Chiesi and Protalix announce Phase III data from BRIGHT study in Fabry disease

Chiesi Global Rare Diseases, a business unit of privately-held Italian drugmaker Chiesi Farmaceutici, and Israeli firm Protalix BioTherapeutics (TASE: PLX) today announced final results from the Phase III BRIGHT study evaluating pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease. Protalix’ shares gained nearly 15% in morning trading.

The Phase III BRIGHT study was a multicenter, multinational, open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of PRX-102 (2mg/kg every four weeks) for 52 weeks. The study involved 30 patients with Fabry disease previously treated with a commercially available enzyme replacement therapy (ERT) – either Replagal (agalsidase alfa) or Fabrazyme (agalsidase beta). PRX-102 is an ERT created from a plant-based platform used to produce a chemically modified stabilized version of the recombinant α-galactosidase-A (GLA) enzyme, which is deficient in Fabry disease patients.

Results showed that treatment with PRX-102 was well tolerated, and Fabry disease assessed by estimated glomerular filtration rate (eGFR) slope and concentration of plasma globotriaosylsphingosine (lyso-Gb3), a biomarker of this disease, was stable. No patients developed treatment-induced anti-drug antibodies to PRX-102.