Protalix BioTherapeutics, Inc. (de) · AMEX:PLX
Protalix BioTherapeutics, Inc. (de) — Investor Presentation (November 2021)
22 slides from Protalix BioTherapeutics, Inc. (de)'s investor presentation, November 2021
Slide 1: Protalix Biotherapeutics Corporate Presentation
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION November 2021
Slide 2: Note Regarding Forward-Looking Statements
Note Regarding Forward-Looking Statements This presentation (the “Presentation”) contains forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements, including, among others, st
Slide 3: Investment Highlights
Investment Highlights Plant cell expressed recombinant proteins with improved therapeutic profiles Revenue Generating Platform FDA approved, commercially marketed drug for Gaucher Proprietary ProCellEx® platform for recombinant protein expression disease. Elelyso® and alfataliglicerase. cGMP manufac
Slide 4: Plant Cell in Suspension Expression System: Executive Summary
Plant Cell in Suspension Expression System for Therapeutic Proteins Development and Industrial Production: Executive Summary Cell lines and Genetic Engineering Simplified and consistent Production Process Unique genetic engineering tools used for producing improved tobacco plants cell lines and Prod
Slide 5: ProCellEx Platform
ProCellEx® Platform Protalix’s ProCellEx® platform uses flexible polyethylene disposable bioreactors and is optimized for plant cell cultures. As opposed to the large stainless steel bioreactors commonly used for recombinant protein production, the ProCellEx® bioreactors are easy to use and maintain
Slide 6: ProCellEx Platform: Proprietary Plant Cells Protein Expression System
ProCellEx® Platform: Proprietary Plant Cells Protein Expression System First and only company to gain FDA approval of a protein produced through plant cell-based expression system Unique capabilities of tailoring genetic engineering and protein engineering tools for pre/post production modifications
Slide 7: From Concept to Market
From Concept to Market Selecting the Gene of Protein drug Pilot production Product Commercial target interest enhancement with ProCellEx® development production protein-drug engineering modifications platform candidate Plant cells Improving Purification and Clinical development Scaling up to genetic
Slide 8: Elelyso for Gaucher Disease
Elelyso® for Gaucher Disease First plant cell derived recombinant protein approved by the FDA Elelyso provides a consistent (and growing) revenue stream for Protalix while validating Validation of the ProCellEx® platform the ProCellEx platform technology and demonstrating the company’s manufacturing
Slide 9: Product Pipeline
Product Pipeline Recombinant proteins with improved therapeutic profiles that target unmet medical needs and established pharmaceutical markets. Discovery and Preclinical Phase 1 Phase 2 Phase 3 Marketing Application pegunigalsidase alfa (PRX-102) Fabry Disease alidornase alfa (PRX-110) Various Resp
Slide 10: Fabry Disease
Fabry Disease Fabry disease is primarily treated with Rare Genetic Disease Growing Market enzyme replacement therapy occurs in one of every (ERT) to replace the missing ~$2.2B+ growing market 40,000 people α-Galactosidase-A enzyme with (expected CAGR ~10%) a recombinant form of the protein via intra
Slide 11: Fabry Disease Competitive Landscape
Fabry Disease Competitive Landscape Product Name Fabrazyme® Replagal® Galafold® Parent Company Sanofi (Genzyme) Takeda (Shire) Amicus Mechanism ERT ERT Pharmacological chaperone Indication Fabrazyme is indicated for use in patients with Replagal is indicated for long-term enzyme Galafold is an α-gal
Slide 12: Pegunigalsidase Alfa Mechanism of Action (MoA)
