Lite Strategy, Inc. · NASDAQ:LITS
Lite Strategy — MEI Pharma — Investor Day (December 2014)
31 slides from Lite Strategy's investor day presentation, Dec 9, 2014
Slide 1: Analyst & Investor Event December 8, 2014
Exhibit 99.1 [ NASDAQ: MEIP ] Analyst & Investor Event December 8, 2014
Slide 2: Forward-Looking Statements
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 diffe
Slide 3: Agenda
Agenda Time Presentation Speaker 6:00 pm Welcome & Introduction Daniel P. Gold, PhD 6:05 pm Pracinostat: A Differentiated HDAC Inhibitor Guillermo Garcia-Manero, MD Update on MDS Pilot Study Guillermo Garcia-Manero, MD Review of Front Line AML Data Guillermo Garcia-Manero, MD 6:25 pm Next Steps for
Slide 4: Introductions
Introductions Guillermo Garcia-Manero, MD Professor, Professor,Department Departmentof ofLeukemia, Leukemia,MD MDAnderson AndersonCancer CancerCenter Center Chief,Section Chief, SectionofofMyelodysplastic MyelodysplasticSyndromes, Syndromes,MD MDAnderson AndersonCancer Cancer Center Center Deputy Ch
Slide 5: Clinical Development Pipeline
Clinical Development Pipeline DRUG CANDIDATE INDICATION / COMBINATION PRE-CLINICAL PHASE I PHASE II PHASE III MyelodysplasticSyndrome Front Line, Int-2 & High-Risk Azacitidine (Vidaza®) Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Pracinostat HDAC Inhibitor MyelodysplasticSyndrom
Slide 6: Pracinostat: A Clinical Perspective
Pracinostat: A Clinical Perspective Guillermo Garcia-Manero, MD MD Anderson Cancer Center
Slide 7: Pracinostat: A Differentiated HDAC Inhibitor
Pracinostat: A Differentiated HDAC Inhibitor • Potent inhibitor of Class I, II and IV HDAC isoenzymes • Tested in 300+ patients in multiple Phase I and Phase II clinic trials Hematologic and solid tumor indications, adult and pediatric patients Well tolerated Manageable side effects consistent with
Slide 8: Evidence of Clinical Activity in MDS and AML
Evidence of Clinical Activity in MDS and AML • Evidence of single-agent activity in elderly AML 1 14% (2/14) CR rate in Phase I dose-escalation study • Evidence of activity in combination with azacitidine in MDS 2 80% (8/10) CR/CRi rate in pilot Phase II study Rapid complete bone marrow responses ob
Slide 9: MDS Pilot Study – Patient Characteristics
MDS Pilot Study – Patient Characteristics Patient 1 2 3 4 5 6 7 8 9 10 Median (range) Age (years) 71 71 63 57 22 70 18 71 52 66 64.5 (18-71) Sex F M F F F M M F F F % BM blasts 18 8 2 9 3 3 6 1 12 9 7 (1-18) Hemoglobin g/dL 11 9.3 10.9 9.5 10.2 9.2 8.2 10.7 11.1 9.9 10.05 (8.2-11.1) WBC K/uL 3.2 1.5
Slide 10: MDS Pilot Study… Three Years Later
MDS Pilot Study… Three Years Later 10
Slide 11: Phase II Study in Front Line AML (MEI-004)
Phase II Study in Front Line AML (MEI-004) Elderly (Age 65 years) Patients with Newly Diagnosed AML Stage 1 (n = 27) Pracinostat plus Azacitidine Three or more patients required to achieve CR/CRi/MLFS to advance to Stage 2 Stage 2 (n = 13) Pracinostat plus Azacitidine AML, acute myeloid leukemia; CR
Slide 12: Treatment Regimen
Treatment Regimen • Pracinostat 60 mg is administered orally 3 days a week (days 1,3 and 5 of each week) for 21 days of each 28 day cycle • Azacitidine is administered subcutaneously or intravenously on days 1-7 or days 1-5 & 8-9 (per site preference) of each 28-day cycle • Dose Modifications: Reduc
Slide 13: Eligibility Criteria
Eligibility Criteria • Key Inclusion: Age 65 years Newly diagnosed de novo, secondary, or treatment-related AML with intermediate or unfavorable-risk cytogenetics based on the Southwest Oncology Group (SWOG) classifications (Slovak et al, 2000). 20% bone marrow blasts Adequate renal, cardiac and liv
Slide 14: Study Evaluations
Study Evaluations • Primary Endpoint: Complete Response (CR) + Complete Response with Incomplete Blood Count Recovery (CRi) + Marrow CR (Morphologic Leukemia Free State, MLFS) • Secondary Endpoints: Overall response rate (CR + CRi + partial response [PR] + PR with incomplete blood count recovery [PR
Slide 15: Patient Disposition
Patient Disposition 50 patients enrolled between December 2013 – December 2014 at 15 sites 41 patients enrolled at data cutoff N = 41 Number of Patients Active 25 Number of Patients Discontinued 16 Reasons for discontinuation: Progressive Disease 6 Adverse Event 6 Other 4 Other – includes patient or
Slide 16: Baseline Characteristics
