Phase III BRIGHT trial of PRX 102 shows drug well tolerated in Fabry Disease.
BioTherapeutics and Chiesi announced final results from the BRIGHT Phase III clinical trial evaluating PRX 102 (pegunigalsidase alfa) 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. Overall, 33 of 182 total treatment-emergent adverse events (TEAEs) reported in nine (30.0%) patients were considered treatment-related; all were mild or moderate in severity and the majority were resolved at the end of the study. There were no serious or severe treatment-related TEAEs and no TEAEs led to death or study withdrawal.
Of the treatment-related TEAEs, 27 were infusion-related reactions (IRRs) and the remainder were single events of diarrhea, erythema, fatigue, influenza-like illness, increased urine protein/creatinine ratio, and urine positive for white blood cells. The 27 IRRs were reported in five (16.7%) patients, all male. All IRRs occurred during the infusion or within two hours post-infusion; no events were recorded between two and 24 hours post-infusion. None of the patients without anti-drug antibodies (ADAs) at screening developed treatment-induced ADAs following the switch to PRX 102 treatment.
Study outcome measures show that plasma lyso-Gb3 concentrations remained stable during the study with a mean change (±SE) of 3.01 nM (0.94) from baseline (19.36 nM ±3.35) to Week 52 (22.23 ±3.60 nM). Mean absolute eGFR values were stable during the 52-week treatment period, with a mean change from baseline of -1.27 mL/min/1.73 m2(1.39). Mean (SE) eGFR slope, at the end of the study, for the overall population, was -2.92 (1.05) mL/min/1.73m2/year indicating stability.
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