Protalix BioTherapeutics, Inc. (de) · AMEX:PLX
Protalix BioTherapeutics, Inc. (de) — Investor Presentation (May 2023)
40 slides from Protalix BioTherapeutics, Inc. (de)'s investor presentation, May 2023
Slide 1: Protalix Biotherapeutics Corporate Presentation
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION May 2023
Slide 2: Disclaimers
Disclaimers THE PURPOSE OF THIS PRESENTATION IS TO INTRODUCE THE RECIPIENT TO PROTALIX BIOTHERAPEUTICS, INC. (“PROTALIX”, “WE” OR “OUR”). THIS PRESENTATION IS FOR INFORMATIONAL PURPOSES ONLY AND DOES NOT CONSTITUTE AN OFFER TO SELL, OR A SOLICITATION OF AN OFFER TO PURCHASE, SECURITIES, NOR SHALL TH
Slide 3: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements that involve risks and uncertainties within the meaning of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Exchange Act. Forward-looking statements are neither histor
Slide 4: Investment Highlights
Investment Highlights A Strong Foundation To Further Expand Into The Rare Disease Space Two Approved Drugs Clinically-Validated Platform Elelyso® (alfataliglicerase in Brazil): FDA approved, commercially Proprietary ProCellEx® platform for recombinant protein expression cGMP marketed drug for Gauche
Slide 5: 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 ELFABRIO® Fabry Disease Approved (US and EU) (pegunigalsi
Slide 6: 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 7: 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 biological Purification and Clinical developme
Slide 8: 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 9: ProCellEx Plant Cell Production: Proven Advantages
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 10: Gaucher Disease
Gaucher Disease ProCellEx® platform validation in lysosomal storage disorders Rare autosomal Glucocerebrosidase (GCD) Gaucher disease Type 1 Standard of care is enzyme recessive disorder enzyme deficiency Symptoms replacement therapy (ERT) Prevalence ranging from Leads to accumulation of Possible sy
Slide 11: 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 12: 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 person fatty substance lead to renal failure, recombin
Slide 13: 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 to 60,000 males Patients who develop anti-drug antibodies or experience infusion-related reactions to current ERT could
Slide 14: 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 the Replagal (agalsidase alpha) is indicated for long- Galafold is
Slide 15: Pegunigalsidase Alfa: Chemically Modified, 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 16: 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 (ng/mL) Plasma Drug Concentration vs. Time Hal
Slide 17: Pegunigalsidase Alfa: 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 18: Summary of Clinical Activity Data from Phase III 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 19: Summary of Safety Data from Phase III 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 20: 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 > (88 treated)1 72 completed 69 enrolled NCT03614234 PB-102-F51: Open-label Extension Study
Slide 21: 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 Approved May 2023 2018 2020 2021 2022 2022 Phase I/II Phase III Phase III Phase III Regulatory n=18 n=22
Slide 22: European Commission Granted Marketing Authorization
European Commission Granted Marketing Authorization to pegunigalsidase alfa Pegunigalsidase alfa 1 mg/kg every two weeks is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase) Safety Pharmacodynamics Eff
Slide 23: Protalix Recent Milestones
Protalix Recent Milestones Q1 Q3 Q1 Q4 Q2 2021 2021 2022 2022 2023 EC approval granted Announced positive Top- Type A meeting with EMA MAA submission BLA Resubmission May 3, 2023 Line results for BRIGHT FDA following CRL in FDA approved May 9, 2023 on Feb 23, 2021 2Q21 TODAY Q1 Q2 Q4 Q2 Q1 2021 2021
Slide 24: Committed Financial and Commercial Partner Chiesi
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 25: Growing Early Stage Pipeline
Growing Early Stage Pipeline Indication Mechanism of Action Status Preclinical studies show: Stable PK profile Long half-life Recombinant uricase enzyme to Uricase Low immunogenicity Severe gout lower uric acid levels and reduce (PRX-115) High specific activity tophi Toxicity study initiated (1Q’22)
