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EpiCept Corporation (EPCT)

May 31, 2015

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Page 1: EpiCept Corporation (EPCT)

1

Corporate Presentation

June 2010

Page 2: EpiCept Corporation (EPCT)

2

Forward-Looking Statements

This presentation contains forward looking statements that involve risks and uncertainties regarding the operations and future results of EpiCept. You should review the company's filings with the Securities and Exchange Commission, including without limitation the company's Form 10-K and Forms 10-Q, which identify specific factors that may cause actual results or events to differ materially from those described in the forward looking statements. The content of this presentation contains time sensitive information that is accurate only as of the date of the presentation. The company undertakes no obligation to revise or update any statements to reflect events or circumstances after the date of this conference call.

Page 3: EpiCept Corporation (EPCT)

3

A specialty pharmaceutical company focused on the development and

commercialization of pharmaceutical products for the treatment of cancer and

pain

Company Description

Page 4: EpiCept Corporation (EPCT)

4

Investment Highlights

Ceplene approved in Europe and launched in UK, Germany and Austria

NDA filed in US, priority review requested

Ceplene is the first efficacious remission maintenance drug for AML

• Met primary endpoint in 320 patient pivotal Phase III trial

• 40% of Ceplene treated patients in first remission were leukemia free at 3 years vs. 26% of control patients (p=0.011)

Substantial upside retained in European partnership with Meda

Market exclusivity for 7 years in US and 10 years in EU

Potential for market expansion in MDS and CML

NP-1 completed Phase II for pain and Crolibulin in Phase I for solid tumors

Azixa partnered with Myriad Pharmaceuticals in Phase II for brain cancer

Page 5: EpiCept Corporation (EPCT)

5

Action Dates

Ceplene

• US NDA filing 2Q 2010

• European commercial launch 2Q 2010

• Canadian NDS approval 3Q 2010

• FDA acceptance of Ceplene NDA / Announced PDUFA Date 3Q 2010

• ODAC meeting 4Q 2010

• US approval Q1 2011

Pipeline

• NP-1 partnership 2H 2010

• Azixa Phase II brain cancer results 2Q 2010

• Crolibulin Phase Ib combination trial initiation 2Q 2010

2010 Upcoming Milestones

Page 6: EpiCept Corporation (EPCT)

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Ceplene®

Reducing the Risk of Relapse in AML

Page 7: EpiCept Corporation (EPCT)

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Ceplene®: EU Approved Label

“Ceplene, administered in conjunction with interleukin-2, is indicated for maintenance of remission in adult patients with acute myeloid leukaemia in first remission to prolong the duration of leukaemia free survival.”

Page 8: EpiCept Corporation (EPCT)

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Meda Partnership

Leading international specialty pharmaceutical company

• Sales and marketing organization of about 1,200 people throughout Europe

• Established sales organizations in the US and more than 40 other countries

• Annual revenues of approximately $1.8 billion

• Oncology/hematology presence with several products, including OnsolisTM indicated for breakthrough pain in cancer patients

Up to $40 million in upfront payments and milestones, plus escalating double digit royalty

EpiCept is responsible for Ceplene's commercial supply

Territory includes Europe, Japan, China, Australia and selected Asian countries

Selective EU launches underway

Page 9: EpiCept Corporation (EPCT)

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Changing the AML Treatment Paradigm

Induction and Consolidation

Therapy

Complete Remission

(70%)

Bone Marrow Transplant

Remission Maintenance: Ceplene+IL-2

Remission Maintenance: Current SOC(no treatment)

(>80% Relapse)

• 90 – 95% mortality at 5 years

• Median survival 6 - 9 months

• 40% Ceplene+IL-2 patients reach LFS at 3 years vs. 26% of SOC patients

• Median improvement of 64 weeks in overall survival

• If successful it is a true cure

• High procedural mortality rate

No Remission

(30%)

Page 10: EpiCept Corporation (EPCT)

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Rationale for the Use of Ceplene with IL-2

IL-2

Activates and expands

T and NK cells

Ceplene (HDC)Protects T and NK cells against inactivation and apoptosis

Elimination of leukemic cells and protection against relapse

- T cells and NK cells can attack and eliminate AML blasts - T and NK cell functions determine relapse risk in AML

NK

NK

AML blast

Page 11: EpiCept Corporation (EPCT)

