Protalix, Chiesi Global Announce Topline Results From The Interim Analysis Of Balance Study

(RTTNews) - Protalix BioTherapeutics, Inc. (PLX) and Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici S.p.A., reported positive initial top line results from the Phase 3 study of pegunigalsidase alfa (PRX-102), dubbed Balance, for the proposed treatment of Fabry disease. The Balance study of PRX-102 in Fabry patients with impaired renal function evaluates the safety and efficacy of 1 mg/kg of PRX-102 dosed every two weeks compared to agalsidase beta (Fabrazyme).

The primary endpoint of the interim analysis is the comparison of mean annualized changes of the eGFR (CKD-EPI) after completion of at least 12 months of treatment between the two treatment arms.

The initial top-line results show that the lower boundary of the confidence interval for the mean difference between the two treatments was below the non-inferiority margin pre-specified for this interim analysis in the ITT analysis set and above such limit in the PP analysis set.

Based on the 12-month data from the Balance study, along with positive data from Phase 3 Bright and Bridge studies, Protalix and Chiesi plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA).

Protalix and Chiesi also plan to submit a Type-A meeting request with the FDA to discuss the path for approval of PRX-102.