anemia, fatigue and gastrointestinal disorders Study enrollment complete (39 patients); continue to follow patients for response and survival One additional marrow CR to date 10

11 Pracinostat: Clinical Activity in AML Evidence of single-agent activity in elderly AML 1 14% (2/14) CR rate in Phase I dose-escalation study Significant activity in combination with azacitidine in elderly AML 2 50 patients enrolled in Phase II study at 15 sites in the U.S. Interim data from 33 evaluable patients reported at ASH o Primary endpoint: CR/CRi/MLFS rate = 45% (15/33) as of Dec 2014 o No patient who achieved a clinical response had progressed 11 1 Garcia Manero et al. Phase 1 Study of the Oral Deacetylase Inhibitor, SB939, in Patients with Advanced Hematologic Malignancies ASH Annual Meeting 2 Garcia-Manero et al. Pracinostat in Combination with Azacitidine Produces a High Rate and Rapid Onset of Disease Remission in Patients with Previously Untreated Acute Myeloid Leukemia ASH Annual Meeting

12 1 st Line Elderly AML Patients (N=33) Phase II Study in Front Line AML (MEI-004) Duration on Study and Response ASH 2014 Response Based on Clinical Review of Efficacy Data CR CRi MLFS PR/PRi Stable Disease Progressive Disease Clinical Benefit AE/Withdrew No Clinical Benefit AE/Withdrew Time to 1 st BM Assessment for Responders Remains on treatment Days on Study 12

13 1 st Line Elderly AML Patients (N=33) Phase II Study in Front Line AML (MEI-004) Duration on Study and Response Today Response Based on Clinical Review of Efficacy Data * CR CRi * * * * MLFS PR/PRi Stable Disease Progressive Disease Clinical Benefit AE/Withdrew No Clinical Benefit AE/Withdrew Time to 1 st BM Assessment for Responders Remains on treatment Months on Study 13 * Patients with improved response since presentation at ASH 2014

14 Phase II Study in Front Line AML: Conclusions Significant clinical activity in elderly patients w/ newly diagnosed AML 52% (17/33) have now achieved primary endpoint o CR = 33% (11/33) to date Majority of clinical responses occur within first two cycles Observed response rate continues to increase with longer follow-up Only one patient who has achieved clinical benefit (SD) has progressed 60-day mortality rate approximately 10% (3/33) Well tolerated in this population of elderly AML patients ( 65 years) To date 12 patients on study > 6 months, reflecting long term tolerability Data support definitive development of Pracinostat in combination with azacitidine in elderly AML patients 14

15 Proposed Phase III Study in Front Line AML Elderly (Age 60 years) Patients with Newly Diagnosed AML Unsuitable for Intensive Therapy Pracinostat + Azacitidine Placebo + Azacitidine Primary endpoint to support accelerated approval: CR Endpoint for full approval: OS ~450 patients, one-to-one randomization Estimated initiation: June

16 Global Market Opportunity in MDS and AML MDS and AML are Growing Markets with Limited Treatment Options HSCT is the only curative treatment; however, the vast majority of patients are ineligible candidates Many patients are also unfit for chemotherapy Most patients who are high-risk and/or can t tolerate intensive therapy receive an HMA (e.g. Vidaza or Dacogen ) In 2013 prior to generic entrants, combined worldwide sales of Vidaza and Dacogen exceeded $1B Pracinostat in combination with HMAs may improve treatment outcomes in a large, unmet market 2013 Epidemiology and Treatment Algorithm * # G7 Markets MDS Prevalence & AML Incidence ~ K Patients High Risk / Int-2 MDS & AML >60 Yrs ~ 50-60K Patients HMA Treated MDS & AML ~ 18-23K Patients Estimate ~30K Pracinostat Target Patients in 7 Key Markets by 2021 General & elderly population growth Increased Rx use in older patients Improved treatment options 16 * Evaluate Pharma # Decision Resources

17 ME-344: Lead Mitochondrial Inhibitor Derived from in-house isoflavone-based technology platform Targets Oxphos pathway resulting in rapid loss of cellular energy and mitochondrial instability via increased ROS Potent inhibitor of multiple human tumor cell lines, including chemotherapy-resistant ovarian cancer stem cells Solid tumor dose-escalation study complete ~ 25% (5/21) experienced progression-free survival (PFS) > 2X longer than prior anti-cancer therapy One confirmed PR in a patient with small cell lung cancer lasting > 2 years Dose limiting toxicity of Grade 3 neuropathy at 15 and 20 mg/kg Generally well tolerated at 10 mg/kg weekly 17

18 Phase Ib Trial of ME-344 Plus Topotecan First Patient Dosed in Cohort Expansion in Oct 2014 Primary Endpoint: Safety & Tolerability of ME Topotecan Patients Eligible for Topotecan Small Cell Lung, Ovarian & Cervical Cancers Confirm MTD (n=14) ME-344 (10 mg/kg weekly) + Topotecan (4 mg/m 2 day 1,8,15) Small Cell Lung Cancer (n=20) ME Topotecan Ovarian Cancer (n=20) ME Topotecan 18

19 PWT143: Highly Selective PI3K Delta Inhibitor Acquired from Pathway Therapeutics in September 2013 Expands drug development pipeline Clinically validated target in hematologic diseases Potential synergies with lead drug candidate Pracinostat Distinct chemical structure and evidence of improved pre-clinical activity compared to other PI3K delta inhibitors in development IND-enabling 2-species tox studies and scale manufacturing methods completed Expect to initiate first-in-human studies in first half of

20 Intellectual Property Pracinostat 3 issued US and 77 issued foreign patents 2 US and 8 foreign applications pending Composition of matter to May 2028 in US, Aug 2026 in EP May 2033 with up to 5 years patent term restoration in US Aug 2031 with up to 5 years Supplementary Protection Certificate in EP ME issued US and 18 issued foreign patents 3 US and 7 foreign applications pending Composition of matter to Sep 2025 in US and EP Sep 2029 with up to 4 years of patent term restoration in US Sep 2030 with up to 5 years Supplementary Protection Certificate in EP PWT143 1 issued US patent 3 US and 29 foreign applications pending Composition of matter to Jan 2031 in US, pending in EP 20

21 Financial Highlights Cash: $78.7 million (as of December 31, 2014) Debt: None Shares outstanding: 33.3 million 21

22 2015 Clinical Milestones Pracinostat Top-line data from Phase II study in front line MDS (March) Full data from Phase II study in front line MDS (June) Full data from Phase II study in front line elderly AML (June) Initiation of Phase III study in front line elderly AML (June) ME-344 Data from Phase Ib trial in small cell lung and ovarian cancers PWT143 Initiation of first-in-human study 22

23 [ NASDAQ: MEIP ] Cowen and Company Health Care Conference March 2015