Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Investor Presentation (January 2011)
34 slides from Protalix Biotherapeutics, Inc. (de)'s investor presentation, Jan 27, 2011
Slide 1: Protalix BioTherapeutics January 2011
Protalix BioTherapeutics January 2011 Confidential
Slide 2: Note Regarding Forward-Looking Statements
Note Regarding Forward-Looking Statements This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements including, among others, sta
Slide 3: Late Stage Biologics Program Targeting a Significant Market
Late Stage Biologics Program Targeting a Significant Market Best-In-Class Commercial Partner Attractive Platform Technology Promising Pipeline Lead Product taliglucerase alfa indicated for Gaucher disease Completed PIII trial and notified of NDA acceptance in July 2010 Open-label extension trial ong
Slide 4: Substantial Value in the Pipeline
taliglucerase alfa Gaucher Disease >$1.25B PRX-102 Fabry Disease >$600M >$350M PRX-105: Acetylcholinesterase Biodefense Applications Oral GCD Program Gaucher Disease >$1.25B COMPOUND & INDICATION RESEARCH PRECLINICAL PHASE I PHASE III FILING APPROVAL PARTNER MKT OPP PRX-106: Anti-TNF Fusion Protein
Slide 5: Key Advantages: Proprietary Technology Platform ProCellEx
Key Advantages Cost effectiveness and scalability Flexible polyethylene bioreactors, low initial capital investment Rapid, horizontal scalability at low cost in compliance with cGMP Requires less costly "hands-on" maintenance Safety and potency No risk of mammalian viral transmission or infection to
Slide 6: Key Milestones and Goals Expected for 2011
Key Milestones and Goals Expected for 2011 2010 Received Orphan Drug designation in Europe Completed FDA submission for taliglucerase alfa Received PDUFA date for taliglucerase alfa Completed preliminary PRX-105 Phase 1 study Granted temporary authorization for use (ATU) for taliglucerase alfa in Fr
Slide 7: Taliglucerase alfa
Taliglucerase alfa Confidential
Slide 8: Gaucher Disease Background
Autosomal recessive disorder caused by mutations in the GBA gene which result in a deficiency of the lysosomal enzyme beta- glucocerebrosidase Patients accumulate large quantities of glucocerebroside in the spleen, liver, lungs and bone marrow that prevent these organs from functioning properly Enzy
Slide 9: Gaucher Disease: A Genetically Inherited Disorder
Macrophages 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 Splenomegaly Outcome: Death - Poor quality of life Gaucher Disease: A
Slide 10: Gaucher Disease: Current Market Dynamics
*Zavesca (r) (Actelion) is a small molecule drug approved for the treatment of Gaucher disease, however usage is extremely limited due to significant side effects Gaucher Disease: Current Market Dynamics 10 ~10,000 patients worldwide Around ~5,500 patients are being treated ~50% global market penetr
Slide 11: Phase III Pivotal Trial Under SPA with FDA
Phase III Pivotal Trial Under SPA with FDA Design Multicenter, world-wide, randomized, double blind, parallel groups, dose-ranging study in 31 untreated patients with significant symptoms of Gaucher disease Patients randomized to one of two dose arms, 30 U/Kg or 60 U/kg, bi-weekly dosing for 9 month
Slide 12: Met Primary Endpoint of % Change in Spleen Volume
Met Primary Endpoint of % Change in Spleen Volume 60 Units/kg 30 Units/kg N=16 N=15 -38.01% +- 9.38* - -37.63 - -56.30 to -20.04 -26.91% +- 7.79* - -27.85 - -42.60 to -15.58 Mean Median Range 9 Months 16.6X 15.4X Mean Baseline X Normal 10.9X 11.3X Mean 9 Months X Normal Range of Spleen volume at bas
Slide 13: 1st Major Secondary Endpoint: Change in Hemoglobin
1st Major Secondary endpoint: Change in Hemoglobin (g/dL) From Baseline - ITT vs. Anemic patients Both anemic and non anemic patients showed an increase in Hb 60 Units/kg n = 16 30 Units/kg n = 15 ITT n= 31 2.2 +- 1.4 1.6 <0.0001* 1.6 +- 1.4 1.3 0.0010* Mean g/dL Median P-value 9 months 22.2% 14.8%
Slide 14: 2nd Major Secondary Endpoint: Liver Volume
2nd Major Secondary Endpoint: Liver Volume % Change from Baseline 60 Units/kg 30 Units/kg 2190.99 376.70 2098.13 1654.07 - 2894.12 2564.07 559.57 2473.19 2000.33 -4121.94 Mean SD Median Range 9 Months (absolute values) -11.11% 6.68 - -12.25 <0.0001* -10.48% 11.27 - -13.87 0.0041* Mean SD Median P-va
Slide 15: 3rd Major Secondary Endpoint: Platelet Counts
3rd Major Secondary Endpoint: Platelet Counts Change from Baseline Baseline range 27,000/ ml - 163,000/ml 60 Units/kg 30 Units/kg N=16 41494 47063 38000 - -15000 to 186000 0.0031* N=15 11427 20214 10000 - -25000 to 59000 0.0460* Mean SD Median Range P-value 9 Months (change from baseline) 72.1% 13.7
Slide 16: Phase III Safety Results
Phase III Safety Results No serious adverse events All AEs were mild or moderate in intensity and the majority of the events spontaneously resolved Headache; hypersensitivity; dizziness; muscle spasm; chest discomfort; nausea; skin irritation; arthalgia No neutralizing antibodies to taliglucerase we
Slide 17: Switchover Study
17 Switchover Study Multicenter, open-label, switchover trial of taliglucerase alfa To assess the efficacy, safety, and lack of deterioration in Gaucher patients currently treated with Cerezyme(r) After 9 months of treatment, patients are enrolling in the extension study First stage of study is comp
