Preliminary Results Indicate Improvement in Kidney Function in Patients Switched from agalsidase alfa (Replagal®) to pegunigalsidase alfa (PRX-102)
A deterioration trend in patients’ kidney function on agalsidase alfa (Replagal®) was reversed to an improvement trend when switched to pegunigalsidase alfa (PRX-102)
Presentation with full Data to be held at the1st Canadian Symposium on Lysosomal Diseases 2018
CARMIEL, Israel I September 21, 2018 I Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®, today announced positive preliminary data from the BRIDGE study of pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease. Additional data will be released at the 1st Canadian Symposium on Lysosomal Diseases 2018.
The BRIDGE study is an open label switch over study evaluating the safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa for at least 2 years and on a stable dose for at least 6 months. Patients are screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, each patient was enrolled and switched from agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. Patients can receive PRX-102 infusions at a home care setup based on the infusion tolerability.
Preliminary data from the first sixteen patients enrolled in the trial demonstrated an improvement in kidney function when switched from agalsidase alfa (Replagal®) to pegunigalsidase alfa (PRX-102). Based on available historical serum creatinine and study 3 month screening period values for approximately 2 years while treated with agalsidase alfa before switching to pegunigalsidase alfa treatment, the annualized estimated glomerular filtration rate (eGFR) slope for patients on Replagal was (negative) –6.8ml/min/1.73m2 . The mean eGFR slope for the same patients following six months of treatment with pegunigalsidase alfa (PRX-102) was changed to be (positive) of +3.7ml/min/1.73m2, these results were statistically significant. Baseline characteristic of these patients were: mean estimated glomerular filtration rate (eGFR) 75.40 and 86.03 mL/min/1.73m2 for males and females, with annualized eGFR slope of -8.0 and -5.1 mL/min/1.73m2/year, respectively.
The BRIDGE study is an open-label, single arm switch-over study to assess the safety and efficacy of pegunigalsidase alfa, 1 mg/kg infused every two weeks, in 22 Fabry patients currently treated with Replagal. Protalix anticipates completing patient enrollment in the BRIDGE trial in the fourth quarter of 2018.
PRX-102 is the Company’s plant cell-expressed recombinant, PEGylated, cross-linked α-galactosidase-A for Fabry disease. In pre-clinical and clinical studies, PRX-102 demonstrated higher stability in plasma, a longer half-life and higher exposure in Fabry disease patients, and a reduction in Gb3 in kidney biopsies in treatment naïve Fabry patients.
About Protalix BioTherapeutics, Inc.
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 U.S. Food and Drug Administration (FDA) in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights. Protalix’s development pipeline includes the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; OPRX-106, an orally-delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.
SOURCE: Protalix BioTherapeutics
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Preliminary Results Indicate Improvement in Kidney Function in Patients Switched from agalsidase alfa (Replagal®) to pegunigalsidase alfa (PRX-102)
A deterioration trend in patients’ kidney function on agalsidase alfa (Replagal®) was reversed to an improvement trend when switched to pegunigalsidase alfa (PRX-102)
Presentation with full Data to be held at the1st Canadian Symposium on Lysosomal Diseases 2018
CARMIEL, Israel I September 21, 2018 I Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®, today announced positive preliminary data from the BRIDGE study of pegunigalsidase alfa (PRX-102) for the treatment of Fabry disease. Additional data will be released at the 1st Canadian Symposium on Lysosomal Diseases 2018.
The BRIDGE study is an open label switch over study evaluating the safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa for at least 2 years and on a stable dose for at least 6 months. Patients are screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, each patient was enrolled and switched from agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. Patients can receive PRX-102 infusions at a home care setup based on the infusion tolerability.
Preliminary data from the first sixteen patients enrolled in the trial demonstrated an improvement in kidney function when switched from agalsidase alfa (Replagal®) to pegunigalsidase alfa (PRX-102). Based on available historical serum creatinine and study 3 month screening period values for approximately 2 years while treated with agalsidase alfa before switching to pegunigalsidase alfa treatment, the annualized estimated glomerular filtration rate (eGFR) slope for patients on Replagal was (negative) –6.8ml/min/1.73m2 . The mean eGFR slope for the same patients following six months of treatment with pegunigalsidase alfa (PRX-102) was changed to be (positive) of +3.7ml/min/1.73m2, these results were statistically significant. Baseline characteristic of these patients were: mean estimated glomerular filtration rate (eGFR) 75.40 and 86.03 mL/min/1.73m2 for males and females, with annualized eGFR slope of -8.0 and -5.1 mL/min/1.73m2/year, respectively.
The BRIDGE study is an open-label, single arm switch-over study to assess the safety and efficacy of pegunigalsidase alfa, 1 mg/kg infused every two weeks, in 22 Fabry patients currently treated with Replagal. Protalix anticipates completing patient enrollment in the BRIDGE trial in the fourth quarter of 2018.
PRX-102 is the Company’s plant cell-expressed recombinant, PEGylated, cross-linked α-galactosidase-A for Fabry disease. In pre-clinical and clinical studies, PRX-102 demonstrated higher stability in plasma, a longer half-life and higher exposure in Fabry disease patients, and a reduction in Gb3 in kidney biopsies in treatment naïve Fabry patients.
About Protalix BioTherapeutics, Inc.
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 U.S. Food and Drug Administration (FDA) in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights. Protalix’s development pipeline includes the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; OPRX-106, an orally-delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.
SOURCE: Protalix BioTherapeutics
Post Views: 21
