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Raptor Pharmaceutical Announces Publication of Phase 3 Results

Datum nieuwsfeit: 03-05-2012
Bron: GlobeNewswire
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Raptor Pharmaceutical Announces Publication of Phase 3 Results in the Clinical Journal of the American Society of Nephrology

Provides an Update on the Nephropathic Cystinosis Program 
NOVATO, Calif., 2012-05-03 19:15 CEST (GLOBE NEWSWIRE) -- 
Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), 
announced the online publication of an article titled, "A Randomized Controlled 
Crossover Trial with a Delayed Release Cysteamine Bitartrate in Nephropathic 
Cystinosis: Effectiveness on WBC Cystine Levels and Comparison of Safety" in 
the Clinical Journal of the American Society of Nephrology ("CJASN"). The CJASN 
is one of the most respected peer reviewed scientific journals among kidney 
related publications.  
The article describes findings from Raptor's open-label, randomized, 
controlled, crossover Phase 3 clinical trial, referred to as RP103-03, the 
top-line results of which were reported by Raptor in July 2011, and presented 
by the clinical investigators as a late breaking poster at the American Society 
of Nephrology ("ASN") – Kidney Week 2011 in Philadelphia last November.  The 
Phase 3 clinical trial enrolled 43 patients and was powered to show that 
Raptor's delayed-release formulation of cysteamine bitartrate, referred to as 
RP103, taken every 12 hours, was non-inferior to the currently-available 
immediate release cysteamine bitartrate treatment, Cystagon®, taken every 6 
hours, for maintenance of white blood cell ("WBC") cystine levels in patients 
who were already well controlled on Cystagon®. The clinical trial also showed 
that RP103 met its primary endpoint at a lower total daily dose than Cystagon®.  
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, and lead author of the publication, 
said, "RP103 met the primary endpoint compared to Cystagon® at a less frequent 
dosing schedule, as well as a reduced total daily dose. As to tolerability, 
none of the severe adverse events reported in the study were considered 
unexpected, given the symptoms associated with the disease itself. The nature 
of the adverse events reported during the study was similar between the 
Cystagon® and the RP103 treatment periods."  
Patrice P. Rioux, M.D., Ph.D., Chief Medical Officer of Raptor, said, "Two 
patients experienced 24% of the total gastrointestinal ("GI") adverse events 
("AEs") recorded in the study, which accounted for 60% of the drug-related GI 
AEs while on RP103. However, I'm pleased to note that following an initial 
period of GI upset upon starting RP103, both of these patients have continued 
to use RP103 in our extension study and have remained on RP103 for almost one 
year. All but one patient out of all the patients who used proton pump 
inhibitors ("PPIs") while on the Cystagon® arm, did not use PPIs on a regular 
basis while on RP103. This may account for the higher total number of GI AEs 
reported under RP103 vs. Cystagon®."  
The RP103 voluntary extension study, referred to as RP103-04, is ongoing with 
38 enrolled patients of the original 41 patients who completed the Phase 3 
clinical trial. Thirty-two of the Phase 3 patients have been on RP103 for at 
least 12 months with some patients now approaching 18 months on RP103. In 
addition, enrollment in the extension study has been expanded to include 13 
children under 6 years old, and 5 patients with a functioning kidney 
transplant. The children are receiving their RP103 by sprinkling the capsule 
contents onto applesauce or administered through a gastric tube. Raptor plans 
to present data from the extension trial later this year.  
In March, 2012, the European Medicines Agency ("EMA") validated Raptor's 
Marketing Authorization Application for RP103 for the potential treatment of 
nephropathic cystinosis. The MAA is now being reviewed by the EMA. Also in 
March, Raptor submitted a New Drug Application ("NDA") to the U.S. Food and 
Drug Administration ("FDA") seeking approval to market RP103 for the potential 
treatment of nephropathic cystinosis. Raptor requested a Priority Review of the 
NDA which, if granted by FDA, could lead to a decision for marketing approval 
for RP103 for the potential treatment of cystinosis in the U.S., in the fourth 
quarter of 2012.  
About Nephropathic Cystinosis 
Nephropathic cystinosis, an orphan disease, is estimated to effect a population 
of 2,000 patients worldwide, including 500 patients in the U.S. and 800 
patients in Europe. Cystinosis patients have inherited a defective cystine 
transporter gene that results in body-wide cellular toxicity resulting from the 
abnormal buildup of the amino acid cystine in the lysosomes. Cystinosis is 
usually diagnosed in the first years of life and requires lifelong therapy. 
Cystine crystals accumulate in various tissues and organs, including the 
kidneys, brain, liver, thyroid, pancreas, muscles and eyes. Left untreated, the 
disease is fatal by the end of the first decade of life. RP103 reduces cellular 
