Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Corporate Update (January 2017)
31 slides from Protalix Biotherapeutics, Inc. (de)'s corporate update, Jan 9, 2017
Slide 1: Protalix BioTherapeutics Corporate Update January 2017
Protalix BioTherapeutics Corporate Update January 2017
Slide 2: 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 Snapshot
3 Protalix Snapshot • FDA approval for plant cell culture based production of a protein - Elelyso® ( afaltaliglicerase ) • 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 infl
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 Significant reduction of capital consumption through sales of alfataliglicerase in Brazil
Slide 5: Pipeline Overview
Pipeline Overview 5 for Fabry Disease PRX - 102 alidornase alfa for Cystic Fibrosis PRX - 110 Oral anti - TNF for Ulcerative Colitis OPRX - 106 Preclinical Phase II Phase III
Slide 6: PRX-102 (pegunigalsidase alfa) for Fabry Disease
PROTALDX Biotherapeutics peg nighsidase alfa (PRX-102) for Fabry Disease
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 quality of life Renal function continues to decline even for patients on long term Enzyme Replacement Therapy (ERT ) Rombach et al 2013 Little functional enzyme every se
Slide 9: PRX-102 PEGylated plant cell derived alpha-galactosidase-A
(PRX - 102 ) 9 PEGylated covalently bound homodimer composes of 2 subunits forming an active and stable enzyme Designed to be superior to the currently approved ERTs Proven Advantages • Larger amounts and longer duration of active enzyme • Enhanced uptake and activity in target organs • Lower format
Slide 10: Days of Active Enzyme
10 14 Days of Active Enzyme 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 4 8 14 12 0 T½ 78.9 2 1.8 0 20 40 60 80 100 Pegunigalsidase alfa (1mg/
Slide 11: PRX-102 Positive impact on Kidney Function
(PRX - 102) 11 Positive impact on Kidney Function Results from Phase I/II Clinical trial 1. Measured as Annualized Rate of Estimated GFR Change (mL/min/1.73 m 2 /year ) 2. Germain et al 2015 Classic Fabry Patients eGFR slope 1 Pegunigalsidase alfa ( eGFR BL 82.4 - 156.3 ) - 1.8* Fabrazyme ® ( eGFR B
Slide 12: PRX-102 Positive impact on cardiac function
(PRX - 102) Positive impact on cardiac function Results from Phase I/II Clinical trial 0 20 40 60 80 100 120 0 6M 12M 0 6M 12M 0 6M 12M EJECTION FRACTION (%) LEFT VENTRICULAR MASS (g) LV MASS INDEX (g/m^2) Classic Fabry patients N=10 0 1 2 3 4 5 6 0 12M 0 12M 0 12M WORST PAIN PAIN ON AVERAGE MEAN IN
Slide 13: PRX-102 well tolerated, low antibody formation
(PRX - 102) 13 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 – all of whom are sero - negative after one year of treatment Favorable safety and tolerability observed throughout
Slide 14: Phase III pivotal trial vs Fabrazyme
(PRX - 102 ) Randomized, double blind, active control study of pegunigalsidase alfa (PRX - 102) 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 : Demon
Slide 15: Supportive switch-over study from Replagal
(PRX - 102) Open label, single arm switch over study to assess the efficacy and safety of pegunigalsidase alfa (PRX - 102) in Fabry patients currently treated with Replagal ® Number of patients to be enrolled : 22 Objective : Safety and Efficacy data of patients switched from Replagal® to PRX - 102
Slide 16: Summary of Global Clinical Program to Support Filing
(PRX - 102 ) 16 Head to Head vs. Fabrazyme ® in Switch Patients Switch - over from Replagal ® FDA 24 mos Superiority Supportive – 12 mos EMA Rest of World 12 mos Comparability (potential for superiority ) Supportive – 12 mos Summary of Global Clinical Program to Support Filing • Ongoing follow - up
Slide 17: PRX-102 market and peak sales potential
