Publications

ProCellEx® Platform

Plant specific N-glycans do not have proven adverse effects in humans.

Shaaltiel Y, Tekoah Y.
Nat Biotechnol. 2016 Jul(pp,.1-3)

Large-scale production of pharmaceutical proteins in plant cell culture-the Protalix experience.

Tekoah Y, Shulman A, Kizhner T, Ruderfer I, Fux L, Nataf Y, Bartfeld D, Ariel T, Gingis-Velitski S, Hanania U, Shaaltiel Y.
Plant Biotechnol J. 2015(pp.1-10)

Establishment of a tobacco BY2 cell line devoid of plant-specific xylose and fucose as a platform for the production of biotherapeutic proteins.

Hanania U, Ariel T, Tekoah Y, Fux L, Sheva M, Gubbay Y, Weiss M, Oz D, Azulay Y, Turbovski A, Forster Y, Shaaltiel Y.
Plant Biotechnol J. 2017(pp.1-10)

Preclinical and first-in-human evaluation of PRX-105, a PEGylated, plant-derived, recombinant human acetylcholinesterase-R.

Atsmon J, Brill-Almon E, Nadri-Shay C, Chertkoff R, Alon S, Shaikevich D, Volokhov I, Haim KY, Bartfeld D, Shulman A, Ruderfer I, Ben-Moshe T, Shilovitzky O, Soreq H, Shaaltiel Y.
Toxicol Appl Pharmacol. 2015(pp.1-10)

Plant-based oral delivery of β-glucocerebrosidase as an enzyme replacement therapy for Gaucher’s disease.

Shaaltiel Y, Gingis-Velitski S, Tzaban S, Fiks N, Tekoah Y,
Aviezer D.
Plant Biotechnol J. 2015(pp.1-8)

PRESENTATIONS OF PRX-115 AT INTERNATIONAL CONFERENCES

Prolonged Plasma Urate-Lowering after a Single Intravenous Administration of PRX-115, aNovel PEGylated Uricase, in Participants with Elevated Urate Levels

Pegunigalsidase alfa

Characterization of a chemically modified plant cell culture expressed human alphagalactosidase-a enzyme for treatment of Fabry disease.

Kizhner T, Azulay Y, Hainrichson M, et al.
Mol Genet Metab. 2015. 114(2):259-267.

Development and Analytical Characterization of Pegunigalsidase Alfa, a Chemically Cross-Linked Plant Recombinant Human α‑Galactosidase‑A for Treatment of Fabry Disease.

Ruderfer R., Shulman A., Kizhner T., AzulayY., Nataf Y., Tekoah Y., and Shaaltiel Y.
Bioconjug Chem. 2018. May 16;29(5):1630-1639.

Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.

Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D.
J Inherit Metab Dis. 2019. May;42(3):534-544.

Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.

Wallace EL, Goker-Alpan O, Wilcox WR, Holida M, Bernat J, Longo N, Linhart A, Hughes DA, Hopkin RJ, Tøndel C,
Langeveld M, Giraldo P, Pisani A, Germain DP, Mehta A,
Deegan PB, Molnar MJ, Ortiz D, Jovanovic A, Muriello M, Barshop BA, Kimonis V, Vujkovac B, Nowak A, Geberhiwot T, Kantola I, Knoll J, Waldek S, Nedd K, Karaa A, Brill-Almon E,
Alon S, Chertkoff R, Rocco R, Sakov A, Warnock DG.
J Med Genet. 2023. Nov 8:jmg-2023-109445.

Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study.

Linhart A, Dostálová G, Nicholls K, West ML, Tøndel C,
Jovanovic A, Giraldo P, Vujkovac B, Geberhiwot T, Brill-Almon E, Alon S, Chertkoff R, Rocco R, Hughes D.
Orphanet J Rare Dis. 2023. Oct 21;18(1):332.

A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies

Myrl Holida, Aleš Linhart, Antonio Pisani, Nicola Longo, François Eyskens, Ozlem Goker-Alpan, Eric Wallace, Patrick Deegan, Camilla Tøndel, Ulla Feldt-Rasmussen, Derralynn Hughes, Anat Sakov, Rossana Rocco, Einat Brill Almon, Sari Alon, Raul Chertkoff, David G. Warnock, Stephen Waldek, William R. Wilcox, John A. Bernat

Presentations of pegunigalsidase alfa at International Conferences

First results of a head-to-head trial of pegunigalsidase alfa vs. agalsidase beta in Fabry disease: 2-year results of the phase 3 randomized, double-blind, BALANCE study.

Oral presentation by Eric Wallace, M.D.
WORLDSymposium: 19th Annual Research Meeting | Orlando, FL, US | February 22–26, 2023

Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study.

Oral presentation by John Bernat, M.D., Ph.D.
WORLDSymposium: 19th Annual Research Meeting | Orlando, FL, US | February 22–26, 2023

Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study.

Poster presentation by John Bernat, M.D., Ph.D.
WORLDSymposium: 19th Annual Research Meeting | Orlando, FL, US | February 22–26, 2023

First results of a head-to-head trial of pegunigalsidase alfa vs. agalsidase beta in Fabry disease: 2-year results of the phase 3 randomized, double-blind, BALANCE study.

Poster presentation by Eric Wallace, M.D.
WORLDSymposium: 19th Annual Research Meeting | Orlando, FL, US | February 22–26, 2023

Long-Term Safety And Efficacy Of Pegunigalsidase Alfa - A Multicenter Extension Study In Adult Patients With Fabry Disease

Abstract
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Tolerability and Infusion Duration of Pegunigalsidase Alfa in Patients with Fabry Disease: Data from 5 Completed Clinical Trials

Abstract
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Switching from Agalsidase Alfa to Pegunigalsidase Alfa to Treat Patients with Fabry Disease: 1 Year of Treatment Data from BRIDGE, a Phase 3 Open-label Study

Poster
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Safety and Efficacy of Pegunigalsidase Alfa Administered Every 4 Weeks in Patients with Fabry Disease: Results from the Phase 3, Open-label, BRIGHT Study

Poster
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Safety and Efficacy of Pegunigalsidase Alfa Administered Every 4 Weeks in Patients with Fabry Disease: Results from the Phase 3, Open-label, BRIGHT Study

Abstract
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Tolerability and Infusion Duration of Pegunigalsidase Alfa in Patients with Fabry Disease: Data from 5 Completed Clinical Trials

Poster
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Safety and Efficacy of Pegunigalsidase Alfa vs Agalsidase Beta on Renal Function in Fabry Disease: 24-Month Results from the Phase III Randomized, Double-blind, BALANCE Study

Abstract
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Safety and Efficacy of Pegunigalsidase Alfa vs Agalsidase Beta on Renal Function in Fabry Disease: 24-Month Results from the Phase III Randomized, Double-blind, BALANCE Study

Poster
The 7th Update On Fabry Disease | Würzburg, Germany | May 29-31, 2022

Lower rate of infusion-related reactions in patients with Fabry disease after switching from agalsidase beta to pegunigalsidase alfa