bioentrepreneurship: effective communications - selling your story
DESCRIPTION
MaRS BioEntrepreneurship Series Event, June 12, 2007 Speaker: Wayne Schnarr, Senior VP, Life Sciences, The EquicomGroup More information including a video: http://www.marsdd.com/bioent/june12TRANSCRIPT
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effective communications
selling your story
MaRS
BioEntrepreneurship
June 12, 2007
Wayne Schnarr
Senior VP, Life Sciences
The Equicom Group
Forward-looking statement
Certain information included in this document is forward-looking and is subject to important risks and uncertainties. The results or events predicted in these statements may differ materially from actual results or events. For additional information with respect to certain of these and other factors, see the reports filed by the various companies mentioned in this presentation with the Securities Commissions of Ontario, Alberta and British Columbia. These companies disclaim any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. This document does not constitute an offer to sell or a solicitation of an offer to buy securities in the United States. No securities have been registered under the United States Securities Act of 1933, as amended or any state securities laws.
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always remember
what are YOU selling
to whom are YOU selling
what is the sales process
never stop selling
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you are selling
or you are on the receiving end of sales pitches
every day
giving lectures
what are you selling
information, passion, ability to think
to whom are you selling
the students in the chairs
what is the sales process
lectures plus
never stop selling
or they fall asleep or don’t attend
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writing grant proposals
what are you selling
your intellectual capabilities
to whom are you selling
granting agencies
what is the sales process
annual submissions
selling never stops
unfortunately not
selling your story to
the financial community &
the health care industry
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the financial community
why do people invest in biotechnology companies?
to make money!
how do VCs make money from investing in biotechnology companies?
through an IPOthrough sale of the company
nobody invests in early stage biotechnology companies for a 10% ROI
“you have to show them the 10-bagger”- Michael Denny
biotech investors buy growth
based on the current and future sales of products and
services
approved products
growth of sales and earnings
no approved productsprogress in preclinical and clinical developmentindependent product validation by partners
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biotech investors balance risk and reward
rewardmulti-billion dollar marketfirst-in-classbest-in-classcure
riskmarket riskclinical riskscientific risk
biotech investors balance risk and reward
rewardwhat are you selling?• a potential reward that justifies the current valuation• a potential reward that is large enough to allow an IPO or M&A transaction
riskwhat are you selling?• you are NOT selling a risk-free opportunity• that you clearly understand the scientific risk• that you can mitigate the clinical risk• that you understand the market risk
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effective presentations:
PowerPoint 101
PowerPoint is on your computer –
which does not mean that you are an expert at creating presentations.
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the presentation should:• be there for the audience, not for the presenter• be useful for multiple audiences• be useful both on the screen and as a handout• not contain every piece of information that is in your business plan• be about 30 slides for a general audience or introductory meeting• have a consistent format, colour scheme and font• be talked to or about, not read• use appropriate animal and human pictures• some slides transmit information
– critical slides SELL
powerpoint 101
what is the hunger?how big is the hunger?
no big hunger …no one cares
need expressed:
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what is the hunger?• what is the specific disease or medical condition• is there a specific subgroup that is being targeted• what are the currently approved drugs, if any, and what are their deficiencies• what is the unmet medical need
how big is the hunger?• be realistic• not every potential patient gets diagnosed or treated• use patient numbers from independent sources• use sales of currently approved drugs where applicable
need expressed
100% validation
<10% validation
Stent Graft
• 400,000 CABG procedures annually• 10 to 20% complications: expensive and life
threatening • 7,000 TMR procedures
• Potentially 30,000 with SPY System
Real-time Cardiac Imaging System
JAMA report on graft failure• Up to 30% of vein grafts used in heart bypass
surgery fail at one year or less
12 peer-reviewed journals - 2000 patient data• Improves clinical outcome: 5 - 8% revisions• Equivalent to X-Ray angiography in real-time
Opportunity and Unmet Need
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Opportunity and Unmet Need
Real-time Vascular Imaging System
• US Market– 300,000 plastic and reconstructive surgeries– 60,000 solid organ transplants– 100,000+ tumor margin detection
• 30% of breast reconstructive surgeries may experience complications
• Poor perfusion: #1 contributor to complications and failed procedures
• No other practical method of assessing tissue or organ perfusion
Coronary Heart Disease
20%of all deaths
oneNumber
killer!
