Transcript Raptor Presentation - Microsoft Internet Information
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Raptor Pharmaceutical Inc.
Protein Therapeutics to Treat Neurodegenerative Brain Diseases and Cancer
Forward-Looking Statements
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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
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
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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)
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Presentation Outline
Opportunities The Science – NeuroTrans TM – Cancer indications: AmpTide TM and HepTide TM Corporate and Project Goals Corporate Structure and Financials Summary
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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 Alzheimer’s Disease, Parkinson’s 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
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CNS Therapeutic Markets are Large and Growing
CNS disorders currently cost the US economy over
$600 billion dollars
annually in supportive care.
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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.
Many CNS Therapies are Stalled in Clinical Development 8
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
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Science Vision
To employ the tissue targeting potential of a class of receptor chaperone proteins, represented by Receptor-Associated Protein, RAP as: – – NeuroTrans TM other tissues - Targeted delivery of therapeutics to the brain and AmpTide TM and HepTide TM -“First in class” targeted cancer therapeutics
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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
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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
Therapeutic Applications
CNS DISEASES CANCER THERAPIES RAP THERAPEUTIC PLATFORM METABOLIC DISEASES VACCINES
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CNS Diseases
NeuroTrans TM Therapies for the Treatment of CNS and Neurodegenerative Diseases
14 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
Selected Members of Lipoprotein Receptor Family
Receptors on the BBB 17 Nature Cell Biology October 1999 volume 1 issue 6 pp E157-E162 DOI:10.1038/14109
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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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RAP is Rapidly Transported Across the Blood-Brain Barrier
10 9 8 7 6 5 4 3 2 1 0 1 minute 3 minutes 5 minutes Negative Control Positive Control RAP Pan, W. 2004 JCS 117:5071
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RAP Linked to Brain Growth Factors: Partnering Opportunities
Alzheimer’s Disease Parkinson’s Disease Amyotropic Lateral Sclerosis (ALS), motor neuron diseases Chronic brain and spinal cord injury (neurogenesis) Huntington’s Disease Rett’s Disease BDNF, NGF, others GDNF, BDNF GDNF, BDNF, CNTF, IGF BDNF, NGF BDNF, FGF2 NGF, BDNF
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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 NeuroTrans TM - Raptor
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Cancer Therapies
AmpTide TM and HepTide TM “First-in-Class” Targeted Therapeutics for Cancer Treatment
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AmpTide
TM
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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AmpTide
TM
Cancer for Treatment of Lung
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
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HepTide
TM
for Delivery of Chemotherapeutics to the Liver
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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)”
Francisco – Hope Rugo, MD Director of Breast Oncology UCSF, BIOInvestorForum, Oct 2006 San
HepTide
TM
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
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RAP-GDNF Parkinson’s RAP-NGF Alzheimer’s HepTide TM Liver cancer AmpTide TM Lung cancer
Discovery Pre IND/CTX Phase clinical Filing I
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12-Month Corporate Goals:
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Raptor
’
s 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
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Corporate Structure
Management Medical and Scientific Advisors Intellectual Property Financial Summary
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Raptor’s Management Team
Christopher M. Starr Ph.D.
CEO and Co-founder
Todd C. Zankel Ph.D.
CSO and Co-founder
– – – 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
Kim R. Tsuchimoto, C.P.A.
CFO
– – – Controller IMSI, SpatiaLight VP Treasurer, BioMarin Pharmaceutical VP Controller BioMarin, Glyko Inc, Licensed CA C.P.A.
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Raptor’s Scientific and Medical Advisors
Guojun Bu, Ph.D.
Technology and Product Development
– – Associate Professor Washington University RAP and LDLR Expert
William Mobley M.D., PH.D.
Neuroscience and NeuroTrans TM Technology
– – Chairman and Professor Neurology and Neurological Sciences, Stanford School of Medicine Director Neuroscience Institute, Stanford School of Medicine
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Raptor’s Scientific and Medical Advisors
Sam Teichman, M.D.
Clinical and Regulatory
– – 20+ years experience on over 40 medical products Held executive positions at ARYx, Genentech, Glycomed, and Mimetix
Rivka Sherman-Gold, Ph.D., M.B.A.
Business Development
Neuroscience
– – – 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
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Raptor’s Scientific and Medical Advisors
Andres Lozano, M.D., Ph.D.
Neurosurgeon
Specialty in Movement Disorders including Parkinson’s 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
Intellectual Property
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APPLICATION NO.
PCT/US02/23923 US10/206,448 US11/202,566 US10/812,849 PCT/US04/19153 PCT06/23104 Filed PCT TITLE Compositions & methods for modulating the BBB Use of chaperone RAP for delivery of therapeutics… Megalin-based delivery of therapeutics...
Compositions comprising RAP variants specific for LRP2 and uses thereof.
Compositions comprising RAP variants specific for Cr-containing proteins… FILING DATE July 25, 02 Aug. 12, 05 Mar. 30, 04 Sept. 16, 05 Set. 18, 06
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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 US$0.60 per share US$3.0M cash as of November 30, 2006 Estimated twelve month spend US$2.6M
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Contact
Christopher M. Starr, Ph.D. Co-Founder and CEO Raptor Pharmaceutical Inc.
(415) 382-1427 [email protected]
www.raptorpharma.com