Kidney Week

Abstract: PO0562

Switching from Agalsidase Alfa to Pegunigalsidase Alfa for Treating Fabry Disease: One Year of Treatment Data from Bridge, a Phase 3 Open-Label Study

Session Information

• CKD Clinical, Outcomes, and Trials - 2
  October 22, 2020 | Location: On-Demand
  Abstract Time: 10:00 AM - 12:00 PM

Category: CKD (Non-Dialysis)

• 2102 CKD (Non-Dialysis): Clinical, Outcomes, and Trials

Authors

• Tøndel, Camilla, Haukeland University Hospital, Bergen, Norway

Camilla Tøndel,

• Linhart, Ales, Charles University, Praha, Czechia

Ales Linhart,

• Dostálová, Gabriela, Charles University, Praha, Czechia

Gabriela Dostálová,

• Nicholls, Kathleen M., The Royal Melbourne Hospital, Melbourne, Victoria, Australia

Kathleen M. Nicholls,

• West, Michael L., Dalhousie University, Halifax, Nova Scotia, Canada

Michael L. West,

• Jovanovic, Ana, Salford Royal, Dept of Inherited Metabolic Disease, Salford, United Kingdom

Ana Jovanovic,

• Giraldo, Pilar, Hospital de Dia Quiron, Zaragoza, Spain

Pilar Giraldo,

• Vujkovac, Bojan, General Hospital Slovenj Gradec, Slovenj Gradec, Slovenia

Bojan Vujkovac,

• Almon, Einat, Protalix Biotherapeutics, Carmiel, Northern, Israel

Einat Almon,

• Alon, Sari, Protalix Biotherapeutics, Carmiel, Northern, Israel

Sari Alon,

• Szlaifer, Mali, Protalix Biotherapeutics, Carmiel, Northern, Israel

Mali Szlaifer,

• Chertkoff, Raul, Protalix Biotherapeutics, Carmiel, Northern, Israel

Raul Chertkoff,

• Hughes, Derralynn, LSDU, Institute of Immunity and Transplantation, Royal Free London NHS Foundation Trust, London, United Kingdom

Derralynn Hughes,

Background

Pegunigalsidase-alfa is a novel, PEGylated α-Galactosidase-A enzyme in development for the treatment of Fabry disease (FD).

Methods

Bridge (PB-102-F30, NCT03018730) is a phase III, open-label, switch-over study, designed to assess the safety and efficacy of pegunigalsidase-alfa (1 mg/Kg EOW) in adult FD patients previously treated with agalsidase alfa for at least 2 years.

Results

This is an interim report of 12-months on-treatment data generated from the first 16 patients (9 males and 7 females) out of the 22 adult patients enrolled. Baseline characteristics: age 24-60 years, the mean estimated Glomerular Filtration Rate (eGFR) 75.45 in males and 85.78 mL/min/1.73m² in females, annualized eGFR slope was -5.04 and -5.18 mL/min/1.73m²/year, respectively, mean residual leucocytes enzymatic activity 5.9% of lab normal mean in males and 27.9% in females, and plasma lyso-Gb3 53.6 and 13.8 nM, respectively. After one year the mean annualized eGFR slope improved from -5.10 mL/min/1.73m²/year while on agalsidase alfa, to -0.23 mL/min/1.73m²/year on pegunigalsidase-alfa. According to Wanner et al. 2018, FD patients with eGFR slope between ≥ -5 and < -3 mL/min/1.73 m²/year are defined as kidney disease progressing and with eGFR slope < -5 mL/min/1.73 m²/year are defined fast progressing. The therapeutic goal is to reach eGFR slope ≥ -3 mL/min/1.73 m²/year for the progressing, and ≥ -5 mL/min/1.73m²/year or more than 50% decrease in progression for the fast progressing. In this interim analysis, 100% of the progressing patients and 66.7% in the fast progressing group achieved the proposed therapeutic goals after switching to pegunigalsidase-alfa. The switch to pegunigalsidase-alfa was safe and well-tolerated. The majority of the patients who completed the study rolled over to a long-term extension study, continuing receiving pegunigalsidase-alfa.

Conclusion

These results suggest a potential benefit of pegunigalsidase-alfa on renal function for FD patients currently treated with agalsidase alfa, to be confirmed by long-term data.

Funding

• Commercial Support –