(PRX - 102) 17 At current rate, market is projected to grow from $ 1.2 B to $ 1.7 B in the next 5 yrs Target – Worldwide Naïve & Switch patients Potential superiority in efficacy based on : Peak Sales Potential of $1B Annually Potential to be gold standard therapy Fabrazyme ® pegunigalsidase alfa eG
Slide 18: alidornase alfa (PRX-110) for Cystic Fibrosis
alidornase alfa (PRX - 110) For Cystic Fibrosis
Slide 19: Cystic Fibrosis
Cystic Fibrosis (CF) Rare genetic disease characterized by a highly viscous mucus most prominently leading to severe lung damage and loss of respiratory function More than 70,000 CF patients worldwide. Growth in number of patients and increase in life expectancy Target Product Annual Sales Reduction
Slide 20: alidornase alfa with Actin Inhibition Resistance
alidornase alfa (PRX 110 ) with Actin Inhibition Resistance 20 Plant - cell derived, 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 concentrations in CF patient
Slide 21: alidornase alfa Clinical Development Plan for CF
alidornase alfa - Clinical Development Plan for CF Phase I – 18 healthy volunteers: Completed • a lidornase alfa was found to be safe and tolerable Phase II - Switch - over study for 15 CF patients previously treated with Pulmozyme ® - Completed • Study duration: 28 days • Efficacy endpoint: FEV1 •
Slide 22: Top Line Results from Interim Analysis of Phase II trial
Top Line Results from Interim Analysis of Phase II trial 22 alidornase alfa (PRX 110 ) was found to be safe and well - tolerated Clinically meaningful improvement in lung function • Mean absolute improvement in ppFEV 1 compared to baseline of 4.1 points * n=13 ** Orkambi ® phase III trial “The preli
Slide 23: Decrease in Sputum DNA Content and Viscosity
Decrease in Sputum DNA Content and Sputum Viscosity upon a lidornase alfa Treatment Initiation 23 * n=7 **n=5 1.42 0.53 0.0 0.2 0.4 0.6 0.8 1.0 1.2 1.4 1.6 Dose Initiation Baseline Treatment 4 weeks mg/mL Mean DNA content* 36.40 3.86 0.0 5.0 10.0 15.0 20.0 25.0 30.0 35.0 40.0 Dose Initiation Baselin
Slide 24: Oral anti-TNF (OPRX-106) for Ulcerative Colitis
Oral anti - TNF (OPRX - 106) For Ulcerative Colitis
Slide 25: 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 26: Oral Anti-TNF OPRX-106
Oral Anti - tumor Necrosis Factor Alpha (anti TNF α ) OPRX - 106 for Inflammatory Diseases 26 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
Slide 27: OPRX-106 Clinical Program
OPRX - 106 Clinical Program 27 Phase I – COMPLETED Safe and well tolerated. Immunomodulation: Regulatory T cell activation showed biological activity in the gut Alteration of systemic immune system without significant systemic absorption Serum cytokine levels remained stable Phase II – ONGOING 15 mi
Slide 28: Sales of Alfataliglicerase in Brazil
Sales of Alfataliglicerase in Brazil 28 Negotiations held with the newly elected government officials in the Brazilian Ministry of Health regarding the potential of alfataliglicerase to become the preferred enzyme replacement therapy for the approximately 700 Gaucher patients treated in Brazil Brazi
Slide 29: Financial Overview
Financial Overview 29 • 124M shares outstanding, as of December 31, 2016 • Dual listed on NYSE MKT and TASE • Strong cash position: ~$63M as of December 31, 2016 • Cash level currently projected to fund operations into late 2019 • $14.9M convertible note due by September 2018, $62.7M convertible not
Slide 30: Protalix has an exciting road ahead
Protalix has an exciting road ahead… 30 x Promising results for pegunigalsidase alfa and alidornase alfa x Clinical development pipeline targeting markets >$8B x R&D focus to advance early pipeline with attractive opportunities for proteins designed for superior clinical profiles x Sales in Brazil s
Slide 31: Moshe Manor, President and CEO
Moshe Manor President and CEO Protalix Biotherapeutics moshe.manor@protalix.com
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