Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Q1 2025 Earnings Presentation
30 slides from Protalix Biotherapeutics, Inc. (de)'s earnings presentation, May 9, 2025
Slide 1: Protalix Biotherapeutics Corporate Presentation
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION J ua ny e 2 20 02 25 4 M 1
Slide 2: Disclaimers
Disclaimers Third-Party Information This presentation also contains estimates and other data made by independent parties and Protalix relating to market size and growth and other data related to the industry in which Protalix operates. This data involves a number of assumptions and limitations, and
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 Established commercial foundation fuels further expansion into the Rare Disease space Elelyso® (alfataliglicerase in Brazil): FDA approved, commercially marketed drug for Gaucher disease Commercial Portfolio Elfabrio® (pegunigalsidase alfa) FDA/European Commission1 approved for
Slide 5: Product Pipeline
Product Pipeline Recombinant proteins designed to potentially target unmet medical needs in established pharmaceutical markets Discovery and Preclinical Phase I Phase II Phase III Marketing Application Commercial Portfolio Gaucher Disease Approved in 23 markets Fabry Disease Approved (US and EU) Dev
Slide 6: Commercial Portfolio
PROTALIXX Biotherapeutics Commercial Portfolio pegunigalsidase alfa {taliglucerase alfa) for injection #200 units/vial | Wuse after dilution
Slide 7: Elelyso for Gaucher Disease
for Gaucher Disease First plant cell derived recombinant protein approved by the FDA Product Elelyso (alfataliglicerase in Brazil) is a proprietary, recombinant form of GCD for long-term treatment of patients with a confirmed diagnosis of type 1 Gaucher disease Gaucher Disease Expressed through our
Slide 8: Elfabrio for Fabry Disease
for Fabry Disease Second plant cell derived recombinant protein approved by the FDA Product Elfabrio (pegunigalsidase alfa): Chemically Modified, Plant Cell Derived, PEGylated, Covalently Fabry Disease Linked Homodimer Approved for marketing by the EC, FDA and others Rare X-linked disease: affecting
Slide 9: Fabry Disease Competitive Landscape
Fabry Disease Competitive Landscape ~$2.2B market (2024) expected to reach over $3.1B (2030), CAGR of 6.6% Product Name Fabrazyme® Replagal® Galafold® Elfabrio® Parent Company Mechanism ERT ERT Pharmacological chaperone ERT Adults & pediatric patients 2+ years Accelerated approval in adults (US). Ap
Slide 10: Global Partnership with Chiesi
Global Partnership with Chiesi Committed Global Partner • International research-focused biopharmaceutical group with sales in excess of €3.4B in 2024 (reflecting 13% growth year-on-year) • Operating in over 30 countries with over 7,000 employees • Strong sales and marketing partner poised to maximi
Slide 11: Development Portfolio
Development Portfolio PRX-115 in Development for Uncontrolled Gout 11
Slide 12: Gout
Gout Most Common Accumulation of Excess Systemic Currently Available Inflammatory Arthritis Uric Acid Disease Therapies Gout affects approximately 15 Hyperuricemia leads to Symptoms: severe acute pain, First-line xanthine oxidase million people in the US accumulation of urate crystals inflammation,
Slide 13: Currently Available Urate-Lowering Therapies
Currently Available Urate-Lowering Therapies Unmet needs remain in uncontrolled gout patients Normal Cell Breakdown Reabsorption or renal excretion Purines Hypoxanthine Xanthine Uric Acid Xanthine oxidase Uric Acid Buildup Diet (Hyperuricemia) Gout Uricase Allantoin Xanthine Oxidase Inhibitors (XOIs
Slide 14: PRX-115: Significant Potential in Uncontrolled Gout
PRX-115: Significant Potential in Uncontrolled Gout $5.6B Uncontrolled Gout (Unmet Need) Global Gout Market1 An estimated 5% of the gout population is considered to have chronic refractory disease Expected CAGR of 6.4% from 2022–2029 Expected CAGR of Uncontrolled gout patients typically do not reach
Slide 15: PRX-115: Chemically Modified, Plant Cell Derived, PEGylated Homotetramer
PRX-115: Chemically Modified, Plant Cell Derived, PEGylated, Covalently Linked Homotetramer • PRX-115 is a PEGylated enzyme designed to potentially have lower immunogenicity by masking immunogenic epitopes and an improved safety profile • PRX-115 is a plant cell-based recombinant Candida Utilis Uric
Slide 16: PRX-115 Phase I Single Ascending Dose Study Design
