Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Corporate Update (March 2018)
30 slides from Protalix Biotherapeutics, Inc. (de)'s corporate update, Mar 21, 2018
Slide 1: Protalix BioTherapeutics Corporate Update March 2018
Protalix BioTherapeutics Corporate Update March 2018
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: Company Highlights
Company Highlights 3 Clinically differentiated and improved recombinant therapeutic proteins • FDA approved ProCellEx® plant cell - based expression system • FDA multi - product and EMA approved manufacturing facility in Israel Pegunigalsidase alfa in Phase III for Fabry disease ~$1.4B growing marke
Slide 4: Corporate Strategy
Corporate Strategy 4 Execution of Phase III Fabry clinical trial program and key data read - outs and potential global approvals Focus on additional partnering opportunities for assets in development Generate revenue through sales of alfataliglicerase in Brazil Advancement of early pipeline products
Slide 5: Pipeline Overview
Pipeline Overview 5 (PRX - 102) Fabry Disease alidornase alfa (PRX - 110) Cystic Fibrosis Oral anti - TNF - α (OPRX - 106) Ulcerative Colitis/ Inflammatory Bowel Disease Preclinical Phase I Phase II Phase III
Slide 6: Pegunigalsidase alfa for Fabry Disease
PROTALDX Biotherapeutics pequieeies alfa amg/kg/2 weeks HM 2mg/kg/4 weeks for Fabry Disease
Slide 7: Fabry disease
Fabry disease 7 Rare genetic lysosomal storage disorder caused by deficiency in the enzyme α - galactosidase A. Lipids accumulate in key organs (kidney, heart, CNS) leading to a progressive and potentially life threatening disease . Disease characterized by: • Progressive kidney disease which can le
Slide 8: Fabry disease remains a high unmet need
Fabry disease remains a high unmet need - Learnings: 8 Limited effect of ERT due to: • Little functional enzyme every second week and incomplete tissue penetration of the infused protein 1 • Presence of antibodies to infused enzyme 1 • Renal function continues to decline even for patients on long te
Slide 9: Pegunigalsidase alfa: substantially greater enzyme exposure than current ERTs
Pegunigalsidase alfa ≥ 14 Days of active enzyme Fabrazyme® time frame* Concentration (ng/mL) 1 10 100 1000 10000 100000 0.0 50.0 100.0 150.0 200.0 250.0 300.0 350.0 Time (hours ) PRX-102 1mg/Kg Fabrazyme 1mg/Kg pegunigalsidase alfa 1mg/kg 14 Days * * Fabrazyme ® ( agalsidase beta) – USPI Higher le
Slide 10: Higher uptake and prolonged activity in target organs
10 Higher uptake and prolonged activity in target organs µg/gr FW = amount of enzyme [µg] per gr of tissue fresh weight [FW], assessed by activity Kizhner T. et al ( 2015 ), A - Molecular Genetics and Metabolism 114 : 259 – 267 Fabry mice model Higher levels of active enzyme in target organs poten
Slide 11: PK modeling shows greater AUC with once-monthly infusion
11 PK modeling shows greater AUC in a single infusion of 2 mg/kg pegunigalsidase alfa vs. two bi - weekly infusions of Fabrazyme® over a 4 - week time frame. First - in - class, once - monthly administration Greater AUC potential for clinical stability with half the infusions
Slide 12: Unique proposition for addressing significant unmet needs
12 Unique proposition for addressing significant unmet needs Two dosing regimens: potential for better efficacy and lower treatment burden x Treatment flexibility for patients x Two independent paths for product superiority 1 mg/kg/ 2 weeks 2 mg/kg/ 4 weeks Superior ERT for patients with progressing
Slide 13: Efficacy and safety seen over months
13 Efficacy & safety seen over 24 months Positive impact on kidney function Pegunigalsidase alfa (eGFR BL 82.4 - 156.3 ) Fabrazyme® (eGFR BL 49 - 170) 2 eGFR slope 1 - 2.2* - 3.8 *n= 7 classic Fabry patients 1. eGFR = e stimated G lomerular F iltration R ate . Measured as annualized rate of Estimate
Slide 14: Clinical advantages recognized by regulatory bodies
Clinical advantages recognized by regulatory bodies Orphan Drug Designation ( ODD) granted by European Commission for significant and plausible clinical advantages compared to authorized therapies • Clinical data demonstrating the stabilization of kidney function • Reduced immunogenicity and periphe
Slide 15: Ex-US collaboration with Chiesi
15 Chiesi, an international privately - held company, strong presence outside US, with focus on the development and commercialization of innovative medicines In exchange for ex - US commercialization rights for pegunigalsidase alfa: • Upfront payment of $ 25 million and additional payments of up to
