DGAP-News: Raptor Pharmaceutical Corp. Begins Dosing in Phase 2b Trial of RP103 for Non-Alcoholic Steatohepatitis ('NASH')

(firmenpresse) - Raptor Pharmaceutical Corp.

25.06.2012 12:30
---------------------------------------------------------------------------

NOVATO, Calif, 2012-06-25 12:30 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP), today
announced that a first patient has been dosed in its Phase 2b clinical trial
evaluating the safety and potential efficacy of RP103 as a potential treatment
of non-alcoholic steatohepatitis ('NASH'), an advanced form of non-alcoholic
fatty liver disease ('NAFLD') in children. The patient was dosed at the
Clinical&Translational Research Institute at the University of California,
San Diego ('UC San Diego'). RP103 is Raptor's proprietary delayed-release
microbead formulation of cysteamine bitartrate.

'NAFLD is the most common serious complication of childhood obesity. We are
seeing a concerning increase in the number of adolescents with advanced liver
disease, creating an urgent need to find an effective treatment for NAFLD,'
said Jeffrey Schwimmer, M.D., the Director of the Fatty Liver Clinic at Rady
Children's Hospital San Diego, and the Principal Investigator for the NASH
Clinical Research Network Pediatric Clinical Center at UC San Diego. 'The
results of a small pilot study were sufficiently encouraging, and we are
excited about the opportunity to test cysteamine in the form of RP103 as a
treatment for NAFLD in children in this large, national study.'

Raptor is conducting this trial within a cooperative research and development
agreement with the National Institute of Diabetes and Digestive and Kidney
Diseases ('NIDDK'), part of the National Institutes of Health. The trial,
called Cysteamine Bitartrate Delayed-Release for the Treatment of Non-alcoholic
Fatty Liver Disease in Children ('CyNCh'), is expected to enroll a total of 160
pediatric participants at ten U.S. centers in the NIDDK-sponsored NASH Clinical

Research Network. NIDDK and Raptor are sharing the costs to conduct the CyNCh
clinical trial.

The primary objective of this randomized, multicenter, double-blind,
placebo-controlled Phase 2b clinical trial is to evaluate whether 52 weeks of
treatment with RP103 in children reverses damage caused by NASH as measured by
changes in NAFLD Activity Score ('NAS'), a histological rating scale of disease
activity. Secondary endpoints will include blood markers for liver health
including alanine transaminase ('ALT') and aspartate transaminase ('AST'), as
well as safety and tolerability.

'The first patient dosed is an important milestone and we are pleased that the
CyNCh clinical trial is underway,' said Ted Daley, President of Raptor. 'Our
collaboration with NIDDK and its NASH Clinical Research Network are invaluable
resources, given our clinical objectives, and we are excited to advance our
program in close partnership with these top NASH clinical investigators.'

The CyNCh clinical trial follows positive results of an open-label Phase 2a
clinical trial which was conducted under a collaboration agreement between
Raptor and the University of California, San Diego ('UC San Diego') at UC San
Diego's General Clinical Research Center. The Phase 2a clinical trial involved
children with biopsy-confirmed diagnosis of moderate to severe NAFLD and
baseline ALT and AST measurements at least twice that of normal levels. These
patients received a prototype formulation of RP103 twice daily for six months,
followed by a six-month post-treatment monitoring period. All patients showed
marked decline in ALT and AST levels during the treatment period with 7 of 11
patients achieving a greater than 50 percent reduction and 6 of 11 patients
with reductions to within normal range. The reductions in ALT and AST were
largely sustained during the 6 month post-treatment phase. Other important
liver function markers showed positive trends with cytokeratin levels
decreasing by an average of 45 percent and adiponectin increasing by 35 percent
during the treatment period.

About NASH

NASH is a more aggressive form of NAFLD that affects approximately 2 to 5
percent of Americans. It occurs in patients who drink little or no alcohol, and
is the most common cause of chronic liver disease in North America. Although
most patients are asymptomatic and feel healthy in early phases of the disease,
NASH causes decreased liver function and can lead to cirrhosis, liver failure
and end-stage liver disease. While 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. There are no currently approved drug therapies for NASH; patients
are limited to lifestyle changes such as diet, exercise and weight reduction to
manage the disease.

