DGAP-News: Raptor Pharmaceutical Provides Update on Phase 3 Clinical Trial of DR Cysteamine for the Potential Treatment of Nephropathic Cystinosis
ID: 419461
06.06.2011 12:30
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Data From Trial Expected to be Released July 2011
NOVATO, Calif., 2011-06-06 12:30 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP), today
announced that on June 3 the last clinical trial patient visits were completed
and final data analysis will commence this week on the Phase 3 clinical trial
of its delayed-release oral formulation of cysteamine bitartrate ('DR
Cysteamine') in patients with nephropathic cystinosis ('cystinosis'). The
Company announced that 41 patients have completed the nine-week study protocol
and the Company expects to report the data from this trial by the end of July
2011. The extension phase of the clinical trial, in which all patients
completing the Phase 3 study may elect to continue on DR Cysteamine therapy, is
ongoing.
The pivotal Phase 3 clinical trial is designed as an outpatient study of the
safety, tolerability, pharmacokinetics ('PK') and pharmacodynamics ('PD') of
every 12-hour DR Cysteamine compared to every 6-hour immediate-release
cysteamine bitartrate in cystinosis patients. DR Cysteamine is designed for
reduced dose frequency and improved tolerability, compared to immediate-release
cysteamine, which is the current standard of care in cystinosis. The
multi-center, randomized, crossover design of this comparative study is a
result of discussions with the U.S. Food and Drug Administration ('FDA') by
which FDA provided significant guidance on trial protocol design, clinical
endpoints, and statistical analyses.
'We are pleased to see our final patients complete the Phase 3 clinical trial
and are gratified that 40 of the 41 patients completing the study have elected
to enter our long-term extension study in which they have chosen to remain on
the twice-a-day DR Cysteamine formulation,' commented Patrice Rioux M.D.,
Ph.D., CMO of Raptor. 'As our earliest enrolled patients began to enter the
extension study in August of last year, some patients have now been treated
with DR Cysteamine for over 9 months; we currently have over 6 months of
extension data on our first 20 patients.'
About Nephropathic Cystinosis
Nephropathic cystinosis is an inborn metabolic error characterized by the
abnormal transport of cystine, an amino acid, out of the lysosomes. Poor
compliance with current treatments for nephropathic cystinosis can cause
serious health consequences, including renal failure and resultant need for a
kidney transplant; growth failure; rickets and fractures; 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.
About Cysteamine and DR Cysteamine
DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral
formulation of cysteamine bitartrate designed to potentially reduce dosing
frequency and reduce gastrointestinal side effects associated with
immediate-release cysteamine bitartrate, which is approved for sale by the FDA
and European Medicines Agency ('EMA') to treat nephropathic cystinosis.
In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diegofor the development DR Cysteamine for
nephropathic cystinosis and cysteamine for other potential indications
including Huntington's Disease, NASH and Batten Disease.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. (Nasdaq:RPTP) ('Raptor') is dedicated to speeding
the delivery of new treatment options to patients by working to improve
existing therapeutics through the application of highly specialized drug
targeting platforms and formulation expertise. Raptor focuses on underserved
patient populations where it can have the greatest potential impact. 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 thrombotic disorder.
Raptor's preclinical programs are based upon bioengineered novel drug
candidates and drug-targeting platforms derived from the human
receptor-associated protein ('RAP') and related proteins that are designed to
target cancer, neurodegenerative disorders and infectious diseases.
For additional information, please visit www.raptorpharma.com.
The Raptor Pharmaceutical Corp. logo is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=7180
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 DR
Cysteamine will reduce dose frequency and improve tolerability compared to
immediate-release cysteamine; that final data review will occur and that the
Company will report initial findings by the end of July, if at all; that all 40
patients will continue to participate in the Company's long-term extension
study in which patients remain on the twice-a-day DR Cysteamine formulation;
and that Raptor will be able to successfully develop DR Cysteamine 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 filed with the SEC
on November 22, 2010; and Raptor's quarterly report on Form 10-Q filed with the
SEC on April 14, 2011; all of which are available free of charge on the SEC's
web site at http://www.sec.gov. 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
lglaser@troutgroup.com
The Ruth Group (media)
Jason Rando
(646) 536-7025
jrando@theruthgroup.com
News Source: NASDAQ OMX
06.06.2011 Dissemination of a Corporate News, transmitted by DGAP -
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Language: English
Company: Raptor Pharmaceutical Corp.
United States
Phone:
Fax:
E-mail:
Internet:
ISIN: US75382F1066
WKN:
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