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Protalix Announces FDA Investigational New Drug Clearance to Commence Once-Monthly Dosing Study of pegunigalsidase alfa (PRX-102) for the Treatment of Fabry Disease
Once Monthly Dosing Would Represent a 50% Reduction in Patient Infusions
Unique Chemical Modifications to pegunigalsidase alfa Result in a Significantly Longer Half-life Allowing for the Potential of Effective Drug Coverage for Four Weeks
Study Planned to Commence in the Third Quarter of 2017
Pegunigalsidase alfa with a 2 mg/kg was found to be safe and well tolerated with no formation of antibodies in the Company’s phase I/II clinical trial of pegunigalsidase alfa for the treatment of Fabry disease. Additionally, in the phase I/II clinical trial, 2 mg/kg of pegunigalsidase alfa demonstrated approximately a 40 times higher circulatory half-life compared with other enzyme replacement therapies, and, as demonstrated in a Fabry mice model, with materially higher active enzyme reaching target organs affected by Fabry disease. Pharmacokinetic (PK) analysis and modeling from the phase I/II clinical trial indicate that pegunigalsidase alfa levels at the second week after infusion remain 10 times higher than published Fabrazyme® levels at the day of infusion. Moreover, the amount of pegunigalsidase alfa in the circulation at weeks three and four, are higher than those of Fabrazyme® during the two-week treatments. These results provide strong rationale for the clinical evaluation of a once-monthly dosing.
Area Under the Curve (AUC) derived from PK data modeling of Fabry patients:
* PK modeling based on Phase I/II data ** Fabrazyme® USPI
“The unique chemical modifications in pegunigalsidase alfa result in significantly longer circulatory half-life, which we believe will provide effective drug coverage for four full weeks,” said
“The Fabry International Network (FIN), a global Fabry patient organization, welcomes the news that Protalix is moving forward with a third phase III clinical study infusing pegunigalsidase alfa 2mg/kg once every 4 weeks,” sated FIN’s President,
The Company plans to enroll up to 30 Fabry patients currently treated with an approved enzyme replacement therapy. Participating patients will be switched to 2 mg/kg of pegunigalsidase alfa once-monthly. A safety and efficacy evaluation will occur at twelve months with additional long term follow-up. The Company expects to commence this study in the third quarter of 2017.
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
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