DGAP-News: Raptor Pharmaceutical Corp. Announces Positive Data on RP103 Extension Study Presented at Kidney Week 2012
(firmenpresse) - Raptor Pharmaceutical Corp.
05.11.2012 12:30
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NOVATO, Calif., 2012-11-05 12:30 CET (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP),
announced that 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, presented on Saturday,
November 3rd a 'Late Breaking' poster entitled 'Extended Treatment with RP103
in Patients with Nephropathic Cystinosis' at Kidney Week 2012.
The poster presented data from Raptor's extension study in which 40 patients
completing the Phase 3 clinical trial elected to continue on RP103 (cysteamine
bitartrate delayed-release) treatment and to be monitored for white blood cell
('WBC') cystine levels, plasma cysteamine levels, estimated glomerular
filtration rate ('eGFR', an indicator of kidney function), quality of life
(PedsQL) and use of antacids.
The data from the extension study demonstrated that patients with nephropathic
cystinosis using RP103 for up to 24 months, maintained WBC cystine levels
continuously below the therapeutic target. After 20 months of treatment, the
total daily dose of RP103 given every 12 hours needed to achieve optimal WBC
cystine levels was on average 72% of the initial immediate release cysteamine
bitartrate dose. Kidney function was well preserved over the length of the
study, as indicated by steady eGFR levels. Additionally, the study showed that
patients taking RP103 are able to reduce concomitant use of anti-acid
medications by nearly half, with 22% of patients taking proton pump inhibitors
('PPI') or histamine 2 ('H2') blockers during the extension vs. 42% upon
initially entering the Phase 3 study when they are taking immediate-release
cysteamine bitartrate.
The extension study also demonstrated significant improvements in all measured
quality of life parameters. From the study onset, the PedsQL 4.0 scores (an
instrument for measuring health-related quality of life in children and
adolescents ages 2 to 18) improved in all patients, including the Total
Functioning Score reflecting a p<0.001 statistical significance, and the Social
Performance Score reflecting a p<0.001 statistical significance. Social
function can be difficult for cystinosis patients treated with cysteamine
bitartrate because of the strong rotten-egg odor associated with the drug's
metabolism and from its other side effects.
Dr. Langman said, 'Cystine reduction therapy is effective but is also a
lifelong commitment. Even brief delays in daily therapy permit toxic cystine
accumulation, exposing tissues to renewed and progressive deterioration. The
currently approved immediate release formulation of cysteamine bitartrate taken
every six hours is effective but difficult to use over the long-term. RP103,
given every 12 hours, offers a better long-term adherence strategy, which is
important in a disease where patient adherence to the strict dosing schedule
has a direct impact on outcomes that are quite severe. Just as important for
the patients and families who make the lifelong commitment to cystine reduction
therapy are the quality of life measurements, which improved for patients on
RP103, across the board.'
About Cystinosis andRP103
Cystinosis is a rare, life-threatening metabolic disorder that causes systemic
toxic cystine accumulation. Toxic cystine accumulation leads to progressive and
irreversible tissue damage and multi-organ failure including renal failure,
blindness, CNS damage, respiratory deficiencies, muscle wasting, and premature
death. Cystinosis is usually diagnosed in the first years of life and requires
lifelong therapy. Left untreated, the disease is fatal by the end of the first
decade of life. Daily cystine depletion is the primary treatment strategy for
cystinosis. Despite a life-long requirement for cystine-depleting agent use to
help preserve multi-organ function, a cumulative inability to maintain adequate
cystine control results in the rapid deterioration of kidney function and
ensuing need for kidney transplantation.
RP103 is an oral, delayed and extended-release medication containing
enteric-coated spheronized micro-beads of cysteamine bitartrate. PROCYSBI(tm) is
the branded name of RP103 for the potential treatment of cystinosis. The NDA
and MAA for RP103 have been submitted and the NDA has been assigned a PDUFA
date of January 30, 2013. RP103 is also in clinical development for the
potential treatment of Huntington's disease and non-alcoholic steatohepatitis
('NASH'). In cystinosis patients, RP103 may reduce cellular toxicity by
continuously removing cystine from the lysosome. RP103, which can also cross
the blood-brain barrier, was engineered specifically to allow release of
cysteamine bitartrate micro-spheres in the duodenum for optimal absorption
while simultaneously enabling administration every 12-hours for the potential
treatment of cystinosis.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. ('Raptor') (Nasdaq:RPTP) seeks to research,
develop, and provide access to medicines that improve life for patients with
severe and rare disorders. Raptor currently has product candidates in clinical
development designed to potentially treat nephropathic cystinosis,
non-alcoholic steatohepatitis, Huntington's Disease, and aldehyde dehydrogenase
deficiency.
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 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 the
FDA and EMA will deliver a decision regarding marketing approval of RP103 for
the potential treatment of cystinosis on January 30, 2013 or the first half of
calendar 2013, respectively; that RP103 will maintain WBC cystine levels
continuously below the therapeutic target; that patients taking RP103 will be
able to reduce concomitant use of anti-acid medications compared to when taking
immediate-release cysteamine bitartrate; that RP103 will improve quality of
life parameters; that RP103 may reduce cellular toxicity by continuously
removing cysteine from the lysosome 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 July 10, 2012; 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(at)troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo(at)evcgroup.com
News Source: NASDAQ OMX
05.11.2012 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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