Replies to post #3655 on PROTALIX BIOTHERAPEUTICS, INC. (PLX)

[midastouch017]

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

[Spideyboy]

Excellent.

Key aspects I like are

- Meeting the non-inferiority endpoint on both the ITT and PP groups!

- Safety and immunogenicity are clearly superior to Fabrazyme on all points!

- Warnock specifically states:

1. This effort is the first randomized, head-to-head comparison in Fabry disease of a new ERT to an established, approved form of ERT. The topline results of the BALANCE study and the entire PRX–102 clinical program indicate that this novel PEGylated ERT has the potential for a long-term clinical benefit for adult Fabry patients.

2. These topline results also indicate that PRX-102 was well-tolerated and support the potential to switch to this novel, investigational ERT from a currently approved ERT


A long road, but finally vindicated. PRX-102 is going to be a great options for Fabry patients!