Raptor Pharmaceutical Inc.
Protein Therapeutics to
Treat Neurodegenerative
Brain Diseases and Cancer
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Forward-Looking Statements
This presentation contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of
1995. These statements relate to future events or our future results of operations or future financial performance, including, but
not limited to, the following: statements relating to our ability to raise sufficient capital to finance our planned operations, our
ability to develop viable drug product candidates, our ability to obtain positive non-clinical trial results, successful clinical
trials of our drug product candidates, our ability to receive necessary marketing clearance approvals from the FDA, our ability
to successfully commercialize our drug product candidates, market acceptance of our drug product candidates and our ability
to successfully compete in the marketplace. In some cases, you can identify forward-looking statements by terminology such
as “may”, “should”, “intends”, “expects”, “plans”, “anticipates”, “believes”, “estimates”, “predicts”, “potential”, or “continue”
or the negative of these terms or other comparable terminology. These statements are only predictions and involve known and
unknown risks, uncertainties and other factors, including the risks that can be found in the section entitled “Risk Factors” on
page 8 of our Form SB-2, filed on June 21, 2006 with the SEC and “Factors That May Affect Future Results” on page 12 of
our Form 10-KSB filed on November 21, 2006 with the SEC (ticker RPTP; Yahoo Finance: RPTP.OB), which may cause our
or our industry’s actual results, levels of activity or performance to be materially different from any future results, levels of
activity or performance expressed or implied by these forward-looking statements. Although we believe that the expectations
reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity or
performance. You should not place undue reliance on these statements, which speak only as of the date that they were made.
These cautionary statements should be considered with any written or oral forward-looking statements that we may issue in
the future. Except as required by applicable law, including the securities laws of the United States, we do not intend to update
any of the forward-looking statements to conform these statements to reflect actual results, later events or circumstances or to
reflect the occurrence of unanticipated events.
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Raptor Company Profile
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Raptor develops protein therapeutics for the treatment of
neurodegenerative brain diseases and cancer.
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Core competencies in protein bioengineering, receptor
biology, non-clinical and clinical development in both
protein and small molecule therapeutics
Corporate and Financial History
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September 2005 - Co-founders Christopher M. Starr, Ph.D. and
Todd C. Zankel, Ph.D. form Raptor Pharmaceutical Inc.
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January 2006 - Asset Acquisition Agreement signed with BioMarin
transferring $16M worth of RAP technology, IP and know-how to
Raptor
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March 2006 - Raptor raises $300K of seed funding, hires CFO
Kim Tsuchimoto, signs facilities lease, and begins PIPE financing
effort based on reverse merger
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May 25, 2006 – Raptor completes reverse merger and finalizes
$5M PIPE (Yahoo: RPTP.OB)
Presentation Outline
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Opportunities
The Science
– NeuroTransTM
– Cancer indications: AmpTideTM and HepTideTM
Corporate and Project Goals
Corporate Structure and Financials
Summary
CNS Therapeutic Markets are Huge
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13.3-16.1 million individuals in the US are afflicted with
CNS disorders; 1.2 million more diagnosed in US each year
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Alzheimer’s Disease, Parkinson’s Disease, Multiple
Sclerosis and Amyotrophic Lateral Sclerosis (ALS) markets
collectively estimated at $30-50B WW, growing at 14%
annually
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Medical Community is unprepared for the growing wave of
unmet medical need and social costs associated with these
diseases: second therapeutic wave for baby boomers
CNS Therapeutic Markets are Large
and Growing
CNS disorders currently cost the US economy over $600
billion dollars annually in supportive care.
“Any effective treatment or cure that can reduce the rapidly
growing financial burden of neurodegenerative disorders
and assist the elderly in maintaining independent living
arrangements will be a major revenue producer for its
developers and distributors”. AdvanceTechnology
Monitors – Novel Approaches to Neurodegenerative
Disorders.
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Many CNS Therapies are Stalled in Clinical
Development
The clinical failure of many potential CNS therapeutics is not
due to a lack of drug potency, but rather to lack of an
effective CNS delivery system.
"There was a great deal of hope for GDNF and there still is a
great deal of interest in finding different ways to provide it
to the brain.” Dr. Anthony Lang, Director of the Movement
Disorders Center at Toronto Western Hospital in Canada.