Slide 18: Regulatory Status
Regulatory Status Rolling NDA submission filed with FDA February 25th, 2011 PDUFA date Submission to Ministry of Health in Israel Information dossier submitted GMP audit of manufacturing facility performed Submission of MAA to EMEA in Europe November 2010 Submission to ANVISA in Brazil November 2010
Slide 19: Expanded Access Program Overview
FDA requested submission of a treatment protocol allowing expanded access to taliglucerase alfa in adult patients with Gaucher disease resulting from the shortage of Cerezyme Protocols approved by FDA and EMEA Patient enrollment on-going in clinical centers around the world Under a treatment protoco
Slide 20: Brazil Program
Total estimate of around 600 Gaucher patients previously treated in Brazil On 8/10/2010, Pfizer entered into a $30 million short-term supply agreement with the Ministry of Health of Brazil Protalix and Pfizer have provided taliglucerase alfa to the Ministry of Health of Brazil for the treatment of p
Slide 21: Commercialization Strategy: Collaboration
Terms Upfront payment of $65 million Additional near-term regulatory milestone payments of up to $50 million Pfizer and Protalix to share net profit and net loss of taliglucerase alfa on a 60% / 40 % basis to Protalix. Certain limited capped expenses Territories Pfizer retains exclusive worldwide ri
Slide 22: Manufacturing and Capacity
Completed GMP Manufacturing Audits by FDA and Israel's Ministry of Health during, 2010 EMEA and ANVISA audits are prerequisite for approval Current facility can supply over 1000 patients Currently building up launch inventory Expansion of manufacturing facility in process with an estimated cost of ~
Slide 23: Taliglucerase Milestones Over the Next Months
Taliglucerase Milestones Over the Next 12 Months PDUFA in US (February 25, 2011) Potential approval in Israel Potential approval in Europe Potential approval in Brazil Additional data from Switchover study Filing for Approval in additional territories around the world 23 Confidential
Slide 24: Pipeline Overview
Pipeline Overview Confidential
Slide 25: PRX-105: Biodefense
25 PRX-105: Biodefense An enzyme present within the synaptic cleft that hydrolyses acetylcholine to choline and acetic acid Protalix has expressed this enzyme and confirmed biological activity in several in- vitro and in vivo assays In development for highly lucrative biodefense market and several o
Slide 26: PRX-102: Fabry's Disease
26 PRX-102: Fabry's Disease Prx-102 is a plant cell expressed, chemically modified and improved version of the human ^-Galactosidase- A enzyme Potential Advantages: Enhanced stability Longer circulatory half life Enhanced activity in target organs Reduced immunogenicity Improved kinetics 2010 Advanc
Slide 27: PRX-106: Anti TNF Protein
Enbrel(tm) (etanercept) product overview: Mode of action: a competitive inhibitor of TNF Expression platform: CHO Indications: Rheumatoid Arthritis and multiple autoimmune indications Market: ~$7.0 billion PRX-106 Amino acid sequence is identical to Enbrel(tm) Current Status: Pre-IND meeting with FD
Slide 28: Oral Delivery of prGCD for Gaucher's Disease
Oral Delivery of prGCD for Gaucher's Disease prGCD is stable in lyophilized carrot cells for months In animal studies prGCD is found in liver and spleen, Gaucher target organs when prGCD carrot cells are fed to rats Active prGCD enzyme is found in target organs following feeding Preliminary PK data
Slide 29: Oral delivery of therapeutic proteins
Oral delivery of therapeutic proteins Long time goal for the bio pharmaceutical industry Currently only very limited success The Plant Cell Advantage The Concept: Plant cell wall (cellulose) serves as protective agent against the gastric environment and can serve as an oral administration vehicle Pr
Slide 30: Financial Overview
Financial Overview Confidential
Slide 31: Financial Status
Financial Status 31 $ in millions Quarter Last Twelve Ending Months Ended 9/30/2010 9/30/2010 [81.2]M Shares Common Stock O/S 9/30/2010 Unaudited Unaudited Cash & Cash Equivalents $44.4 $44.4 Revenues 3.2 5.9 R & D Expenses* 3.5 28.9 G & A Expenses 1.4 7.6 Operating Cash Use (9.7) 28.7 (1) (1) Inclu
Slide 32: Protalix Shareholder Perspectives
Protalix Shareholder Perspectives 32 Top Holders Total Holdings % of Total Outstanding * Biocell 14.5mm 17.9% Techno-Rov 6.2mm 7.6% Baillie Giffold & Co. 5.8mm 7.2% Named Executive Officers 7.1mm 8.7% * Based on shares outstanding as of 9/30/10 10Q. 1.3mm shares subject to named executive officer 10
Slide 33: Late Stage Biologics Program Targeting a Significant Market
Late Stage Biologics Program Targeting a Significant Market Best-In-Class Commercial Partner Attractive Platform Technology Promising Pipeline Lead Product taliglucerase alfa indicated for Gaucher disease Completed PIII trial and notified of NDA acceptance in July 2010 Open-label extension trial ong
Slide 34: Q&A
PROTALDX< Biotherapeutics Q&A
Protalix BioTherapeutics — Investor Presentation (January 2011) on 6ix