Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Corporate Update (June 2016)
31 slides from Protalix Biotherapeutics, Inc. (de)'s corporate update, Jun 7, 2016
Slide 1: Protalix BioTherapeutics Corporate Update June 2016
Protalix BioTherapeutics Corporate Update June 2016
Slide 2: Forward-looking statements
This presentation contains forward - looking statements within the meaning of Section 27A of the Securities Act of 1933, as amende d, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward - looking statements including, among others, statemen ts regarding expectations as to
Slide 3: Protalix Snapshot
3 Protalix Snapshot • FDA approval for plant cell culture based production of a protein - Elelyso® • Protein production site approved by FDA, EMA and other major regulatory bodies world - wide • Phase II candidates: • Inhaled protein for Cystic Fibrosis • Oral protein for inflammatory diseases • Pha
Slide 4: Strategy Highlights
Strategy Highlights 4 Focus on development of proprietary proteins with superior clinical profiles Execution of late stage clinical studies Advancement of early pipeline products into clinical development
Slide 5: Pipeline Overview
Pipeline Overview 5 for Fabry Disease PRX - 102 AIR DNAse TM for Cystic Fibrosis PRX - 110 Oral anti - TNF for Ulcerative Colitis OPRX - 106 Phase I Phase II Phase III Pegunigalsidase alfa* *pending WHO publication
Slide 6: Pegunigalsidase alfa for Fabry Disease (PRX-102)
for Fabry Disease (PRX - 102) Pegunigalsidase alfa
Slide 7: Fabry Disease
Fabry Disease 7 Rare genetic lysosomal storage disorder caused by deficiency in the enzyme α - galactosidase A. ~5,000 patients treated worldwide Lipids accumulate in key organs (kidney, heart, CNS ) leading to a progressive and potentially life threatening disease. Renal and Cardiac failures are th
Slide 8: Fabry Disease Remains a High Unmet Need
Fabry Disease Remains a High Unmet Need 8 Hypertension and cardiomyopathy Renal insufficiency and renal failure Impact on pain and gastrointestinal function Renal function continues to decline even for patients on long term Enzyme Replacement Therapy (ERT ) Rombach et al 2013 Most pronounced 4 - 7 d
Slide 9: Pegunigalsidase alfa (PRX-102): A Chemically Modified Plant Cell Derived Enzyme
Pegunigalsidase alfa (PRX 102) : A Chemically Modified Plant Cell Derived Recombinant Human α - galactosidase - A 9 PEGylated covalently bound homodimer active and stable enzyme Designed to be superior to the currently approved ERTs Proven Advantages • Larger amounts and longer duration of active
Slide 10: Pegunigalsidase alfa: 14 Days of Active Enzyme
10 10 100 1000 10000 100000 0 100 200 300 Concentration ( ng/mL) Days ~1/2 day ~14 days Plasma pegunigalsidase alfa concentration vs. time 1mg/Kg Pegunigalsidase alfa time frame Fabrazyme ® time frame 2 4 6 8 10 14 12 0 T½ 78.9 2 1.8 0 20 40 60 80 100 PRX-102 (1mg/kg) Fabrazyme (1mg/kg) Replegal (0.
Slide 11: Results from Phase I/II Clinical Trial in Fabry Patients
11 104.8 106.2 102.7 0 50 100 BL 6M 12M Positive impact on Kidney Function *measured as Annualized Rate of Estimated GFR Change (mL/min/ 1.73 m 2 /year ) ** Germain et al 2015 *** Phase I/II Doses 0.2 , 1 , 2 mg/kg ; For pegunigalsidase alfa 1 mg/kg dose (n= 6 ). eGFR slope was - 0.30 Pegunigalsidas
Slide 12: Favorable safety and tolerability observed
12 Pegunigalsidase alfa was well tolerated, with 98% of events being mild and moderate Only 3 patients (19%) tested positive for treatment - induced anti - drug antibodies % antibodies Pegunigalsidase alfa 19% Fabrazyme 74%* “As PRX - 102 has a different PK profile, which is probably the cause for t
Slide 13: Phase III pivotal trial initiated
Randomized, double blind, active control study of PRX - 102 (pegunigalsidase alfa ) compared to Fabrazyme ® in Fabry patients previously treated with Fabrazyme ® Number of patients to be enrolled : 78 52 to be switched to pegunigalsidase alfa 26 to remain on Fabrazyme ® Objective : Demonstrate super
Slide 14: Supportive Clinical Trial
Open label, single arm switch over study to assess the efficacy and safety of PRX - 102 (pegunigalsidase alfa) in Fabry patients currently treated with Replagal ® Number of patients to be enrolled : 22 Objective : Efficacy and safety data of patients switched from Replagal ® to PRX - 102 over 12 mon
Slide 15: Summary of Global Clinical Program to Support Filing
15 Pivotal Randomized Controlled Study vs Fabrazyme ® Open Label Switch - Over from Replagal ® FDA 24 mos Superiority Supportive – 12 mos EMA Rest of World 12 mos Interim non - inferiority (potential for superiority) Supportive – 12 mos • Ongoing follow - up of Phase I/II naïve patients • Pediatric