11
54 millionlow HDL
Americans with
There are
Top 3 drug categories 2004 annual sales
5 blockbuster
Cytostatics(Cancer)
Antiulcerants(GI tract)
Cholesterol & triglyceride
reducers
World’s Largest Drug Market
drugs$23.8B
$25.5B
$30.2B
}
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23| IPO presentation | iCo Therapeutics
Nothing disruptive
No blockbusters
The Old Ophthalmic World Order
Allergan & Alcon
23| IPO presentation | iCo Therapeutics
24| IPO presentation | iCo Therapeutics
efficacy in treating wAMD 2006 F2007
Decimated incumbent therapies
Revenue
95%
Now Lucentis is a Big Success
*Source: Genentech documents and Rodman & Renshaw analyst reports
$940 million*
$380million*
24| IPO presentation | iCo Therapeutics
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25| IPO presentation | iCo Therapeutics
targets one growth factor (VEGF)
monthly injections to retain efficacy
Lucentis is a big success, but it is not over
Only Requires
25| IPO presentation | iCo Therapeutics
26| IPO presentation | iCo Therapeutics
wAMDis not
Our Focus is DME
DME(diabetic macular edema)
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27| IPO presentation | iCo Therapeutics
wAMD DME
VEGF bFGF IGF-I EPO HGF integrins
wAMD vs DME
Acute event
Elderly
Disease progression
Average onset
Gradual deterioration
Working years
Predominantly VEGFPathology
28| IPO presentation | iCo Therapeutics
Proliferation of new blood vessels in PDR
More blood brought to area
Vessels are permeable
Blood leaks into retina area
Causes swelling & deformation of retina
DME: Leaking Vasculature at Back of Eye
28| IPO presentation | iCo Therapeutics
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29| IPO presentation | iCo Therapeutics
Normal Retina Retina with DME
DME: Disease State
30| IPO presentation | iCo Therapeutics
• Even well-timed and adequate laser treatment only effectively controls edema in 50% of patients with CSME and repeated laser therapy (more than three or four treatments) is contraindicated dueto cumulative destruction of the visual field. Patients with diffuseor cystic macular edema tend to have a poorer response to laser.
• Kenalog is gaining usage in these patients, and those with vitreous traction are candidates for vitrectomy.
Kenalog or Vitrectomy
Laser Photocoagulation
Unsuccessful
Successful
DME1.6 MM pts
DR5.3 MM pts
Recurrent
Kenalog or Vitrectomy
DH Insight Briefing – Ophthalmology | November, 2005 pg. 44
DME Treatment Options are Currently Limited
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T S X : M S
Inappropriate immune attack on the protective coating (myelin) surrounding the nerves of the brain and spinal cord
MBP8298 suppresses immune attack at the most common molecular target
Multiple Sclerosis (MS)
T S X : M S
Geography Genetics
of all MS patients have HLA-DR2 or HLA-DR4 genes (our responder group)
75%up to
MS Susceptibility Factors
2.5 million patients
T S X : M S
DR2/DR4
DR2/DR4
DR2/DR4
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T S X : M ST S X : M S
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T S X : M SMS has Two Major Populations
MBP8298 Phase II trial
MBP8298 Phase III trials
Market40-45%of MS patients
Market40-45%of MS patients
Relapsing Remitting MS(RRMS)
Secondary Progressive MS (SPMS)
50% convert in 10 yrs
90% convert in 25 yrs
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T S X : M SMS has Two Major Markets
US$5.8B current market
Blockbuster market potential
Relapsing Remitting MS(RRMS)
Secondary Progressive MS (SPMS)
~500,000 treated patients annually of >1 million patients
Few patients treated of>1 million patients
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T S X : M SMS has Two Major Markets
Approved ProductsBiogen Idec: Avonex®
Bayer Schering Pharma AG: Betaseron®
Teva: Copaxone®
Merck Serono S.A: Rebif®
Biogen Idec/Elan: Tysabri®
Relapsing Remitting MS(RRMS)
Secondary Progressive MS (SPMS)
US$5.8B currentmarket
Approved ProductsBetaseron® (No proven delay in progression, only approved with relapses)Novantrone® (Cardiotoxicity limits use to 2 – 3 years)
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Blockbuster market potential
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B R E A K I N G T H R O U G H
6 millionpatients in the worldAnnual incidence: 700,000
arrhythmias 1 in 4 adults will get AF (age 40+)
* Wang et al. Circulation: Journal of the American Heart Association. August 2004.
AF Incidence
B R E A K I N G T H R O U G H
Current treatment drawbacks
drugs heat ablation
treats symptoms only
30 – 60% effectivenessdiminishes over time
serious side effects
used in <3% of casesonly when drugs fail
high procedural risks:PV stenosisThrombosisEsophageal perforation
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B R E A K I N G T H R O U G H
Healthcare ramifications and risks
1
2
3
4
>15%
>20%
415,000 (U.S.)
US$6.6 billion
leading cause of stroke
leads to chronic heart failure
leading cause of hospitalizations
cost to healthcare
* Donald M Lloyd-Jones, Md, ScM, FACC. Medscape Cardiology 8 (2). 2004.
no practical solution!