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Ceplene: Mechanism of Action in AML

NKp46

Activating Receptors

Myeloid Cell

NK Cell

H2 receptor

Cytotoxicity

O2

AML Blasts

NKG2D

NADPH oxidase

Response to IL-2

Apoptosis

Page 12: EpiCept Corporation (EPCT)

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Ceplene: Mechanism of Action in AML

Lysed

NKp46

Activating Receptors

Myeloid Cell

NK Cell

H2 receptor

Cytotoxicity

Ceplene (HDC)

NKG2D

NADPH oxidase

Response to IL-2

Page 13: EpiCept Corporation (EPCT)

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Pivotal Phase III Study Design, Trial Endpoints and Analyses

• Primary endpoint: Leukemia-free survival (LFS) after 3 years

• Secondary endpoints:

• Overall survival (OS)

• LFS in CR1 and CR>1 subgroups

• LFS rates at 6, 12, 18, 24 and 36 months

• Safety and quality-of-life (QLQ-C30)

AML Remissions(n=320)

18 Months 18 MonthsEndpointAnalysis

Ceplene+IL-2 (n=160)10 cycles over 18 months followed by 18 months of observation

Randomized

• Key prognostic variables tested by multivariate analyses

• Additional follow-up at 5 and 6 years

Standard of Care (No Treatment)(n=160)

Page 14: EpiCept Corporation (EPCT)

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Results of the Pivotal Phase III Study:Leukemia-Free Survival

Overall Population CR1 Population

Page 15: EpiCept Corporation (EPCT)

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Results of the Pivotal Phase III Study: Overall Survival

Months from Randomization

Ove

rall

Su

rviv

al (

%)

P = 0.118

CR1 Population

Ceplene/IL-2 (n=129) Control (n=132)

Ceplene/IL-2 (n=160) Control (n=160)

Overall Population

P = 0.161

100

80

60

40

20

0

100

80

60

40

20

00 12 24 36 48 7260 0 12 24 36 48 7260

Months from RandomizationMonths from Randomization

Ove

rall

Su

rviv

al (

%)

P = 0.118

CR1 Population

Ceplene/IL-2 (n=129) Control (n=132)

Ceplene/IL-2 (n=160) Control (n=160)

Overall Population

P = 0.161

100

80

60

40

20

0

100

80

60

40

20

00 12 24 36 48 7260 0 12 24 36 48 7260

Months from Randomization

Overall Population CR1 Population

Page 16: EpiCept Corporation (EPCT)

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Summary: Clinical Benefit of Ceplene+IL-2

• Phase III study met primary endpoint of LFS rate at 3 years

Overall Population CR1 Population

3 Year LFS

Log Rank

Treatment: 34%Control: 24%p = 0.008

Treatment: 40% Control: 26%p = 0.011

5 Year LFS

Log Rank

Treatment: 30%Control: 21%p = 0.017

Treatment: 34% Control: 22%p = 0.025

3 Year OS

Log Rank

Treatment: 48%Control: 44%p = 0.161Median Improvement: 24 weeks

Treatment: 55% Control: 46%p = 0.118Median Improvement: 64 weeks

Page 17: EpiCept Corporation (EPCT)

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Ceplene: US Regulatory Status

Pre-NDA meeting completed

NDA filed June, 2010

82% concurrence of FDA and EMEA approvals since 2006(1)

Key issues to be addressed:

1. Approval based on single pivotal trial

2. Acceptability of Leukemia Free Survival as primary endpoint

3. Trial design - Isolation of Ceplene’s contribution to efficacy vs. IL-2

(1) Source: FDA – Center for Drug Evaluation & Research, New Drug Review: 2009 (FDA/CMS Summit December 3, 2009), includes New Molecular Entities only

Page 18: EpiCept Corporation (EPCT)

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FDA Issue 1: Single Pivotal Trial

Robust statistical outcome of pivotal study (p<0.008)

Significant unmet medical need

Orphan indication

Numerous precedents for approval based on a single randomized pivotal trial for orphan indications and/or high unmet medical need if the data demonstrates substantial evidence of efficacy (Folotyn, Istodax, Velcade)

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FDA Issue 2: Acceptability of LFS as Appropriate Primary Endpoint

Data supports LFS as a surrogate marker for survival

Median improvement of 64 weeks on overall survival

No adverse effect seen on survival

Numerous precedents in oncology for accelerated approval based on response rate, progression free survival, time to progression (Gleevec, etc.)