Slide 16: Robust Phase III pivotal clinical program
16 1mg/kg 2 weeks Head to Head vs. Fabrazyme® in Switch Patients 2mg/kg 4weeks Switch - over from Fabrazyme® and Replagal ® 1mg/kg 2 weeks Switch - over from Replagal® FDA 24 mos Superiority 12 mos Safety and efficacy 12 mos Supportive EMA Rest of World 12 mos Comparability (potential for superiorit
Slide 17: Potential to be gold standard therapy
17 Potential to be gold standard therapy Peak Sales Potential over $ 1B Annually ( >50% market share) » 1 mg/kg /2 weeks Potential superiority in efficacy Fabrazyme® pegunigalsidase alfa eGFR slope - 3.8 - 2.2* Half life 2 hours ~80 hours Active enzyme ½ day >14 days Antibody presence 74% 0%** *N=7
Slide 18: Oral anti-TNF-α OPRX-106 for Inflammatory Bowel Disease
Oral anti - TNF - α OPRX - 106 for Inflammatory Bowel Disease
Slide 19: Inflammatory Bowel Disease (IBD)
IBD are autoimmune inflammatory diseases of the digestive tract including Ulcerative Colitis (UC ) and Crohn’s Disease (CD ) • Debilitating: severe diarrhea, abdominal pain, fatigue and weight loss • Affects > 2.5 million people across US and Europe UC market ~ $5.5B; CD market ~ $3.0B Key players i
Slide 20: IBD remains an area of high unmet need
Despite the wide array of available treatments, there are major drawbacks: Loss of response to anti – TNF α drugs • Clinical efficacy is lost in ~25 - 45% of all patients who were primary responders • Most likely attributed to the high level of neutralizing antibodies Safety risks • Black box warnin
Slide 21: OPRX-106: plant cell expressed oral anti-TNF-α
Developed via Protalix’s platform for orally delivered proteins whereby the plant cell wall protects the protein and serves as a natural oral administration vehicle. Administered orally, OPRX - 106 is biologically active in the gut with no anti - drug antibody formation and without systemic absorpti
Slide 22: Positive Clinical Results OPRX-106
22 61% 72% 72% 89% 28 % 67% Geboes score Fecal calprotectin Improved rectal bleeding Improved Mayo score Clinical remission Clinical response Positive Clinical Results OPRX - 106 Safe and well - tolerated. Adverse events (AEs) were mild to moderate and transient No anti drug antibodies were detected
Slide 23: Oral anti-TNF-α biologically locally active in the gut
Inflammatory Bowel Disease market >$8.0B High clinical response and remission rates in patients with moderate disease No formation of anti - drug antibodies No systemic absorption Will potentially not carry the safety risks of current therapies. Overcomes the drawbacks of injected or infused anti -
Slide 24: Alidornase alfa (PRX-110) for Cystic Fibrosis
alidornase alfa (PRX - 110 ) for Cystic Fibrosis
Slide 25: Cystic Fibrosis and alidornase alfa
25 Cystic Fibrosis (CF) is a rare genetic disease characterized by a highly viscous mucus most prominently leading to severe lung damage and loss of respiratory function. Over 70,000 CF patients world - wide Leading product, Pulmozyme®, DNase enzyme, ~$700M annual sales with significant growth poten
Slide 26: Phase II trial demonstrates clinically meaningful lung function improvement
26 Mean absolute change in ppFEV1 * N=16 Phase II trial demonstrates clinically meaningful lung function improvement alidornase alfa ( PRX - 110 )
Slide 27: Clinically meaningful lung function improvement and next steps
27 Clinically meaningful lung function improvement as a result of effective mucociliary clearance Extraordinary reduction of the presence of Pseudomonas aeruginosa (P.sa) infections as a result of alidornase alfa treatment - All P.sa positive patients showed an >75% reduction of which 60% experience
Slide 28: Financial Overview
Financial Overview 28 • ~ 145M shares outstanding, as of December 31, 2017 • Dual listed on NYSE American and TASE • Cash position: ~$ 51M as of December 31, 2017 • Cash level currently projected to fund operations into 2020 • $5.9M convertible note due by September 2018, ~$59M convertible note due
Slide 29: Significant achievements to date and upcoming goals
Protalix had significant achievements to date 29 x Strong positive two year data for pegunigalsidase alfa x Promising results for OPRX - 106 and alidornase alfa x Clinical development pipeline targeting markets ~$10B x R&D focus to advance early pipeline with attractive opportunities for proteins de
Slide 30: Moshe Manor, President and CEO
Moshe Manor President and CEO Protalix BioTherapeutics moshe.manor@protalix.com
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