[ NASDAQ: MEIP ] Needham Healthcare Conference April 8-9, 2014

Transcription

1 [ NASDAQ: MEIP ] Needham Healthcare Conference April 8-9, 2014

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 with comprehensive clinical development programs in hematology and solid tumors Epigenetic Modification Cancer Cell Metabolism Signaling Data from ongoing Phase II trials of lead drug candidate Pracinostat anticipated in 2014 Single-agent activity from first-in-human trial of ME-344 Strong intellectual property protection extending past 2025 Exclusive worldwide rights to entire pipeline of drug candidates 3

4 Management Team EXECUTIVE MANAGEMENT Daniel Gold, PhD President & Chief Executive Officer Former Chief Scientific Officer & Founder of Favrille Robert Mass, MD Chief Medical Officer Former Head of Medical Affairs, BioOncology at Genentech Thomas Zech Chief Financial Officer Former Chief Financial Officer at Pacira Pharmaceuticals David Urso Senior Vice President, Corporate Development & General Counsel Former Principal, Forward Ventures & COO, Tioga Pharmaceuticals Wendy Levin, MD, MS Vice President, Clinical Development & Medical Affairs Former Director, Clinical Development Oncology/Hematology at Pfizer Ofir Moreno, PhD Vice President, Research & Development Former Chemist at Dendreon, Corvas, Amgen & Merck Kelly Powell Vice President, Business Development Former Business Development at Amylin Pharmaceuticals Pete De Spain Vice President, Investor Relations Former IR at Prometheus, Favrille, Anadys & Sequenom BOARD OF DIRECTORS Christine White, MD Lead Director Former Head of Global Medical Affairs, Biogen Idec Charles Baltic, JD Co-Head of Healthcare at Needham & Co. Leah Cann, MBA Two-time Wall Street Journal All-Star Analyst Nick Glover, PhD Former President & CEO of YM BioSciences Daniel Gold, PhD President & CEO of MEI Pharma Thomas Reynolds, MD, PhD Former Chief Medical Officer, Seattle Genetics William Rueckert Former Chairman of Novogen Limited 4

5 SIGNALLING PROGRAM CANCER METOBOLISM PROGRAM EPIGENTICS PROGRAM Clinical Development Pipeline DRUG CANDIDATE INDICATION PRE-CLINICAL PHASE I PHASE II PHASE III MDS: Front Line Intermediate-2 and high-risk patients Pracinostat HDAC Inhibitor MDS: Refractory Patients refractory to Vidaza or Dacogen AML: Front Line Elderly patients not suited for induction therapy ME-344 Mitochondrial Inhibitor Small Cell Lung Cancer / Ovarian Cancer PWT143 PI3K Delta Inhibitor Hematologic Cancers 5

6 Pracinostat: Potential Best-in-Class Oral HDAC Inhibitor Tested in 200+ patients in multiple Phase I & Phase II clinic trials Hematologic & solid tumor indications, adult & pediatric patients Readily manageable side effects consistent with drugs of this class Best-in-class pharmacokinetic profile, broadly active Clinical evidence of single-agent activity in elderly AML* 14% (2/14) CR rate in Phase I dose-escalation trial Clinical evidence of synergistic activity in combination with Vidaza (azacitidine) in myelodysplastic syndrome (MDS) # 8 out of 10 patients achieved CR or CRi 5 out of 10 patients achieved complete cytogenetic bone marrow response 5 out of 10 patients went on to bone marrow transplantation 6 * G. Garcia Manero, et al. Phase 1 Study of the Oral Deacetylase Inhibitor, SB939, in Patients with Advanced Hematologic Malignancies ASH Annual Meeting # G. Garcia-Manero, 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

7 Pracinostat: Front Line MDS Study Intermediate-2 and High-Risk Patients MDS Patients Previously Untreated Intermediate-2 or High Risk Group 1 (n=50) Pracinostat plus Vidaza Group 2 (n=50) Placebo plus Vidaza Primary Endpoint: CR Secondary endpoints: overall response rate, hematologic improvement, clinical benefit rate, duration of response, progression-free survival, rate of leukemic transformation, overall survival & safety profile Expect to complete enrollment in Q3 2014, unblind study in Q