PRX-115 Phase I Single Ascending Dose Study Design Cohort 8 Cohort 7 Study Scheme Cohort 6 Primary Endpoint: safety and tolerability Cohort 5 Secondary Endpoints: PK, PD (uric acid levels) Cohort 4 Dose escalation meeting by blinded Safety Monitoring Committee (SMC) following completion of each coho
Slide 17: PRX-115 Phase I Single Ascending Dose Study Summary
PRX-115 Phase I Single Ascending Dose Study Summary First-in-Human trial enrolled 64 subjects with elevated uric acid levels across 8 dose cohorts Safety and Immunogenicity Phase 2 Planning In Progress Favorable tolerability profile 12 of 48 PRX-115-treated subjects experienced a study drug-related
Slide 18: PRX-115 Summary
PRX-115 Summary Recombinant PEGylated Uricase Enzyme for Potential Treatment of Uncontrolled Gout Addressable Market Asset Overview • Recombinant PEGylated uricase enzyme produced via Approximately 15 million US gout patients, of which ~5% ProCellEx® plant cell-based expression system considered to
Slide 19: Growth Strategy
Growth Strategy 19
Slide 20: Research to Fuel Next Stage of Growth
Research to Fuel Next Stage of Growth Goal: Within 3 years, 4-5 discovery to Phase II programs in the pipeline ProCellEx® platform Research Priority Indications Leveraging existing platforms Rare disease focus Strategy 20
Slide 21: Expansion of ProCellEx Platform for Drug Delivery
Expansion of ProCellEx® Platform for Drug Delivery Exploring platform expansion to include drug delivery modalities for optimized delivery of therapeutic proteins • Unmet need: efficient and targeted delivery of therapeutic proteins • Potential applications: • Package recombinant proteins in deliver
Slide 22: Focus on High Unmet Needs in Rare Disease Space
Focus on High Unmet Needs in Rare Disease Space Therapeutic Area Strategy: Focus on Rare Disease Space Rare Genetic and • Protalix conducted systematic analysis to identify potential key areas of Non-Genetic focus in rare disease space • Both genetic and non-genetic opportunities Extended • Potentia
Slide 23: Well Capitalized to Advance Protalix to Next Phase
Well Capitalized to Advance Protalix to Next Phase CASH STREAMS FROM $10.0 in Q12025 revenue from selling goods, up 17% over Q1 2024 STRONG PARTNERSHIPS REVENUE $53M in revenue (FY 2024) $34.7M (at March 31, 2025); approaching cash flow postive, CASH & CASH RUNWAY sufficient to support ongoing opera
Slide 24: Experienced Leadership Team
Experienced Leadership Team DROR BASHAN SHOSHI TESSLER, PH.D. EYAL RUBIN YARON NAOS ORI KALID, PH.D. President & CEO VP, Clinical Dev & Regulatory Affairs SVP & CFO SVP of Operations VP of R&D Mr. Bashan has served as our Dr. Tessler joined Protalix in October Mr. Rubin has served as our SVP and Mr.
Slide 25: Accomplished Board of Directors
Accomplished Board of Directors ELIOT FORSTER, PH.D. DROR BASHAN POL F. BOUDES, M.D. GWEN A. MELINCOFF Chairman President & CEO, Director Director Director AHARON SCHWARTZ, AMOS BAR SHALEV SHMUEL “MULI” BEN ZVI, PH.D. PH.D. Director Director Director 25
Slide 26: Appendix
Appendix 26
Slide 27: Participant Demographics and Baseline Characteristics
Participant Demographics and Baseline Characteristics Parameter Statistic Pooled PRX-115 Pooled Placebo Overall n 48 16 64 Age (years) Mean 35.9 33.4 35.3 SD 12.3 10.8 11.9 Sex n(%) Female 16(33.3) 5(31.3) 21(32.8) Male 32(66.7) 11(68.8) 43(67.2) Race n(%) American Indian or Alaska Native 0 0 0 Asia
Slide 28: Overall Summary of Adverse Events
Overall Summary of Adverse Events* Pooled Pooled Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Cohort 6 Cohort 7 Cohort 8 PRX-115 Placebo N 6 6 6 6 6 6 6 6 48 16 TEAE n(%) 5(83.3) 6(100.0) 5(83.3) 3(50.0) 6(100.0) 5(83.3) 3(50.0) 4(66.7) 37(77.1) 13(81.3) Related TEAE n(%) 1(16.7) 5(83.3) 3 (50.0) 1(
Slide 29: PRX-115 Pharmacokinetic Profile
PRX-115 Pharmacokinetic Profile Dose-dependent increase in PRX-115 exposure with single administration PRX-115 Mean Plasma Concentrations Throughout 85-day Follow-up Period (n=48) 14 12 Plasma PRX-115 (µg/mL) Cohort 1 10 Cohort 2 Detectable exposures 8 Cohort 3 were observed in plasma Cohort 4 for u
Slide 30: PRX-115 Pharmacodynamic Results
PRX-115 Pharmacodynamic Results Rapid reduction of plasma uric acid below 6 mg/dL following single administration of PRX-115 Uric Acid Mean Plasma Concentrations Throughout 85-day Follow-up Period (n=64) 10.0 9.0 Placebo Plasma Urate (mg/dL) 8.0 Cohort 1 7.0 Effect of PRX-115 on plasma uric Cohort 2
Protalix BioTherapeutics, Inc. (DE) — Q1 2025 Earnings Presentation on 6ix