PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Results Show

PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Results Show

Protalix BioTherapeutics‘ investigational therapy PRX-102 (pegunigalsidase alfa) improved kidney function in Fabry disease patients, according to preliminary Phase 3 clinical results.

The company announced that additional positive preliminary data of its BRIDGE clinical trial will be presented during the Canadian Symposium on Lysosomal Diseases, taking place Oct. 5-6 in Sherbrooke, Quebec.

Michael L. West, MD, principal investigator of the trial in Nova Scotia, Canada, will discuss the results in an oral presentation titled “Pegunigalsidase Alfa-a Novel Enzyme Replacement Therapy for the Treatment of Fabry Disease: Preliminary Results from the Phase III Bridge Study.

PRX-102, developed by Protalix, is an investigational enzyme replacement therapy (ERT) that compensates for the lack of the alpha-galactosidase A (αGAL-A) enzyme in Fabry patients.

The absence of αGAL-A leads to the damaging accumulation of two fat molecules — Gb3 and lysoGb3 — in tissues such as the heart, kidneys, nervous system, eyes, and skin.

According to Protalix, PRX-102 is more stable and active than currently available ERTs — Fabrazyme (agalsidase beta), developed by Sanofi Genzyme, and Replagal (agalsidase alpha), developed by Shire — because it was chemically modified to be an improved version of the enzyme.

Previous data from Phase 1/2 studies showed that PRX-102 is safe and prevented kidney and heart dysfunction in Fabry disease patients. Also, results from the ongoing Phase 3 BALANCE study (NCT02795676) have so far demonstrated that PRX-102 is superior to Fabrazyme in stabilizing kidney function, and is less suppressed by pre-existing neutralizing antibodies than Fabrazyme.

Now the company will present preliminary data of the ongoing open-label, switch-over Phase 3 BRIDGE trial (NCT03018730) evaluating the safety and effectiveness of PRX-102 in Fabry disease patients previously treated with Replagal for at least two years.

After an initial three-month period of observation, the participants are switched from Replagal to 1 mg/kg of PRX-102, administered every two weeks for a year.

The preliminary results concern the first 14 enrolled patients (seven men and seven women) who have completed six months of PRX-102 treatment. The patients’ age range is 27-60 years, and 10 of them showed signs of kidney damage at the beginning of the study.

Preliminary data has shown that PRX-102 treatment greatly improved patients’ kidney function by reverting the decline in the glomerular filtration rate — a measure of how well the kidneys are cleaning the blood.

“The unique pegunigalsidase-alfa [PRX-102] characteristics and these preliminary results further support the potential advantage of pegunigalsidase-alfa over the currently available FD [Fabry disease] therapies,” the researchers wrote.

The trial is still recruiting in Canada, Europe, the U.K., and Australia, and aims to enroll up to 22 patients.

PRX-102 was designated an orphan drug for the treatment of Fabry disease by the European Commission in 2017, and was granted fast-track designation by the U.S. Food and Drug Administration this year.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.
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Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.
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