Raptor Pharmaceutical Corp. Announces Publication of Results from Phase 2a Trial of DR Cysteamine for Treatment of Cystinosis

By Raptor Pharmaceutical Corp., PRNE
Sunday, January 10, 2010

Potential to Improve Treatment with Less Frequent Administration and Better Tolerability

NOVATO, California, January 11 - Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP),
today announced the publication of results from a Phase 2a clinical trial of
a prototype formulation of its proprietary delayed-release cysteamine
bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis
("cystinosis"). Ranjan Dohil, M.D., Professor of Pediatrics at the University
of California, San Diego
, was lead author of the study to be published in the
Journal of Pediatrics and available online at
www.ncbi.nlm.nih.gov/pubmed/19775699?itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSum&ordinalpos=1.

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

The Phase 2a study demonstrated proof-of-concept for DR Cysteamine, which
is Raptor's proprietary, delayed-release, enteric-coated microbead
formulation of immediate release cysteamine bitartrate contained in a gelatin
capsule. Immediate-release cysteamine bitartrate ("IR Cysteamine") is the
current standard of care for treating cystinosis. The results indicated that
when given twice daily, the prototype DR Cysteamine formulation was effective
at maintaining low white blood cell ("WBC") cystine levels (<2 nmol
half-cystine/mg protein) in subjects with cystinosis. Results also indicated
that the prototype DR Cysteamine effectively maintained trough WBC cystine
levels within a satisfactory range when patients received approximately 60%
of the previous total daily dose of IR Cysteamine.

Dr. Dohil stated, "We are seeking to improve tolerability and reduce
dosing frequency requirements of IR Cysteamine which have been documented
challenges for cystinosis patients, leading to widely reported instances of
poor treatment compliance. We believe the results of the Phase 2a study bring
us significantly closer to a potential treatment solution for cystinosis
patients. The results from our trial indicate prototype delayed-release
cysteamine formulation lead to improved tolerability and efficacy when
administered twice-daily and at a lower total daily dose than IR Cysteamine.
Based on these results, I believe that Raptor's final DR Cysteamine
formulation has the potential to improve compliance and long-term treatment
outcomes for cystinosis patients."

Based on these Phase 2a results, Raptor conducted a Phase 2b clinical
trial using its final commercial formulation of DR Cysteamine and recently
announced the following top-line Phase 2b results: DR Cysteamine demonstrated
improved tolerability and the potential to reduce total daily dosage and
administration frequency compared to IR Cysteamine.

    - Pharmacokinetic evaluation showed that DR Cysteamine had a terminal
      half-life more than three times longer than the terminal half-life
      of IR Cysteamine.
    - Twice-daily DR Cysteamine may achieve the same pharmacodynamic result
      while using a total daily dose 30% lower than IR Cysteamine
      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 IR Cysteamine.
    - The proprietary, final formulation of DR Cysteamine confirmed earlier
      clinical trials conducted by Dr. Dohil using an enteric-coated
      prototype formulation of cysteamine bitartrate, which was funded by the
      Cystinosis Research Foundation ("CRF").

During the first quarter of 2010, Raptor plans to meet with the Food and
Drug Administration ("FDA") and European Medicines Agency ("EMEA") to discuss
plans for a repeat-dose, pivotal, Phase 3 clinical trial in cystinosis
patients. Upon receiving FDA and EMEA agreements on protocol, Raptor intends
to initiate its Phase 3 clinical trial at multiple sites in the US and
Europe.

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 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 the results of the Phase 2a study bring Raptor significantly
closer to a potential treatment solution for cystinosis patients; that
Raptor's DR Cysteamine formulation has the potential to improve compliance
and long-term treatment outcomes for this highly unmet medical need; that
twice-daily DR Cysteamine may achieve the same pharmacodynamic result while
using a total daily dose 30% lower than IR Cysteamine administered four times
daily; that Raptor intends to initiate its Phase 3 clinical trial at multiple
sites in the US and Europe; 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 and the joint proxy statement/prospectus on Form S-4
filed with the SEC on August 19, 2009, all of which are available free of
charge on the SEC's web site at 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:

    Kim R. Tsuchimoto, CFO
    +1-415-382-1390
    ktsuchimoto@raptorpharma.com

    The Ruth Group

    Sara Ephraim Pellegrino (investors)
    +1-646-536-7002
    spellegrino@theruthgroup.com

    Janine McCargo (media)
    +1-646-536-7033
    jmccargo@theruthgroup.com

Kim R. Tsuchimoto, CFO of Raptor Pharmaceutical Corp., +1-415-382-1390, or ktsuchimoto at raptorpharma.com; Sara Ephraim Pellegrino, investors, +1-646-536-7002, or spellegrino at theruthgroup.com, Janine McCargo, media, +1-646-536-7033, or jmccargo at theruthgroup.com, both of The Ruth Group for Raptor Pharmaceutical Corp.

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