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“By 2007, drug delivery will account for 39% of all
pharmaceutical sales” John Waslif, analyst with UK
research firm, Visiongain
Science Vision
To employ the tissue targeting potential of a class of receptor
chaperone proteins, represented by Receptor-Associated
Protein, RAP as:
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NeuroTransTM - Targeted delivery of therapeutics to the brain and
other tissues
AmpTideTM and HepTideTM -“First in class” targeted cancer
therapeutics
Receptor-Associated Protein (RAP)
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37kDa human protein
RAP has a normal function in receptor biosynthesis for
lipoprotein receptor synthesis
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Binds to lipoprotein receptors on cell surfaces
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Lipoprotein receptors are the therapeutic targets for RAP
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RAP Receptors are Found in Most
Tissues
Examples of lipoprotein
receptors in contact
with blood
LRP 1
LRP2, Megalin
VLDLR
LRP5/6
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Therapeutic Applications
CANCER
THERAPIES
CNS DISEASES
RAP
THERAPEUTIC
PLATFORM
METABOLIC
DISEASES
VACCINES
CNS Diseases
NeuroTransTM
Therapies for the Treatment of CNS and
Neurodegenerative Diseases
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The Brain’s Extensive Capillary Network
Makes it Ideal for Drug Delivery
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Popular, But False Concept of the
Blood-Brain Barrier (BBB)
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The BBB Contains a Variety of Active
Transport Systems
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Selected Members of Lipoprotein
Receptor Family
Receptors
on the BBB
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Nature Cell Biology October 1999 volume 1 issue 6 pp E157-E162 DOI:10.1038/14109
How are RAP-Drug Fusions Made?
RAP cDNA
Protein Therapeutic cDNA
Standard
Bacterial or
Mammalian Cell
Production
RAP- drug fusion protein
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How Does RAP Deliver Drugs to the
Brain?
RAP fusion injected into blood
RAP receptor
Blood Brain Barrier
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Vd (μL/gm)
RAP is Rapidly Transported Across the
Blood-Brain Barrier
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1
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Negative Control
Positive Control
RAP
1 minute
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3 minutes
5 minutes
Pan, W. 2004 JCS 117:5071
RAP Linked to Brain Growth Factors:
Partnering Opportunities
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Alzheimer’s Disease
BDNF, NGF, others
Parkinson’s Disease
GDNF, BDNF
Amyotropic Lateral Sclerosis
(ALS), motor neuron diseases
GDNF, BDNF, CNTF, IGF
Chronic brain and spinal cord
injury (neurogenesis)
BDNF, NGF
Huntington’s Disease
BDNF, FGF2
Rett’s Disease
NGF, BDNF
Approaches to Delivering Therapeutics
Across the BBB
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Pharmacological
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Neurosurgical and invasive approaches
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Polymer wafer implants, catheter-based pump and direct intrathecal
administration, BBB disruption - chemotherapeutics
Physiological - potential BBB protein transporters Company
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Lipophilic agents, nanoparticle technology, glycomimics,
phospholipid microparticles - small synthetic molecules
Transferrin/Insulin systems – Armagen
Bacterial protease inhibitor – Angiochem
Modified Diphtheria toxin – toBBB
RAP based NeuroTransTM - Raptor
Cancer Therapies
AmpTideTM and HepTideTM
“First-in-Class” Targeted Therapeutics for
Cancer Treatment
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AmpTideTM Targets a Cell Surface
Cancer Tumor Protein
Matriptase is a protein-cleaving enzyme … that can trigger
formation of tumor cells.
Matriptase is also the first known cell surface enzyme that
acts as an oncogene – a protein that alone prompts
uncontrolled cell division in tumors
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AmpTideTM for Treatment of Lung
Cancer
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Product Profile:
– Novel MatRAP target for treatment of lung cancer
Status:
– Ongoing pre-clinical studies
– Evaluation and selection of first clinical target
Market:
– Total $10B market for all epithelial cancers
– Potential $500M for each indication
HepTideTM for Delivery of
Chemotherapeutics to the Liver
“ The immense unique challenges of primary liver cancer
make it imperative for investigators to identify the most
promising agents..” – Alan Venook MD Director Mt. Zion
Cancer Center Clinical Research, UCSF Cancer Chemo.