toxicity by continuously removing cystine from the lysosome.  
About Cysteamine and RP103 
RP103 is Raptor's proprietary, delayed and extended release, oral medication 
designed to potentially treat the underlying metabolic cause of cystinosis. 
RP103 is an enteric coated, microbead formulation of cysteamine bitartrate that 
has been formulated to be sprinkled onto food for administration to patients 
too young to take oral capsules. As demonstrated in the Phase 3 clinical trial 
detailed in the publication in CJASN, the every 6 hour cysteamine dosing 
treatment may be reduced substantially to twice daily on RP103.  
In December 2007, Raptor obtained an exclusive, worldwide license from the 
University of California, San Diego for the development of RP103 for 
nephropathic cystinosis and for cysteamine for other potential indications 
including Huntington's Disease, currently in a Phase 2/3 clinical trial in 
France, and non-alcoholic steatohepatitis ("NASH") currently in a Phase 2b 
clinical trial in the US. Raptor has been granted orphan product designation 
for RP103 for the potential treatment of nephropathic cystinosis by the EMA and 
About Raptor Pharmaceutical Corp. 
Raptor Pharmaceutical Corp. (Nasdaq:RPTP) ("Raptor") seeks to research, 
produce, and deliver medicines that improve life for patients with severe, rare 
disorders. 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.  
Raptor's preclinical programs are based upon bioengineered novel drug 
candidates and drug-targeting platforms derived from the human 
receptor-associated protein and related proteins that are designed to target 
cancer and infectious diseases.  
For additional information, please visit 
The Raptor Pharmaceutical Corp. logo is available at  
                           FORWARD LOOKING STATEMENTS 
This document contains forward-looking statements as that term is defined in 
the Private Securities Litigation Reform Act of 1995. These statements relate 
to future events or our future results of operation or future financial 
performance, including, but not limited to the following statements: that 
Raptor will complete its extension trial or present data from its extension 
trial later this year; that the every 6 hour cysteamine dosing treatment will 
be reduced to twice daily on RP103; that FDA could render a decision regarding 
marketing approval of RP103 for the potential treatment of cystinosis in the 
U.S., in the fourth quarter of 2012; and that Raptor will be able to 
successfully develop RP103 or any of its other product candidates. These 
statements are only predictions and involve known and unknown risks, 
uncertainties and other factors, which may cause the Company's actual results 
to be materially different from these forward-looking statements. Factors which 
may significantly change or prevent the Company's forward looking statements 
from fruition include: that Raptor may be unsuccessful in developing any 
products or acquiring products; that Raptor's technology may not be validated 
as it progresses further and its methods may not be accepted by the scientific 
community; that Raptor is unable to retain or attract key employees whose 
knowledge is essential to the development of its products; that unforeseen 
scientific difficulties develop with the Company's process; that Raptor's 
patents are not sufficient to protect essential aspects of its technology; that 
competitors may invent better technology; that Raptor's products may not work 
as well as hoped or worse, that the Company's products may harm recipients; and 
that Raptor may not be able to raise sufficient funds for development or 
working capital. As well, Raptor's products may never develop into useful 
products and even if they do, they may not be approved for sale to the public. 
Raptor cautions readers not to place undue reliance on any such forward-looking 
statements, which speak only as of the date they were made. Certain of these 
risks, uncertainties, and other factors are described in greater detail in the 
Company's filings from time to time with the Securities and Exchange Commission 
(the "SEC"), which Raptor strongly urges you to read and consider, including: 
Raptor's annual report on Form 10-K, as amended by Form10-K/A, filed with the 
SEC on November 11, 2011 and December 19, 2011, respectively; and Raptor's 
quarterly report on Form 10-Q filed with the SEC on April 9, 2012; all of which 
are available free of charge on the SEC's web site at 
Subsequent written and oral forward-looking statements attributable to Raptor 
or to persons acting on its behalf are expressly qualified in their entirety by 
the cautionary statements set forth in Raptor's reports filed with the SEC. 
Raptor expressly disclaims any intent or obligation to update any 
forward-looking statements.  
         CONTACT: Trout Group (investors) 
         Lauren Glaser 
         (646) 378-2972 
         EVC Group (media) 
         Janine McCargo 
         (646) 688-0425 
Ticker_symbol :
ISIN US75382F1066

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