Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Non-Deal Roadshow (September 2013)
30 slides from Protalix Biotherapeutics, Inc. (de)'s roadshow presentation, Sep 11, 2013
Slide 1: Protalix BioTherapeutics Roadshow Presentation September 2013
Protalix BioTherapeutics Roadshow Presentation September 2013 David Aviezer, Ph.D., MBA President & Chief Executive Officer Yossi Maimon, CPA, MBA Chief Financial Officer
Slide 2: Note Regarding Forward-Looking Statements
This presentation contains forward - looking statements within the meaning of Section 27 A of the Securities Act of 1933 , as amended, and Section 21 E of the Securities Exchange Act of 1934 , as amended. The forward - looking statements including, among others, statements regarding expectations as
Slide 3: Protalix Investment Thesis
▪ Collaboration with for ELELYSO™ commercialization ▪ Rights for Israeli and Brazilian market fully owned by Protalix ▪ Long - term commercial agreement with Brazilian Government ▪ Plant cell - based protein expression system – ProCellEx ® ▪ Significant advantages over existing expression systems ▪
Slide 4: Protalix Pipeline
Protalix Pipeline Compound Indication Research Pre clinical Phase I Phase II Phase III Approved Partner Market taliglucerase alfa (i.v.) Gaucher Disease >$ 1.25 B PRX - 105 (Acetyl Cholinesterase) Biodefense & CNS >$700M PRX - 102 ( Alpha Galactosidase) Fabry Disease ~$900M PRX - 112 ( Oral Glucocer
Slide 5: ProCellEx Proprietary Technology Platform
ProCellEx ® Proprietary Technology Platform 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 poten
Slide 6: ELELYSO for Gaucher Disease
ELELYSO for Gaucher Disease 㻢
Slide 7: 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 8: Current Market Dynamics
*Zavesca ® (Actelion) is a small molecule drug approved for the treatment of Gaucher disease, however usage is extremely limi ted due to significant side effects • Approximately 12 , 000 patients worldwide • Approximately 6 , 000 patients are being treated • ~ 50 % global market penetration • Orphan
Slide 9: ELELYSO: Approval Status
ELELYSO: Approval Status • U.S. FDA approved enzyme replacement therapy for the long – term treatment of adults with a confirmed diagnosis of type 1 Gaucher disease • Priced in the U.S. 25 % below the cost of Cerezyme® United States • August 2010 Pfizer entered into a $ 30 million short - term sup
Slide 10: ELELYSO: Commercialization Agreement with Terms
ELELYSO: Commercialization Agreement with Terms • Pfizer and Protalix share net profit and net loss of ELELYSO on a 60 % / 40 % basis. Only certain limited capped expenses allowed Territories • Pfizer retains exclusive worldwide rights outside of Israel and Brazil • Protalix retains exclusive commer
Slide 11: Gaucher Personal Support and Supply Continuity Program
Gaucher Personal Support • Reimbursement support ▪ Help with benefit verification and pre - authorization for treatment ▪ $ 0 out - of - pocket costs for eligible patients • Infusion support ▪ Assists with administration of at - home infusions ▪ Schedules transportation to and from infusions and hel
Slide 12: ELELYSO: Approved in Israel
ELELYSO: Approved in Israel • Approved by Israeli Ministry of Health – September 27 , 2012 • Approved for reimbursement via the national health basket as of January 2013 ▪ Agreements in place with payers ▪ Currently , for the first quarter of commercial sales in Israel, ELELYSO has generated $ 1.0 M
Slide 13: ELELYSO / UPLYSO: Brazil
ELELYSO / UPLYSO TM : Brazil • Protalix and Brazil’s Ministry of Health enter into supply and technology transfer agreement for UPLYSO (Marketing name in Brazil) • Fiocruz (an arm of the Brazilian Ministry of Health) is required to complete purchase of at least ~$ 280 million worth of UPLYSO to reac
Slide 14: PRX-102 for Fabry Disease
PROTALIDX Biotherapeutics PRX-102 for Fabry Disease
Slide 15: Fabry Overview
Fabry Overview • Fabry Disease Second most common lysosomal storage disease Accumulation of Gb 3 ( α - Galactosidase - A substrate) causes multiple system disorders Main target organs affected – heart, kidney and brain • α - Galactosidase - A A non - covalently linked Homo - dimer of 404 aa
Slide 16: PRX-102: Potential Best-in-Class Fabry Treatment (Enzyme Kinetics)