Cooperative group studies use LFS as primary endpoint for AML

Page 20: EpiCept Corporation (EPCT)

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FDA Issue 3: Trial Design - Isolation of Ceplene’s Contribution to Efficacy vs. IL-2

Only Ceplene+IL-2 pivotal trial successful for remission maintenance

Six separate IL-2 monotherapy AML trials have failed to demonstrate IL-2 benefit

Meta and Bayesian analysis of background IL-2 monotherapy “contribution” concluded incremental contribution of Ceplene beyond IL-2 background at p<0.05

Page 21: EpiCept Corporation (EPCT)

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IL-2 Monotherapy Trials vs. Ceplene+IL-2

Trial NAge

(years)

Monthly IL-2 Dose

MIU/m2

P-values

Blaise et al. 78 <50 120 0.54

CALGB 9720 163 ≥60 81 0.47

CCG-2961 289 ≤21 52 0.49

CALGB 19808 214 <60 91 0.13

ALFA 9801 161 50-70 25 0.88

EORTC/GIMEMA 288 <61 12 - 24 0.57

Ceplene+IL-2

Phase III Study 320 18-84 15 0.01

Page 22: EpiCept Corporation (EPCT)

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IL-2 Monotherapy vs. Standard of Care

Dis

ease

-Fre

e S

urvi

val

Pro

babi

lity

No further treatment

P=0.47

Time (years)

Dis

ease

-Fre

e S

urvi

val

Pro

babi

lity

No further treatment

P=0.47

Time (years)

100

80

60

Standard of careIL-2

0 20 40 60

0.0

0.2

0.4

0.6

0.8

1.0

Months from 2nd randomization

P(E

ven

t Fre

e S

urv

iva

l)

Arm1Arm2

p= 0.88

IL-2No treatment

P=0.88

Months from IL-2 randomization

Dis

ea

se

-fre

e s

urv

iva

l

Ov

era

ll s

urv

iva

l

Blaise et al. CALGB 9720 ALFA 9801

Years from IL-2 randomization

CALGB 19808 CCG 2961

Note: Kaplan analysis curve for EORTC/GIMEMA trial not yet published

Page 23: EpiCept Corporation (EPCT)

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Ceplene: Market Opportunity

US

Prevalence 38,000

New Patients/Year 13,000(1)

Complete Remissions Achieved (70%) 9,100

Less Bone Marrow Transplants (20-25%) (2,275)

Annual New Patient Candidates 6,825

Median Annual Cost/Patient $ 35-40K

Ceplene Peak Market Potential $235-270 MM

(1) National Cancer Institute (2) European LeukemiaNet (ELN)

Page 24: EpiCept Corporation (EPCT)

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Ceplene US Commercialization Strategy

• Orphan protection granted, 7 years market exclusivity

• First and only product to prevent AML relapse

• No competing products in late stage development

• Continue targeting top KoLs through symposia, conferences, etc.

• Initiate pre-launch Ceplene experience program

• Outreach to patient advocate groups

• Planning self launch in US with 20-25 MSLs/sales representatives

• Target audience 1500 hematologists

• Target top 200 US hospitals

• Gain inclusion of Ceplene+IL-2 in AML treatment guidelines

Page 25: EpiCept Corporation (EPCT)

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Ceplene Summary

Ceplene+IL-2 is first and only therapeutic regimen to prevent relapse and prolong LFS in AML patients in remission

Ceplene+IL-2 LFS rate at 3 years of 40% vs. 26% for control (p=0.011) is clinically relevant for AML patients in first remission

Cycles of daily Ceplene+IL-2 s.c. injections at home are well-tolerated and are not an impediment to AML patients’ QoL or functional status

No competing therapy in Phase II or III clinical development

European partnership established, launches underway

Pursuing North American regulatory approvals

Orphan drug designation provides 7 years market exclusivity in US and 10 in EU

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EpiCept™ NP-1: Product Profile

• Phase III ready topical cream for the relief of pain associated with post- herpetic neuropathy (PHN), diabetic neuropathy and chemotherapy

• 4% amitriptyline plus 2% ketamine combination provides pain relief equivalent to current therapies

