Raptor Pharmaceutical Inc.

1
Raptor Pharmaceutical Inc.

• Protein Therapeutics to Treat Neurodegenerative
  Brain Diseases and Cancer

2
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 industrys
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.
3
Raptor Company Profile

• Raptor develops protein therapeutics for the
  treatment of neurodegenerative brain diseases and
  cancer.
• Core competencies in protein bioengineering,
  receptor biology, non-clinical and clinical
  development in both protein and small molecule
  therapeutics

4
Corporate and Financial History

• September 2005 - Co-founders Christopher M.
  Starr, Ph.D. and Todd C. Zankel, Ph.D. form
  Raptor Pharmaceutical Inc.
• January 2006 - Asset Acquisition Agreement signed
  with BioMarin transferring 16M worth of RAP
  technology, IP and know-how to Raptor
• March 2006 - Raptor raises 300K of seed funding,
  hires CFO Kim Tsuchimoto, signs facilities lease,
  and begins PIPE financing effort based on reverse
  merger
• May 25, 2006 Raptor completes reverse merger
  and finalizes 5M PIPE (Yahoo RPTP.OB)

5
Presentation Outline

• Opportunities
• The Science
• NeuroTransTM
• Cancer indications AmpTideTM and HepTideTM
• Corporate and Project Goals
• Corporate Structure and Financials
• Summary

6
CNS Therapeutic Markets are Huge

• 13.3-16.1 million individuals in the US are
  afflicted with CNS disorders 1.2 million more
  diagnosed in US each year
• Alzheimers Disease, Parkinsons Disease,
  Multiple Sclerosis and Amyotrophic Lateral
  Sclerosis (ALS) markets collectively estimated at
  30-50B WW, growing at 14 annually
• 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

7
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.

8
Many CNS Therapies are Stalled in
ClinicalDevelopment

• 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.
By 2007, drug delivery will account for 39 of
all pharmaceutical sales John Waslif, analyst
with UK research firm, Visiongain
9
Science Vision

• To employ the tissue targeting potential of a
  class of receptor chaperone proteins, represented
  by Receptor-Associated Protein, RAP as
• NeuroTransTM - Targeted delivery of
  therapeutics to the brain and other tissues
• AmpTideTM and HepTideTM -First in class
  targeted cancer therapeutics

10
Receptor-Associated Protein (RAP)

• 37kDa human protein

• RAP has a normal function in receptor
  biosynthesis for lipoprotein receptor synthesis

• Binds to lipoprotein receptors on cell surfaces

• Lipoprotein receptors are the therapeutic targets
  for RAP

11
RAP Receptors are Found in Most Tissues

• Examples of lipoprotein
• receptors in contact
• with blood

LRP 1 LRP2, Megalin VLDLR LRP5/6
12
Therapeutic Applications
CANCER THERAPIES
CNS DISEASES
RAP THERAPEUTIC PLATFORM
METABOLIC DISEASES
VACCINES
13
CNS Diseases
NeuroTransTM Therapies for the Treatment of
CNS and Neurodegenerative Diseases
14
The Brains Extensive Capillary Network Makes it
Ideal for Drug Delivery
15
Popular, But False Concept of the Blood-Brain
Barrier (BBB)
16
The BBB Contains a Variety of Active Transport
Systems
17
Selected Members of Lipoprotein Receptor Family
Receptors on the BBB
Nature Cell Biology   October 1999 volume 1 issue
6 pp E157-E162 DOI10.1038/14109
18
How are RAP-Drug Fusions Made?
RAP- drug fusion protein
19
How Does RAP Deliver Drugs to the Brain?
RAP receptor
Blood Brain Barrier
20
RAP is Rapidly Transported Across the Blood-Brain
Barrier
Vd (µL/gm)
Pan, W. 2004 JCS 1175071
21
RAP Linked to Brain Growth Factors Partnering
Opportunities
Alzheimers Disease BDNF, NGF, others
Parkinsons Disease GDNF, BDNF
Amyotropic Lateral Sclerosis (ALS), motor neuron diseases GDNF, BDNF, CNTF, IGF
Chronic brain and spinal cord injury (neurogenesis) BDNF, NGF
Huntingtons Disease BDNF, FGF2
Retts Disease NGF, BDNF
22
Approaches to Delivering Therapeutics Across the
BBB

• Pharmacological
• Lipophilic agents, nanoparticle technology,
  glycomimics, phospholipid microparticles - small
  synthetic molecules
• Neurosurgical and invasive approaches
• Polymer wafer implants, catheter-based pump and
  direct intrathecal administration, BBB disruption
  - chemotherapeutics
• Physiological - potential BBB protein
  transporters - Company
• Transferrin/Insulin systems Armagen
• Bacterial protease inhibitor Angiochem
• Modified Diphtheria toxin toBBB
• RAP based NeuroTransTM - Raptor

23
Cancer Therapies
AmpTideTM and HepTideTMFirst-in-Class
Targeted Therapeutics for Cancer Treatment
24
AmpTideTM Targets a Cell Surface Cancer Tumor
Protein
25
AmpTideTM for Treatment of Lung Cancer

• 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

26
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)54S87
• 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

27
HepTideTM for the Treatment of Primary Liver
Cancer

• 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 (gt1M patients WW)
• Life expectancy without liver transplant (lt5 of
  patients qualify) is 3-5 months following
  diagnosis

28
Pipeline of Products

• Product Pre- IND/CTX Phase
• Indication Discovery clinical Filing
  I

• RAP-GDNF
• Parkinsons
• RAP-NGF
• Alzheimers
• HepTideTM
• Liver cancer
• AmpTideTM
• Lung cancer

29
12-Month Corporate Goals

• 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)

30
Raptors Hybrid Business

• Build value through internal development programs
  to drive early stage products toward
  clinic/partnerships

• Leverage parallel product development
  capabilities of CNS and cancer programs

• In-license complementary technology and products

• Provide for new composition of matter patent
  life product life-cycle management

31
Corporate Structure

• Management
• Medical and Scientific Advisors
• Intellectual Property
• Financial Summary

32
Raptors Management Team

• Christopher M. Starr Ph.D.
• CEO and Co-founder
• Todd C. Zankel Ph.D.
• CSO and Co-founder
• Kim R. Tsuchimoto, C.P.A.
• CFO

• 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.

33
Raptors Scientific and Medical Advisors

• Guojun Bu, Ph.D.
• Technology and Product Development
• William Mobley M.D., PH.D.
• Neuroscience and NeuroTransTM Technology

• 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

34
Raptors Scientific and Medical Advisors

• Sam Teichman, M.D.
• Clinical and Regulatory
• Rivka Sherman-Gold, Ph.D., M.B.A.
• Business Development
• Neuroscience

• 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, JJ,
  Schering-Plough, SKB, Centocor, Elan

35
Raptors Scientific and Medical Advisors

• Andres Lozano, M.D., Ph.D.
• Neurosurgeon
• Specialty in Movement Disorders including
  Parkinsons Disease

• 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

36
Intellectual Property
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
37
Financial Summary

• 29,633,333 shares of common stock outstanding
• (27 owned by management and insiders)
• 9,193,333 common stock purchase warrants
  outstanding exercisable at US0.60 per share
• US3.0M cash as of November 30, 2006
• Estimated twelve month spend US2.6M

38
Contact

• Christopher M. Starr, Ph.D.
• Co-Founder and CEO
• Raptor Pharmaceutical Inc.
• [phone]
• cstarr_at_raptorpharma.com
• www.raptorpharma.com