raptor pharmaceutical inc

38
1 Raptor Pharmaceutical Inc. Protein Therapeutics to Treat Neurodegenerative Brain Diseases and Cancer

Upload: kyle

Post on 25-Feb-2016

50 views

Category:

Documents


3 download

DESCRIPTION

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

TRANSCRIPT

Page 1: Raptor Pharmaceutical Inc

1

Raptor Pharmaceutical Inc.

Protein Therapeutics to Treat Neurodegenerative

Brain Diseases and Cancer

Page 2: Raptor Pharmaceutical Inc

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.

Page 3: Raptor Pharmaceutical Inc

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

Page 4: Raptor Pharmaceutical Inc

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)

Page 5: Raptor Pharmaceutical Inc

5

Presentation Outline

Opportunities The Science

– NeuroTransTM

– Cancer indications: AmpTideTM and HepTideTM

Corporate and Project Goals Corporate Structure and Financials Summary

Page 6: Raptor Pharmaceutical Inc

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

Page 7: Raptor Pharmaceutical Inc

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.

Page 8: Raptor Pharmaceutical Inc

8

Many CNS Therapies are Stalled in ClinicalDevelopment

"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.

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.

“By 2007, drug delivery will account for 39% of all pharmaceutical sales” John Waslif, analyst with UK research firm, Visiongain

Page 9: Raptor Pharmaceutical Inc

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

Page 10: Raptor Pharmaceutical Inc

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

Page 11: Raptor Pharmaceutical Inc

11

RAP Receptors are Found in Most Tissues

LRP 1LRP2, MegalinVLDLRLRP5/6

Examples of lipoproteinreceptors in contact with blood

Page 12: Raptor Pharmaceutical Inc

Therapeutic Applications

RAPTHERAPEUTIC

PLATFORM

CNS DISEASES

METABOLIC DISEASES VACCINES

CANCER THERAPIES

Page 13: Raptor Pharmaceutical Inc

13

CNS Diseases

NeuroTransTM

Therapies for the Treatment of CNS and Neurodegenerative Diseases

Page 14: Raptor Pharmaceutical Inc

14

The Brain’s Extensive Capillary Network Makes it Ideal for Drug Delivery

Page 15: Raptor Pharmaceutical Inc

15

Popular, But False Concept of the Blood-Brain Barrier (BBB)

Page 16: Raptor Pharmaceutical Inc

16

The BBB Contains a Variety of Active Transport Systems

Page 17: Raptor Pharmaceutical Inc

17 Nature Cell Biology   October 1999 volume 1 issue 6 pp E157-E162 DOI:10.1038/14109

Selected Members of Lipoprotein Receptor Family

Receptors on the BBB

Page 18: Raptor Pharmaceutical Inc

18

How are RAP-Drug Fusions Made?

Protein Therapeutic cDNA

StandardBacterial or Mammalian Cell Production

RAP- drug fusion protein

RAP cDNA

Page 19: Raptor Pharmaceutical Inc

19

How Does RAP Deliver Drugs to the Brain?

RAP fusion injected into blood

Blood Brain Barrier

RAP receptor

Page 20: Raptor Pharmaceutical Inc

20

RAP is Rapidly Transported Across the Blood-Brain Barrier

0123456789

10

1 minute 3 minutes 5 minutes

Negative ControlPositive ControlRAPV

d (μ

L/gm

)

Pan, W. 2004 JCS 117:5071

Page 21: Raptor Pharmaceutical Inc

21

RAP Linked to Brain Growth Factors: Partnering Opportunities

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

Page 22: Raptor Pharmaceutical Inc

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

Page 23: Raptor Pharmaceutical Inc

23

Cancer Therapies

AmpTideTM and HepTideTM

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

Page 24: Raptor Pharmaceutical Inc

24

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

Page 25: Raptor Pharmaceutical Inc

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

Page 26: Raptor Pharmaceutical Inc

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)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

Page 27: Raptor Pharmaceutical Inc

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 (>1M patients WW)

– Life expectancy without liver transplant (<5% of patients qualify) is 3-5 months following diagnosis

Page 28: Raptor Pharmaceutical Inc

28

Pipeline of Products

Product Pre- IND/CTX PhaseIndication Discovery clinical Filing IRAP-GDNF Parkinson’sRAP-NGF Alzheimer’sHepTideTM

Liver cancerAmpTideTM

Lung cancer

Page 29: Raptor Pharmaceutical Inc

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)

Page 30: Raptor Pharmaceutical Inc

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

Page 31: Raptor Pharmaceutical Inc

31

Corporate Structure

Management

Medical and Scientific Advisors

Intellectual Property

Financial Summary

Page 32: Raptor Pharmaceutical Inc

32

Raptor’s 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.

Page 33: Raptor Pharmaceutical Inc

33

Raptor’s 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

Page 34: Raptor Pharmaceutical Inc

34

Raptor’s 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, J&J, Schering-Plough, SKB, Centocor, Elan

Page 35: Raptor Pharmaceutical Inc

35

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

Page 36: Raptor Pharmaceutical Inc

36

Intellectual Property

APPLICATION NO. TITLE FILING DATEPCT/US02/23923US10/206,448

Compositions & methods for modulating the BBB

July 25, 02

US11/202,566 Use of chaperone RAP for delivery of therapeutics…

Aug. 12, 05

US10/812,849PCT/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

Page 37: Raptor Pharmaceutical Inc

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

Page 38: Raptor Pharmaceutical Inc

38

Contact

Christopher M. Starr, Ph.D. Co-Founder and CEO Raptor Pharmaceutical Inc.(415) [email protected] www.raptorpharma.com