Slide 16: Peak Sales Potential of $1B Annually
16 At current rate, market is projected to grow from $1.2B to $1.7B in the next 5 years Target – Worldwide Naïve & Switch patients Potential superiority in efficacy based on: Fabrazyme ® Pegunigalsidase alfa eGFR slope - 1.89 - 0.32 Half life 2 hours ~80 hours Active available enzyme ½ day 14 days A
Slide 17: AIR DNase (PRX-110) For Cystic Fibrosis
AIR DNase TM (PRX - 110) For Cystic Fibrosis
Slide 18: Cystic Fibrosis (CF)
Cystic Fibrosis (CF) Rare genetic disease characterized by a highly viscous mucus most prominently leading to severe lung damage and loss of respiratory function ~70,000 CF patients worldwide. Growth in number of patients and increase in life expectancy Target Product 2015 Sales Reduce mucus viscosi
Slide 19: AIR DNase
AIR DNase TM Plant cell derived chemically modified recombinant DNase with A ctin I nhibition R esistance 19 Chemically modified DNase enzyme resistant to inhibition by actin thus designed to enhance the enzyme’s efficacy in CF patients’ sputa. Actin , a potent inhibitor of DNase, found in high conc
Slide 20: Reduction of mucus viscosity in human sputum samples
AIR DNase TM : Demonstration of reduction of mucus viscosity in human sputum samples 20 N=6 Rheology Data Analysis
Slide 21: AIR DNase Clinical Development Plan for Cystic Fibrosis
AIR DNase TM Clinical Development Plan for Cystic Fibrosis Phase I – 18 healthy volunteers: Completed • AIR DNase TM found to be safe and tolerable Phase II in CF patients – Initiated • Switch - over study for 15 patients previously treated with Pulmozyme ® • Study duration: 28 days • Efficacy endpo
Slide 22: Oral anti-TNF (OPRX-106) For Ulcerative Colitis
Oral anti - TNF (OPRX - 106) For Ulcerative Colitis
Slide 23: Oral Delivery of Therapeutic Proteins
Oral Delivery of Therapeutic Proteins Requirements for oral delivery of protein therapeutics • Protein must survive the gastric environment • Protein must be released from within the cells into the intestine • Protein must be able to act within the intestine The plant cell advantage: • Plant cell wa
Slide 24: Anti-TNF for Inflammatory Diseases
Anti - tumor Necrosis Factor Alpha (anti - TNF α ) for Inflammatory Diseases Anti - TNF market >$30B with multiple blockbuster products (injections and IV infusions ) Multiple indications: • Ulcerative Colitis (~$5.5B) • Rheumatoid Arthritis ( ~ $ 1 7 B ) • Psoriasis ( ~ $5.7B ) • Crohn’s Disease (
Slide 25: Oral Anti-TNF OPRX-106 for Inflammatory Diseases
Oral Anti - tumor Necrosis Factor Alpha (anti TNF α ) OPRX - 106 for Inflammatory Diseases 25 Potential to locally deliver higher doses with fewer side effects in an oral formulation OPRX - 106 Inflammatory Bowel Disease Animal Model P=0.001 Non - diseased control OPRX - 106 OPRX - 106 TNBS saline s
Slide 26: OPRX-106 Phase I Healthy Volunteers - completed
OPRX - 106 Phase I Healthy Volunteers - completed 26 Safe and well tolerated. Immunomodulation: Regulatory T cell activation showed biological activity in the gut: CD4/CD25, CD4/Foxp3/CD25, CD8/CD25, CD8/Foxp3/CD25) Alteration of systemic immune system without significant systemic absorption Serum c
Slide 27: OPRX-106 Phase II Expected to commence by July 2016
OPRX - 106 Phase II 27 Expected to commence by July 2016 Study design: • 15 mild to moderate untreated ulcerative colitis patients • Oral once daily administration - 8 week treatment duration • Evaluating two doses for: • Safety and Tolerability • Pharmacokinetics • Efficacy parameters: Mayo score,
Slide 28: Financial Overview
Financial Overview 28 • ~ 100M shares outstanding, as of March 31, 2016 • Dual listed on NYSE MKT and TASE • Strong cash position: ~$67M as of March 31 , 2016 • Cash level currently projected to finance the company into 2018, through significant milestones • $69M convertible note due by September 20
Slide 29: Promising results for flagship product for Fabry disease
Promising results for flagship product for Fabry disease Three molecules in clinical development Phase III with pegunigalsidase alfa Phase II with AIR DNaseTM Phase II with Oral anti-TNF Clinical stage pipeline targeting markets that are >$8 billion R&D focus to advance early pipeline with attractiv
Slide 30: Upcoming milestones
Finalize enrollment in Phase III pegunigalsidase alfa (PRX-102) studies in Fabry Enroll patients in Phase II study for AIR DNaseTM (PRX-110) in Cystic Fibrosis Initiate Phase II study for oral anti TNF (OPRX-106) in Ulcerative Colitis Report results from Phase II for AIR DNase TM Report results from
Slide 31: Moshe Manor, President and CEO
Moshe Manor President and CEO Protalix Biotherapeutics moshe.manor@protalix.com
Protalix BioTherapeutics — Corporate Update (June 2016) on 6ix