Protalix BioTherapeutics, Inc. (de) · AMEX:PLX
Protalix BioTherapeutics, Inc. (de) — Investor Presentation (December 2022)
26 slides from Protalix BioTherapeutics, Inc. (de)'s investor presentation, December 2022
Slide 1: Protalix Biotherapeutics Corporate Presentation
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION December 2022
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 of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are subject
Slide 3: Investment Highlights
Investment Highlights Plant cell expressed recombinant proteins with improved therapeutic profiles Revenue Generating, FDA-Approved Drug Clinically-Validated Platform FDA approved, commercially marketed drug for Gaucher disease. Proprietary ProCellEx ® platform for recombinant protein expression Ele
Slide 4: Product Pipeline
Product Pipeline Recombinant proteins designed to have potentially improved therapeutic profiles that target unmet medical needs and established pharmaceutical markets Discovery and Preclinical Phase I Phase II Phase III Marketing Application Pegunigalsidase alfa (PRX-102) Fabry Disease Uricase (PRX
Slide 5: ProCellEx: Differentiated Protein Plant Cell Expression Platform
ProCellEx®: Protalix’s Differentiated Protein Plant Cell Expression Platform Unique Genetic Engineering Tools Optimized for Complexity Generates improved tobacco plant cell lines expressing plant unique expression cassettes designed Ability to express proteins that are difficult to express in to pro
Slide 6: From Concept to Market
From Concept to Market Selecting the Gene of interest Protein drug Pilot production with Product development Commercial target protein- engineering enhancement ProCellEx® platform production drug candidate modifications Plant cells genetically Improving Purification and Clinical development Scaling
Slide 7: ProCellEx Platform: Proprietary Protein Plant Cell Expression System
ProCellEx® Platform: Proprietary Protein Plant Cell Expression System Unique capabilities of tailored genetic engineering and protein engineering tools for customized pre/post production modifications, with capacity for commercial scale-up Agrobacterium mediated cell DEVELOPMENT OF transformation us
Slide 8: ProCellEx Plant Cell Production: Proven Advantages Over Other Technologies
ProCellEx® Plant Cell Production: Proven Advantages Over Other Cell- Based Production Technologies Flexible polyethylene disposable bioreactors optimized for plant cell cultures Large-Scale Plant Cell Production Advantages • Rapid product roll-out and development • No risk of viral contamination fro
Slide 9: Gaucher Disease
Gaucher Disease ProCellEx® platform validation in lysosomal storage disorder Rare autosomal Glucocerebrosidase (GCD) Gaucher disease Type 1 Standard of care is enzyme recessive disorder enzyme deficiency Symptoms replacement therapy (ERT) Occurs in approximately Leads to accumulation of Possible sym
Slide 10: Platform Validation: Elelyso for Gaucher Disease
Platform Validation: Elelyso® for Gaucher Disease First plant cell derived recombinant protein approved by the FDA Validation of the ProCellEx® platform Elelyso provides a consistent (and growing) revenue stream for Protalix while validating the ProCellEx platform technology and demonstrating Elelys
Slide 11: Fabry Disease
Fabry Disease Rare X-linked α-galactosidase-A Multisystemic Disease Standard of care is ERT recessive disease enzyme deficiency LSD occurring in Leads to accumulation of the Progressive disease that can Patients are injected with approximately 1 in 40,000 fatty substance lead to renal failure, recom
Slide 12: Pegunigalsidase Alfa Has Significant Potential in Fabry Disease
Pegunigalsidase Alfa Has Significant Potential in Fabry Disease Patients typically require regular, long-term treatment regimen to replace Prevalence: deficient enzyme 1 in 40,000 Patients who develop anti-drug antibodies or experience infusion-related reactions to current ERT could benefit from an
Slide 13: 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 (agalsidase beta) is indicated for Replagal (agalsidase alpha) is indicated for long- Galafold is an
Slide 14: Pegunigalsidase Alfa: Chemically Modified, Plant Cell Derived, PEGylated Homodimer
Pegunigalsidase Alfa: Chemically Modified, Plant Cell Derived, PEGylated, Covalently Linked Homodimer • Pegunigalsidase alfa is a PEGylated enzyme designed to potentially have lower immunogenicity and an improved safety profile • Covalent linked via short 2kD PEG having two reactive ends results in
