Raptor Pharmaceutical Announces Publication of Phase 3 Results in the Clinical Journal of the American Society of Nephrology
Raptor Pharmaceutical Corp.
03.05.2012 19:15
---------------------------------------------------------------------------
Provides an Update on the Nephropathic Cystinosis Program
NOVATO, Calif., 2012-05-03 19:15 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP),
announced the online publication of an article titled, 'A Randomized Controlled
Crossover Trial with a Delayed Release Cysteamine Bitartrate in Nephropathic
Cystinosis: Effectiveness on WBC Cystine Levels and Comparison of Safety' in
the Clinical Journal of the American Society of Nephrology ('CJASN'). The CJASN
is one of the most respected peer reviewed scientific journals among kidney
related publications.
The article describes findings from Raptor's open-label, randomized,
controlled, crossover Phase 3 clinical trial, referred to as RP103-03, the
top-line results of which were reported by Raptor in July 2011, and presented
by the clinical investigators as a late breaking poster at the American Society
of Nephrology ('ASN') - Kidney Week 2011 in Philadelphia last November. The
Phase 3 clinical trial enrolled 43 patients and was powered to show that
Raptor's delayed-release formulation of cysteamine bitartrate, referred to as
RP103, taken every 12 hours, was non-inferior to the currently-available
immediate release cysteamine bitartrate treatment, Cystagon(r), taken every 6
hours, for maintenance of white blood cell ('WBC') cystine levels in patients
who were already well controlled on Cystagon(r). The clinical trial also showed
that RP103 met its primary endpoint at a lower total daily dose than Cystagon(r).
Craig B. Langman, M.D., Head of Kidney Diseases, and the Isaac A. Abt, M.D.,
Professor of Kidney Diseases and Tenured Professor of Pediatrics, Northwestern
University Feinberg School of Medicine, and lead author of the publication,
said, 'RP103 met the primary endpoint compared to Cystagon(r) at a less frequent
dosing schedule, as well as a reduced total daily dose. As to tolerability,
none of the severe adverse events reported in the study were considered
unexpected, given the symptoms associated with the disease itself. The nature
of the adverse events reported during the study was similar between the
Cystagon(r) and the RP103 treatment periods.'
Patrice P. Rioux, M.D., Ph.D., Chief Medical Officer of Raptor, said, 'Two
patients experienced 24% of the total gastrointestinal ('GI') adverse events
('AEs') recorded in the study, which accounted for 60% of the drug-related GI
AEs while on RP103. However, I'm pleased to note that following an initial
period of GI upset upon starting RP103, both of these patients have continued
to use RP103 in our extension study and have remained on RP103 for almost one
year. All but one patient out of all the patients who used proton pump
inhibitors ('PPIs') while on the Cystagon(r) arm, did not use PPIs on a regular
basis while on RP103. This may account for the higher total number of GI AEs
reported under RP103 vs. Cystagon(r).'
The RP103 voluntary extension study, referred to as RP103-04, is ongoing with
38 enrolled patients of the original 41 patients who completed the Phase 3
clinical trial. Thirty-two of the Phase 3 patients have been on RP103 for at
least 12 months with some patients now approaching 18 months on RP103. In
addition, enrollment in the extension study has been expanded to include 13
children under 6 years old, and 5 patients with a functioning kidney
transplant. The children are receiving their RP103 by sprinkling the capsule
contents onto applesauce or administered through a gastric tube. Raptor plans
to present data from the extension trial later this year.
In March, 2012, the European Medicines Agency ('EMA') validated Raptor's
Marketing Authorization Application for RP103 for the potential treatment of
nephropathic cystinosis. The MAA is now being reviewed by the EMA. Also in
March, Raptor submitted a New Drug Application ('NDA') to the U.S. Food and
Drug Administration ('FDA') seeking approval to market RP103 for the potential
treatment of nephropathic cystinosis. Raptor requested a Priority Review of the
NDA which, if granted by FDA, could lead to a decision for marketing approval
for RP103 for the potential treatment of cystinosis in the U.S., in the fourth
quarter of 2012.
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 that 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 years 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 end of 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. As demonstrated in the Phase 3 clinical trial
detailed in the publication in CJASN, the every 6 hour cysteamine dosing
treatment may be reduced substantially to twice daily on RP103.
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. Raptor has been granted orphan product designation
for RP103 for the potential treatment of nephropathic cystinosis by the EMA and
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 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 complete its extension trial or present data from its extension
trial later this year; that the every 6 hour cysteamine dosing treatment will
be reduced to twice daily on RP103; that FDA could render a decision regarding
marketing approval of RP103 for the potential treatment of cystinosis in the
U.S., in the fourth quarter of 2012; 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@troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo@evcgroup.com
News Source: NASDAQ OMX
03.05.2012 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
---------------------------------------------------------------------------
Raptor Pharmaceutical Corp.
