Protalix BioTherapeutics, Inc. (de) · AMEX:PLX
Protalix BioTherapeutics, Inc. (de) — Investor Presentation (March 2023)
35 slides from Protalix BioTherapeutics, Inc. (de)'s investor presentation, March 2023
Slide 1: Protalix Biotherapeutics Corporate Presentation
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION March 2023
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 cGM
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 biological Purification and Clinical developme
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 the Replagal (agalsidase alpha) is indicated for long- Galafold is
Slide 14: 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 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 (ng/mL) Plasma Drug Concentration vs. Time Hal
Slide 16: 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 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 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 2018 May 2023 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 Q4 Q2 2021 2021 2022 2022 2023 Announced positive Top- Type A meeting with EMA MAA submission BLA Resubmission EC Decision May 3, 2023 Line results for BRIGHT FDA following CRL in FDA PDUFA May 9, 2023 on Feb 23, 2021 2Q21 TODAY Q1 Q2 Q4 Q2 Q
Slide 22: 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 23: Growing Early Stage Pipeline
Growing Early Stage Pipeline Mechanism of Indication Status Upcoming Milestones Action Preclinical studies show: Stable PK profile Long half-life Recombinant uricase Uricase Low immunogenicity Topline phase I results Severe gout enzyme to lower uric acid (PRX-115) High specific activity anticipated
Slide 24: Well Capitalized to Mature the Potential of PRX-102
Well Capitalized to Mature the Potential of PRX-102 CASH REVENUE STOCK INFORMATION $22.2M (4Q’22) $25.3M in revenue (FY2022) NYSE American PLX Tel Aviv Stock Exchange PLX FINANCING NET BURN RATE Successfully completed a Note Exchange Outstanding Shares 57,353,4301 $5.6 M/Q in 3Q’21 to effectively ex
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 March 2023
Slide 27: Appendix
Appendix Corporate Presentation I March 2023 27
Slide 28: 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 29: 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, 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 extracellu
Slide 30: Phase I/II Study
Phase I/II Study Substantial reduction & high correlation between two Fabry disease biomarkers: Gb 3 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 Overal
Slide 31: 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 32: 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 33: 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 34: 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 March 2023 34
Slide 35: 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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