8 Pracinostat: Refractory MDS Study Failure After Initial HMA Therapy MDS Patients Failure to Initial HMA Therapy (Vidaza or Dacogen ) Group 1a (n=29) Primary or Secondary Failures Group 2a (n=29) Stable Disease Add Pracinostat to Initial HMA Therapy (Vidaza or Dacogen ) If two or more patients respond, enroll stage 2 Group 1b (n=9) Primary or Secondary Failures Group 2b (n=9) Stable Disease Primary Endpoint: Clinical Improvement Rate (CR + PR + HI) Preliminary data expected at ASH in December

9 Pracinostat: Front Line AML Study Elderly Patients Not Suited for Intensive Chemotherapy Elderly (Age 65 Years) Patients with Newly Diagnosed AML Stage 1 (n=27) Pracinostat plus Vidaza If three or more patients respond, enroll Stage 2 Stage 2 (n=13) Pracinostat plus Vidaza Primary Endpoint: CR + CRi + morphologic leukemia free state Preliminary data expected at ASH in December

10 HDAC + HMA Single-Arm Combination Studies HDAC Inhibitor HMA N ORR CR+CRi Pracinostat Vidaza 10 90% 80% Panobinostat 1 Vidaza 26 31% 8% Panobinostat 2 Vidaza 16 50% 25% Mocetinostat 3 Vidaza 20 61% 50% Entinostat 4 Vidaza 27 44% 7% Vorinostat 5 Vidaza 40 75% 35% Vorinostat 6 Dacogen 61 21% 15% Vorinostat 7 Vidaza 11 82% 55% Valproic Acid 8 Vidaza 65 44% 22% Valproic Acid 9 Dacogen 54 22% 22% Valproic Acid 10 Vidaza 53 42% 28% 10 1 Tan PT, Blood 2011; 118: Abstract Ottmann OG, Blood 2011; 118: Abstract Luger SM, J Clin Oncol 2013; 31: Abstract Gore SD, Blood 2006; 108: Abstract Silverman LR, Blood 2013; 122: Abstract Kirschbaum M, Blood 2009; 114: Abstract Silverman LR, J Clin Oncol 2008; 26; Abstract Raffoux E, OncoTarget 2010 May;1: Garcia-Manero G, Blood 2006 Nov;108: Soriano AO, Blood 2007 Oct;110:

11 Pracinostat & SB991 HDAC1 Inhibitory Activity Degradation of DNMT1* 250 kda 150 kda 100 kda 75 kda 50 kda 37 kda DNMT1 75 kda 50 kda 37 kda actin 11 * MDA-MB-231 human breast cancer cells were treated for 24 hours with HDACi as shown. Protein levels were assayed by Western blotting.

12 Plasma concentration (nm) Pracinostat: Enhanced PK and Exposure in Clinical Studies 1000 HDAC Time (h) 12

13 Pracinostat & SB991 HDAC6 Inhibitory Activity Acetylation of α-tubulin 13

14 Pracinostat: Market Opportunity MDS and AML are growing markets with limited treatment options 14 * Evaluate Pharma

15 Pracinostat: Potential in Solid Tumors Data Presented at AACR on Sunday* Activated Transcription Factor 3 (ATF-3) acts as a tumor suppressor in certain human tumors, including bladder and colon cancer Decreased ATF-3 expression in bladder cancer is associated with tumor progression and a reduced survival rate in patients Pracinostat treatment of human bladder cancer cells in vitro results in reactivation of ATF-3 expression and inhibition of colony formation and cell growth Pracinostat reactivation of ATF-3 expression is observed in xenograft model and correlates with tumor response 15 * D Sooraj, et al. Activated Transcription Factor 3 (ATF-3) Expression is a Potential Marker of Tumor Response to the HDAC Inhibitor Pracinostat AACR Annual Meeting

16 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 Decreased ATP induces activation of AMP kinase leading to dual mtor / AKT inhibition Potent inhibitor of multiple human tumor cell lines, including chemotherapy-resistant ovarian cancer stem cell 16

17 ME-344: First-in-Human Clinical Study Dose-escalation study in patients with solid refractory tumors 38% (8/21) of evaluable patients achieved stable disease (SD) or better One confirmed partial remission (PR) in a patient with small cell lung cancer (SCLC) Seven patients with SD as best response Generally well tolerated at 10 mg/kg weekly Dose limiting toxicity of Grade 3 neuropathy at 15 and 20 mg/kg 17

