Corporate Presentation
September 2013
Safe Harbor Statement
During the course of this presentation we will make statements that constitute
forward-looking statements. These statements may include operating expense
projections, the initiation, timing and results of pending or future clinical trials,
the actions or potential action of the FDA, the status and timing of ongoing
research, corporate partnering activities and other factors affecting Adherex
Technologies’ financial condition or operations. Such forward looking
statements are not guarantees of future performance and involve risk,
uncertainties and other factors that may cause actual results, performance or
achievements to vary materially from those expressed or implied in such
statements. These and other risk factors are listed from time to time in reports
filed with the SEDAR and the Securities and Exchange Commission, including
but not limited to, reports on Forms 10-Q and 10-K. Adherex does not intend
to update any forward looking information to reflect actual results or changes
in the factors affecting forward-looking information.
Company Overview
Biopharmaceutical company dedicated to the discovery and development of
novel cancer therapeutics
Two late stage oncology clinical products: Sodium Thiosulfate (STS) and
Eniluracil (EU)
STS: pending favorable data will file for NDA
EU: pending partnering discussions advance to Phase III
US based management team
Headquarters in Research Triangle Park, NC
Ticker: ADHXF – USA, AHX – Toronto
Market Cap: $10 MM; 25.1 MM shares outstanding
$1.0 MM in cash at 6/30/13, no debt
Large insider ownership with aligned shareholder incentives
Management and Board of Directors
Rosty Raykov – Chairman and CEO
Bear Stearns, Tiedemann Group, John Levin & Co., Alchem and DCML Co.
Chris Rallis – Director
30 years of business development, legal and operating experience at
Wellcome and Triangle Pharmaceuticals. Currently, executive in residence
with Pappas Ventures.
Steve Skolsky – Director
30 years of operating experience, including Head of Glaxo Welllcome’s
Division of HIV/Oncology, Chief Executive at Trimeris and Sequoia
Pharmaceuticals. Currently, Global Head of Clinical and Data Operations at
Quintiles.
STS Investment Highlights
STS is a chemo-protectant agent being developed exclusively in children to prevent hearing
loss caused by cisplatin
Received Orphan Drug Designation in 2004 with 7.5 years exclusivity upon approval
Phase III trial conducted by Children’s Oncology Group is fully enrolled with data on 135
patients expected in October 2013
Phase III trial conducted by SIOPEL6 in children with liver cancer 80/102 patients enrolled
Clinical trial costs covered by government grants
Adherex has exclusive rights to data from both studies
Potential for Rare Pediatric Disease Voucher: upon approval of STS
6 month priority review to any other new NDA or BLA application
Voucher can be transferred or sold with no restrictions
Intellectual property: Use-patent as chemo-protectant in-licensed from OHSU
Issued European and Japanese patents expire 2021, US pending
prosecution
Platinum Hearing Loss is Frequent, Severe and Irreversible
Platinum drugs are widely used anti cancer agents in pediatric oncology
Produce profound, irreversible, cumulative hearing loss
Destroy the cochlear hair cells of inner ear
Effect can be seen after as little as the second or third dose
Hearing loss (ototoxicity) is a dose-limiting side effect
Up to 2,000 children receive platinum based chemotherapy every year in the
US: 40-90% develop irreversible ototoxicity *
Loss of high frequency hearing sensitivity - loss of high frequency
consonants (s/f/th/p/k/h/t)
Background noise compounds disability in critical settings - distance hearing
and hearing in the classroom
Infants and young children at critical stage of development lack speech
language development and literacy
Older children and adolescents lack social-emotional development
and educational achievement
*Neuwelt and Brock. J Clin Oncol 2010;28:1630-1632
Ototoxicity in Children Treated with Cisplatin and/or
Carboplatin
90
80
Ototoxicity (%)
61% bilateral hearing loss
(ASHA criteria) at the end
of treatment
41% required hearing aids
that only partially restore
hearing
22% of patients had dose
reductions due to
ototoxicity
N=67 age 8 m -20 years
100
88
75
70
67
60
50
50
40
30
20
10
11
0
Medulloblastoma
*Gilmer-Knight et al., Journal of
Clinical Oncology
Osteosarcoma
Neuroblastoma
PNET
Germ cell
Current Approach to
Platinum Induced
Hearing Loss
Hearing Loss
Detected or
Communicated
by Patient
Continue Course
Leads to more severe hearing loss
Detectable hearing loss generally
begins after about two or three cycles
of cisplatin, and may continue to occur
for months (or longer) after use is
discontinued
Hearing Loss Diagnosed
Dose Modify
Switch therapies
Less effective dosing/treatment, potentially
shortening survival
Target and Proposed STS Mechanism
Antitumor Effect
NH3
NH3 Pt
Cl
Cl
Ototoxicity Effect
NH3
NH3
Pt
Cl
Protein
STS
NH3
Cl
NH3
Pt
Cl
Protein
Cl
Requires both Cl unbound to
crosslink DNA
Binding to plasma proteins
occurs within first hour which
inactivates one binding site
Free cDDP (unbound) short t1/2
:1.5 hr
Requires one Cl unbound to
affect cochlear hair cells
Binding to plasma proteins
occurs within first hour which
inactivates one binding site
STS will bind second site
preventing ototoxicity
STS Protects Against Cisplatin Ototoxicity in the Rat
Change from baseline hearing threshold. Effect of STS
(8 g/m2 IV) 4 hrs, 8 hrs, or 12 hrs after administration of
cisplatin (6 mg/kg IA)
Delayed administration of STS after platinum
agents in animals reduces ototoxicity
*Dickey DT et al. J Pharmacol Exp Therapeut 2005;314:1052-1058
Cisplatin and STS animal PKs
Cisplatin clearance is complete by 6 hrs with or without STS, when STS levels are at or higher
than clinically achievable levels
Cisplatin Pharmacokinetics
nu/nu mice administered 4 mg/kg CDDP i.p.