AF Business OpportunityUntreated Pool New Cases/Year
2.2 million 160,000
2.1 million 145,000
4.3 million 305,000
>$2 BillionAnnual Business Opportunity
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solution to the hunger -
the valueproposition
need addressed:
what is your product?• type of drug, device, diagnostic• what is the mechanism of action
what is the value of your product?• the value is in the data• human data is more valuable than animal data• if you have human data, eliminate or minimize the animal data• consider the audience when assessing the complexity and presentation of the data being used
need addressed
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Neuradiab Survival Data
Clinically compelling difference
42%
1980 to
2004
2005
91Surgery + Radiation + Temozolomide + Neuradiab
Surgery + Radiation + Temozolomide
Surgery + Radiation
Survival in weeks
75604515 30 105900
weeks
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64weeks
53weeks
iCo–007for the treatment of
DME
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45| IPO presentation | iCo Therapeutics
iCo-007: Method of Action (MOA) - VEGF Plus
Signal through c-Raf
Growth factors
initiate signal
c-Raf
Retina
45| IPO presentation | iCo Therapeutics
VEGF
HGF
EPO
46| IPO presentation | iCo Therapeutics
iCo-007: MOA - VEGF Plus
iCo-007 inhibits the production of c-raf, thereby preventing the signaling of growth factors, which in turn prevents the production of new and permeable blood vessels
Growth factors
Modulatesignal
Retina
46| IPO presentation | iCo Therapeutics
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47| IPO presentation | iCo Therapeutics
iCo-007: MOA - VEGF Plus
iCo-007Inhibits c-Raf production which prevents cell growth and permeability
ribosome
The production of c-RafSignal pathway Signal pathway interrupted
The inhibition of c-Raf
mRNA
47| IPO presentation | iCo Therapeutics
48| IPO presentation | iCo Therapeutics
c-Raf immunostainingof porcine eye
107189Treated
Control
120
100
80
60
40
20
0Saline D7
34μgD7
180μgD14
180μg
% Saline Control
Preclinical Evidence: Inhibition of c-Raf
48| IPO presentation | iCo Therapeutics
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49| IPO presentation | iCo Therapeutics
Preclinical Evidence: Inhibition of new blood vessel growth in mouse eye
Saline 14μg ISIS 15770
50| IPO presentation | iCo Therapeutics
Single intravitreal
injection
Preclinical Evidence: 3 month dosing achievable
(90 μg)
Half life
44 days
50| IPO presentation | iCo Therapeutics
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T S X : M SMBP8298: Overview
* Based on previous clinical trial results
IV ’push’ every six monthsConvenient administration
Synthetic peptide for specific responder group (up to 75% of MS patients)
Designer drug
Delayed median time to progression for 5 years in responder groupLong-term efficacy*
Epitope-specific tolerance, not general immunosuppressantUnique mechanism
Side effect: minor injection site irritationVery safe*
Indications: SPMS & RRMSOnly novel agent for SPMS in Phase III trials
T S X : M ST S X : M S
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MBP8298 slows the progression of MS
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T S X : M S
Analysis of CSF autoantibodies guided development• RRMS: Autoantibodies detectable only during relapse events• SPMS/PPMS: Autoantibodies continuously present – useful indicator of drug effect
Drug concept: Induction of antigen-specific tolerance• Observed in early vaccine development• High dose intravenous administration of soluble antigen
MBP8298 was designed to replicate the most common antibody target• IV administration suppressed CSF autoantibody levels in most patients• HLA-DR2-restricted T-cells target the same sequence
HLA-DR genes• Direct the fine specificity of immune responses • HLA-DR2 and -DR4 predispose to MS and make up the majority of patients• Easy genetic test for HLA type
MBP8298: Drug discovery/ development for progressive MS
T S X : M SMBP8298 Replicates the Myelin Target
MBP829817 amino acid peptideIdentical to the natural
sequenceBlood Vessel Blood Brain
BarrierNerve Fiber
T-cells & B-cells from immune system attack 82-98 portion of myelin in HLA-DR2 (and other) patients1
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T S X : M SMBP8298 Treatment Induces Tolerization
Blood Vessel Blood Brain Barrier
Nerve Fiber
MBP8298synthetic peptide identical to dominant site of immune attack
500mg dose every six months
“Classic” Tolerization Principle:
“Reverse” of vaccination
Established in vaccine research 50+ years ago
Shown to cure or prevent EAE animal models of MS
2
T S X : M SImmune System is Tolerized
Tolerization Result:
Eliminates antibodies to MBP8298 for six+ months
Requires dose every six months
Clinical delay in disease progression
Blood Vessel Blood Brain Barrier
Nerve Fiber
3
4
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T S X : M SPublished Efficacy Results
“Long-term follow-up treatment and assessment of patients in this responder group showed a median time to progression of 78 months for MBP8298 treated patients compared with 18 months for placebo-treatment (Kaplan–Meier analysis, P = 0.004…)”
T S X : M SFive Year Delay in Progression
Placebo18 months MBP8298
78 months
Phase II Trial: Kaplan-Meier Analysis of HLA-DR2 and/or DR4 patients at 84 Months
% N
ot P
rogr
esse
d
Endpoint: Time to 1st confirmed progression on EDSS
MBP8298
Placebo
20
40
60
80
100
0
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Insulin - Exploding Demand
2006
2012
6,000 kg
16,000 kgWho is going to fill the gap?
