Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — CIBC Healthcare Conference 2007
29 slides from Protalix Biotherapeutics, Inc. (de)'s conference presentation, Nov 2, 2007
Slide 1: CIBC Healthcare Conference
CIBC Healthcare Conference November 5 th , 2007 Dr. David Aviezer President and CEO
Slide 2: Safe Harbor Statement
Safe Harbor Statement To the extent that statements in this press release are not strictly historical, all such statements are forward- looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject t
Slide 3: Company Highlights
Company Highlights prGCD for Gaucher Disease Proprietary Protein Expression System Targeting Commercially Viable Proteins World Class Management & Board Lead product candidate targeting a large, growing market Similar to Cerezyme, the only approved enzyme replacement therapy Currently in Phase III t
Slide 4: Experienced Management and World Class Board and Advisors
Experienced Management and World Class Board and Advisors Title Experience Name Advisor Prof. Ari Zimran Advisor Prof. Gad Galili Director of the Gaucher clinic, Shaarei Zedek Medical Center, Jerusalem. World opinion leader in Gaucher Disease Advisor Prof. Aaron Ciechanover Chairman, Dept. of Molecu
Slide 5: Company Snapshot
Company Snapshot Proprietary ProCellEx™ protein expression system based on plant cell-based expression technology Lead product prGCD - plant expressed Glucocerebrosidase for Gaucher Disease currently in Phase III clinical trials Deep pipeline focused on complex therapeutic proteins for novel and bio
Slide 6: Protalix Biotherapeutics
. Protalix Biotherapeutice
Slide 7: ProCellEx Protein Expression System
ProCellEx™ Protein Expression System Technology Platform Proprietary plant cell-based protein expression system Comprehensive set of technologies and capabilities for the development of recombinant proteins from initial nucleotide cloning to large scale protein production Enables expression of prote
Slide 8: Key Advantages of the ProCellEx System
Key Advantages of the ProCellEx System Enables Penetration of Patent Protected Markets Cost Effectiveness and Scalability Significant Advantages Over Mammalian Cell- based Systems Avoids infringing on the method-based patents of other proteins developed with mammalian cell-based expression systems,
Slide 9: Gaucher Disease: A genetically inherited disorder
Gaucher Disease: A genetically inherited disorder Macrophages Outcome: Bone Bone Marrow Liver Lung Spleen Source: Beutler and Grabowski, The Metabolic and Molecular Bases of Inherited Disease 2001 Skeletal pathology Pulmonary hypertension/ infiltration Hepatomegaly Anemia Thrombocytopenia Splenomega
Slide 10: The Enzymatic Defect in Gaucher Disease
HO CH 2 CH CH CH = CH (CH 2 ) 12 CH 3 OH O O OH HO OH OH O OH OH HO OH O=C CH 2 CH 2 CH 2 (CH 2 ) n CH 3 N OH CH 2 CH CH CH = CH (CH 2 ) 12 CH 3 O=C CH 2 CH 2 CH 2 (CH 2 ) n CH 3 N OH Glucose Ceramide Glucosyl Ceramide + The Enzymatic Defect in Gaucher Disease Acid -Glucosidase Glucocerebrosidase
Slide 11: Gaucher Disease: Attractive Market Characteristics
Gaucher Disease: Attractive Market Characteristics Comment Characteristic Cerezyme ® Growth ($m) Large Market Size Attractive Patient Profile Steady Growth Prospects Competition No cure for Gaucher, ongoing chronic therapeutic treatment Treatment cost of ~$200,000 p.a., Less than 5,000 treated patie
Slide 12: prGCD: Equal to Superior Biological Activity
prGCD: Equal to Superior Biological Activity prGCD has equal to superior enzymatic activity degrading the natural substrate when compared to Cerezyme® : Cerezyme and prGCD cellular uptake -0.05 0.00 0.05 0.10 0.15 0.20 0.25 0.30 0.35 0.40 0.45 0 u/ml 1.6 u/ml 3.2 u/ml 6.4 u/ml Enzyme concentration C
Slide 13: Similar three Dimensional structure
Similar three Dimensional structure 3-D crystal structure of prGCD solved by a team of world renowned scientists from the Weizmann Institute of Science and compared to Cerezyme® structure.
