Protalix BioTherapeutics Inc. (NYSE: PLX) has reported topline results from the BALANCE pivotal Phase III clinical trial evaluating pegunigalsidase alfa (PRX–102), 1 mg/kg, administered every two weeks. “We are pleased to announce positive topline results from the BALANCE Phase III clinical trial and would like to thank the Fabry disease patients and their families, as well as the study investigators and their teams. Our robust clinical development program, from the Phase I\II clinical trial, through the three Phase III clinical trials and the related extension studies, required substantial time and effort from study participants who showed a strong level of commitment allowing our clinical development program to move forward,” said Dror Bashan, Protalix’s President and Chief Executive Officer. “Based on results from our clinical program, we believe that PRX–102, as a PEGylated enzyme replacement therapy with potentially two different dosing regimens, may be a valuable new treatment option for individuals suffering from Fabry disease.”
“This is an important milestone both for the Fabry community and Protalix in a long and productive journey. We thank all of our collaborators for their contributions and support throughout this journey,” said Einat Brill Almon, Ph.D., Protalix’s Sr. Vice President and Chief Development Officer. “We believe that this multi-year study demonstrates the potential for switching from agalsidase beta to PRX–102 in the treatment of patients with Fabry disease. The study met our pre–defined criteria for non-inferiority of the primary endpoint of kidney function in a head–to–head active comparison on both the Intent–to–Treat (ITT) and Per Protocol (PP) analysis sets. These topline results show that PRX–102 was comparable to agalsidase beta in controlling eGFR decline, which is a key measure of Fabry disease progression, and continue to demonstrate a favorable tolerability profile for PRX–102. Combined with previous Phase III results from our BRIGHT and BRIDGE studies, as well as the results from our Phase I/II study and its long-term extension, we believe we have a compelling and consistent dataset from both treatment–naïve and ERT–experienced patients. Given these results, we plan, together with Chiesi, our commercialization partner, to work with regulatory agencies on the applicable submissions, hopefully bringing PRX–102 to approval as a new PEGylated enzyme replacement therapy for all adult Fabry patients.”
Out of the patients that completed the trial, 69 have opted with advice of the treating physician to continue receiving PRX-102 1mg/kg every other week in a long term extension study.