Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Corporate Update (January 2018)
28 slides from Protalix Biotherapeutics, Inc. (de)'s corporate update, Jan 5, 2018
Slide 1: Protalix BioTherapeutics Corporate Update January 2018
Protalix BioTherapeutics Corporate Update January 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 FDA approved ProCellEx ® plant cell - based expression system being utilized to develop clinically differentiated and improved recombinant therapeutic proteins. FDA multi - product and EMA approved manufacturing facility in Carmiel , Israel. Pegunigalsidase alfa in Phase III for
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 alfataliglucerase 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 (OPRX - 106) – Ulcerative Colitis/ Inflammatory diseases Preclinical Phase I Phase II Phase III
Slide 6: Pegunigalsidase alfa for Fabry Disease
PROTALDX Biotherapeutics pequigestces alfa amg/kg/2 weeks HM 2mg/kg/4 weeks for Fabry Disease
Slide 7: Fabry disease remains a high unmet need
Fabry disease remains a high unmet need 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 . “Little functional enzyme every second we
Slide 8: Unique proposition for addressing significant unmet needs
8 Unique proposition for addressing significant unmet needs A chemically modified plant cell derived PEGylated enzyme. Designed to be superior to the currently approved ERTs Two dosing regimens: potential for better efficacy and lower treatment burden x Treatment flexibility for patients x Two indep
Slide 9: 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: PK modeling shows greater AUC with monthly dosing
10 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 11: Phase I/II data: positive impact on kidney function
11 Phase I/II – 24 month data 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 Excellent safety and tolerability profile • Favorable safety and tolerability observed throughout ~35 patient years • 19% formation
Slide 12: Continuous reductions observed over months
Continuous reductions observed over 24 months 0 2 4 6 8 10 12 14 0 10 20 30 40 50 60 70 80 90 100 0 5 10 15 20 25 Treatment Duration - Months Gb3 ( ug /mL) Lyso - Gb 3 (ng/mL) Lyso Gb3 Gb3 Gastrointestinal symptoms Mean Score 0.00 0.50 1.00 1.50 2.00 2.50 3.00 BL Month 12 Month 24 SEVERITY OF ABDOMI
Slide 13: Continuous clinical stability observed over months
Continuous clinical stability observed over 24 months Stable Renal Function n=11 n=11 0 20 40 60 80 100 120 140 160 0 5 10 15 20 25 eGFR (mL/ min/ 1.73m 2 ) Treatment duration (Months) Individual eGFR CKD - EPI trend - lines 0.0 20.0 40.0 60.0 80.0 100.0 120.0 BL 12M 24M BL 12M 24M BL 12M 24M EF (%)
Slide 14: EU Orphan Drug Designation granted
Clinical advantage recognized with granting of EU Orphan Drug Designation (ODD ) Although other therapies for Fabry Disease are approved for use in the EU, the European Commission granted Orphan Drug Designation (ODD) for pegunigalsidase alfa for the treatment of Fabry disease based on: Pegunigalsid
Slide 15: Ex-US exclusive collaboration with Chiesi Farmaceutici
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 1 mg/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 superiori
Slide 17: Potential to be gold standard therapy
17 Potential to be gold standard therapy Peak Sales Potential over $ 1 B 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
Slide 18: alidornase alfa (PRX-110) for Cystic Fibrosis
alidornase alfa (PRX - 110) for Cystic Fibrosis
Slide 19: Cystic Fibrosis and alidornase alfa
19 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 20: Phase II trial demonstrates lung function improvement
20 Mean absolute change in ppFEV1 * N=16 Phase II trial demonstrates clinically meaningful lung function improvement alidornase alfa (PRX 110 )
Slide 21: Clinically meaningful lung function improvement
21 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 22: Oral anti-TNF OPRX-106 for Ulcerative Colitis
Oral anti - TNF OPRX - 106 for Ulcerative Colitis
Slide 23: Oral anti-TNF platform OPRX-106
With Protalix’ platform for orally delivered proteins, the plant cell wall protects the protein and serves as a natural oral administration vehicle OPRX 106 is a plant cell - expressed recombinant anti - TNF fusion protein Anti - TNF market >$30B with multiple blockbuster products all of which are i
Slide 24: Clinical Program for Ulcerative Colitis
24 Phase I – 15 Healthy Volunteers - COMPLETED Safe and well tolerated. Alteration of systemic immune system with no systemic absorption Phase II – ONGOING Enrollment completed – 24 mild to moderate ulcerative colitis patients First 14 patients completed the study and four patients still ongoing Ora
Slide 25: Positive interim results of OPRX-106
Positive interim results of 14 patients: Improvement for majority of patients OPRX 106 Parameter Number of patients Clinical Response 1 8/14 57% Clinical Remission 2 5/14 36% Improved rectal bleeding score 3 11/14 79% Fecal calprotectin 12/14 86% Geboes score 9/14 64% OPRX 106 was safe and well - to
Slide 26: Financial Overview
Financial Overview 26 • ~144M shares outstanding, as of December 31, 2017 • Dual listed on NYSE MKT and TASE • Cash position: ~$50.5M 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 by No
Slide 27: Protalix has an exciting road ahead
Protalix has an exciting road ahead… 27 x Two year data for pegunigalsidase alfa x Promising results for alidornase alfa and OPRX 106 x Clinical development pipeline targeting markets ~$7B x R&D focus to advance early pipeline with attractive opportunities for proteins designed for superior clinical
Slide 28: Moshe Manor, President and CEO
Moshe Manor President and CEO Protalix BioTherapeutics moshe.manor@protalix.com
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