[ NASDAQ: MEIP ] August 2014

Transcription

1 [ NASDAQ: MEIP ] August 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 focused on the clinical development of three wholly owned drug candidates Pracinostat: Oral HDAC inhibitor with clinical activity in MDS and AML o Data from three Phase II studies anticipated by Q ME-344: Novel mitochondrial inhibitor with single-agent activity o Data from Phase Ib study expected in Q 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 Cash sufficient to fund operations through

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 METABOLISM PROGRAM EPIGENETICS 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 AML: Front Line Elderly patients not suited for induction therapy MDS: Refractory Patients refractory to azacitidine or decitabine 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 Potent inhibitor of Class I, II and IV HDAC isoenzymes Tested in 250+ patients in multiple Phase I and Phase II clinic trials Hematologic and solid tumor indications, adult and pediatric patients Readily manageable side effects consistent with drugs of this class Best-in-class pharmacokinetic profile, broadly active SB991, the major in vivo metabolite of Pracinostat, demonstrates higher activity than Pracinostat Combined on target IC 50 activity for HDAC1 predicted to be >24 hours 6

7 Pracinostat: Clinical Activity in MDS and AML Evidence of single-agent activity in elderly AML* 14% (2/14) CR rate in Phase I dose-escalation study Evidence of activity in combination with azacitidine in MDS # 80% (8/10) CR/CRi rate in pilot Phase II study Rapid complete bone marrow responses observed 50% (5/10) achieved complete cytogenetic bone marrow response 50% (5/10) went on to bone marrow transplantation Evidence of activity in combination with azacitidine in elderly AML 67% (6/9) rapid overall response rate in ongoing Phase II study 7 * 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

8 Pracinostat: Front Line MDS Study (MEI-003) Intermediate Risk-2 or High Risk MDS Patients Previously Untreated w/ HMA n=50 n=50 Pracinostat + Azacitidine Placebo + Azacitidine 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 & tolerability Data expected in Q * 24 sites activated

9 Pracinostat: Front Line AML Study (MEI-004) Elderly (Age 65 Years) Patients w/ Newly Diagnosed AML Not Suited for Intensive Chemotherapy Stage 1 (n=27) Pracinostat + Azacitidine If three or more patients achieve CR/CRi Stage 2 (n=13) Pracinostat + Azacitidine Primary Endpoint: CR + CRi + MLFS Secondary endpoints: overall response rate, complete cytogenetic/molecular response, duration of response, progression-free survival, overall survival, safety & tolerability 9 * 15 sites activated

10 2 (22) 1 (11) Pracinostat: 3 (33) Neutropenic Peripheral fever 2 Blood (22) Count 1 (11) Dynamics Only toxicities reported in at least one patient are shown 1 (11) in Pilot MDS Study 1 (11) 1 (11) 9 (100) 4 (44) 3 (33) 1(11) 1 (11) 7 (77) 4 (44) 3 (33) 2 (22) 1 (11) 1 (11) Hypomagnesemia 2 (22) 0 Thrombocytopenia 2 (22) 2 (22) Peripheral Blood Count Dynamics s. fficacy and G. Garcia-Manero, Only et al. counts Very high during rates the of first clinical 3 cycles and cytogenetic ( C ) of therapy response are with shown the combination of the histone deacetylase inhibitor Pracinostat (sb939) and 5-azacitidine in high-risk myelodysplastic syndrome ASH Annual Meeting ined as CR + Conclusions

11 Pracinostat: Refractory MDS Study (MEI-005) MDS Patients Who Failed Initial HMA Therapy Primary or Secondary Failures Stage 1 (n=29) Pracinostat + Azacitidine Stable Disease Stage 1 (n=29) Pracinostat + Azacitidine If two or more patients respond Stage 2 (n=9) Pracinostat + Azacitidine Stage 2 (n=9) Pracinostat + Azacitidine Primary Endpoint: Clinical Improvement Rate (CR + PR + HI) Secondary endpoints: overall response rate, complete response, hematologic improvement, duration of response, progression-free survival, time to progression, overall survival, safety & tolerability 11 * 16 sites activated

12 Pracinostat: Market Opportunity in MDS & AML MDS and AML are growing markets with limited treatment options 12 * Evaluate Pharma

13 Pracinostat: Potential in Solid Tumors Data Presented at AACR in April 2014 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 13 * 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

14 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 cells 14

15 ME-344: Lead Mitochondrial Inhibitor 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 PR in a patient with small cell lung cancer (104+ weeks) 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 15

16 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 104 Duration of Last Prior Therapy Duration of ME * Patients Achieving a PR or SD; As of August 8, 2014

17 Phase Ib Trial of ME-344 Plus Topotecan Data Anticipated in Q 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) Small Cell Lung Cancer (n=20) ME Topotecan Platinum Refractory Ovarian Cancer (n=20) ME Topotecan 17 * 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

18 Determination of Sensitivity & Resistance to ME-344 Why Do Cells Die? Sensitive Human Colorectal Cell Lines: IC 50 > 100nM Resistant Human Colorectal Cell Lines: IC 50 < 50uM 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 Scale manufacturing methods completed Anticipate completion of IND-enabling 2-species tox studies by end 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: $54.7 million (as of 3/31/14) Sufficient to fund operations through 2015 Debt: None Common stock outstanding: 21.6 million 21

22 Clinical Milestones Pracinostat Initiation of Phase II trial in refractory MDS Orphan Drug Designation for AML Stage one CR/CRi rate met in front line AML trial Completion of enrollment in front line MDS trial (Q3 2014) Stage one responses in refractory MDS trial (2H 2014) Additional data from front line AML trial (ASH 2014) Top line data from front line MDS trial (Q1 2015) ME-344 Initiation of Phase Ib trial + Topotecan MTD established/cohort expansion in Phase Ib trial (2H 2014) Data from Phase Ib trial in small cell lung & ovarian cancers (Q2 2015) PWT143 Initiation of first-in-human study (1H 2015) 22

23 [ NASDAQ: MEIP ] August 2014