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Protalix BioTherapeutics Reports Positive Phase I/II Interim Clinical Data on the 1mg/kg Cohort of PRX-102 for Fabry Disease
Positive efficacy data across all disease parameters
Positive safety data with low level of antibody formation
End of Phase II meeting with
"We are very pleased with the positive results from the 1mg cohort for PRX-102," commented Mr.
The phase I/II clinical trial of PRX-102 for the treatment of Fabry disease is an open-label, dose-ranging study treating up to 18 naïve male and female adult patients. The three dose cohorts include dosage groups of 0.2 mg/kg, 1mg/kg and 2mg/kg with intravenous infusions of PRX-102 every two weeks, with a six-month efficacy follow up period.
This interim analysis includes 6 patients enrolled in the 1mg/kg dose group at six months of treatment. The interim safety analysis includes 18 patients enrolled in three dose cohorts of 0.2mg/kg, 1mg/kg and 2mg/kg.
Interim Efficacy Results
Based on an analysis of kidney biopsies with randomized blinded scoring (n=4), PRX-102 demonstrated a reduction in renal peritubular capillary Gb3 of 86% using a quantitative Barisoni Lipid Inclusion Scoring System (BLISS).
Reductions of plasma Lyso-Gb3 and plasma Gb3 concentrations were also observed. Males (n=4) demonstrated a -67.5 ng/mL and a -5.3 µg/mL change, Females (n=2) demonstrated a -9.2 ng/mL mean change in Lyso-Gb3 and a -0.23 µg/mL mean change in plasma Gb3, respectively.
Furthermore, all patients had stable cardiac function after only six months, as measured by left ventricular mass (LVM), left ventricular mass index (LVMI) and ejection fraction (EF). Stable kidney function was also observed, as measured by estimated glomerular filtration rate (eGFR) and urine protein.
The safety analysis for adverse events represents a total of 15 patient years. PRX-102 was well tolerated, with the majority of adverse events being mild and moderate. Only one of the patients evaluated for safety experienced hypersensitivity, and only three patients, or approximately 19%, developed antibodies.
"The data presented from the 1mg cohort of PRX-102 continues to be very encouraging," said Dr. Derralynn Hughes of the Lysosomal Storage Disease Unit,
Enrollment in the phase I/II clinical trial of PRX-102 was completed in early
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
Established in 1984 in
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