NOVATO, Calif., Jul 25, 2011 (GlobeNewswire via COMTEX) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") RPTP +3.57% today announced that its Phase 3 clinical trial of Delayed Release or DR Cysteamine, known as study drug RP103 ("RP103"), for the treatment of nephropathic cystinosis, met the primary endpoint of non-inferiority compared to Cystagon(R), immediate-release cysteamine bitartrate. The comparison was based on white blood cell ("WBC") cystine levels, the established efficacy surrogate biomarker and sole primary endpoint in the clinical trial. The Company also reported that there were no unexpected serious safety concerns experienced by patients in the trial attributable to RP103.
Nephropathic cystinosis is a severe ultra-orphan inherited condition which results in premature death if not treated. The current standard of care for cystinosis is oral Cystagon(R), immediate-release cysteamine bitartrate, which must be taken strictly every 6 hours, including a middle-of-the-night dose. Lack of compliance with the strict dosing schedule of Cystagon(R) has been widely reported to be a significant challenge in the therapeutic management of cystinosis patients. RP103 is Raptor's proprietary, twice-daily formulation of cysteamine bitartrate, designed for reduced dose frequency and improved tolerability for the treatment of cystinosis. Raptor's pivotal Phase 3 clinical trial was designed as an outpatient study of the pharmacodynamics, pharmacokinetics, safety and tolerability of RP103 compared to Cystagon(R) in cystinosis patients. The clinical trial was conducted at eight clinical research centers in the US and Europe.
Of 41 patients who completed the Phase 3 protocol, 38 were included in the evaluable data set, 3 not being fully compliant with the protocol. The age range of study participants was 6-26 years, with 87% of patients below 16 years old. On average, the peak WBC cystine level measured in patients treated with Cystagon(R) was 0.54 +/- 0.05 nmol 1/2 cystine/mg protein, compared to an average peak value of 0.62 +/- 0.05 nmol 1/2 cystine/mg protein for patients treated with RP103. The mean difference was 0.08 nmol 1/2 cystine/mg protein, with a 95.8% confidence interval of 0.00-0.16 (one sided p=0.021). As stipulated in the Statistical Analysis Plan, the non-inferiority endpoint of the clinical trial would be achieved when the upper end of the confidence interval around the mean difference of WBC cystine levels did not exceed an absolute value of 0.3. The upper end of the confidence interval in the Phase 3 clinical trial was determined to be 0.16, thus achieving the non-inferiority endpoint. More:
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