Protalix BioTherapeutics, Inc. a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell‑based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research focused healthcare Group (Chiesi Group), announced final results from the BRIGHT Phase III clinical trial evaluating pegunigalsidase alfa (PRX‑102) for the potential treatment of Fabry disease. The results indicate that treatment with 2 mg/kg of PRX-102 administered by intravenous (IV) infusion every four weeks was well tolerated, and Fabry disease assessed by estimated glomerular filtration rate (eGFR) slope and plasma lyso-Gb3 concentration was stable.
“We are excited to share the final data from the BRIGHT study, an important milestone in the progress of our PRX-102 clinical program,” said Dror Bashan, Protalix’s President and Chief Executive Officer. “The availability of this data for review by the U.S. Food and Drug Administration, the European Medicines Agency and other regulators is another step forward towards the anticipated approval of PRX-102 as a potential good alternative for adult Fabry patients in both the regular 1 mg\kg every two weeks as well as the 2 mg\kg every four weeks regimen.”
PRX-102 is a plant cell-expressed recombinant, PEGylated, cross-linked α‑galactosidase‑A product candidate. The BRIGHT Phase III clinical trial (NCT03180840) was a multicenter, multinational open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of treatment with 2 mg/kg of PRX-102 administered every four weeks for 52 weeks (a total of 14 infusions). The study enrolled 30 adult patients with Fabry disease (24 males and 6 females) with mean (SD) age of 40.5 (11.3) years, ranging from 19 to 58 years, who previously received an approved enzyme replacement therapy (ERT) for at least three years on a stable dose administered every two weeks (agalsidase alfa – Replagal® or agalsidase beta – Fabrazyme®). The most common Fabry disease symptoms at baseline were acroparesthesia, heat intolerance, angiokeratomas and hypohydrosis.
All patients who participated in the study received at least one dose of PRX-102, and 29 patients completed the study. Of these 29 patients, 28 received the intended regimen of 2 mg/kg of PRX-102 every four weeks throughout the entire study, while one patient was switched to 1 mg/kg of PRX-102 every two weeks per protocol at the 11th infusion. One patient withdrew from the study after the first infusion due to a traffic accident.
First infusions of PRX-102 were administered under controlled conditions at the investigational site. Based on pre-specified criteria in the study protocol, patients were able to receive their PRX-102 infusions at a home care setup once the Investigator and Sponsor Medical Monitor agreed that it was safe to do so.