DGAP-News: Raptor Pharmaceutical Completes Enrollment in Pivotal Phase 3 Clinical Trial of DR Cysteamine for the Potential Treatment of Nephropathic Cystinosis
ID: 334119
24.01.2011 12:30
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Over 30 Patients Expected to Complete Eight-Week Study Protocol
NOVATO, Calif., 2011-01-24 12:30 CET (GLOBE NEWSWIRE) --Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP), today
announced it has completed enrollment in its Phase 3 clinical trial of its
proprietary delayed-release oral formulation of cysteamine bitartrate ('DR
Cysteamine') in patients with nephropathic cystinosis ('cystinosis').
The pivotal Phase 3 clinical trial is designed as an outpatient study of the
safety, tolerability, pharmacokinetics ('PK') and pharmacodynamics ('PD') of DR
Cysteamine dosed every twelve hours in patients with cystinosis, compared to
the current standard of care, immediate-release cysteamine bitartrate, which
requires dosing every six hours. Raptor expects over 30 patients to complete
the eight-week study protocol. All patients completing the Phase 3 clinical
trial have the option of enrolling in a long-term follow-on study where they
continue to receive DR Cysteamine twice daily for the extent of the study.
'As defined in our statistical analysis plan, an interim statistical analysis
of intra-patient variance after 20 patients had completed the study, led us to
determine that our patient enrollment is complete. With enrollment completed,
we anticipate that we will be able to meet our goal of reporting top line data
from this clinical trial in the first quarter of 2011,' remarked Patrice P.
Rioux, M.D., Ph.D., Chief Medical Officer of Raptor. 'Patient compliance is
paramount to improving therapeutic control and achieving optimal long-term
treatment outcome, and we believe twice-daily DR Cysteamine has the potential
to be as efficacious as immediate-release cysteamine bitartrate for cystinosis
patients, but with a more convenient dosing schedule and potentially improved
tolerability.'
The randomized, crossover design of the pivotal Phase 3 clinical trial is a
result of discussions with the U.S. Food and Drug Administration ('FDA')
through which the FDA provided significant guidance on trial protocol design,
clinical endpoints, and statistical analyses plans. The primary endpoint of the
multi-center, global clinical trial is the steady-state white blood cell
('WBC') cystine levels of patients taking DR Cysteamine compared to
immediate-release cysteamine bitartrate. Secondary endpoints are the safety and
tolerability of DR Cysteamine and the comparability of steady-state PK of DR
Cysteamine and immediate-release cysteamine bitartrate.
'DR Cysteamine has the potential to address two critical issues with the
current treatment regimen for cystinosis patients, which result in sub-optimal
disease control in many of them. I believe this new formulation of cysteamine
bitartrate could greatly improve the quality of life and long-term health
outcomes of these patients,' said Craig Langman, M.D., The Isaac A Abt MD
Professor of Kidney Diseases at the Feinberg School of Medicine, Northwestern
University, and Lead Investigator in Raptor's Phase 3 clinical trial.
In November 2009, Raptor completed its Phase 2b clinical trial of DR Cysteamine
in cystinosis. DR Cysteamine demonstrated improved tolerability and the
potential to reduce total daily dosageand administration frequency compared to
immediate-release cysteamine bitartrate. Immediate-release cysteamine
bitartrate is the only drug therapy approved for marketing by the FDA and
European Medicines Agency ('EMA') for this indication. Despite being the
standard of care, gastrointestinal side effects and a strict around-the-clock,
every 6 hour dosing schedule for immediate-release cysteamine bitartrate create
tolerability and compliance issues for cystinosis patients that DR Cysteamine
is designed to address.
About Nephropathic Cystinosis
Nephropathic cystinosis is an inborn metabolic error characterized by the
abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to
treat 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 gastrointestinal side effects associated with immediate-release
cysteamine bitartrate, which is approved for sale by the FDA and EMA to treat
nephropathic cystinosis, a rare, genetic lysosomal storage disease.
In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diego for 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
Raptor expects over 30 patients to complete the eight-week study protocol; that
the patients enrolled in the Phase 3 clinical trial will enroll in a long-term
follow-on study where they continue to receive DR Cysteamine twice daily for
the extent of the study; that Raptor will meet its goal of reporting top line
data from the Phase 3 clinical trial in the first quarter of 2011; that Raptor
believes that twice-daily DR Cysteamine has the potential to be as efficacious
as immediate-release cysteamine bitartrate for cystinosis patients, but with a
more convenient dosing schedule and potentially improved tolerability; that DR
Cysteamine has the potential to address two critical issues with the current
treatment regimen for cystinosis patients; that DR Cysteamine could greatly
improve the quality of life and long-termhealth outcomes of cystinosis
patients; that patient compliance is paramount to improving therapeutic control
and achieving optimal long-term treatment outcome; 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 January 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
24.01.2011 Dissemination of a Corporate News, transmitted by DGAP -
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The issuer is solely responsible for the content of this announcement.
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Language: English
Company: Raptor Pharmaceutical Corp.
United States
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ISIN: US75382F1066
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Datum: 24.01.2011 - 12:30 Uhr
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News-ID 334119
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