• Over 1,300 patients treated in seven clinical trials

• Effective and well-tolerated in chronic use

• Better side effect profile than oral therapies

• Skin irritation is infrequent; fatigue and drowsiness are rare and mild

• NP-1 represents a large, low risk commercial opportunity

Page 27: EpiCept Corporation (EPCT)

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EpiCept™ NP-1: PHN Clinical Data

NP-1 (n=32)

Placebo(n=46)

Baseline (mean)

6.47+1.65 6.5+1.60

Day 21(mean) 3.28+ 2.1 4.35+2.2

Mean Change 3.19 + 2.0

p=0.026

2.15+1.8

Efficacy vs. placebo tested in 150 PHN patients in dose response trial

Dose-response trial met primary endpoint; p=0.026

Strong analgesic effect

Additive to or can replace standard of care

Effective in persistent pain

Supports advancement to Phase III

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EpiCept™ NP-1: PHN Clinical Data Phase II, Non-inferiority design, vs. gabapentin and placebo in PHN for 4 weeks

Met primary endpoint: • Efficacy superior to placebo, p =.044• Not inferior to gabapentin, p =.84

Results indicate at least equivalent efficacy to unit market leader

Better side effect profile

Supports advancement to Phase III

Primary Endpoint: Mean pain score

NP-1n=135

Placebon=76

Gabapentinn=138

Baseline 6.04 ± 1.25 6.37 ± 1.32 6.32 ± 1.17

At the end of week 4 3.54 ± 1.98 4.22 ± 2.30 3.55 ± 1.93

Mean difference in pain score

2.42 ± 0.18

p =.044

1.88 ± 0.23 2.47 ± 0.18

ITT Population

Page 29: EpiCept Corporation (EPCT)

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2008 US market size: 4.7 million patients, $2.6 billion in sales

2018 est. US market size: 6.1 million patients; $5.1 billion in sales

Key market drivers: aging population and unmet medical need due to ineffective therapy

Competing products: gabapentin (generic), Lyrica (Pfizer), Cymbalta (Eli Lilly), Lidoderm (Endo)

• No current therapy effective in more than 50% of patients• Oral therapies have CNS side effects• Lidoderm patch not convenient for many areas prone to pain

May be used in combination with other therapies

Patent protection to 2021 plus US orphan drug designation in PHN

EpiCept™ NP-1: Market Opportunity

Page 30: EpiCept Corporation (EPCT)

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Crolibulin

• Small molecule, vascular disruption agent (VDA), disrupts newly formed vascular cells and blood flow to tumor

• Superior anti-tumor (apoptotic) activity compared to other VDAs

• Enhanced efficacy in combination with Cisplatin or other chemotherapeutics

• Active on multi-drug resistant cells

• DLT/MTD identified

• Activity in resistant hepatocellular carcinoma

• Objective evidence of anti-tumor response by CT perfusion

• Combination trial to be initiated Q2 2010

• Strong intellectual property position

Page 31: EpiCept Corporation (EPCT)

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Azixa™ (MPC-6827): Myriad Pharmaceuticals

Small molecule, apoptosis inducer with VDA activity, concentrates in the CNS

Two Phase I studies completed in brain cancer: primary and metastatic

Treatment schedule: Once a week for 3 weeks on 28 day cycle

Reported Phase I results

- Reached Maximum Tolerated Dose (MTD)

- Signs of efficacy: measurable reduction in tumor size in primary glioblastoma and patients with secondary metastases

Myriad Pharmaceuticals conducting Phase II trials dosing in primary and metastatic brain cancer

Page 32: EpiCept Corporation (EPCT)

32

Investment Highlights

Ceplene approved in Europe and launched in UK, Germany and Austria

NDA filed in US, priority review requested

Ceplene is the first efficacious remission maintenance drug for AML

• Met primary endpoint in 320 patient pivotal Phase III trial

• 40% of Ceplene treated patients in first remission were leukemia free at 3 years vs. 26% of control patients (p=0.011)

Substantial upside retained in European partnership with Meda

Market exclusivity for 7 years in US and 10 years in EU

Potential for market expansion in MDS and CML

NP-1 completed Phase II for pain and Crolibulin in Phase I for solid tumors

Azixa partnered with Myriad Pharmaceuticals in Phase II for brain cancer

Page 33: EpiCept Corporation (EPCT)

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