About Cysteamine and RP103

RP103 is Raptor's proprietary delayed release oral medication currently being
investigating in several indications. RP103 is an enteric coated, microbead
formulation of cysteamine bitartrate.

In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diego for the development of RP103 and other
forms of cysteamine for the potential treatment of non-alcoholic
steatohepatitis ('NASH') currently in a Phase 2b clinical trial in the U.S.,
described above, Huntington's Disease currently in the Phase 2/3 clinical trial
in France and for the development of RP103 for nephropathic cystinosis which
Raptor has recently filed for marketing approval in the U.S. and E.U. The U.S.
Food and Drug Administration ('FDA') has accepted for filing Raptor's New Drug
Application ('NDA') for RP103 for the potential treatment of nephropathic
cystinosis and assigned the user fee goal date of January 30, 2013. Raptor's
E.U. marketing application of RP103 for the potential treatment of nephropathic
cystinosis is under review by the EMA, and Raptor expects a decision in the
first half of calendar 2013. In 2010, the Company acquired an exclusive
worldwide license to intellectual property covering the use of transglutaminase
inhibitors, a class of molecules chemically similar to cysteamine, in the
potential treatment of Huntington's Disease and other neurological disorders
from the Weizmann Institute of Science in Israel and Niigata University in
Japan. Raptor has been granted orphan product designation for RP103 for the
potential treatment for nephropathic cystinosis by the European Medicines
Agency and FDA and for the potential treatment of Huntington's Disease by the
FDA.

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 www.raptorpharma.com.

The Raptor Pharmaceutical Corp. logo is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=7180

FORWARD LOOKING STATEMENTSThis 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
NIDDK will be able to complete enrollment and complete the Phase 2b clinical
trial; that Raptor will be able to supply clinical material and funding for the
Phase 2b clinical trial; if the Phase 2b clinical trial is completed, that the
resulting clinical trial results will meet its primary objectives and secondary
clinical endpoints; that the Phase 2b clinical trial will emulate the results
of the Phase 2a open label study; that RP103 will prevent significant GSH
depletion and potentially reverse NASH-related liver damage; that the FDA and
EMA will deliver a decision regarding marketing approval of RP103 for the
potential treatment of nephropathic cystinosis on January 30, 2013 or the first
half of calendar 2013, respectively; 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 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



25.06.2012 Dissemination of a Corporate News, transmitted by DGAP -
a company of EquityStory AG.
The issuer is solely responsible for the content of this announcement.

DGAP's Distribution Services include Regulatory Announcements,
Financial/Corporate News and Press Releases.
Media archive at www.dgap-medientreff.de and www.dgap.de

---------------------------------------------------------------------------

Language: English
Company: Raptor Pharmaceutical Corp.


United States
Phone:
Fax:
E-mail:
Internet:
ISIN: US75382F1066
WKN:

End of Announcement DGAP News-Service

---------------------------------------------------------------------------

Themen in dieser Pressemitteilung:


Unternehmensinformation / Kurzprofil:
Leseranfragen:
PresseKontakt / Agentur:
Bereitgestellt von Benutzer: EquityStory
Datum: 25.06.2012 - 06:30 Uhr
Sprache: Deutsch
News-ID 1127167
Anzahl Zeichen: 0

contact information:
Contact person:
Town:

Phone:

Kategorie:

Business News

Anmerkungen:


Diese Pressemitteilung wurde bisher 160 mal aufgerufen.


Die Pressemitteilung mit dem Titel:
"DGAP-News: Raptor Pharmaceutical Corp. Begins Dosing in Phase 2b Trial of RP103 for Non-Alcoholic Steatohepatitis ('NASH')
"
steht unter der journalistisch-redaktionellen Verantwortung von

Raptor Pharmaceutical Corp. (Nachricht senden)

Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).

Alle Meldungen von Raptor Pharmaceutical Corp.