- Protalix BioTherapeutics Inc 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 PRX‑102 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.
PLXProtalix BioTherapeutics Inc
$2.60-%
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