|View printer-friendly version|
|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
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms “anticipate,” “believe,” “estimate,” “expect,” “plan” and “intend” and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk. Factors that might cause material differences include, among others: failure or delay in the commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to monitor patients adequately during or after treatment; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and lack of sufficient funding to finance clinical trials; the risk that the results of the clinical trials of our product candidates will not support our claims of superiority, safety or efficacy, that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to the amount and sufficiency of our cash and cash equivalents; risks related to the successful conclusion of our negotiations with the