Company estimates 13
Fermentation is One Alternative
$
4 to 6 yearsLong lead times
2006: 6,000 kg of insulin
2012: 16,000 kg of insulin
1 (est.)Capital invested in existing manufacturing plants
2.5XMore capital required
B.2
14
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Supplying World Insulin Demand
15,0003 commercial farms
Supplyfor the entire planetin 2012
15
acres or
1 commercial farm
1 mile
0
Economics of Plant-Produced Insulin
insulin expression oftotal seed protein
acre produces 1 kg of insulin
acres to supply 2012 projected insulin demand
est. capital cost for 1,000 kgof plant-produced insulin
Enabling technology to meet insulin demand
% Capital cost reductioncompared to fermentation
1.2 %~1.015,000$80M
Floret Seed
Safflower
16
7
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Insulin - Chemical Equivalence
Safflower-derived insulin:Molecular mass 5807 Da
Safflower-derived insulin:
• chemically equivalent to commercially-available human insulin
• folds identically to commercially-available human insulin
V8 protease fingerprinting
Commercial pharmaceutical-grade insulin:Molecular mass 5807 Da
Electrospray Mass Spectrometry
17
Insulin - Functional Equivalence (mouse)
Saline
USP Insulin (pharma grade standard)
Insulin (Humulin® R-Eli Lilly)
Insulin (SemBioSys)
Safflower Insulin Tolerance Test
18
error bars = +/- SEM20
40
60
80
100
120
0 50 100 150Minutes Post Injection
% In
itial
Blo
od G
luco
se
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the action plancreating value:
you have already sold them:• the unmet medical need• the value proposition
what is left to sell them?• that you can increase the value of the product by appropriately spending their money
action plan
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Reversesplaque build-up
Regulatory Path
There is a clear
Conclusions from the meeting:
• Insulin can follow the abbreviated 505(b)(2) rule
• First human trial will be a Phase II for pharmacokinetics & pharmacodynamics
• 50 subjects, 1 month study
• Second human trial will be aPhase III for longer-term safety
• 500 subjects, 6 months, 2 arms safflower insulin and Humulin®
• 500 subjects, 6 months, 1 arm safflower insulin only
• No special regulations related to plant, QC/OA requirements cover all host related issues
safflower-derived insulin
Met with the FDA for a pre IND consultation in October 2006
processregulatory
for
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68| IPO presentation | iCo Therapeutics
- 6 month follow-up
- Open label
- Secondary efficacy endpoint
Dr. Philip RosenfeldMiami FL
Trial Design
- 15-30 patients
- Single administration
- Ascending dosage
- 3 planned centres
Dr. Scott CousinsDurham NC
Dr. David BoyerBeverly Hills CA
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69| IPO presentation | iCo Therapeutics
In-license
Initiate Phase 1
- 15-30 patient trial
- Dose escalation study
Phase 1Results P2
Out License
iCo-007: Achievements and Milestones
H22007
H22008
H22005
Safety
H12007
- IND Accepted
- Manufacturing scale-up
- License from ISIS- World wide rights
- All therapeutic indications
- Minimal upfront payment- Back-end loaded milestones
- Royalty rate doesn’t impede partnering
NDAP3
T S X : M SOngoing Clinical Development
MAESTRO-01Pivotal Phase III SPMS trial – Canada and Europe
• Powered for HLA DR2 & DR4 responder group• Placebo-controlled, double blind, 2-year treatment period• Recruitment is complete
• Includes approximately 550 patients • Interim analysis: mid-2008
MAESTRO-02Open-Label, Follow-on Portion to MAESTRO-01
• After patients have completed 2 years of treatment in MAESTRO-01, patients may chose to receive MBP8298
• MAESTRO-02 will primarily evaluate long-term safety • Support regulatory submissions
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T S X : M SOngoing Clinical Development
MAESTRO-03Pivotal Phase III SPMS trial – United States
• Received FDA clearance to proceed with pivotal phase III• Placebo-controlled, double blind, 2-year treatment period• Powered for HLA DR2 & DR4 responder group• Up to 510 patients
MINDSET-01Phase II RRMS trial – Europe
• Fifteen month, double-blind, placebo-controlled • Up to 215 patients from 30 sites• Followed by 12-month active treatment open label extension
period
T S X : M S
LeadInvestigatorsDr. Mark FreedmanOttawa General HospitalCanada
Dr. Carolyn YoungThe Walton CentreClinical Trials CentreLiverpool, UK
Dr. Tomas OlssonKarolinskaUniversitetssjukhuset, SolnaStockholm, Sweden
Professor Hans-Peter Hartung Neurologische KlinikHeinrich-Heine-UniversitätDüsseldorf, Germany
MAESTRO-01 Phase III SPMS TrialT S X : M S
Trial sites around the world
Canada & Europe• 48 trial sites• 10 countries
72
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T S X : M STarget Clinical Timelines
2007 2008 2009 2010 2011
MBP8298 Timelines
Phase III SPMS Trial MAESTRO-01
Phase III SPMS Trial (US Trial)
MAESTRO-03
Phase II RRMS Trial MINDSET-01
Trial Data Analysis & submission
Enrollment TrialRegulatorySubmission
