Protalix stock soars 33% as PRX–102 for Fabry disease equals Sanofi's Fabrazyme in trial
Apr. 04, 2022 8:06 AM ETProtalix BioTherapeutics, Inc. (PLX)SNY
By: Ravikash, SA News Editor
Protalix BioTherapeutics (NYSE:PLX) and Chiesi Global Rare Diseases said PRX–102 was as good as Sanofi 's (SNY) Fabrazyme for treating Fabry disease in a late stage study.
Protalix and Chiesi, a unit of Chiesi Farmaceutici, plan to resubmit a biologics license application to the FDA for PRX–102, a PEGylated enzyme replacement therapy, in H2 2022.
The companies were evaluating PRX–102 (pegunigalsidase alfa) 1 mg/kg administered every two weeks against Fabrazyme (agalsidase beta) in patients with Fabry disease with deteriorating renal function in a phase 3 trial called BALANCE.
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," said Einat Brill Almon, Protalix's senior vice president and chief development officer.
The study enrolled 78 patients who were previously treated with Fabrazyme for at least one year. Patients were randomized on a 2:1 ratio for switching to PRX–102 or continuing on Fabrazyme. A total of 77 patients were treated; 52 with PRX–102 and 25 with Fabrazyme.
The companies said PRX–102 met the criteria for non-inferiority of the primary goal of kidney function.
The companies noted that Treatment-related adverse events were reported for 21 (40.4%) patients in the PRX–102 arm compared to 11 (44.0%) in the agalsidase beta arm.
In March, the companies reported data from a 52-week study, dubbed BRIGHT, of PRX-102 to treat Fabry disease — an inherited disorder that results from deficient activity of the lysosomal a–Galactosidase–an enzyme causing progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide in blood vessel walls throughout the body.
PLX +33.33% to $1.40 premarket April 4