Slide 26: Well Capitalized to Advance Protalix to Next Phase
Well Capitalized to Advance Protalix to Next Phase CASH REVENUE $33.0M (1Q’23) $25.3M in revenue (FY2022) FINANCING NET BURN RATE Successfully completed a Note Exchange $5.6 M/Q in 3Q’21 to effectively extend maturity from 2021 to 2024 and lower principal CASH RUNWAY DEBT Cash Runway to 2Q’251 $28.7
Slide 27: Focus on Innovative Approaches in Rare Disease Space
Focus on Innovative Approaches in Rare Disease Space Specific Scientific Expertise for Protein Therapeutics In-House Discovery Pipeline based on Protein Capabilities Leveraging ProCellEx platform and PEGylation capabilities for highly innovative opportunities Reinforce protein capabilities Focus on
Slide 28: A Strong Foundation To Further Expand Into The Rare Disease Space
A Strong Foundation To Further Expand Into The Rare Disease Space 2 approved products: Gaucher and Fabry Rare Genetic Diseases Network & Expertise Strong Clinical and Reg. Expertise for Biologics Revenue Generating – Stream of revenues Strong Partnerships Validated Platforms: ProCellEx & PEGylation
Slide 29: 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 30: 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 May 2023 30
Slide 31: Appendix
Appendix Corporate Presentation I May 2023 31
Slide 32: Evolving Protalix: Addressing High Unmet Needs in the Rare Disease Space
Evolving Protalix: Addressing High Unmet Needs in the Rare Disease Space Leveraging track record of success into other rare diseases Strategy Vision Next Steps Protalix Now Initial Success May 2012: 2023: Reinforce Protein Discovery Building a significant pipeline Protalix’s 1st approved product Cap
Slide 33: Uricase (PRX-115) for Severe Gout
Uricase (PRX-115) for Severe Gout PRX-115 is an investigational, PEGylated enzyme expressed via ProCellEx® for intravenous (IV) administration of recombinant uricase being designed for the treatment of severe gout Overview • Gout is the most common form of chronic inflammatory disability, generally
Slide 34: Long Acting DNase I (PRX-119) for NETs-related diseases
Long Acting (LA) DNase I (PRX-119) for NETs-related diseases PRX-119 is an investigational, 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 (NE
Slide 35: Phase I/II Study
Phase I/II Study Substantial reduction & high correlation between two Fabry disease biomarkers: Gb3 and lyso-Gb3 Reduction of Gb3 in the kidney 10 Baseline 6 month Responder analysis: Mean score ± SE 8 Portion of patient achieved reduction in BLISS score 6 4 90 2 12/14 80 0 11/14 Male Female Overall
Slide 36: BRIDGE Phase III Trial Summary
BRIDGE Phase III Trial Summary Switch from agalsidase alfa to pegunigalsidase alfa Safety Data Efficacy Data Favorable tolerability and immunogenicity profile Most TEAEs were mild or moderate in severity, with all AEs being transient 2 patients (9.1%) withdrew from treatment due to hypersensitivity
Slide 37: BRIGHT Phase III Trial Summary
BRIGHT Phase III Trial Summary Pegunigalsidase Alfa Administered at 2 mg/kg Every 4 Weeks Safety Data Efficacy Data Stable Fabry disease progression, measured by eGFR slope and plasma lyso-Gb3, Favorable tolerability profile throughout pegunigalsidase alfa therapy • Most TEAEs were mild to moderate
Slide 38: BALANCE Phase III Trial Summary
BALANCE Phase III Trial Summary Demonstrated favorable tolerability, immunogenicity and activity profiles Safety Data Efficacy Data Favorable tolerability and immunogenicity profile The pre-specified non-inferiority margin was met and demonstrated that PRX-102 is statistically non-inferior to agalsi
Slide 39: BALANCE Study – Primary Efficacy Analysis
BALANCE Study – Primary Efficacy Analysis Evaluation of Renal Function (A) median eGFR values over time (B) primary efficacy analysis of the median difference in eGFR slope between baseline and 24 months Corporate Presentation I May 2023 39
Slide 40: BALANCE Study Results
BALANCE Study Results Treatment Emergent Adverse Events Pegunigalsidase alfa Agalsidase beta Overall Conclusions: n=52 n=25 N=77 ▪ Pegunigalsidase alfa showed non-inferiority to agalsidase beta based Any TEAE on the median eGFR annualized slope, a key measure of Fabry disease progression Events, n (
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