Open Label Extension
Trial Completion
Phase III Trial
Interim Analysis
Data Analysis & submission
Data Analysis & submission
Interim Analysis
Enrollment Trial
T S X : M SNear-Term Milestones
Interim analysis in MAESTRO-01 trial (mid-2008)
Initiate enrolment in Phase III MAESTRO-03 trial in United States (mid-2007)
Complete enrolment in Phase II MINDSET-01 RRMS trial in Europe (mid-2007)
Completion of MINDSET-01 Phase II trial (mid-2008)
Potential Partnerships
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Milestones
Q3 2005 Q4 2005 Q1 2006
X
X
X
X
X
X
U.S. sales partner
Installed in 25 U.S. hospitals
Initiate studies for expanded indications
Initiate North American multi-centre trial
Initiate combo trial with multi-national partner
File for Canadian and European approval
SPY System
OPTTX System
Pipeline Strategy & Corporate OverviewReal-time medical imaging in the
operating room
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Real-time Image Guidance in the Operating Room
Pipeline Product Strategy
Design / POPFDA Clearance
Trialsfor FDA
Market Development Launch
Install base in US 70+ devices
Install base in US 150+ devices
Launch H2-07
Launch H1-08
Launch 2008
Launch H1-08
FDA clearance
Q3-07
Human POP
Human POPH2-07
Human POPH2-07MINI
Other Urological Procedures
Prostate
Plastic / Recon / Transplant
TMR
Cardiac
SurgicalUrology
FDA cleared Q1 2007
Health Canada Approved
MIS
$900 million
Recurring revenue model
Average kit price $700Cost of goods $200
Marketed products opportunity &
Real-time Image Guidance in the Operating Room
$600MM+860,000 / year $250MM*
400,000 / year$50MM*60,000 / year
*Selling Price Country-specific
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$1 Billion
Real-time Image Guidance in the Operating Room
$700MM1,000,000 / year $250MM*
400,000 / year$50MM*60,000 / year
Pipeline product opportunity & MINI
Recurring revenue model
Average kit price $700Cost of goods $200
*Selling Price Country-specific
Structure: Newly Diagnosed GBM Endpoints
Multi-Center Pivotal Trial Design
Randomized1 arm is standard of care1 arm adds Neuradiab as adjunct therapy
310 patients per arm30-40 sitesLarger than Temodar study
PrimaryOverall SurvivalFinal analysis at 456 events
Secondary1 and 2 year survivalProgression-free survival
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Multicenter
Exploratory AnalysisPotential to see incremental benefit in non-Temodarresponders
80
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FDA consultation CRO selection
CRO selected by Bradmerfor Phase III trial
Prologue Research International
Oncology specialist; former oncology clinical op’s team from Pharmacia-Adria
Big pharma and biotech clients
Experience with radiopharmaceutical trials
1181
Trial Design Recent Events
FDA approved Bradmer’s plan for Phase III trialNovember 2006End of Phase II meeting
Q2 2007Final protocol and manufacturing data to be submitted
Laureate Pharma MDS Nordion
cGMP Manufacturing – Transition to Commercial
Antibody Producer Radiolabeling Partner
1182
Status Report:Antibody scale-up complete
Sufficient supply to complete Phase IIIcGMP antibody product completed in January
> 2 dozen successful equivalence testsFinal formulation, optimization of radiolabel process ongoing at NordionClinical trial material to be released mid year
82
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Neuradiab Pivotal Trial Timeline
2006 2007 2008 2009 2010
Trial Preparation Mfg / FDA / Site
Prep / Svc Providers
Trial LaunchMid 2007
Enrollment Complete
2011
NDAsubmission
License from DukeQ4 2005
Full DataAnalysis
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“Open Label” Trial
can you do it all in a few slides?
yes!
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PAC-113
Uncontrolled Infectious Disease
Healthy State Immune Compromised
Immune system controls growth of resident
fungus and bacteria
75%present in up to
of the population
Impaired immune system permits attack by resident
fungus and bacteria
High probability of recurrence
Patients being treated for cancer and diabetes
30% of asthma patients treated with corticosteroids
90% of AIDS patients (and up to 43% of HIV patients)
Oral CandidiasisCandida albicans
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Current Treatments Deficient
Current treatments are ineffective, cause drug resistance, cause severe side effects
A novel, safe and effective treatment has the potential to generate US$300 – US$400 million per year worldwide.
Amphotericin BSystemic
Severe side effects
NystatinTopical
52% efficacy
AzolesSystemic/Topical
Potential for resistance
Fighting Infectious Disease: PAC-113
Based on natural antimicrobial peptide occurring in saliva
PAC-113, delivered as mouthwash, binds to fungus surface and kills quickly
Highly active against Candida, greater than 95% efficacy in vitro
45
Four safety and efficacy trials completedIndication: gingivitis (gum disease)300+ patientsConducted in the U.S. by Periodontix Inc.
