Raptor Pharmaceutical to present DR Cysteamine trial data at Lysosomal Disease Network Symposium

Feb 10 2010

Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP), today announced the presentation of cystinosis data at the Annual Lysosomal Disease Network WORLD Symposium 2010, being held February 10-12 in Miami, Florida. Biomarker data from a Phase IIb pilot study of Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") will be the subject of a poster titled, "Correlation of Plasma Cysteamine and WBC Cystine Levels at Steady State in Patients Treated with Cysteamine Bitartrate." The results will also be published in an upcoming issue of the journal Molecular Genetics and Metabolism. 

The data supports the effectiveness of pre-dose plasma cysteamine concentration in determining the correct cysteamine dose for nephropathic cystinosis ("cystinosis") patients. Pre-dose plasma cysteamine concentration is an easier biomarker to measure in clinical settings compared to cystine levels in white blood cells ("WBC"s). Researchers in the pilot study found that pre-dose plasma cysteamine concentration has the potential to be a viable alternative to WBC cystine levels as a predictive biomarker for proper cysteamine dosing.

Patrice P. Rioux, M.D., Ph.D., Chief Medical Officer said, "We are pleased to share our most recent biomarker findings with the Lysosomal Disease Network, and have taken advantage of this opportunity to further contribute to the study and treatment of cystinosis. Our positive Phase IIb DR Cysteamine clinical trial data demonstrated improved tolerability in cystinosis patients and potential efficacy with less frequent dosing and a lower total daily dosage compared to standard of care, immediate-release cysteamine bitartrate. If we can avoid nocturnal dosing and improve compliance issues associated with today's standard of care, we believe we have the potential to improve both quality of life and overall treatment results for cystinosis patients."

Cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to treat cystinosis can cause serious health consequences, including renal failure and resultant kidney transplant, growth failure, rickets, photophobia and blindness. Symptom onset typically occurs within the first year of life, when cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes.

Separately, Raptor announced the establishment of a Dutch subsidiary, called Raptor Pharmaceuticals Europe BV, to be used as a base for the Company's European clinical, regulatory, commercial and business development activities.