Raptor Pharmaceutical Reports Positive Interim Phase 2a Clinical Data in Non-Alcoholic Steatohepatitis (NASH)
October 12 2009 - 6:30AM
PR Newswire (US)
Primary Endpoint Achieved in Completed Treatment Phase NOVATO,
Calif., Oct. 12 /PRNewswire-FirstCall/ -- Raptor Pharmaceutical
Corp. ("Raptor" or the "Company") (NASDAQ:RPTPD), announced
positive findings from the completed treatment phase of its
open-label Phase 2a clinical trial of delayed-release cysteamine
bitartrate ("DR Cysteamine") in adolescent patients with
non-alcoholic steatohepatitis ("NASH"), a progressive form of liver
disease believed to affect 2% to 5% of the U.S. population. At the
completion of the initial six-month treatment phase, the study
achieved the primary endpoint: mean blood levels of alanine
aminotransferase ("ALT"), a common biomarker for NASH, were reduced
by over 50%. Additionally, over half of the study participants had
achieved normalized ALT levels by the end of the treatment phase.
(Logo: http://www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO )
There are no currently approved drug therapies for NASH, and
patients are limited to lifestyle changes such as diet, exercise
and weight reduction to manage the disease. DR Cysteamine
represents an important potential treatment option for patients
with NASH. Although NASH is most common in insulin-resistant obese
adults with diabetes and abnormal serum lipid profiles, its
prevalence is increasing among juveniles as obesity rates rise
within this patient population. Although most patients are
asymptomatic and feel healthy, NASH causes decreased liver function
and can lead to cirrhosis, liver failure and end-stage liver
disease. Under a collaboration agreement between Raptor and the
University of California, San Diego ("UC San Diego"), the
open-label Phase 2a trial of a prototype formulation of DR
Cysteamine is being conducted at UC San Diego's General Clinical
Research Center and entails six months of treatment followed by a
six-month post-treatment monitoring period. Eligible patients with
baseline ALT and aspartate aminotransferase ("AST") measurements at
least twice that of normal levels were enrolled to receive
twice-daily, escalating oral doses of up to 1,000 mg of DR
Cysteamine. The trial currently has enrolled eleven NASH patients
between 11-18 years old. No major adverse events were reported
during the six-month treatment phase. Trial subjects continue to be
monitored during the six-month post-treatment period currently
underway. Full results are being submitted for peer review by
Raptor and UC San Diego, and are expected to be presented in 2010.
Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UC San
Diego and principal investigator for the NASH study, stated, "We
were encouraged by the results of this study. The degree of ALT and
AST reductions are indicative of likely improvements in severity of
fatty liver damage. The trial results are consistent with ALT and
AST reductions normally seen in patients that achieve at least 10%
weight loss, even though study participants did not show a
significant change in body mass index. DR Cysteamine appears to be
a promising candidate for NASH and we look forward to further
analyzing these patients during the post-treatment phase." Raptor's
chief medical officer, Patrice Rioux, M.D., Ph.D., said, "These
interim results have established proof-of-concept and support
further clinical development of DR Cysteamine in NASH. This is an
area of significant unmet need, especially with growing numbers of
obese children diagnosed with the disorder. While the clinical
hurdle is usually high for studies in children and adolescents, we
are satisfied with the long-term safety demonstrated in this age
group by the currently-marketed immediate-release cysteamine
bitartrate formulation. This safety track record, coupled with our
interim Phase 2a efficacy data, gives us a great sense of
encouragement as we advance DR Cysteamine through the clinic."
Under a license with UC San Diego, Raptor is developing DR
Cysteamine for cystinosis, NASH and other potential therapeutic
indications. Cysteamine is known to be a scavenger of reactive
oxygen species and potent antioxidant, most likely through its
ability to increase intracellular glutathione levels. Cysteamine
has also demonstrated potential efficacy in preclinical and
clinical studies in Huntington's Disease, Batten Disease and other
indications. About Raptor Pharmaceutical Corp. Raptor
Pharmaceutical Corp. (NASDAQ:RPTPD) ("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 a non-opioid solution designed to
potentially treat chronic pain 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
http://www.raptorpharma.com/. 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: Raptor's and UC San Diego's
ability to complete the clinical trial in NASH patients, DR
Cysteamine's ability to treat NASH, ALT and AST as a biomarker to
determine the efficacy of a treatment for NASH, Raptor's ability to
further develop DR Cysteamine in NASH and other indications. 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 the joint proxy statement/prospectus on Form S-4 filed
with the SEC on August 19, 2009; Raptor's annual report on Form
10-K filed with the SEC on March 27, 2009; Raptor's quarterly
report on Form 10-Q filed with the SEC on August 11, 2009; Raptor's
wholly-owned subsidiary's, Raptor Pharmaceuticals Corp. ("RPC")
Registration Statement on Form S-1, as amended, that was declared
effective on August 7, 2008; RPC's annual report on Form 10-K filed
with the SEC on October 30, 2008, as amended by that Form 10-K/A
filed with the SEC on December 23, 2008; and RPC's quarterly report
on Form 10-Q filed with the SEC on July 15, 2009, 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. For more information, please contact:
Karl Cahill, Investor Relations (858) 531-6100 The Ruth Group Sara
Ephraim Pellegrino (investors) (646) 536-7002 Janine McCargo
(media) (646) 536-7033
http://www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGODATASOURCE:
Raptor Pharmaceutical Corp. CONTACT: Karl Cahill, Investor
Relations, +1-858-531-6100, ; or Sara Ephraim Pellegrino
(investors), +1-646-536-7002, or Janine McCargo (media),
+1-646-536-7033, , both of The Ruth Group Web Site:
http://www.raptorpharma.com/
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