18 ME-344: First-in-Human Clinical Study Duration of Prior Therapy Compared to ME-344* Small Cell Lung Cancer Carcinoid of the Ileum Urothelial Cervical Cervical Leiomyosarcoma Non-Small Cell Lung Ovarian Non-Small Cell Lung Weeks Duration of Last Prior Therapy Duration of ME * Patients Achieving a PR or SD; As of April 1, 2014

19 Phase Ib Trial of ME-344 Plus Topotecan Expected to Initiate this Quarter Primary Endpoint: Safety & Tolerability of ME Topotecan Patients Eligible for Topotecan Small Cell Lung, Ovarian & Cervical Cancers Establish MTD (n=12) ME-344* (10 mg/kg weekly) + Topotecan (4 mg/m 2 day 1,8,15) * If DLT s are noted in > 33% (5/12) patients, ME-344 will be reduced to 5 mg/kg and evaluated in another 12 patients Small Cell Lung Cancer (n=20) ME Topotecan Platinum Refractory Ovarian Cancer (n=20) ME Topotecan 19

20 Determination of Sensitivity & Resistance to ME-344 Sensitive Human Colorectal Cell Lines: IC 50 <100nM Resistant Human Colorectal Cell Lines: IC 50 <50uM 20

21 PWT143: Highly Selective PI3K Delta Inhibitor Acquired from Pathway Therapeutics in September 2013 Expands drug development pipeline, footprint in hematology Clinically validated target in hematologic diseases Distinct chemical structure & evidence of improved pre-clinical activity compared to other PI3K delta inhibitors in development Potential synergies with lead drug candidate Pracinostat Minimal investment required to complete IND-enabling studies Anticipate completion of IND-enabling studies by end of

22 PWT143: Potent Inhibitor of Leukemia and Lymphoma Cell Lines Cell Line Tumor Type IC50 (um) in Proliferation Assays PWT143 CAL-101 PCI Daudi Burkitt s Lymphoma Ramos Burkitt s Lymphoma Farage DLBCL (GCB) SU-DHL-5 DLBCL (GCB) WSU-NHL DLBCL (GCB) or FL RL DLBCL (GCB) SU-DHL-10 DLBCL (GCB) no data U-2932 DLBCL (ABC) no data SR Lymphoma (indeterminate origin) 1.46 > GRANTA-519 Mantle cell lymphoma ND HL-60 Promyelocytic leukemia CCRF-CEM T-cell lymphoblastic leukemia MOLT-4 T-cell lymphoblastic leukemia 1.40 > CCRF-SB B-cell lymphoblastic leukemia

23 Intellectual Property Pracinostat Three issued U.S. and 74 issued foreign patents Three U.S. and 12 foreign applications pending Composition of matter to 2028, methods of use to 2025 in U.S. ME-344 Two issued U.S. and 18 issued foreign patents Three U.S. and seven foreign applications pending Composition of matter and methods of use to 2025 PWT143 One issued U.S. patent One U.S. and 15 foreign applications pending Composition of matter to 2031, methods of use expected to 2032 in U.S. 23

24 Financial Highlights Cash: $59.8 million (as of 12/31/13) Sufficient to fund operations through 2015 Debt: None Common stock outstanding: 21.6 million Fully diluted shares: 27.9 million Warrants: 4.9 million shares Weighted average strike price: $

25 2014 Clinical Milestones Pracinostat Initiation of Phase II trial in refractory MDS Orphan Drug Designation for AML Completion of enrollment in front line MDS trial (Q3 2014) Stage I responses anticipated in refractory MDS trial (Q3 2014) Stage I responses anticipated in front line AML trial (Q3 2014) Preliminary data from front line AML trial (ASH 2014) Preliminary data from refractory MDS trial (ASH 2014) Top line data from front line MDS trial (Q1 2015) ME-344 Initiation of Phase Ib trial in small cell lung & ovarian cancer (Q2 2014) PWT143 Completion of IND-enabling studies (Year End 2014) 25

26 [ NASDAQ: MEIP ] Needham Healthcare Conference April 8-9, 2014