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[ NASDAQ: MEIP ] Wedbush PacGrow Healthcare Conference August 11, 2015

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Page 1: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

[ NASDAQ: MEIP ]

Wedbush PacGrow Healthcare Conference

August 11, 2015

Page 2: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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.

Page 3: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

3

MEI Pharma (Nasdaq: MEIP)

• San Diego-based oncology company with three wholly owned drug

candidates

• All three drug candidates in the clinic with data expected by year end

• Strong intellectual property protection for all drug candidates

• Strong cash position: $70.5 million as of March 31, 2015

• Experienced management team with proven oncology drug

development experience

Page 4: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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DRUG CANDIDATE INDICATION / COMBINATION PRE-CLINICAL PHASE I PHASE II PHASE III

EP

IGE

NE

TIC

SP

RO

GR

AM

PracinostatHDAC Inhibitor

Acute Myeloid LeukemiaFront Line, ElderlyAzacitidine (Vidaza®)

Myelodysplastic SyndromeFront Line, Int-2 & High-RiskAzacitidine (Vidaza®)

Myelodysplastic SyndromeRefractory to HMAAzacitidine (Vidaza®) or

Decitabine (Dacogen®)

MyelofibrosisFront Line & Relapsed/RefractoryRuxolitinib (Jakafi®)

CA

NC

ER

ME

TA

BO

LIS

M

PR

OG

RA

M

ME-344Mitochondrial Inhibitor

Small Cell Lung CancerAdvanced or MetastaticTopotecan (Hycamtin®)

Ovarian CancerAdvanced or MetastaticTopotecan (Hycamtin®)

SIG

NA

LIN

G

PR

OG

RA

M

PWT143PI3K Delta Inhibitor

Hematologic Cancers

Clinical Development Pipeline

Page 5: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

5

Pracinostat in MDS: What We Learned

• Evidence of activity in combination with azacitidine in pilot study1

89% (8/9) overall response rate; combination “very well tolerated”

• Top-line data from randomized Phase II study released in March 2015

Addition of Pracinostat to azacitidine did not improve rate of complete

remission compared to azacitidine alone

Combination of Pracinostat plus azacitidine resulted in high rate of early

drug discontinuations, primarily due to tolerability

Patients receiving Pracinostat plus azacitidine for > 4 cycles achieve

prolonged progression-free & overall survival compared to

azacitidine alone (HRs < 0.6); data still maturing

o Data submitted for presentation at ASH in December 2015

1 Quintás-Cardama et al. ASH 2012: Abstract 3821

Page 6: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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Azacitidine Azacitidine + Vorinostat

CR rate 24% (22/92) 15% (14/91)

Off Tx due to toxicity / side

effect / complication9% (8/92) 24% (22/91)

Relapse-free survival, on

Tx > 6 months (median)7 months 13 months (P = .11)

North American Intergroup Randomized

Phase II MDS Study (SWOG S1117)1

1 Sekeres et al. ASH 2014: LBA - 5

Conclusions / Opinion:

• “Are combination regimens in MDS too toxic? Or do we need to manage

toxicities better?”

• “ORR is not the right endpoint for large MDS trials. We need to focus on

durable responses, and on OS.”

Page 7: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15

Response Based on Clinical

Review of Efficacy Data

CR

CRi

MLFS

PR/Pri

Stable Disease

Progressive Disease

No On-Study Assessment

Time to 1st Bone Marrow

Assessment for Responders

Ongoing

Months on Study

1stLin

e E

lderly A

ML P

atients

(N

=50)

Phase II Study of Pracinostat in AMLDuration on Study and Best Response1

1 Garcia-Manero et al. EHA 2015: Abstract P568

Page 8: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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MDS (MEI-003) MDS (MEI-003) AML (MEI-004)

Azacitidine(n=51)

Pracinostat +

Azacitidine(n=51)

Pracinostat +

Azacitidine(n=50)

Cycle 1 4 5 3

Cycle 2 0 8 4

Total 4 (8%) 13 (25%) 7 (14%)

Phase II Study of Pracinostat in AML vs. MDSDiscontinuations in First Two Cycles Due to Tolerability*

* Includes adverse events, patient decisions/informed consent withdrawals and “other”

Page 9: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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Phase II Study of Pracinostat in AMLOverall Survival by Risk Group1

Patients are censored as of the date of last contact to determine survival status on or after May 15, 2015. CNSR, censored.