STS Pharmacokinetics
nu/nu mice administered 3.5 g/kg STS i.p.
STS Level
1 min
15 min
Mouse # 1
222.0 mg/dL
941.0 mg/dL
Mouse # 2
180.0 mg/dL
5.85 mg/dL
Mouse # 3
133.5 mg/dL
n/a
Mouse # 4
145.8 mg/dL
1131.0 mg/dL
Mouse # 5
not detectable
1246.0 mg/dL
Mouse # 6
177.1 mg/dL
975.0 mg/dL
*Gregory Reaman et al
Time-Dependent Tumor Protection of STS after
Administration of Cisplatin in Nude Mice
STS allows for anti-tumor activity when given properly
*Harned TM et al. Clin Cancer Res 2008;14:533-540
COG ACCL0431: Randomized Study of STS for
Prevention of Cisplatin-induced Hearing Loss
Newly diagnosed children with hepatoblastoma, germ cell tumor,
osteosarcoma, neuroblastoma, and medulloblastoma
Study Chair: David Freyer, DO, MS
135 randomized patients fully enrolled and study completed in
1Q 2012
Futility analysis conducted and reviewed by COG DSMC August 2011
with recommendation at the time to continue study
Expect data in October 2013
COG ACCL0431: Randomized Study of STS for Prevention
of Cisplatin-induced Hearing Loss
Note: Patient must first be
enrolled on the COG hearing
assessment study, ACCL05C1
Newly diagnosed germ cell tumor,
hepatoblastoma,
medulloblastoma,
neuroblastoma, or osteocarcoma
Diagnosis
Planned treatment program includes  200 mg/m2 cisplatin
(administered according to the disease-specific regimen)
Study entry onto ACCL0431
Randomization
Sodium thiosulfate given intravenously over
15 minutes starting 6 hours after completion
of each cisplatin infusion (STS Arm)
No sodium thiosulfate treatment
given (Observation Arm)
Protocol therapy ends when patient completes
planned treatment regimen containing cisplatin
Audiology done with
each cycle of therapy
80% power to detect
22.5% vs 45%
change in hearing
80% power to rule out
>12% difference in
3 yr EFS
Secondary objectives
measurement of
nephrotoxicity and
neurotoxicity
SIOPEL 6: Efficacy of STS in Reducing Ototoxicity in Patients
Receiving Cisplatin for Standard Risk Hepatoblastoma
Newly diagnosed children with standard risk hepatoblastoma
Study Chair: Peppy Brock, MD
80 randomized patients fully enrolled out of 102
Interim evaluations of efficacy of the chemotherapy carried out and reviewed by IDMC
after 20, 40, 60 and 80 patients are evaluable for response
Early stopping will be considered in case of concerns on efficacy of chemotherapy in
either treatment arm
The first two interim safety analysis after 20 and 40 patients were conducted with
IDMC recommending study to continue
Two interim and one final efficacy analyses planned for early stopping in case of a
greater than expected difference between treatment arms in terms of hearing loss
SIOPEL 6: Efficacy of STS in Reducing Ototoxicity in Patients
Receiving Cisplatin for Standard Risk Hepatoblastoma
Diagnostic biopsy
Tumour storage
Radiological
staging
RRR if required
Registration to
remote data entry
site via web
R
A
N
D
O
M
I
S
A
T
I
O
N
cDDP 80 mg/m2
4 Cycles
cDDP 80 mg/m2
STS: 10-20 gm/m2
depending on age/weight
4 Cycles
80% power to detect 60% vs 35% hearing loss
Two interim and one final efficacy analyses planned
for early stopping in case of a greater than expected
difference between treatment arms in terms of
hearing loss
D
E
L
A
Y
E
D
S
U
R
G
E
R
Y
cDDP
2 Cycles
cDDP + STS
2 Cycles
STS: Development Timeline
Event
FDA Type C Clinical Development Meeting
Timing

Presented to Pediatric ODAC 
Mar 2011
ODAC recognized challenge of demonstrating STS does not reduce efficacy of cisplatin and
agreed adult study would not be appropriate
Nov 2011
COG ACCL0431 Clinical Data
Oct 2013
SIOPEL 6 Interim Efficacy Analysis
H1 2014
FDA Type C Clinical Meeting – Agree Data Acceptable for NDA
H1 2014
FDA Pre NDA Meeting
mid 2014
NDA Submission
1Q 2015
STS Market Opportunity
Pediatric Market Opportunity
12,000 children develop cancer in the US every year
2,000 children will receive platinum-based chemotherapy, 3x ROW
Pricing to be determined based on available therapies in the market
Competitive Position
Significance of injury increases value of STS
Limited competition – hearing aids and cochlear implants do not prevent hearing loss
Hearing aids cost $2000 to $6000 each
Cochlear implants cost up to $75,000 each
Third party market research shows strong adoption characteristics*
Physician approval very high
Payors feedback positive
*Campbell Alliance and Panel Intelligence market research, analysis and surveys
Summary
STS has Two Potential Near Term Value Drivers
Positive data from COG Phase III trial will support filing of NDA
Upon approval of NDA, receipt of Rare Pediatric Disease Voucher
Attractive Commercial Market Opportiunity
2,000 patients in the US (3x ROW), limited competition
Third party market research shows potential for strong adoption
Eniluracil is active and well tolerated
Indications where 5-FU is administered potentially in excess of $1
BLN market
Seek partnership for further development of Eniluracil