1 / 38

Raptor Pharmaceutical Inc.

Raptor Pharmaceutical Inc. Protein Therapeutics to Treat Neurodegenerative Brain Diseases and Cancer. Forward-Looking Statements.

kyle
Download Presentation

Raptor Pharmaceutical Inc.

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript


  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 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.

  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 • 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

  7. CNS Therapeutic Markets are Large and Growing CNS disorders currently cost the US economy over $600 billiondollars 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 CANCERTHERAPIES CNS DISEASES RAP THERAPEUTIC PLATFORM METABOLIC DISEASES VACCINES

  13. CNS Diseases NeuroTransTMTherapies for the Treatment of CNS and Neurodegenerative Diseases

  14. The Brain’s 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 DOI:10.1038/14109

  18. RAP cDNA Protein Therapeutic cDNA Standard Bacterial or Mammalian Cell Production How are RAP-Drug Fusions Made? RAP- drug fusion protein

  19. RAP fusion injected into blood 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 117:5071

  21. RAP Linked to Brain Growth Factors: Partnering Opportunities

  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 HepTideTM“First-in-Class” Targeted Therapeutics for Cancer Treatment

  24. 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 AmpTideTM Targets a Cell Surface Cancer Tumor Protein

  25. 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 AmpTideTM for Treatment of Lung Cancer

  26. “ 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 HepTideTM for Delivery of Chemotherapeutics to the Liver

  27. 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 HepTideTM for the Treatment of Primary Liver Cancer

  28. Pipeline of Products Product Pre- IND/CTX Phase Indication Discovery clinical Filing I RAP-GDNF Parkinson’s RAP-NGF Alzheimer’s 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. 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

  31. Corporate Structure • Management • Medical and Scientific Advisors • Intellectual Property • Financial Summary

  32. 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. Raptor’s Management Team

  33. Guojun Bu, Ph.D. Technology and Product Development William Mobley M.D., PH.D. Neuroscience andNeuroTransTM 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 Raptor’s Scientific and Medical Advisors

  34. 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, J&J, Schering-Plough, SKB, Centocor, Elan Raptor’s Scientific and Medical Advisors

  35. 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 Raptor’s Scientific and Medical Advisors

  36. Intellectual Property

  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 US$0.60 per share • US$3.0M cash as of November 30, 2006 • Estimated twelve month spend US$2.6M

  38. Contact Christopher M. Starr, Ph.D. Co-Founder and CEO Raptor Pharmaceutical Inc. (415) 382-1427 cstarr@raptorpharma.com www.raptorpharma.com

More Related