Protalix BioTherapeutics Inc. (NYSEAMERICAN: PLX) has announced final results from the BRIGHT third phase clinical study evaluating pegunigalsidase alfa for possible Fabry disease treatment.
PRX-102 treatment well tolerated
The findings demonstrate that treatment with 2 mg/kg PRX-102 given via IV infusion every 4 weeks was well tolerated, and Fabry disease was stable as measured by the slope of the estimated glomerular filtration rate (eGFR) and plasma lyso-Gb3 concentration.
CEO Dror Bashan said, “We are excited to share the final data from the BRIGHT study, an important milestone in the progress of our PRX-102 clinical program. The availability of this data for review by the U.S. Food and Drug Administration, the European Medicines Agency and other regulators is another step forward towards the anticipated approval of PRX-102 as a potential good alternative for adult Fabry patients in both the regular 1 mg\kg every two weeks as well as the 2 mg\kg every four weeks regimen.”
PRX-102 is a recombinant, PEGylated, cross-linked galactosidaseA product candidate expressed in plant cells. The BRIGHT third Phase clinical study (NCT03180840) was a multinational, multicenter, open-label, switch-over trial designed to assess the efficacy, safety, and pharmacokinetics of 2 mg/kg PRX-102 given after four weeks for 52 weeks, totaling 14 injections.
30 Fabry disease adults recruited in the study
The study included 30 adult subjects with Fabry disease (six females and 24 males) ranging from 19 years to 58 years who had received prior and approved enzyme replacement therapy (ERT) on a steady therapeutic dose given every two weeks (agalsidase beta – Fabrazyme or agalsidase alfa – Replagal). At baseline, the most prevalent Fabry disease symptoms were heat intolerance, acroparesthesia, hypohidrosis, and angiokeratomas.
All subjects who took part in the study were given at least one PRX-102 dose, and 29 people finished the study. Throughout the study, 28 of the 29 patients received the planned protocol of 2 mg/kg PRX-102 each four weeks, while one subject was shifted to 1 mg/kg PRX-102 every two weeks for each regimen at the 11th injection. Due to a traffic accident, one subject withdrew from the trial after the first infusion.