Safety Established Clinically
Well tolerated
No drug related adverse events
Dose related improvement in clinical endpoints
PAC-113 Clinical Strategy
Initiated Phase I/II trial
Q1-06
Results from Phase I/II trial
Q1-07
Initiate dosing in Phase II trial
Q3-07
Clinical objectives:Safety and tolerability
Efficacy in eliminating or reducing clinical signs and symptoms of infectionMicrobiological response
PAC-113 NystatinHead to head comparisonRandomized, examiner-blinded, parallel design
44 HIV patients per arm
14-day treatment phase 14-day follow-up period day 28 follow-up visit
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Significant Market Potential
– C. albicans– C. glabrata– C. parapsilosis– C. tropicalis
90% of all AIDS patients30% of asthmatics on steroidspatients on chemotherapy /
radiation
US $300-$400M US $1.6B
Treatment of topical fungal infections
Treatment of oral candidiasis
PAC-113 has demonstrated activity against:
PAC-G31P
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Uncontrolled Immune Response
Healthy State Diseased State
1.Pathogens induce an inflammatory response by the immune system
2.One component of the inflammatory response is neutrophilinfiltration
3. Excess neutrophilscan create serious medical complications
ARDSAsthmaCOPD
Pneumonia
Regulating Immune Responses: PAC-G31P
Cytokines bind to CXCR1 and CXCR2
receptors, attracting and activating
neutrophils
PAC-G31P closely resembles cytokines and blocks CXCR 1/2
cytokine
neutrophil
CXCR1
CXCR2
48
Low
dos
e PA
C-G
31P
Endo
toxi
nco
ntro
l
PAC-G31P Effective in Animal ModelsN
eutr
ophi
lco
unt
30
0
15
October 2005, The Journal of Leukocyte Biology
Nor
mal
ani
mal
Hig
h do
se P
AC-G
31P
PAC-G31P Clinical Strategy
Currently manufacturing clinical materials
Proceed with preclinical toxicology work for systemic administration
Proof-of-concept clinical study in Asia in 2007 in collaboration with a third party
Start North American Phase I single dose clinical trial in healthy volunteers by the end of 2007
Manufacturing
Ongoing
Toxicity Study
H1-07
Asian Clinical Study
H1-07
North American Ph. I
H2-07
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Markets
ARDS:– Mortality is 30% to 40%– No approved drugs for prevention or treatment
Asthma:– 1.8 million emergency room visits in 2004– Total cost to the U.S. in 2004 was $16.1 billion
COPD:– 4th leading cause of death in the U.S.– The cost to the U.S. in 2004 was in excess of $37 billion
150,000people affected
annually in the U.S.
10.7 millionAmerican adults were believed to have COPD (2003)
COPDARDS20.5 millionAmericans were
estimated to have Asthma (2004)
Asthma
are you finished selling?
never!
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don’t stop selling
What is left to sell?managementboard and other advisorsintellectual propertymanufacturingquality of current shareholdersfinancial situationexit opportunitiesinvestment highlightsetc.
Product Pipeline
OTC / Topical RxStratoDerm™
DHA Rich Oil
GLA Rich Oil
ImmunoSphere™
DermaSphere®
Apo AI
Insulin
Nutritional supplements
Nutritional supplements
Animal health
Personal care
Atherosclerosis
Diabetes
2008
2010
2014
2008
2010
Non
-pha
rmac
eutic
alP
harm
aceu
tical
Class Product Indication Launch
2008
35
2005
51
Corporate Overview
36
Established: 1994: Spin out –University of Calgary
Stock Market: TSX: SBS.TO
Market cap: $67MM
Cash: $28.8MM (31/03/07)
Burn rate: - 2006: $1.0 MM per month- Cash to early 2009
Employees: 65 (19 Ph.D.s)
Board of Directors
Richard Smith (Chairman) Former President & CEO Dow AgroSciences Canada, Inc.
Andrew Baum Director, President & CEOSemBioSys Genetics Inc.
Alexander R. Giaquinto, Ph.D. Former, Sr. VP, Global ComplianceSchering-Plough
Douglass Given M.D., Ph.D. Partner, Bay City Capital
Nancy Harrison Former Senior VPVentures West Management Inc.
David Howard ChairpersonAngiotech Pharmaceuticals, Inc.
37
52
Patents
Protecting the platform11 U.S. patents and applicationsProduction of recombinant proteins in plants using the oilbody-oleosin technology platform
Protecting the tools8 U.S. patents and applicationsTools and techniques to make our products
Protecting the products16 U.S. patents and applicationsComposition of matter, manufacturing method, and method of use claims directed to formulations comprising oilbodies
As of January 12, 2007
patents issued(19 U.S.)
patents pending(16 U.S.)
139
139
38
Upcoming Milestones
39
Initiation of new pharmaceutical product development program Complete Dermasphere Facility and begin full scale manufacturing and salesExecute commercialization plan for ImmunoSphere™product and launch in Q1 2008
Other
Q3 2007 Q3 2007
Q2 2007 - Q1 2008
Animal Data in Model System – ArabidopsisAchieve commercial levels of Apo AI expression in safflowerPartnership Opportunities
Apo A1Q3 2007Q3 2007Q4 2007 - Q2 2008
Submit insulin IND to FDA Initiate Phase II trials with completion by Q2, 2008Partnership Opportunities
Q4 2007 Q1 - Q2 2008Q2 - Q4 2008
Insulin
53
105| IPO presentation | iCo Therapeutics
Vancouver, BC, CanadaHead Office
$600,000 monthBurn-rate
$2.7 millionCash on Hand
$7.5 millionRaised to Date
2005Founded
Corporate Data
106| IPO presentation | iCo Therapeutics
Andrew Rae, MBAFounder & CEO
John Clement, PhDFounder & Chief Technical & Development Officer
Santa Jeremy Ono, PhDChief Scientific Officer
Peter Hnik, MD, MHSc.Chief Medical Officer
John Meekison, BA, CIM, P. Log.Founder & Chief Financial Officer
Sidney Himmel, CAChairmanPresident and Chief Executive Officer, Trigon Uranium Corp.
