Raptor Pharmaceuticals Doses First Patient in Phase 2b Clinical Trial of DR Cysteamine for Cystinosis



    NOVATO, Calif., June 1 /CNW/ -- Raptor Pharmaceuticals Corp. ("Raptor" or
the "Company") (OTC Bulletin Board:   RPTP), today announced that it has dosed
the first patient in its Phase 2b clinical trial, conducted in collaboration
with the University of California, San Diego ("UCSD"), to evaluate Raptor's
proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") capsules
in nephropathic cystinosis ("cystinosis"), a rare genetic lysosomal storage
disease. Under Raptor's open Investigational New Drug application ("IND"),
UCSD is performing the Raptor-sponsored trial at its General Clinical Research
Center.
    

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

    
    The Phase 2b clinical trial will enroll up to six cystinosis patients
with a history of good compliance using the currently available
immediate-release form of cysteamine bitartrate, the only drug cleared for
marketing by the U.S. Food and Drug Administration ("FDA") and European
Medicines Agency ("EMEA") to treat cystinosis. The clinical trial will
evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of a
single dose of DR Cysteamine in patients.  Data from the study are expected in
the third quarter of 2009.  Raptor plans to follow the Phase 2b clinical study
with a pivotal, Phase 3 clinical study in cystinosis patients anticipated to
commence in the fourth quarter of 2009.

    DR Cysteamine is Raptor's proprietary enteric-coated micro-bead
formulation of cysteamine bitartrate contained in a gelatin capsule and
designed for increased absorption, less frequent dosing and improved
tolerability relative to the current standard of care. Published studies have
shown that fewer than  25% of patients taking immediate-release cysteamine
bitartrate are fully-compliant due to the drug's strict every-six hour dosing
schedule and dose-limiting side effects, including gastrointestinal ("GI")
problems such as stomach aches, nausea and vomiting.

    Patrice Rioux, M.D., Ph.D., Raptor's Chief Medical Officer stated, "We
are pleased that enrollment is now underway in our Phase 2b clinical trial in
cystinosis.  This is an underserved patient population and there is a
compelling need for an improved treatment option.  Many patients are unable to
take adequate levels of the drug to halt the progression of their disease,
which gradually destroys the organs in the body. We believe DR Cysteamine
could have positive effects on the long-term health of cystinosis patients as
well as the quality of life for patients, their families and caregivers."

    Bruce Barshop, M.D., Ph.D., Professor of Clinical Pediatrics at UCSD and
principal investigator for the cystinosis clinical trial, commented, "The
initiation of this cystinosis trial offers us an opportunity to evaluate the
potential for an improved treatment invented here at UCSD and further
developed by Raptor. We're pleased to collaborate with Raptor in this effort
to advance the standard of care in cystinosis."
    

    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
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
for use of cysteamine and DR Cysteamine for the treatment of non-alcoholic
steatohepatitis ("NASH"), a progressive liver disease believed to affect 2-5%
of the U.S. population.  In October, 2008 Raptor announced the initiation of a
Phase 2a clinical study of cysteamine bitartrate in NASH patients, in
collaboration with UCSD.  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 Pharmaceuticals Corp.

    
    Raptor Pharmaceuticals Corp. ("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 to treat nephropathic
cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease
("HD"), and aldehyde dehydrogenase ("ALDH2") deficiency.

    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 be able to fully enroll and complete the clinical trial in
cystinosis patients; that data will be available in the third quarter of 2009;
 that Raptor will start a Phase 3 clinical trial in cystinosis patients in the
fourth quarter of 2009, if at all; that DR Cysteamine will increase
absorption, require less frequent dosing or improve tolerability relative to
the current standard of care; that DR Cysteamine will have a positive effect
on cystinosis patients and increase their quality of life;  and that any of
the Company'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 its Registration Statement
on Form S-1, as amended, that was declared effective on August 7, 2008; its
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 its Form 10-Q
filed with the SEC on April 13, 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 the Company'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




    




For further information:

For further information: Karl Cahill, Investor Relations,
+1-858-531-6100, kcahill@raptorpharma.com; or 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
Web Site: http://www.raptorpharma.com

Organization Profile

RAPTOR PHARMACEUTICAL CORP.

More on this organization


Custom Packages

Browse our custom packages or build your own to meet your unique communications needs.

Start today.

CNW Membership

Fill out a CNW membership form or contact us at 1 (877) 269-7890

Learn about CNW services

Request more information about CNW products and services or call us at 1 (877) 269-7890