1 Garcia-Manero et al. EHA 2015: Abstract P568

Page 10: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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AZA-AML-0011 MEI-0042

Conventional

Care Regimens(n=247)

Azacitidine(n=241)

Pracinostat +

Azacitidine(n=50)

CR rate 21.9% 19.5% 42%

60-day mortality rate 18.2% 16.2% 10%

One-yearsurvival rate 34.2% 46.5% 60%

Overall survival 6.5 months10.4 months

(P = .1009)?

International Phase III Study of Azacitidine in

Newly Diagnosed AML (AZA-AML-001)

1 Dombret et al. Blood. 2015 Jul 16;126(3):291-92 As of ASH abstract submission on August 4, 2015

Page 11: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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• Novel mechanism of action directly targets mitochondrial OXPHOS

complex I1, resulting in rapid loss of cellular energy (ATP)

• Evidence of single agent activity in Phase I dose-escalation study in

refractory solid tumors2

• Phase 1b combination study with topotecan in relapsed/refractory

ovarian and small cell lung cancers closed to enrollment

Initial survival estimates expected in Q4 2015

• New pre-clinical data shows significantly enhanced anti-tumor

activity when combined with VEGFR tyrosine-kinase inhibitor (TKI)3

ME-344: Novel Mitochondrial Inhibitor

1 Lim et al. Am J Cancer Res. 2015;5(2):689-7012 Bendell et al. Cancer. 2015 Apr 1;121(7):1056-633 Manevich et al. AACR 2015: Abstract 5409

Page 12: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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Spontaneous Breast Tumor ModelNintedanib (VEGFR/PDGFR/FGFR TKI) + ME-344*

0

200

400

600

800

1000

1200

0 1 2 3 4 5 6 7 8 9 10

Weeks

Vehicle BIBF ME BIBF+MENint

Tumor Growth Inhibition

ME-344 Nint + ME-344

* Collaboration with Miguel Quintela-Fandino, MD, PhD, Spanish National Cancer

Research Centre, Madrid

Page 13: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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0

200

400

600

800

1000

1200

1400

T0 T1 T2 T3 T4 T5 T6 T7 T8 T9 T10 T11 T12 T13 T14 T15

Vehicle ME344 BIBF BIBF+ME344Nint Nint + ME-344

Weeks

Human KRas Mutated Lung XenograftNintedanib (VEGFR/PDGFR/FGFR TKI) + ME-344*

Tumor Growth Inhibition

* Collaboration with Miguel Quintela-Fandino, MD, PhD, Spanish National Cancer

Research Centre, Madrid

Page 14: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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PWT143: Highly Selective PI3K Delta Inhibitor

• First PI3K delta inhibitor (Idelalisib) approved in July 2014

Recommended starting dose: 150 mg orally, twice daily

• PWT143 has shown superior pre-clinical activity compared to other

PI3K delta inhibitors, including Idelalisib

• First-in-human study initiated in June 2015

Measurable plasma levels as well as significant on target activity

observed at 10 mg starting dose level; PK results suggest daily dosing

• PK/PD data submitted for presentation at ASH in December 2015

Page 15: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

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Upcoming Clinical Milestones

Pracinostat

Overall survival estimate from Phase II study in AML (Q4 2015)

ME-344

Initial survival estimates from Phase Ib study + topotecan (Q4 2015)

Initiation of Phase II study + TKI (Q1 2016)

PWT143

PK data from first-in-human study (Q4 2015)

Initiation of Phase I study in hematologic cancer (Q1 2016)

Page 16: Wedbush PacGrow Healthcare Conference August 11, … Presentation...Acute Myeloid Leukemia Front Line, Elderly Azacitidine (Vidaza®) Myelodysplastic Syndrome Front Line, Int-2 & High-Risk

[ NASDAQ: MEIP ]

Wedbush PacGrow Healthcare Conference

August 11, 2015