Protalix BioTherapeutics, Inc. (de) · AMEX:PLX
Protalix BioTherapeutics, Inc. (de) — Investor Presentation (March 2021)
22 slides from Protalix BioTherapeutics, Inc. (de)'s investor presentation, March 2021
Slide 1: Protalix Biotherapeutics Corporate Presentation March 2021
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION MARCH 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: ProCellEx Platform
ProCellEx® Platform 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, customized for each individual protein candidate
Slide 5: Advantages of Proprietary Plant Cell-Based Platform (ProCellEx)
Advantages of Proprietary Plant Cell-Based Platform (ProCellEx®) Biologic Optimization Can Handle Complexity Experienced internal interdisciplinary capabilities Ability to express certain proteins that are to improve biologic dynamics (e.g., glycosylation difficult to express in other systems profil
Slide 6: 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 7: 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) Licensed to S
Slide 8: 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 ~$2B+ growing market 40,000 people α-Galactosidase-A enzyme with (expected CAGR ~10%) a recombinant form of the protein via intrave
Slide 9: Fabry Disease Competitive Landscape
Fabry Disease Competitive Landscape Product Name Fabrazyme® Replagal® Galafold® Parent Company Sanofi (Genzyme) Shire (acquired by Takeda) Amicus Mechanism ERT ERT Pharmacological chaperone Indication Fabrazyme is indicated for use in patients with Replagal is indicated for long-term enzyme GALAFOLD
Slide 10: Pegunigalsidase Alfa is Designed For (MOA)
Pegunigalsidase Alfa is Designed For (MOA) ▪ Pegunigalsidase is a PEGylated enzyme designed to be potentially superior to the currently approved ERTs ▪ Covalent linked via short 2Kd PEG, stable enzyme with extended circulatory and tissue half-life and enzyme ▪ Continuous coverage/presence of enzyme
Slide 11: Potential Advantages of pegunigalsidase alfa (PRX-102)
Potential Advantages of pegunigalsidase alfa (PRX-102) PEGUNIGALSIDASE ALFA • Delivery of active enzyme into the target organ. • Reduction of globotriaosylceramide (Gb3) burden in kidney biopsies which demonstrates potential efficacy based on proven Mechanism of Action. • Clinical improvement measur
Slide 12: Phase I/II Study - Development Rationale
Phase I/II Study - Development Rationale High levels of active available enzyme → 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 weeks
Slide 13: Phase I/II Study: Reduction of Fabry Biomarkers
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 14: 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 Interim results follow up 78 Randomized Double Blind expected H1 2021 (basis 100% Enrolled Head-to-Head vs. for EMA MAA Submission) Fabrazyme® 24 mos. 1mg / kg 2 weeks Open
Slide 15: Clinical Program Overview
Clinical Program Overview Pegunigalsidase Alfa PRX-102 Mechanism of Action ➢ Delivered to lysosome to reduce accumulated substrate ➢ Increased exposure ➢ Low Immunogenicity Phase I/II ➢ Safety ➢ Reduction of Gb3 & Lyso Gb3 ❖ Safety ❖ Safety ❖ Safety ❖ eGFR slope improvement ❖ Potential Superiority ❖
Slide 16: Protalix Recent and Upcoming Expected Milestones
Protalix Recent and Upcoming Expected Milestones Q2 2020 Q1 2021 H1 2021 Top-Line results of Equity Financing gross Interim results of BRIDGE proceeds of $40.2M BALANCE for EMA MAA submission BLA submission under Q4 2020 Partnership with accelerated approval Final results (CSR) of SarcoMed USA to An
Slide 17: 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 18: Alidornase alfa (PRX-110): Actin Inhibition Resistant DNase
Alidornase alfa (PRX-110): Actin Inhibition Resistant DNase OVERVIEW POSITIONING • Alidornase alfa is a plant cell expressed recombinant human DNase I • Biologically active and safe in humans, alidornase alfa is being chemically modified to resist inhibition by actin, thus enhancing developed for ot
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, generall
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 half-life in the circulation for the treatment of NETs related diseases Overview • Neutrophil extracellular traps (NETs) are web-
Slide 21: Well Capitalized with Strong Institutional & Banking Support
Well Capitalized with Strong Institutional & Banking Support CASH REVENUE $38.5M (preliminary 4Q’20) $12M in revenue (3Q’20) FINANCING NET BURN RATE Recent successful financing for gross proceeds of ~$40M $6M/Q, declining as patients shift over to extension study (1Q’21) UBS O’Connor CASH RUNWAY DEB
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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