Slide 14: Protalix Biotherapeutics
Protalix Biotherapeutice
Slide 15: Glycan consistency
Fg *‘Protalix « Biotherapeutics oy Glycan consistency a ee
Slide 16: Glucocerebrosidase Binds to its Target Cells via the Mannose Receptor
Glucocerebrosidase Binds to its Target Cells via the Mannose Receptor Macrophage Mannose receptor Mannose residues GCD
Slide 17: Plant cell technology produces a ready to use bioactive enzyme
Protalix plant cell technology produces a “ready to use” bioactive enzyme: Protalix technology does not require additional glycan processing Mammalian technology requires multiple post-expression modifications: Active - Mannose exposed glycan structure Mannose GlcNAc Fucose Xylose Galactose Sialic a
Slide 18: Batch to Batch Consistency of Glycoforms produced by ProCellEx
Batch to Batch Consistency of Glycoforms produced by the ProCellEx™ system
Slide 19: Successful Phase I Clinical Study
Successful Phase I Clinical Study Design Single-center, non-randomized, open label performed at Hadassah Medical Center, Jerusalem Study performed under FDA IND approval. Objectives Primary : To evaluate the safety of three escalating doses of human prGCD in healthy volunteers Secondary : To determi
Slide 20: Pharmacokinetic data: prGCD vs. Cerezyme
Pharmacokinetic data: prGCD vs. Cerezyme ( IV administration ) Preclinical –Primate data Human data prGCD data: T1/2 13-20 minutes T1/2 10.5-14.5 minutes Cerezyme (published data) T1/2 6.8-8 minutes T1/2 ~ 3.6-10.4 minutes
Slide 21: prGCD Phase III Trial
prGCD Phase III Trial – Launched Aug 07 Multi-center world wide study Efficacy and safety in 30 untreated patients with significant symptoms of Gaucher disease treated with prGCD for 9 months Special Protocol Assessment approved by FDA in July 2007 Randomized, double blind, parallel groups, dose-ran
Slide 22: prGCD Clinical Development Timeline
prGCD Clinical Development Timeline 2007 2008 2009 Initiated Phase III Clinical Trials NDA Submission Phase III Efficacy Data Drug Approval
Slide 23: prGCD for Gaucher Disease
prGCD for Gaucher Disease prGCD Plant cell expressed recombinant GCD for enzyme replacement therapy Enzyme replacement therapy is gold standard of care Substantially similar amino acid, glycan and three dimensional structure compared to Cerezyme Major proof of concept for ProCellEx™ Protein Expressi
Slide 24: Leveraging our Platform into Attractive Market Opportunities
Leveraging our Platform into Attractive Market Opportunities Phase of Development Indication Market Opportunity Market Dynamic Pipeline Candidate R & D, Animal Studies R & D, Animal Studies Phase III in progress Follicle Stimulating Hormone for Female Infertility Biodefense and organophosphate poiso
Slide 25: Monoclonal Antibodies Expression
Monoclonal Antibodies Expression Protalix system represents an alternative platform for hard-to- express proteins in mammalian systems, including monoclonal antibodies IgG1-CHO IgG4-Protalix IgG1-Protalix Control Heavy Chain Light Chain Human IgG Standard Recombinant IgG produced by Protalix’s plant
Slide 26: Strategic Collaborations
Strategic Collaborations In addition to internal, proprietary development, Protalix also seeks to share risk and enter into partnerships for the early-stage co-development for biotherapeutic proteins Framework agreement for R&D collaboration on two proteins using ProCellEx system TEVA to be granted
Slide 27: Strong Intellectual Property Protection
Strong Intellectual Property Protection: 50+ Patents & Applications 6 patent families in the in house portfolio Cell & tissue culturing device, system and method Expression of lysosomal Enzymes in plant cells Large scale bioreactor system delivering proteins through oral administration Expression of
Slide 28: Company Highlights
Company Highlights prGCD for Gaucher Disease Proprietary Protein Expression System Targeting Commercially Viable Proteins World Class Management & Board Lead product candidate targeting a large, growing market Similar to Cerezyme, the only approved enzyme replacement therapy Currently in Phase III t
Slide 29: Contact
Contact: Dr. David Aviezer President & CEO david@protalix.com
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