Protalix Biotherapeutics, Inc. (de) · AMEX:PLX
Protalix Biotherapeutics, Inc. (de) — Investor Presentation (December 2022)
18 slides from Protalix Biotherapeutics, Inc. (de)'s investor presentation, December 2022
Slide 1: Protalix Biotherapeutics Corporate Presentation
PROTALIX BIOTHERAPEUTICS CORPORATE PRESENTATION December 2022 KOL Presentation I 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: Pegunigalsidase alfa (PRX-102) for Fabry Disease
KOL Event December 2022 Pegunigalsidase alfa (PRX-102) for Fabry Disease Myrl D. Holida, PA-C University of Iowa Health Care, Iowa City, Iowa 4
Slide 5: Disclaimer
Disclaimer Myrl Holida has been or is currently involved in clinical trials with Sanofi-Genzyme, Sangamo, AVROBIO, Amicus Therapeutics, Takeda, Protalix Biotherapeutics, Chiesi and Idorsia. No direct funding is received for these trials as they are institution directed. He has also been a consultant
Slide 6: Myrl D. Holida, PA-C
Myrl D. Holida is a Physician Assistant with 35 years of experience in patient care, initially treating Pediatric Bone Marrow Transplant (BMT) patients with end stage malignancies and Lysosomal Storage Disorder patients in a clinical trial setting. Myrl’s experience includes many levels of research
Slide 7: Fabry Disease, a Multisystemic Disease
Fabry Disease, a Multisystemic Disease Fabry Disease Fabry disease (FD) is a progressive, X-linked inherited disorder of glycosphingolipid metabolism due to deficient or absent lysosomal a-galactosidase-A activity and intracellular accumulation of glycosphingolipids (especially globotriaosylceramide
Slide 8: Pegunigalsidase Alfa
Pegunigalsidase Alfa Chemically modified, plant cell derived, PEGylated, covalently linked homodimer Pegunigalsidase Alfa Pegunigalsidase alfa is a PEGylated enzyme designed to potentially have lower immunogenicity and an improved safety profile Covalent linked via short 2000 Da PEG having two r
Slide 9: Pegunigalsidase Alfa: Robust Clinical Development Program
Pegunigalsidase Alfa: Robust Clinical Development Program Overall, >140 patients received at least 1 infusion of pegunigalsidase alfa in the clinical program Hundreds of patient-years exposure to the treatment (~300 patient-years at 1 mg/kg E2W; ~100 patient-years at 2 mg/kg E4W) Pegunigalsidase Alf
Slide 10: Robust Clinical Development Program Supports Regulatory Submissions in Various Regions
Robust Clinical Development Program Supports Regulatory Submissions in Various Regions Worldwide, multicenter, program Collaboration with TOP Fabry disease KOLs and patient groups Phase I/II Regulatory Anticipated Year of Completion: Approvals 1H 2023 2018 2020 2021 2022 2022 Phase I/II Phase III Ph
Slide 11: Phase I/II study with ERT Naïve Fabry disease patients
Phase I/II study with ERT Naïve Fabry disease patients Up to 6 years of pegunigalsidase alfa treatment; include treatment under long-term extension study Pharmacokinetics Pharmacodynamics Clinical Activity Data Pharmacokinetics Profiles Mean (±SE) Absolute eGFR Values Over Time Reduction of Gb3 in t
Slide 12: BRIDGE Phase III Trial Summary
BRIDGE Phase III Trial Summary Switch from agalsidase alfa to pegunigalsidase alfa Clinical Activity Data Safety 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 hype
Slide 13: BRIGHT Phase III Trial with Alternative Dosing Regimen - Summary
BRIGHT Phase III Trial with Alternative Dosing Regimen - Summary Pegunigalsidase Alfa Administered at 2 mg/kg Every 4 Weeks Pharmacokinetics Clinical Activity Data Safety Data Pegunigalsidase alfa concentration over time Stable eGFR throughout 1 year of Favorable tolerability profile: 100000 pegunig
Slide 14: BALANCE, a Phase III, Randomized, Double-blind, Active-Controlled Study
BALANCE, a Phase III, Randomized, Double-blind, Active-Controlled Study Assessed safety and efficacy of pegunigalsidase alfa vs. agalsidase beta in adult patients with Fabry disease with deteriorating renal function Study Design Study Assessments Efficacy Assessments: • The primary efficacy endpoint
Slide 15: BALANCE, a Phase III Trial – Efficacy Data Summary
BALANCE, a Phase III Trial – Efficacy Data Summary Assessed safety and efficacy of pegunigalsidase alfa vs. agalsidase beta in adult patients with Fabry disease with deteriorating renal function Efficacy Assessment: Evaluation of Renal Function Primary efficacy analysis of the difference in median o
Slide 16: BALANCE, a Phase III Trial – Safety Data Summary
BALANCE, a Phase III Trial – Safety Data Summary Assessed safety and efficacy of pegunigalsidase alfa vs. agalsidase beta in adult patients with Fabry disease with deteriorating renal function Immunogenicity Data Safety Data Rates of ADA+ patients and nAb+ patients over time (ITT analysis set) • Sim
Slide 17: BALANCE, a Phase III Trial Summary
BALANCE, a Phase III Trial Summary Assessed safety and efficacy of pegunigalsidase alfa vs. agalsidase beta in adult patients with Fabry disease with deteriorating renal function Summary • Pegunigalsidase alfa showed non-inferiority to agalsidase beta based on the median eGFR annualized slope, a key
Slide 18: Summary & Current Status of Pegunigalsidase Alfa Clinical Development Program
Summary & Current Status of Pegunigalsidase Alfa Clinical Development Program Ongoing Clinical Studies and Expanded Access Programs: • PB-102-F60 - Open label extension study with 1 mg/kg E2W: 90 active patients, some for >6 years • BALANCE (from both arms), BRIDGE and phase I/II trial completers wh
Protalix BioTherapeutics — Investor Presentation (December 2022) on 6ix