Protalix BioTherapeutics and Chiesi Global Rare Diseases have reported final data from the Phase III BRIGHT clinical trial of pegunigalsidase alfa (PRX‑102) to potentially treat Fabry disease.
PRX‑102 is a plant cell culture-expressed and chemically updated stabilised form of the recombinant α‑Galactosidase‑A enzyme.
The multinational, multicentre, open-label, switch-over trial analysed the safety, efficacy and pharmacokinetics of intravenous (IV) doses of 2mg/kg PRX-102 given every four weeks for 52 weeks.
It enrolled 30 adult Fabry disease patients aged 19 to 58 years, who were earlier treated with an approved enzyme replacement therapy (ERT) for a minimum of three years on a stable dose given every two weeks.
According to the findings, treatment with 2mg/kg IV doses of PRX-102 given every four weeks was found to be well tolerated.
Furthermore, Fabry disease, as evaluated by estimated glomerular filtration rate (eGFR) slope and plasma lyso-Gb3 concentration, was reported to be stable.
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In the trial, 30% of subjects had treatment-emergent adverse events (TEAEs), which were found to be all mild or moderate in nature.
No serious or severe TEAEs linked to the treatment and no TEAEs that caused death or withdrawal from the trial were reported.
No subjects developed anti-drug antibodies following treatment with PRX-102, the company said.
Protalix BioTherapeutics president and CEO Dror Bashan said: “We are excited to share the final data from the BRIGHT study, an important milestone in the progress of our PRX-102 clinical programme.
“The availability of this data for review by the US 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.”
In September 2019, the companies completed subject enrolment in the Phase III BALANCE trial of pegunigalsidase alfa (PRX 102) for treating Fabry disease.