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Protalix BioTherapeutics Reports Positive Interim Data From Phase I/II Clinical Trial of PRX-102 for the Treatment of Fabry Disease
Meaningful Clinical Benefits Demonstrated Across All Key Disease Parameters
Favorable Safety Profile
"We are very enthusiastic about the encouraging efficacy and safety data presented today 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 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.
The interim efficacy analysis includes 6 patients enrolled in the 0.2mg/kg dose group at six months of treatment (for Gb3 in renal peritubular capillaries n=5). The interim safety analysis includes 12 patients; 6 patients enrolled in the 0.2mg/kg dose group and 6 patients enrolled in the 1mg/kg dose group.
Interim Efficacy Results
Based on an analysis of kidney biopsies with randomized blinded scoring, PRX-102 demonstrated a reduction in renal peritubular capillary Gb3 of 82.2% for males and 65.4% for females using a quantitative Barisoni Lipid Inclusion Scoring System (BLISS) for a combined reduction of 75.5%. Applying the semi quantitative scoring method, commonly used by approved enzyme replacement therapies, PRX-102 demonstrated a reduction of 69.6% in abnormal capillary score. Using the well-accepted Brief Pain Inventory scale, a 100% reduction in Worst Pain and an average of 78.8% improvement on patients' Impact On Functioning were observed. Furthermore, all patients had stable cardiac function, with favorable trends 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, with favorable trends after only six months, as measured by estimated glomerular filtration rate (eGFR) and urine protein.
The safety analysis for adverse events represents a total of 6.7 patient years. PRX-102 was well tolerated, with the majority of events being mild and moderate. Only one of the 12 patients evaluated for safety experienced hypersensitivity. Six patients receiving the 0.2mg/kg dose and 2 patients receiving the 1m/kg dose were evaluated for antibody formation. Of these 8 patients, only 2 patients, or 33% of the 0.2 mg/kg dose cohort, developed antibodies.
"The safety and clinical benefits of PRX-102 demonstrated in the interim results are extremely exciting and promising. Meaningful improvement or stability was seen across all disease parameters studied, including Gb3 levels, renal function, pain and cardiac parameters," said Professor
Dr. Derralynn Hughes of the Lysosomal Storage Disease Unit,
Currently, patients are being treated in all three dose groups of the Phase I/II trial, and the Company expects to conclude enrollment by the end of first quarter of 2015. All patients that have completed the initial six-month efficacy follow-up have opted to be enrolled in the extension studies. The Company is planning to present additional interim results at the WORLD symposium which is being held
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
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: unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; slower than expected rates of patient recruitment; 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 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; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for regulatory approval; delays in the approval or potential rejection of any applications we file with the
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