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)

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.

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

The Taliglucerase alfa Story

Taliglucerase alfa: An enzyme replacement therapy using plant cell expression technology.

Grabowski GA, Golembo M, Shaaltiel Y.
Mol Genet Metab 2014;112:1–8.

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 Oct;13(8):1199-208.

Immunogenicity of glycans on biotherapeutic drugs produced in plant expression systems-The taliglucerase alfa story.

Rup B, Alon S, Amit-Cohen BC, Brill Almon E, Chertkoff R, Tekoah Y, Rudd PM.
PLoS One. 2017 Oct 31;12(10):e0186211.

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 Sep;15(9):1120-1129.

Sequential Genome Editing and Induced Excision of the Transgene in N. tabacum BY2 Cells.

Sheva M, Hanania U, Ariel T, Turbovski A, Rathod VKR, Oz D, Tekoah Y, Shaaltiel Y.
Front Plant Sci. 2020 Nov 25;11:607174.

Taliglucerase alfa – clinical experience

Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease.

Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Elstein D,
Paz A, Brill-Almon E, Chertkoff R.
Blood Cells Mol Dis. 2015 Jan;54(1):9-16.

Long-term efficacy and safety results of taliglucerase alfa through 5 years in adult treatment-naïve patients with Gaucher disease.

Zimran A, Durán G, Giraldo P, Rosenbaum H, Giona F,
Petakov M, Terreros Muñoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R.
Blood Cells Mol Dis. 2019; Sep;78:14-21.

Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.

Zimran A, Brill-Almon E, Chertkoff R, Petakov M,
Blanco-Favela F, Muñoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D.
Blood. 2011 Nov 24;118(22):5767-73.

A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.

Pastores GM, Petakov M, Giraldo P, Rosenbaum H, Szer J, Deegan PB, Amato DJ, Mengel E, Tan ES, Chertkoff R,
Brill-Almon E, Zimran A.
Blood Cells Mol Dis. 2014 Dec;53(4):253-60.

Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naïve patients with Gaucher disease.

Zimran A, Durán G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Muñoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E.
Am J Hematol. 2016 Jul;91(7):656-60.