Raptor Pharmaceutical Completes DR Cysteamine Phase 2b Clinical Trial in Cystinosis

Raptor Pharmaceutical Completes DR Cysteamine Phase 2b Clinical Trial in
Cystinosis
Goal of Trial Achieved Demonstrating Potentially Less Frequent Administration
Using Lower Daily Dose

NOVATO, Calif., Nov. 23 /PRNewswire-FirstCall/ -- Raptor Pharmaceutical Corp.
("Raptor" or the "Company") (Nasdaq: RPTP), today announced results from a
Phase 2b clinical trial of its proprietary delayed-release cysteamine
bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis
("cystinosis"). The trial, conducted at the University of California, San
Diego ("UCSD") General Clinical Research Center, evaluated safety,
tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR
Cysteamine in nine cystinosis patients.

(Logo: http://www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO )

Phase 2b Clinical Trial Highlights:
-- DR Cysteamine demonstrated improved tolerability and the potential to
reduce total daily dosage and administration frequency compared to
immediate-release cysteamine bitartrate.
-- Pharmacokinetic evaluation showed that DR Cysteamine had a terminal
half-life more than three times longer than the terminal half-life of
immediate-release cysteamine bitartrate capsules.
-- Twice-daily DR Cysteamine may achieve the same pharmacodynamic result
while using a daily dose 30% lower than immediate-release cysteamine
bitartrate capsules administered four times daily.
-- No adverse events recorded during the clinical trial were determined
by
the principal investigator to be possibly or probably related to DR
Cysteamine. Nine adverse events recorded in the clinical trial were
determined to be possibly or probably related to immediate-release
cysteamine bitartrate.

Raptor plans to meet with the Food and Drug Administration ("FDA") and
European Medicines Agency ("EMEA") to discuss the results of its phase 2b
study and its plans for a repeat-dose, pivotal, phase 3 clinical trial in
cystinosis patients. Upon receiving FDA and EMEA concurrence, Raptor intends
to initiate its phase 3 clinical trial at multiple sites in the US and Europe
in the first quarter of 2010.
DR Cysteamine is Raptor's proprietary, enteric-coated micro-bead formulation
of immediate release cysteamine bitartrate capsules, the current standard of
care for treating cystinosis.
Bruce Barshop, M.D., Ph.D., Professor of Clinical Pediatrics at UCSD and
principal investigator for the cystinosis clinical trial of DR Cysteamine,
stated, "The excellent tolerability of DR Cysteamine, as demonstrated in this
study, represents a major step forward in potentially improving the treatment
of cystinosis. Raptor's formulation has the potential to improve dosing
compliance and long-term outcomes for cystinosis patients."
Raptor's phase 2b clinical trial followed earlier clinical trials with an
enteric-coated cysteamine prototype conducted by Ranjan Dohil, M.D., Associate
Professor of Pediatrics at UCSD and funded by the Cystinosis Research
Foundation ("CRF"). The CRF also supported Raptor's phase 2b clinical trial.
Patrice P. Rioux, M.D., Ph.D., chief medical officer of Raptor, said, "The
results from our phase 2b trial are consistent with previous clinical findings
from Dr. Dohil and support our plans to provide a twice-daily cysteamine
therapy for cystinosis patients. We look forward to continuing our development
program of DR Cysteamine in cystinosis with a phase 3 clinical trial."
Cystinosis is an inborn metabolic error characterized by the abnormal
transport of cystine, an amino acid, out of the lysosomes. Failure to treat
cystinosis can cause serious health consequences, including renal failure and
resultant kidney transplant, growth failure, rickets, 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 DR Cysteamine
DR Cysteamine is an enteric-coated micro-bead formulation of cysteamine
bitartrate. Raptor obtained an exclusive, worldwide license to DR Cysteamine,
as well as orphan drug designation from the FDA for DR Cysteamine for the
potential treatment of nephropathic cystinosis, through its December 2007
acquisition of Encode Pharmaceuticals. In March 2008, Raptor acquired an
exclusive worldwide license to intellectual property from the University of
California, San Diego ("UCSD") covering use of cysteamine and DR Cysteamine
for the potential treatment of non-alcoholic steatohepatitis ("NASH"), a
progressive liver disease believed to affect 2-5% of the U.S. population. In
October 2009, Raptor and UCSD announced positive interim data from the
six-month treatment phase of its Phase 2a clinical study of cysteamine
bitartrate in NASH patients. In November 2008 Raptor announced a collaboration
with Centre Hospitalier Universitaire d'Angers, in France, to study DR
Cysteamine in Huntington's Disease, a rare, genetic neurological 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 a non-opioid solution designed to potentially treat chronic
pain.

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.

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 will start a Phase 3 clinical trial in cystinosis patients at multiple
US and European sites in the first quarter of 2010, if at all; that DR
Cysteamine may allow for a 30% reduction in a patient's total daily cysteamine
dose compared to the current standard of care; that DR Cysteamine can be
administered in two doses per day instead of four, to achieve the same
pharmacodynamic result; that tolerability of DR Cysteamine, as demonstrated in
this study, potentially represents a major step forward in the care of
cystinosis patients; that Raptor's formulation could result in greatly
improved dosing compliance and better long-term outcomes for cystinosis
patients; and that any of Raptor's clinical and preclinical drug candidates
will result in approved therapeutics. 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 at raising funds to continue its development programs;
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; and that Raptor's products may not work as well as hoped or
worse, that the Company's products may harm recipients. 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 current report on
Form 8-K as filed with the SEC on November 17, 2009; 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; and
Raptor's quarterly report on Form 10-Q filed with the SEC on August 11, 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
kcahill@raptorpharma.com

The Ruth Group
Sara Ephraim Pellegrino (investors) / Janine McCargo (media)
(646) 536-7002 / (646) 536-7033
spellegrino@theruthgroup.com / jmccargo@theruthgroup.com

SOURCE Raptor Pharmaceutical Corp.

Karl Cahill, Investor Relations, +1-858-531-6100, kcahill@raptorpharma.com,
Sara Ephraim Pellegrino (investors), +1-646-536-7002,
spellegrino@theruthgroup.com, or Janine McCargo (media), +1-646-536-7033,
jmccargo@theruthgroup.com, both of The Ruth Group