Transcript Slide 1

Rodman & Renshaw Annual
Global Investment Conference
September 2011
Forward-Looking Statements
This presentation contains forward-looking statements that involve substantial
risks and uncertainties. These statements are often, but not always, made
through the use of words or phrases such as "anticipates," "expects," "plans,"
"believes," "intends," and similar words or phrases. All statements, other than
statements of historical facts, included in this presentation regarding our
strategy, future operations, outlook, milestones, the success of Arno’s product
development, potential advantages of Arno’s product candidates, future
financial position, future financial results, plans and objectives of management
are forward-looking statements. We may not actually achieve these plans,
intentions or expectations and Arno cautions investors not to place undue
reliance on our forward-looking statements. Actual results or events could differ
materially from the plans, intentions and expectations disclosed in the forwardlooking statements we make. Various important factors that could cause actual
results or events to differ materially from the forward-looking statements that
we make. Such factors include, among others, risks that the results of clinical
trials will not support our claims or beliefs concerning the effectiveness of our
product candidates, our ability to finance the development of our product
candidates, regulatory risks, and our reliance on third party researchers and
other collaborators. Arno is providing this information as of the date of this
presentation and does not undertake any obligation to update any forwardlooking statements as a result of new information, future events or otherwise.
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What Differentiates Arno Therapeutics
• Three oncology drug candidates with unique MOAs
• Highly differentiated products for unmet medical
needs with substantial market potential
• Leadership team with records of success in oncology
drug development and commercial outcomes
• Strong patent position on all assets
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Global Product Success
Glenn R. Mattes, President & CEO
 25+ years of R&D, commercialization and managerial experience ; Taxotere®, Lovenox®,
Remicade®, Prezista®, Doxil®, Procrit®/Eprex®
J. Chris Houchins, Chief Operating Officer
 20+ years of global drug development experience; Celebrex®, Aromasin®, Ellence®,
Emcyt®, Temodar®, Intron®/Peg-Intron®, Caelyx®
Stefan Proniuk, PhD, MBA, Senior Director Product Development
 15+ years of global CMC drug development experience, multiple NCEs and approved
products; Triaminic®, Niravam ®
Alex Zukiwski, MD, Chief Medical Officer
 16+ years of global pharmaceutical industry experience; Taxotere®, Xeloda®,
Procrit®/Eprex®, Velcade®, Doxil®, Yondelis®, FluMist®/Fluenz®
Arie Belldegrun, MD, Executive Chairman of the Board

Professor and Chair of Urologic Oncology, UCLA
Founder of Agensys and Cougar Biotechnology (Zytiga™)
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Potential Labeling Drives Development
HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use
AR-42 safely and effectively. See full prescribing information for AR-42.
AR-42 tablets for oral administration
----------------------------RECENT MAJOR CHANGES-------------------------Indications and Usage, XXXXXXXXXX XXXXXXX XXXXX XXXXX (1)
Warnings and Precautions, Rash (5.2)
Warnings and Precautions, Fatigue (5.2)
--------------------------INDICATIONS AND USAGE---------------------------AR-42 is a pan-histone deacetylase (HDAC) inhibitor indicated for the treatment of patients
with:
– XXXXXXXXX XXXXXXXXX XXXXXX XXXXXXX XXXXXXXXXXXXXX who require
therapeutic intervention but are not candidates for curative surgical resection(1).
– XXXXXXX who require therapeutic intervention but are not candidates for curative
surgical intervention or radiation therapy(1).
AR-42 in combination with XXXXXXXX is indicated for the treatment of patients with
XXXXXXXXXX XXXXXXX XXXXX XXXXX after progression of at least one prior platinum
containing chemotherapy regimen (1).
