Protalix BioTherapeutics and Chiesi Farmaceutici (Chiesi Group) have concluded patient enrolment in the Phase III BALANCE clinical trial of pegunigalsidase alfa (PRX 102) to treat Fabry disease.
Pegunigalsidase alfa is an investigational, plant cell culture-expressed enzyme made via chemical modification of the recombinant alpha-Galactosidase-A protein.
The drug candidate is intended to address the unmet clinical need for Fabry disease, which is an X-linked inherited condition caused by deficient lysosomal enzyme alpha-galactosidase A activity.
In a mouse model of the disease, pegunigalsidase alfa demonstrated favourable enzyme activity levels in affected organs.
The head-to-head, randomised, double-blind, active control Phase III BALANCE trial is designed to investigate the safety and efficacy of the drug candidate in patients with Fabry disease and progressing kidney disease.
It involves patients who received prior treatment with agalsidase beta for around one year and on a stable dose for a minimum of six months.
Participants will be administered with intravenous infusions of 1mg/kg pegunigalsidase alfa or agalsidase beta every two weeks for 24 months.
Protalix BioTherapeutics president and CEO Dror Bashan said: “We are excited to complete enrolment in our third Phase III study of PRX‑102 for the treatment of Fabry disease. This achievement marks a major milestone in our development of PRX‑102.
“As we have already disclosed, we are targeting a BLA submission under an accelerated approval pathway for PRX‑102 in the first quarter of 2020 based on data from our completed Phase I/II clinical studies of PRX‑102 and from our ongoing Phase III BRIDGE clinical study.”
In addition to BALANCE, pegunigalsidase alfa is being studied in Phase III BRIDGE and BRIGHT trials.
The BRIDGE trial is assessing 1mg/kg pegunigalsidase alfa given every two weeks in patients previously treated with agalsidase alpha.
Meanwhile, the BRIGHT trial involves 2mg/kg pegunigalsidase alfa administered every four weeks in patients who were formerly on enzyme replacement therapy.