Baseline Characteristics Number of Patients at Baseline n = 41 (%) Age (years) Median 76 Range 69 - 84 Gender Male 24 (59) Female 17 (41) AML Disease Status Newly diagnosed de novo 29 (71) Secondary (AHD & treatment related) 12 (29) ECOG Status 0-1 33 (80) 2 8 (20) Bone Marrow Blasts at Baseline Med
Slide 17: Treatment Emergent Adverse Events in 10% of Patients
Treatment Emergent Adverse Events in 10% of Patients (all causality) All Grades (%), n=41 Grades 3-4 (%), n=41 Hematologic Febrile Neutropenia 12 (29) 10 (24) Thrombocytopenia 11 (27) 10 (24) Anemia 9 (22) 4 (10) Neutropenia 4 (10) 4 (10) Leukopenia 4 (10) 1 (2) Non-Hematologic Nausea 18 (44) 2 (5)
Slide 18: Treatment Emergent Adverse Events Leading to Drug Discontinuation
Treatment Emergent Adverse Events Leading to Drug Discontinuation Discontinuation AE Term Grade (Cycle/Day) Outcome Peripheral Motor Neuropathy 3 3/1 Resolved Parainfluenza 3 3/22 Resolved Prolonged QTc/AF 3 2/15 Resolved Subdural Hematoma 5 3/22 Fatal Sepsis 5 2/3 Fatal Sepsis 5 2/14 Fatal AE, adve
Slide 19: Response
Response Interim Response Assessment n=33* (%) CR/CRi/MLFS (Primary endpoint) 15 (45) CR 9 (27) CRi 4 (12) MLFS 2 (6) PR/PRi 3 (10) Stable Disease 4 (12) Progressive Disease 6 (18) Clinical Benefit** 1 (3) No Clinical Benefit 4 (12) *All patients who have had at least 1 on-study disease assessment O
Slide 20: Evolution of Clinical Responses: Interim Efficacy and Duration on Study
Evolution of Clinical Responses: Interim Efficacy and Duration on Study 20
Slide 21: Event Free Survival
Event Free Survival 10f==— 094 is %- -- 084 % 074 ob- -- —<@ - -Go- ------- 064 054 044 034 024 014 00+ Propartion of Subjects Event Froe 21 wf wo 4
Slide 22: Overall Survival
Overall Survival Proportion of Subjects Alive 22 107 09+ 087 0.77 067 0.574 047 037 024 017 00+ —- 3-3 a — om. 26 ! Oo me— eee — ee ee eK KH eRe eK Ke Ke OO ' ' 1 "00 -© T T T T T T T T T T 0 1 2 3 4 5 6 8 9 10
Slide 23: Bone Marrow Response in MDS and AML
Bone Marrow Response in MDS and AML 50 40 30 MDS Pilot Study 20 10 0 -10 -20 -30 -40 -50 -60 -70 -80 -90 -100 n=9 -110 200 180 160 Phase II AML Study 140 120 100 80 60 40 20 0 -20 -40 -60 -80 -100 -120 n = 27; 6 patients had no on-study BM performed 23
Slide 24: Conclusions
Conclusions • Pracinostat in combination with azacitidine demonstrates significant clinical activity in elderly patients with newly diagnosed AML To date, 15 of 33 patients (45%) achieved the primary endpoint of CR+CRi+MLFS No patient who achieved a clinical response has progressed Most clinical res
Slide 25: Conclusions (cont’d)
Conclusions (cont’d) • Pracinostat in combination with azacitidine was well tolerated in this population of elderly AML patients The most common treatment-emergent AEs included neutropenia, febrile neutropenia, thrombocytopenia, nausea, fatigue and anemia Adverse events resulting in dose reductions
Slide 26: Pracinostat: Looking Ahead
Pracinostat: Looking Ahead Robert D. Mass, MD Chief Medical Officer
Slide 27: Proposed Phase III AML Study Designs
Proposed Phase III AML Study Designs Elderly (Age 60 years) Patients with Newly Diagnosed AML Unsuitable for Intensive Therapy Pracinostat Placebo + + Azacitidine Azacitidine • Primary endpoint: CR Secondary endpoints: OS, ORR, transfusion independency rate, duration of response, PFS, tolerability a
Slide 28: Phase II Front Line MDS Study (MEI-003)
Phase II Front Line MDS Study (MEI-003) Intermediate Risk-2 or High Risk MDS Patients Previously Untreated w/ HMA Pracinostat Placebo + + Azacitidine Azacitidine • Primary Endpoint: CR Secondary endpoints: overall response rate, hematologic improvement, clinical benefit rate, duration of response, p
Slide 29: Phase II Front Line MDS Study (MEI-003)
Phase II Front Line MDS Study (MEI-003) Patient Demographics 1 2 3 Study MEI-003 Pub Pub (N=102) (N=99) (N=179) Age Median 70 yrs 69 yrs 69 yrs <65 29% NR 32% >65 71% NR 68% Gender Male 69% 72% 74% Female 31% 27% 26% PS 0 34% NR 44% 1 57% NR 48% 2 9% NR 7% IPSS INT-2 65% 11% 43% High 35% 9% 46% 1 Ba
Slide 30: 2015 Clinical Milestones
2015 Clinical Milestones Pracinostat Top line data from Phase II study in front line MDS (March) Full data set from Phase II study in front line MDS (June) Initiation of Phase III study in front line elderly AML (June) ME-344 Data from Phase Ib trial in small cell lung & ovarian cancers PWT143 Initi
Slide 31: Q&A Analyst & Investor Event December 8, 2014
[ NASDAQ: MEIP ] Q&A Analyst & Investor Event December 8, 2014