DGAP-News: Raptor Pharmaceutical Corp. to Present at Cystinosis Research Foundation Day of Hope Fami
(businesspress24) - Raptor Pharmaceutical Corp.
12.04.2012 12:30
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NOVATO, Calif., 2012-04-12 12:30 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP) today
announced that Ted Daley, President, will present an update on its
investigational drug candidate, Cysteamine Bitartrate Delayed-Release Capsules
(RP103), for the potential treatment of nephropathic cystinosis at the
Cystinosis Research Foundation ('CRF') Day of Hope Family Conference to be held
from April 20-21. The presentation will include data from the Raptor's Phase 3
clinical trial of RP103 for the potential treatment of cystinosis as well as a
regulatory update. Mr. Daley's presentation will take place at Balboa Bay Club
in Newport Beach, California on Friday, April 20th at 11:30 a.m. Pacific Time.
Mr. Daley stated, 'Raptor always looks forward to these opportunities to share
our progress with cystinosis families as part of our ongoing mission to serve
this extraordinary community. It will be especially gratifying to be able to
report to the Cystinosis Research Foundation Family Conference that we have
recently filed our New Drug Application for RP103, since it was the CRF that
funded the original clinical studies that first demonstrated the potential
benefits of RP103.'
'This is a remarkable time for the cystinosis community,' said Nancy Stack,
President and co-founder of the CRF. 'We believe that we are closer than ever
to FDA approval of a delayed-release formulation of cysteamine, and researchers
continue to make important discoveries about cystinosis gene and stem cell
therapies. We feel fortunate that we can again come together as families to
support each other and recognize the brilliant researchers who have dedicated
their lives to our children and celebrate the significant advances they have
made in cystinosis research.'
About Nephropathic Cystinosis
Nephropathic cystinosis, an orphan disease, is estimated to effect a population
of 2,000 patients worldwide, including 500 patients in the U.S. and 800
patients in Europe. Cystinosis patients have inherited a defective cystine
transporter gene which results in body-wide cellular toxicity resulting from
the abnormal buildup of the amino acid cystine in the lysosomes. Cystinosis is
usually diagnosed in the first year of life and requires lifelong therapy.
Cystine crystals accumulate in various tissues and organs, including the
kidneys, brain, liver, thyroid, pancreas, muscles and eyes. Left untreated, the
disease is fatal by the first decade of life. RP103 reduces cellular toxicity
by continuously removing cystine from the lysosome.
About Cysteamine and RP103
RP103 is Raptor's proprietary delayed and extended release oral medication
designed to potentially treat the underlying metabolic cause of cystinosis.
RP103 is an enteric coated, microbead formulation of cysteamine bitartrate that
has been formulated to be sprinkled onto food for administration to patients
too young to take oral capsules. Raptor has been granted orphan product
designation for RP103 by the EMA and FDA.
In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diego for the development of RP103 for
nephropathic cystinosis and for cysteamine for other potential indications
including Huntington's Disease currently in a Phase 2/3 clinical trial in
France and non-alcoholic steatohepatitis ('NASH') currently in a Phase 2b
clinical trial in the US.
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 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
FDA will approve a delayed-release formulation of cysteamine, if at all; that
researchers continue to make important discoveries about cystinosis gene and
stem cell therapies, if at all; 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 and Amendment No. 1 to its Annual Report on
Form 10-K/A filed with the SEC on November 14, 2011 and December 19, 2011,
respectively; and Raptor's quarterly report on Form 10-Q filed with the SEC on
April 9, 2012, 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.comEVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo(at)evcgroup.com
News Source: NASDAQ OMX
12.04.2012 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,
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Media archive at www.dgap-medientreff.de and www.dgap.de
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Language: English
Company: Raptor Pharmaceutical Corp.
United States
Phone:
Fax:
E-mail:
Internet:
ISIN: US75382F1066
WKN:
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