Nephropathic cystinosis is usually diagnosed in infancy and requires lifelong therapy.
Raptor currently has product candidates in clinical development designed to potentially treat
nephropathic cystinosis, Non-alcoholic Steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase deficiency ("ALDH2"), and thrombotic disorder.
Moreover, determination of PMN leukocyte cysteine concentrations in individuals with a family history of
nephropathic cystinosis is important for identifying heterozygotes for this heritable disease.
Raptor obtained an exclusive, worldwide license to DR Cysteamine, as well as orphan drug designation from the FDA for DR Cysteamine for the potential treatment of
nephropathic cystinosis, through its December 2007 acquisition of Encode Pharmaceuticals.
Tests for linear trend and multiple comparisons (Student-Newman-Keuls test) were used to examine the existence of any linear trend across
nephropathic status groups and the differences between groups.
PROCYSBI (TM): A $50 million loan agreement was completed with Raptor Pharmaceuticals (NASDAQ: RPTP) to help fund the commercialization of PROCYSBI (TM), the branded name of RP103 for the potential treatment of
nephropathic cystinosis, and to advance the company's development programs.
Cysteamine ([beta]-mercaptoethylamine) depletes cystine from cystinotic cells and is used for treatment of children with
nephropathic cystinosis (5).
Raptor's lead orphan drug is DR Cysteamine for the treatment of
nephropathic cystinosis, a genetic disease marked by abnormal buildup of cysteine in the cells which can result in organ and brain damage.
Raptor currently has product candidates in clinical development designed to potentially treat
nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase ("ALDH2") deficiency, and a non-opioid solution designed to potentially treat chronic pain and thrombotic disorder.
Raptor currently has product candidates in clinical development designed to treat
nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase ("ALDH2") deficiency, and a non-opioid solution designed to treat chronic pain and potentially thrombotic disorder.
By the end of 2009, we expect to announce clinical trial data on our lead product candidate, DR Cysteamine, from a Phase 2a clinical trial in non-alcoholic steatohepatitis ("NASH") and a Phase 2b clinical trial in
nephropathic cystinosis ("cystinosis").
Raptor" or the "Company") (OTC Bulletin Board: RPTP), today announced that it has dosed the first patient in its Phase 2b clinical trial, conducted in collaboration with the University of California, San Diego ("UCSD"), to evaluate Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") capsules in
nephropathic cystinosis ("cystinosis"), a rare genetic lysosomal storage disease.
