Benzinga | Jun 2, 2021 12:11PM EDT

Protalix Shares Hits 52-Week Low After Adverse Event Reported In Fabry Disease Trial With PRX-102

* Protalix BioTherapeutics Inc (NYSE:PLX) and Chiesi Farmaceutici S.p.A. have provided an update on the clinical development of pegunigalsidase alfa (PRX--102) for the proposed treatment of Fabry disease.

* PRX--102 is currently being studied in Phase 3 BALANCE trial evaluating 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 study is the comparison of mean annualized changes (slope) 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 in the Intention to Treat analysis set and above such limit in the Intention to Treat analysis set.

* At the time of this analysis (n=77), two patients discontinued participation due to treatment-emergent adverse events - one discontinued participation due to a related adverse event. No deaths were registered.

* Unblinded final data are anticipated in Q2 of 2022 after all remaining patients have completed the 24-month treatment period.

* Despite reporting safety issues, the company will move forward with marketing application in Europe PRX--102 in Fabry disease.

* Regarding the regulatory process in the U.S., the companies plan to submit a Type--A meeting request with the FDA to discuss the path for approval of PRX--102.

* In April, the companies received FDA Complete Response Letter for PRX102 for Fabry disease, though no safety concerns were noted in the letter.

* Price Action: PLX shares are down 13.8% at $2.57 during the market session on the last check Wednesday.