Raptor Pharmaceutical (RPTP) Announces Positive Data on RP103
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Raptor Pharmaceutical Corp. (Nasdaq: RPTP), announced that Craig B. Langman, M.D., Head of Kidney Diseases, and the Isaac A. Abt, M.D., Professor of Kidney Diseases and Tenured Professor of Pediatrics, Northwestern University Feinberg School of Medicine, presented on Saturday, November 3rd a "Late Breaking" poster entitled "Extended Treatment with RP103 in Patients with Nephropathic Cystinosis" at Kidney Week 2012.
The poster presented data from Raptor's extension study in which 40 patients completing the Phase 3 clinical trial elected to continue on RP103 (cysteamine bitartrate delayed-release) treatment and to be monitored for white blood cell ("WBC") cystine levels, plasma cysteamine levels, estimated glomerular filtration rate ("eGFR", an indicator of kidney function), quality of life (PedsQL) and use of antacids.
The data from the extension study demonstrated that patients with nephropathic cystinosis using RP103 for up to 24 months, maintained WBC cystine levels continuously below the therapeutic target. After 20 months of treatment, the total daily dose of RP103 given every 12 hours needed to achieve optimal WBC cystine levels was on average 72% of the initial immediate release cysteamine bitartrate dose. Kidney function was well preserved over the length of the study, as indicated by steady eGFR levels. Additionally, the study showed that patients taking RP103 are able to reduce concomitant use of anti-acid medications by nearly half, with 22% of patients taking proton pump inhibitors ("PPI") or histamine 2 ("H2") blockers during the extension vs. 42% upon initially entering the Phase 3 study when they are taking immediate-release cysteamine bitartrate.
The extension study also demonstrated significant improvements in all measured quality of life parameters. From the study onset, the PedsQL 4.0 scores (an instrument for measuring health-related quality of life in children and adolescents ages 2 to 18) improved in all patients, including the Total Functioning Score reflecting a p<0.001 statistical significance, and the Social Performance Score reflecting a p<0.001 statistical significance. Social function can be difficult for cystinosis patients treated with cysteamine bitartrate because of the strong rotten-egg odor associated with the drug's metabolism and from its other side effects.
The poster presented data from Raptor's extension study in which 40 patients completing the Phase 3 clinical trial elected to continue on RP103 (cysteamine bitartrate delayed-release) treatment and to be monitored for white blood cell ("WBC") cystine levels, plasma cysteamine levels, estimated glomerular filtration rate ("eGFR", an indicator of kidney function), quality of life (PedsQL) and use of antacids.
The data from the extension study demonstrated that patients with nephropathic cystinosis using RP103 for up to 24 months, maintained WBC cystine levels continuously below the therapeutic target. After 20 months of treatment, the total daily dose of RP103 given every 12 hours needed to achieve optimal WBC cystine levels was on average 72% of the initial immediate release cysteamine bitartrate dose. Kidney function was well preserved over the length of the study, as indicated by steady eGFR levels. Additionally, the study showed that patients taking RP103 are able to reduce concomitant use of anti-acid medications by nearly half, with 22% of patients taking proton pump inhibitors ("PPI") or histamine 2 ("H2") blockers during the extension vs. 42% upon initially entering the Phase 3 study when they are taking immediate-release cysteamine bitartrate.
The extension study also demonstrated significant improvements in all measured quality of life parameters. From the study onset, the PedsQL 4.0 scores (an instrument for measuring health-related quality of life in children and adolescents ages 2 to 18) improved in all patients, including the Total Functioning Score reflecting a p<0.001 statistical significance, and the Social Performance Score reflecting a p<0.001 statistical significance. Social function can be difficult for cystinosis patients treated with cysteamine bitartrate because of the strong rotten-egg odor associated with the drug's metabolism and from its other side effects.
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