03.05.2012 19:15
---------------------------------------------------------------------------
Provides an Update on the Nephropathic Cystinosis Program
NOVATO, Calif., 2012-05-03 19:15 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP),
announced the online publication of an article titled, 'A Randomized Controlled
Crossover Trial with a Delayed Release Cysteamine Bitartrate in Nephropathic
Cystinosis: Effectiveness on WBC Cystine Levels and Comparison of Safety' in
the Clinical Journal of the American Society of Nephrology ('CJASN'). The CJASN
is one of the most respected peer reviewed scientific journals among kidney
related publications.
The article describes findings from Raptor's open-label, randomized,
controlled, crossover Phase 3 clinical trial, referred to as RP103-03, the
top-line results of which were reported by Raptor in July 2011, and presented
by the clinical investigators as a late breaking poster at the American Society
of Nephrology ('ASN') - Kidney Week 2011 in Philadelphia last November. The
Phase 3 clinical trial enrolled 43 patients and was powered to show that
Raptor's delayed-release formulation of cysteamine bitartrate, referred to as
RP103, taken every 12 hours, was non-inferior to the currently-available
immediate release cysteamine bitartrate treatment, Cystagon(r), taken every 6
hours, for maintenance of white blood cell ('WBC') cystine levels in patients
who were already well controlled on Cystagon(r). The clinical trial also showed
that RP103 met its primary endpoint at a lower total daily dose than Cystagon(r).
Craig B. Langman, M.D., Head of Kidney Diseases, and the Isaac A. Abt, M.D.,
Professor of Kidney Diseases and Tenured Professor of Pediatrics, Northwestern
University Feinberg School of Medicine, and lead author of the publication,
said, 'RP103 met the primary endpoint compared to Cystagon(r) at a less frequent
dosing schedule, as well as a reduced total daily dose. As to tolerability,
none of the severe adverse events reported in the study were considered
unexpected, given the symptoms associated with the disease itself. The nature
of the adverse events reported during the study was similar between the
Cystagon(r) and the RP103 treatment periods.'
Patrice P. Rioux, M.D., Ph.D., Chief Medical Officer of Raptor, said, 'Two
patients experienced 24% of the total gastrointestinal ('GI') adverse events
('AEs') recorded in the study, which accounted for 60% of the drug-related GI
AEs while on RP103. However, I'm pleased to note that following an initial
period of GI upset upon starting RP103, both of these patients have continued
to use RP103 in our extension study and have remained on RP103 for almost one
year. All but one patient out of all the patients who used proton pump
inhibitors ('PPIs') while on the Cystagon(r) arm, did not use PPIs on a regular
basis while on RP103. This may account for the higher total number of GI AEs
reported under RP103 vs. Cystagon(r).'
The RP103 voluntary extension study, referred to as RP103-04, is ongoing with
38 enrolled patients of the original 41 patients who completed the Phase 3
clinical trial. Thirty-two of the Phase 3 patients have been on RP103 for at
least 12 months with some patients now approaching 18 months on RP103. In
addition, enrollment in the extension study has been expanded to include 13
children under 6 years old, and 5 patients with a functioning kidney
transplant. The children are receiving their RP103 by sprinkling the capsule
contents onto applesauce or administered through a gastric tube. Raptor plans
to present data from the extension trial later this year.
In March, 2012, the European Medicines Agency ('EMA') validated Raptor's
Marketing Authorization Application for RP103 for the potential treatment of
nephropathic cystinosis. The MAA is now being reviewed by the EMA. Also in
March, Raptor submitted a New Drug Application ('NDA') to the U.S. Food and
Drug Administration ('FDA') seeking approval to market RP103 for the potential
treatment of nephropathic cystinosis. Raptor requested a Priority Review of the
NDA which, if granted by FDA, could lead to a decision for marketing approval
for RP103 for the potential treatment of cystinosis in the U.S., in the fourth
quarter of 2012.
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 that 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 years 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 end of 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. As demonstrated in the Phase 3 clinical trial
detailed in the publication in CJASN, the every 6 hour cysteamine dosing
treatment may be reduced substantially to twice daily on RP103.
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. Raptor has been granted orphan product designation
for RP103 for the potential treatment of nephropathic cystinosis by the EMA and
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 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 complete its extension trial or present data from its extension
trial later this year; that the every 6 hour cysteamine dosing treatment will
be reduced to twice daily on RP103; that FDA could render a decision regarding
marketing approval of RP103 for the potential treatment of cystinosis in the
U.S., in the fourth quarter of 2012; 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@troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo@evcgroup.com
News Source: NASDAQ OMX
03.05.2012 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
---------------------------------------------------------------------------