Slide 15: Clinical Development Rationale Informed by Phase I/II Study
Clinical Development Rationale Informed by Phase I/II Study High levels of active available enzyme potentially improved clinical benefit Results of the Phase I/II Clinical Trial: Demonstrated stability throughout 2-week infusion interval Concentration Half-Life (ng/mL) Plasma Drug Concentration vs
Slide 16: Pegunigalsidase Alfa: Robust Completed Clinical Development Program
Pegunigalsidase Alfa: Robust Completed Clinical Development Program Hundreds of patient years exposure to the treatment Phase I/II Study Phase III BRIDGE Study Phase III BRIGHT Study Phase III BALANCE Study n=18, 3+ 9 months n=22, 12 months n=30, 12 months n=78, 24 months Head-to-head, randomized, a
Slide 17: Summary of Clinical Activity Data from Phase III Clinical Program
Summary of Clinical Activity Data from Phase III Clinical Program Program demonstrates clinical activity of pegunigalsidase alfa Clinical trial population (n=22) Clinical trial population (n=30) Clinical trial population (n=78) randomized 2:1; PRX-102:agalsidase Substantial improvements in plasma ly
Slide 18: Summary of Safety Data from Phase III Clinical Program
Summary of Safety Data from Phase III Clinical Program All trials to date show favorable tolerability and immunogenicity profiles Patient population (n=22) Patient population (n=30) Patient population (n=78) Favorable tolerability profile Favorable tolerability profile Favorable tolerability profile
Slide 19: Ongoing Extension Clinical Studies and Expanded Access
Ongoing Extension Clinical Studies and Expanded Access NCT03566017 PB-102-F60: Open-Label Extension Study – 1 mg/kg Every Two Weeks Phase I/II 10 completed 10 enrolled 97 enrolled > 20 completed > 18 enrolled > (90 treated) 72 completed 69 enrolled NCT03614234 PB-102-F51: Open-label Extension Study
Slide 20: Clinical Program Supports Regulatory Submissions
Robust Clinical Development Program Supports Regulatory Submissions Worldwide, multicenter, program Collaboration with TOP Fabry disease KOLs and patient groups Regulatory Phase I/II Applications Anticipated Approvals 1H 2023 2018 2020 2021 2022 2022 Phase I/II Phase III Phase III Phase III Regulato
Slide 21: Recent and Upcoming Expected Milestones
Protalix Recent and Upcoming Expected Milestones Q1 Q3 Q1 H2 2021 2021 2022 2022 Announced positive Top- Type A meeting with EMA MAA submission BLA Resubmission Line results for BRIGHT FDA following CRL in on Feb 23, 2021 2Q21 TODAY Q1 Q2 Q4 Q2 Q1/Q2 2021 2021 2021 2022 2023 FDA issued a CRL for Las
Slide 22: Committed Financial and Commercial Partner Chiesi Farmaceutici
Committed Financial and Commercial Partner Global Partnership with Chiesi Farmaceutici S.p.A. Up to $1 billion in potential milestone payments • International research-focused pharmaceuticals and healthcare group with ~$3B in revenue • Operating in 30 countries with over 6,000 Tiered royalties of 15
Slide 23: Growing Early Stage Pipeline
Growing Early Stage Pipeline Mechanism of Indication Status Upcoming Milestones Action Preclinical studies show: Stable PK profile Recombinant uricase Long half-life Uricase Severe Gout enzyme to lower uric Low immunogenicity Initiate phase I study 1Q’23 (PRX-115) acid levels High specific activity
Slide 24: Well Capitalized to Mature the Potential of PRX-102
Well Capitalized to Mature the Potential of PRX-102 CASH REVENUE STOCK INFORMATION $20.8M (3Q’22) $21.2M in revenue (YTD 2022) NYSE American PLX Tel Aviv Stock Exchange PLX FINANCING NET BURN RATE Successfully completed a Note Outstanding Shares 50,665,5981 $5.7 M/Q Exchange in 3Q’21 to effectively
Slide 25: Experienced Leadership Team
Experienced Leadership Team DROR BASHAN EINAT BRILL ALMON, PH.D. EYAL RUBIN YARON NAOS YAEL HAYON, PH.D. President & CEO Senior Advisor (former SVP, Chief SVP & CFO SVP of Operations VP of R&D Development Officer) Mr. Bashan has served as our Dr. Almon joined Protalix in Mr. Rubin has served as our
Slide 26: Accomplished Board of Directors
Accomplished Board of Directors ZEEV BRONFELD DROR BASHAN POL F. BOUDES, M.D. GWEN A. MELINCOFF Chairman President & CEO, Director Director Director AHARON SCHWARTZ, PH.D. AMOS BAR SHALEV SHMUEL “MULI” BEN ZVI, PH.D. Director Director Director Corporate Presentation I December 2022
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