William Jarosz, JDCartesian Capital Group, LLC
Richard Barker, PhDDirector General of the Association of the British Pharmaceutical Industry
Richard GlickmanCo-founder, CEO and Chairman, Aspreva Pharmaceuticals
George Lasezkay, JD Principal, Turning Point Consultants, LLC
Julia Levy, PhDCo-founder of QLT
Alan C. Bird, MDEmeritus Professor, UCL
David Boyer, MDRetina-Vitreous Associates Medical Group
Philip Rosenfeld, MD, PhDProfessor, Bascom Palmer Eye Institute, University of Miami, School of Medicine
Jason Slakter, MDClinical Professor, NYU School of Medicine
Non-Executive Directors Strategic Advisory Board
Extensive public company and life science experience | Solid operational and product development expertise | Ophthalmic specific expertise
Management and Directors
Management
54
107| IPO presentation | iCo Therapeutics
PfizerEyetech(wAMD)
NovartisGenentech(wAMD)
BayerRegeneron(wAMD)
MerckSirna(wAMD)
NovartisQLT(wAMD)
AlconAmgen(Ophthalmic Therapies)
PfizerAngiosyn(wAMD)
AllerganSirna(wAMD + other ophthalmic diseases)
OSIEyetech(wAMD)
Hot Therapeutic Arena
108| IPO presentation | iCo Therapeutics
Investment Highlights
Very attractive valuationManagement team / advisory board with extensive ocular experience
Initial market - $100 million potentialPhase 2 commencing now
iCo 008 targets multiple indications
>$1 billion potential Phase 1 commencing now
iCo-007 has large market/blockbuster potential
55
An example
Investor Presentation
June 2007
56
Forward-looking statement
Certain information included in this document is forward-looking and is subject to important risks and uncertainties. The results or events predicted in these statements may differ materially from actual results or events. For additional information with respect to certain of these and other factors, see the reports filed by ARIUS Research Inc. with the Securities Commissions of Ontario, Alberta and British Columbia. ARIUS Research Inc. disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. This document does not constitute an offer to sell or a solicitation of an offer to buy securities in the United States. No securities have been registered under the United States Securities Act of 1933, as amended or any state securities laws.
“The mission of ARIUS is to discover and develop the next wave of antibody drugs to
address the needs of patients and physicians for safe, effective treatments.”
Breakthrough antibody drugs
57
Hitting a target doesn’t guarantee success
1000’s of high affinity Antibodies have been identified
In the last decade,
only ~20 havebeen approved and
successful
There is a target within the target
The desired effect (cell death / cell signaling) will only be triggered by
specific areas within the targeted antigen
58
ARIUS focuses on
“Does the antibody kill cancer cells while leaving normal cells alone?”
we have successfully If YES, then,
hit the target within the target
FunctionFIRST™
Select antibodies for function(In vivo activity)
Immunize with primary human tumor
ARIUS Discovery Paradigm
Generate Antibodies
Identify target and eliminate those with prior patents
Patent ARIUS antibody and all epitopes on target with positive efficacy
Proprietary antibody discovery and selection platform
59
FunctionFIRST™ yields results
400A pipeline of
antibodies
lead candidatesThree
partnershipsFive
$400 Millionin potential milestone payments
Genentech Oxford bioMedica
Takeda PDL BioPharma
Medarex
Our Partners
60
Genentech exclusive antibody license
Top oncology antibody company with blockbuster successes:
• Avastin©• Rituxan©• Herceptin©
Partnership signed March 2006• Upfront licensing fee• Milestone payments based
on clinical milestones• Royalties
Prevents tumor growth and enhances survival in breast cancer models
Tum
or V
olum
e (m
m3 )
Days Post-ImplantationPe
rcen
t Su
r viv
al (
% )Antibody Dosing: 15 mg/kg i.p. 3x /week x 10 doses
0 50 100 150 2000
20
40
60
80
100
120Isotype controlAR7BD-33-11A
Treatment period
0
250
500
750
1000
1250
1500
0 10 20 30 40 50 60 70 80
Treatment period
Other leaders partnered with ARIUS
Takeda• Japan’s largest pharma
• Multi-product collaboration
• $1M in cash upfront/$1M equity
• Fund research activities for life of deal
PDL BioPharma• Leader in antibody humanization
• Partnership to discover and develop antibodies
• Option for in-license antibodies
Medarex• Joint research program using
Medarex’s UltiMab Human Antibody Development System
• The combination of technologies eliminates the humanization step in the FunctionFIRST™ platform
Oxford BioMedica• Agreement to jointly develop products
for cancer therapy
• Oxford characterized the antibodies and identify cognate antigens
• Liver cancer target identified - 37LRP –(AACR 2006) and being developed
61
Our Products
CD44 Cancer Stem Cell ProgramAntibody AR001
CD44 cancer stem cell program
0
100
200
300
400
500
600
700
800
0 10 20 30 40 50
Buffer Control
20mg/kg
10mg/kg
2mg/kg
0.2mg/kg
Treatment Period
• Aberrant expression of CD44 occurs in a variety of tumors
• CD44 implicated as a functional cancer stem cell marker in leukemia, breast and prostate cancer
• Arius lead antibody targets the CD44 antigen
• ARH460-16-2 efficacy shown in breast and prostate tumors
Produces Breast Cancer Regression
Tum
or V
olum
e (m
m3 )
Days Post-Implantation
Perc
ent
Sur v
ival
(% )
0 20 40 60 80 100 120 140
0
20
40
60
80
100
120
Buffer Control Isotype Control ARH460-16-2
Treatment period post-treatment period
Increased Survival in Breast Cancer
62
CD44 – cancer stem cell marker
Cancer stem cells are the factory of the tumour
Chemotherapy shrinks tumour
Tumour returns
Targeting cancer stem cells may be more effective
Cancer building ability is reduced
Survival is extended
AR001 demonstrates Significant inhibition oftumor growth and metastases in a dose response, human liver cancer model
CD44 cancer stem cell program
63
Trop-2 Signal Transduction ProgramAntibody AR002
Trop-2 signal transduction program
0
200
400
600
800
1000
1200
1400
0 10 20 30
Days Post-Implantation
Tum
or V
olum
e (m
m 3 )
BufferAR002
Treatment Period
• Trop-2 identified as a major determinant of tumor growth and metastasis
• Over-expression of Trop-2 increases growth rates in several types of cancer
• ARIUS Trop-2 antibodies are the first to demonstrate efficacy
Inhibits tumor growth and increases survival in prostate cancer models
Days Post Implantation0 20 40 60 80 100
Per
cent
Sur
viva
l (%
)