Enzyme Kinetics - Michaelis Menten Kinetics pNP-alpha-D-Galactpyranoside (mM) 0 2 4 6 8 10 12 14 16 18 20 22 24 26 Reaction Rate (µM/min) 1 2 3 PRX102 Sample K M (µM ) V max (µM/min ) k cat (sec - 1 ) k cat / K M (sec - 1 * mM - 1 ) Replagal ® 4443 3.31 52.96 0.011 PRX - 102 3285 3.72 59.53 0.018 Im
Slide 17: Bio-distribution and efficacy of PRX-102 in Fabry mice
Bio - distribution and efficacy of PRX - 102 in Fabry mice following repeat dosing Experiment Four injections once every two weeks with either 2 mg/kg of PRX - 102 or placebo 㻝㻣 Gb 3 clearance from tissues following repeated administration of PRX - 102 or placebo PRX - 102 : Potential Best - in - Cl
Slide 18: Improved In-vivo Activity: PRX-102 vs. Replagal
PRX - 102 exhibits higher activity levels in target organs over time in Fabry mice after a single injection Single injection of either PRX - 102 or Replagal ® Improved In - vivo Activity: PRX - 102 vs. Replagal 㻝㻤 PRX - 102 : Potential Best - in - Class Fabry Treatment
Slide 19: PRX-102: Clinical Perspectives
• Aug 2012 – Protalix Received FDA clearance of Investigational New Drug (IND) application • Multi - center submissions to IRBs – Ongoing • Phase I/II trial in Fabry Patients – Initiated under IND • Ongoing phase I/II trial in Fabry patients • Patients currently being treated and/or recruited in sit
Slide 20: Other Pipeline Programs
PROTALIDX Biotherapeutics Other Pipeline Programs www.protalix.com
Slide 21: GCD Enzyme Oral Delivery
GCD Enzyme Oral Delivery • Oral delivery of therapeutic proteins Long - time goal of the biopharmaceutical 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 a
Slide 22: PRX-112: Plant Cell Expressed GCD for Oral ERT
PRX - 112 : Plant Cell Expressed GCD for Oral ERT 㻞㻞 Development Status • Study conducted under Israeli MOH approval • Study performed in Shaarei Zedek (Jerusalem) and Rambam (Haifa) medical centers in Israel • Scheduled study completion – Q 3 2013 • Study Report – Q 4 2013 Overview • Edible carrot
Slide 23: PRX-106: Overview
Enbrel ® (etanercept) product overview: • Mode of action: a competitive inhibitor of TNF • Expression platform: CHO • Indications: Rheumatoid Arthritis and multiple autoimmune indications • Market: ~$ 8 billion PRX - 106 : Overview 㻞㻟 Enbrel ® Overview • Main function: TNF α “ trap” for anti - infla
Slide 24: Oral Administration of Plant Cells Expressing Anti-TNF for Colitis
Oral Administration Of Plant Cells Expressing Anti - TNF Fusion Protein (PRX - 106 ) For The Treatment Of Colitis 㻞㻠 Colon Pathology • Project Highlights: Oral Plant Cell platform Targets disease site IP advantage • Expressing PRX - 106 • Experiment: TNBS animal model of colitis Induction of
Slide 25: Oral PRX-106 Alleviates ConA Immune-Mediated Hepatitis
* p< 0.02 ; relative to saline; & p< 0.0005 , relative to negative control # p< 0.03 , relative to negative control Orally Administered Plant Cell PRX - 106 Alleviates ConA Immune - Mediated Hepatitis 0 1000 2000 3000 4000 5000 6000 7000 u/L ALT AST * * &* &* &* * * Experiment: ConA liver inflammati
Slide 26: Oral PRX anti-TNF Summary
Oral PRX 106 anti - TNF Summary Positive results from two models: Hepatitis (Con - A model) - Two consecutive experiments (~ 50 animals) ▪ Oral PRX - 106 reduced inflammation(INF γ and liver enzymes depletion) ▪ Oral PRX - 106 reduced necrosis in the liver Colitis (TNBS model) – Two consecutive expe
Slide 27: Financial Overview and Upcoming Milestones
Financial Overview and Upcoming Milestones
Slide 28: Financial Status
Financial Status $ in millions Six months Six months ended ended 6/30/2013 6/30/2012 ~ 93.5M Shares Common Stock O/S 9/15/2013 Unaudited Unaudited Cash & Cash Equivalents $33.1 $59.3 Total Revenues 7.0 3.9* R&D Expenses, net 11.8 15.7 G&A Expenses 4.3 5.1 Net Loss $11.2 $21.3* * Excludes $25M mil
Slide 29: Financial Overview
Financial Overview • ~ 93.5 M shares outstanding ( 6.30.2013 ) • US, Israeli and EU institutional holders • Publicly traded on NYSE MKT and TASE • Substantial tax benefits 㻞㻥
Slide 30: Upcoming Milestones
Upcoming Milestones Next 12 months • Receive additional marketing approvals for ELELYSO • Report data from clinical trial of oral prGCD for Gaucher disease • Complete recruitment for Phase I/II trial of PRX - 102 in Fabry patients • Report data from Phase I/II trial of PRX - 102 in Fabry patients •
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