Pegunigalsidase Alfa Mechanism of Action (MoA) • Pegunigalsidase is a PEGylated enzyme designed to be potentially superior to the currently approved ERTs, primarily on safety and immunogenicity that has the potential to be translated to efficacy • Covalent linked via short 2kD PEG having two reactiv
Slide 13: Clinical Program Overview
Clinical Program Overview Pegunigalsidase Alfa PRX-102 Mechanism of Action Ø Delivered to lysosome to reduce accumulated substrate Ø Increased exposure Ø Low Immunogenicity Phase I/II Ø Safety Ø Reduction of Gb3 & Lyso Gb3 v Safety v Safety v Safety v eGFR slope improvement v Potential Superiority v
Slide 14: Phase I/II Study - Development Rationale
Phase I/II Study - Development Rationale High levels of active available enzyme à potentially improved clinical benefit • Chemically modified plant cell derived PEGylated covalently bound homodimer • In Phase I/II clinical trial, pegunigalsidase alfa has been observed to be stable throughout 2-week
Slide 15: Phase I/II Study: Biomarker Reduction
Phase I/II Study Substantial reduction & high correlation between two Fabry disease biomarkers Gb3 Burden in Kidney Biopsies (BLISS) Responder analysis: 10 Portion of patient achieved reduction in BLISS score Baseline 6 month 8 Mean score ± SE 90 6 80 12/14 -85.0% 4 11/14 -47.7% -67.8% 70 10/14 2 60
Slide 16: Pegunigalsidase alfa (PRX-102) Current Clinical Trials
Pegunigalsidase alfa (PRX-102) Current Clinical Trials Design Number of Patients Next Data Read-Out Completed 1mg/kg 2 weeks Randomized Final Outcomes Data Double Blind Head-to-Head 78 Expected 1H 2022 vs. Fabrazyme® 24 mos. 1mg/kg 2 weeks Open Label 22 Final results Switch Over from Replagal® repor
Slide 17: Protalix Recent and Upcoming Expected Milestones
Protalix Recent and Upcoming Expected Milestones Q2 2020 Q1 2021 Top-Line results of Equity Financing gross BRIDGE proceeds of $40.2M BLA submission under Q4 2020 Q3 2021 Q1 2022 accelerated approval Final results (CSR) of Type A meeting with FDA EMA MAA pathway for BRIDGE following CRL in 2Q21 subm
Slide 18: Committed Commercial Partner
Committed Commercial Partner Global Partnership with Chiesi Farmaceutici S.p.A. Up to $1+ billion in potential milestone payments Chiesi’s 6,000 employees and ~$2.5B in revenue (2019) provides Tiered royalties of 15-35% (ex-US); Protalix with the strong Sales & 15-40% (US) Marketing partner to maxim
Slide 19: Uricase (PRX-115) for Refractory Gout
Uricase (PRX-115) for Refractory Gout PRX-115 is a PEGylated enzyme expressed via ProCellEx® for intravenous (IV) administration of recombinant Uricase, designed for safe and efficacious treatment of refractory gout. Overview • Gout is the most common form of chronic inflammatory disability, general
Slide 20: Long acting (LA) DNase I (PRX-119) for NETs related diseases
Long acting (LA) DNase I (PRX-119) for NETs related diseases PRX-119 is a cutting edge PEGylated recombinant human DNase I, expressed via ProCellEx® , designed to elongate DNase half-life in the circulation for the treatment of NETs related diseases. Overview • Neutrophil extracellular traps (NETs)
Slide 21: Well Capitalized to Mature the Potential of PRX-102
Well Capitalized to Mature the Potential of PRX-102 CASH REVENUE $48.7 M (3Q’21) $12.1 M in revenue (3Q’21) FINANCING NET BURN RATE Successfully completed a Note Exchange in 3Q’21 to $6.0M/Q, declining as patients shift over to extension extend maturity from 2021 to 2024 and lower principal study UB
Slide 22: Experienced Leadership Team
Experienced Leadership Team DROR BASHAN EINAT BRILL ALMON, PH.D. EYAL RUBIN YARON NAOS YAEL HAYON, PH.D. President & CEO SVP, Chief Development Officer SVP & CFO SVP of Operations VP of R&D Mr. Bashan has served as our Dr. Almon joined Protalix in Dec Mr. Rubin has served as our SVP Mr. Naos joined
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