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Product Candidates
Discovery
AR-42
Preclinical
Phase I
Phase II
Phase III
Registration
Hematologic Malignancies and Solid Tumors
AR-12 Solid Tumors and Lymphomas
AR-67
Glioblastoma Multiforme
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AR-42 Profile
 Pan-DAC inhibitor; oral formulation
 In vitro and in vivo activity demonstrated in multiple tumor
types
– Greater potency and efficacy vs. vorinostat in AML
models
– Encouraging results in multiple cancers and leukemic
stem cells
 Phase I/II study in hematological malignancies and solid
tumors is currently ongoing
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AR-42 Profile
 Phase II program anticipated to begin in 2Q2012
 Potential Indications
– Non-small cell lung cancer
– Prostate cancer
– Cranial-spinal axis tumors
– Carcinoma of the urothelium
– Hepatocellular carcinoma
– Acute myelogenous leukemia
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AR-42 Phase I/II Study
 Dose escalation phase to define MTD in study subjects with hematological
malignancies
– N~20 patients
 A solid tumor dose escalation arm is being added
– Tumors of “interest”; N = ~21
 MTD expansion phase
– N~30 patients, 10 subjects in each cohort (chronic lymphocytic leukemia,
multiple myeloma and Hodgkin’s disease)
 Trial Design
– Standard 3+3 study dose escalation study, oral administration 3x/week
on non-consecutive for 3 consecutive weeks of a 28 day treatment cycle
– Assessment in both phases for biomarkers of target inhibition, including
histone and tubulin acetylation, as well as other potential markers for
treatment changes or toxicity
– Study endpoints: Safety , PK, PD, preliminary efficacy
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Minimal Regrowth of Ben-Men-1-LucB
Xenografts After AR-42 Treatment
+ AR-42 (months)
+ Normal diet (months)
0
1
6
1
2
3
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AR-12 Profile
 Potential first-in-class; targeted PDK-1 inhibitor;
oral formulation
 Phase I/II study in solid tumors and lymphomas
currently enrolling
 Potential Indications
– NSCLC
– Breast cancer
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AR-12 MOA
Targets PI3K/Akt Pathway
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AR-12 Combination Potential
 Preclinical data show increased activity and/or reversal of
resistance when combined with:
 Tarceva® (non-small cell lung cancer, in vitro and in vivo)
 Iressa® (non-small cell lung cancer, in vitro and in vivo)
 Avastin ® (colon cancer, in vivo)
 Herceptin ® (breast cancer, in vitro)
 Tamoxifen (ER positive and negative breast cancer, in vitro and in vivo)
 Gleevec ® (chronic myelogenous leukemia, in vitro)
 Nexavar ® (nasopharyngeal cancer, in vitro)
Tseng PH et al., Mol Pharmacol. 2006 Nov;70(5):1534-41
Cen L et al., British J Cancer (2007) 97, 785-791
Ping-Hui Tseng et al, Blood (2005) 105: 4021-4027
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AR-12 Phase I/II Study
• Dose-escalation to determine MTD and/or
active biologic dose in subjects with solid
tumors and lymphomas
– Study is ongoing
– Early evidence of biologic activity
• Phase II expansion will allow for efficacy/safety
assessments in tumors of interest
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AR-67 Profile
 Third-generation camptothecin analogue
 Phase II glioblastoma multiforme study currently
enrolling
 Phase II colorectal carcinoma study scheduled to start
4Q2011
 Potential Indications
– Glioblastoma multiforme
– Colorectal cancer
– Non-small cell lung cancer
– Small cell lung cancer
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AR-67 Summary
 Tert-butyl-silyl camptothecin derivative with substantially
improved lactone stability, and increased lipophilicity
– ~ 85% remains in active “lactone” form
– potential increased central nervous system penetration
– potential for more predictable metabolism/toxicity
 Phase I study demonstrated anti-tumor activity; acceptable
safety (no drug related diarrhea); linear pharmacokinetics
 Phase II GBM study proceeding after reaching interim
assessment
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Response Three Years after Initiation
(Two Years after Last AR-67 Infusion)
Pre-treatment / Baseline 3/3/08
AR-67 Phase I, Subject 21
Three years later 2/28/11
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Market Potential
Compound
AR-42
AR-12
AR-67
Indication
NSCLC
prostate
AML
bladder cancer
multiple myeloma
cranial/spinal tumors
breast cancer
NSCLC
GBM
colorectal cancer
US Incidence
220,000
240,000
12,900
69,000
20,500
13,000
232,000
220,000
10,000
140,000
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Upcoming Milestones
 Expand AR-42 Phase I/II study into solid
tumors of interest
 Complete AR-12 Phase I enrollment; identify
indications for Phase II testing
 Complete AR-67 Phase II GBM study
enrollment
 Initiate AR-67 Phase II colorectal cancer study
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Reasons to Invest in Arno
 Promising & differentiated oncology pipeline
 Efficient, well-defined clinical/regulatory
pathway with planned near term inflection points
and potential expedited global registration
programs
 Senior management has record of success in
oncology
 New roadmap to value creation
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