0
20
40
60
80
100
120 Buffer ControlAR002.
Treatment Period
64
Cell Proliferation
Erbitux, Herceptin, VectibixErbitux, Herceptin, Vectibix
Tarceva, IressaLapatinib, TarcevaTarceva, , IressaIressaLapatinibLapatinib, ,
AvastinAvastin
GleevacGleevacGleevac
GleevacGleevacGleevac
SHP-2Shc
VEGFRVEGFREGFREGFR PDGFRPDGFR
MEK
RAF
Grb2
SOS
RAS
MAPK
Antibodies
Small molecule inhibitors
TROPTROP--22
AR47A6.4.2AR47A6.4.2
Novel target in key cancer pathway
CD59 Immune Modulator ProgramAntibody AR003
65
CD59 immune modulator program
Increases survival in lung cancer models
Shrinks tumors in breast cancer
• CD59 widely expressed in malignant tumors, allowing cancer cells to evade immune system
• AR36A36.11.1 could help activate immune system in addition to targeting cells directly
• AR36A36.11.1 has been demonstrated effective in a number of tumor types
MDA-MB-468 in female athymic nude mice
0
50
100
150
200
250
300
350
400
30 35 40 45 50 55 60 65 70 75 80
No TreatmentVehicleAR003 (20mg/kg)Taxotere (30mg/kg)
Treatment Period
Tum
or V
olum
e (m
m3 )
Days Post Implantation
Treatment Period
Days Post-Implantation0 20 40 60 80 100
Surv
ival
(%)
0
20
40
60
80
100
120 Buffer Control AR003
Epitope location for function-blocking antibodies
Epitope location for Non-function-blocking antibodies
Epitope location for ARIUS Antibodies
CD59 immune modulator program
66
Arius antibody pipeline
Robust and growing intellectual property estate
16 issued and allowed patents- Composition of matter of antibodies- Methods of treatment- Antibody discovery technology platform
61 published patent applications
149 patents pending
67
Opportunities
9 licensing deals in 90 days!
AstraZeneca and Regeneron
Pharmaceuticals
US$120Mplus royalties
Genentech and Seattle Genetics
US$860Mplus royalties
Genentech and BioInvent
US$190Mplus royalties
GSK and Antitope Limited
PDL BioPharmaand Trellis Bioscience
Cambridge Antibody and
iCoTherapeutics
Medarexand
Compugen
Wyeth and Raven
Biotechnologies
Pfizer and Elusys
Therapeutics
68
Multiple acquisitions in last 12 months
May 2006Merck
acquiresAbmaxis
US$80M
May 2006Merck
acquiresGlycoFi
US$400M
March 2007Eisai
acquiresMorphotek
US$350M
November 2006GenentechacquiresTanox
US$919M
August 2006 AstraZeneca
acquires Cambridge Antibody
US$1.3B
December 2006GSK
acquiresDomantis
US$454M
ARIUS Milestones 2007
• Sign additional strategic collaboration/partnering agreements
• Advance our programs:– Complete pre-clinical toxicity studies for stem cell program
– Pre-IND meeting with the FDA
– Develop expression cell lines for other 2 lead programs and transfer to cGMP manufacturer
– Advance IND enabling studies for 2 additional programs
• Expand intellectual property and library of antibody drug candidates
69
About ARIUS
Headquarters: Toronto, Canada
Symbol: TSX:ARI
Employees: 40 current (18 last year)
Cash: $19 million
Burn-rate: $2.3 million (Q1, 2007)
ManagementDavid S. YoungChairman, President and Chief Executive Officer
Helen FindlayExecutive Vice President and Chief Business Officer
Warren WhiteheadChief Financial Officer
Susan HahnDirector of Development
Daniel PereiraVice President of Research
Daniel RubensteinChief Medical Officer
Robert GundelChief Scientific Officer
Board of DirectorsWilliam T. BodenhamerDirector
Carl L. GordonDirector
Joe ZakrewzskiDirector
Diane KalinaDirector
Dan AndersenBoard Observer
Chau Q. KhuongBoard Observer
Management and Board
70
Investor Presentation
June 2007
closing thoughts
71
if you had only one slide
need expressed: hunger
market opportunity: greed
need addressed: value proposition
creating value: the action plan
entrepreneurs
life sciences