Pharm. (2004)54:S87
“The Future for cancer chemotherapy is the combination of
targeting molecules with proven chemotherapeutic agents
(to reduce toxicity)” – Hope Rugo, MD Director of Breast
Oncology UCSF, BIOInvestorForum, Oct 2006 San
Francisco
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HepTideTM for the Treatment of
Primary Liver Cancer
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Product Profile:
– Targeted delivery of chemotherapeutics to treat
hepatocellular carcinoma (primary liver cancer)
Status:
– Ongoing pre-clinical studies
Market:
– 10-15K patients diagnosed each year in US with primary
liver cancer (>1M patients WW)
– Life expectancy without liver transplant (<5% of patients
qualify) is 3-5 months following diagnosis
Pipeline of Products
Product
Indication
RAP-GDNF
Parkinson’s
RAP-NGF
Alzheimer’s
HepTideTM
Liver cancer
AmpTideTM
Lung cancer
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Discovery
Preclinical
IND/CTX
Filing
Phase
I
12-Month Corporate Goals:
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Establish university collaboration on BBB
– Stanford University, RAP-NGF for the treatment of
neurodegenerative diseases
In-license complementary targeting technology
Establish collaboration on brain disease therapeutics
Present NeuroTransTM technology – IBC Conference, San
Diego, Dec 2006
Present Stanford pre-clinical data
Present cancer data
Establish collaboration(s) on cancer
Bring in additional $5M (warrants/PIPE)
Raptor’s Hybrid Business
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Build value through internal development programs to drive
early stage products toward clinic/partnerships
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Leverage parallel product development capabilities of CNS
and cancer programs
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In-license complementary technology and products
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Provide for new “composition of matter” patent life –
product “life-cycle” management
Corporate Structure
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Management
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Medical and Scientific Advisors
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Intellectual Property
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Financial Summary
Raptor’s Management Team
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Christopher M. Starr Ph.D.
 CEO and Co-founder
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Todd C. Zankel Ph.D.
 CSO and Co-founder
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Kim R. Tsuchimoto, C.P.A.
 CFO
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SVP, CSO, Co-Founder
BioMarin Pharmaceutical
VP Research Glyko Inc
NRC Associate Follow, NIH
Senior Director, Research
BioMarin Pharmaceutical
NIH Fellow Berkley, CA
Ph.D. Columbia University
Controller IMSI, SpatiaLight
VP Treasurer, BioMarin
Pharmaceutical
VP Controller BioMarin, Glyko
Inc, Licensed CA C.P.A.
Raptor’s Scientific and Medical
Advisors
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Guojun Bu, Ph.D.
 Technology and Product
Development
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William Mobley M.D., PH.D.
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Neuroscience and
NeuroTransTM Technology
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Associate Professor Washington
University
RAP and LDLR Expert
Chairman and Professor
Neurology and Neurological
Sciences, Stanford School of
Medicine
Director Neuroscience Institute,
Stanford School of Medicine
Raptor’s Scientific and Medical
Advisors
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Sam Teichman, M.D.
 Clinical and Regulatory
Rivka Sherman-Gold, Ph.D., M.B.A.
 Business Development
 Neuroscience
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20+ years experience on over 40
medical products
Held executive positions at
ARYx, Genentech, Glycomed,
and Mimetix
CBO Diatos, CEO Alydar,
Director of BD Abgenix
Associate Director of BD
Athena Neurosciences
Licensing experience Pfizer,
Amgen, J&J, Schering-Plough,
SKB, Centocor, Elan
Raptor’s Scientific and Medical
Advisors
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Andres Lozano, M.D., Ph.D.
 Neurosurgeon
 Specialty in Movement Disorders
including Parkinson’s Disease
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Professor and RR Tasker Chair
in Functional Neurosurgery and
Canada Research Chair in
Neuroscience at the University
of Toronto
Head of Applied and
Interventional Research at the
Toronto Western Research
Institute
Over 200 Publications and
extensive clinical trial expertise
Intellectual Property
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APPLICATION NO.
TITLE
FILING DATE
PCT/US02/23923
US10/206,448
Compositions & methods for
modulating the BBB
July 25, 02
US11/202,566
Use of chaperone RAP for delivery of
therapeutics…
Aug. 12, 05
US10/812,849
PCT/US04/19153
Megalin-based delivery of
therapeutics...
Mar. 30, 04
PCT06/23104
Compositions comprising RAP
variants specific for LRP2 and uses
thereof.
Sept. 16, 05
Filed PCT
Compositions comprising RAP
variants specific for Cr-containing
proteins…
Set. 18, 06
Financial Summary
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29,633,333 shares of common stock outstanding
(27% owned by management and insiders)
9,193,333 common stock purchase warrants outstanding
exercisable at US$0.60 per share
US$3.0M cash as of November 30, 2006
Estimated twelve month spend US$2.6M
Contact
Christopher M. Starr, Ph.D.
Co-Founder and CEO
Raptor Pharmaceutical Inc.
(415) 382-1427
cstarr@raptorpharma.com
www.raptorpharma.com
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