Protalix BioTherapeutics Completes Enrollment in Phase 3 Trial

Protalix BioTherapeutics, Inc. recently announced the completion of enrollment in the Phase 3 BRIGHT clinical trial of pegunigalsidase alfa, or PRX‑102, for the treatment of Fabry disease, via intravenous (IV) infusions of 2 mg/kg administered every 4 weeks. PRX‑102 is the company’s plant cell-expressed recombinant, PEGylated, cross-linked α‑galactosidase‑A drug candidate.

“We are grateful to the participants and our investigators for their dedication and support of the study,” said Moshe Manor, Protalix’s President and Chief Executive Officer.  “Completion of enrollment marks a key milestone for us. As Fabry disease is a chronic illness with no cure that requires enzyme replacement therapy infusions every 2 weeks under the current standard of care, the possibility of doubling the time between infusions has the potential to greatly enhance the quality of life for some portion of these patients.”

The BRIGHT study is a 12-month, open-label switchover study to assess the safety, efficacy and pharmacokinetics (PK) of pegunigalsidase alfa 2 mg/kg administered every 4 weeks in up to 30 Fabry patients previously treated with an enzyme replacement therapy (ERT): Fabrazyme or Replagal. To determine eligibility for participation in the study, candidates were screened to identify and select Fabry patients with stable kidney disease. Patients that matched the criteria were enrolled in the study and switched from their current treatment of intravenous (IV) infusions every 2 weeks to 2 mg/kg of PRX‑102 every 4 weeks for 12 months.

Patients participating in the study are being evaluated to, among other disease parameters, determine if their kidney disease has not further deteriorated while being treated with the four‑week dosing regimen as measured by eGFR and Lyso Gb3, as well as other parameters. In addition, participating patients are being evaluated to assess the safety and tolerability of PRX‑102.

To date, substantially all patients that were enrolled in the BRIGHT study remain on the 4‑week dosing regimen, and all of the patients that completed the study opted, with the advice of the treating physician, to continue treatment under the 4‑week dosing regimen in a long-term extension study.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx.  Protalix’s unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner.  Protalix’s first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the US FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights. Protalix’s development pipeline includes the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human alpha‑GAL‑A protein for the treatment of Fabry disease; OPRX‑106, an orally delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the US and outside the US, for the development and commercialization of pegunigalsidase alfa.