Raptor Pharmaceutical Corp. Provides Program Update on RP103 (DR Cysteamine) for Nephropathic Cystinosis
Raptor Pharmaceutical Corp.
21.12.2011 12:30
---------------------------------------------------------------------------
On Track to File Marketing Applications in US and EU in the First Quarter of
2012
NOVATO, Calif., 2011-12-21 12:30 CET (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP) announced
today a program update for its lead investigational compound, RP103 for the
potential treatment of nephropathic cystinosis. RP103 is Raptor's proprietary,
enteric-coated, microbead delayed-release oral formulation of cysteamine
bitartrate designed to potentially reduce dosing frequency and reduce
gastrointestinal side effects associated with the currently approved
immediate-release cysteamine bitartrate.
Following the successful completion of Raptor's pivotal Phase 3 clinical trial
of RP103, which was announced in July 2011, the Company has been conducting a
planned voluntary extension study to monitor white blood cell ('WBC') cystine
levels and collect long-term safety and quality of life data. Of the 40
patients who entered the safety study after completing the Phase 3 clinical
trial, 38 are currently still enrolled. All of these 38 patients have now been
taking RP103 in the extension study for at least 6 months, with some patients
having been in the extension study for as long as 15 months. Raptor intends to
include at least 6 months of safety data for all Phase 3 completers who remain
in the extension study, with its New Drug Application ('NDA') and Marketing
Authorization Application ('MAA') filings that are expected to be filed in the
first calendar quarter of 2012. The Company plans to keep the extension study
open to all enrolled patients until RP103 becomes locally commercially
available.
Based on the positive results of Raptor's Phase 3 clinical trial and on the
findings of its bioequivalence study, which demonstrated similar drug exposure
whether administered in whole capsule or sprinkled onto applesauce, US and
European regulatory agencies approved the Company's expanded enrollment in the
extension study to include patients who did not qualify for the Phase 3
clinical trial. These patients include children 1-6 years old and patients who
have undergone a kidney transplant. Ten of an anticipated 18 additional
patients have already enrolled in the expanded extension study. The Company
also plans to study RP103 in cystinosis patients who have either stopped taking
or have not been adequately controlled with the currently-marketed,
immediate-release cysteamine bitartrate. While the data for these additional
patient groups are not required to be included in Raptor's NDA and MAA filings,
the Company expects to include them in subsequent updates to these marketing
applications.
'The data from these additional study patients will be helpful for physicians
in determining optimal treatments in a broader population of patients with
nephropathic cystinosis than we were able to study in our Phase 3 clinical
trial,' said Patrice P. Rioux, M.D. Ph.D., Raptor's Chief Medical Officer.
Raptor has completed pre-submission meetings with the US Food and Drug
Administration ('FDA') and the European Medicines Agency ('EMA') and is on
track to submit marketing applications in both markets in the first calendar
quarter of 2012. Raptor plans to file for 'Priority Review' in the US and
'Accelerated Approval' in the EU. If granted, the Company could potentially
receive approval for the sale of RP103 for the treatment of nephropathic
cystinosis in both the US and the EU in the fourth calendar quarter of 2012. In
anticipation of the potential approval in 2012, Raptor is actively building its
US and European commercial infrastructure and pre-commercial programs to market
RP103.
About Nephropathic Cystinosis
Nephropathic cystinosis is a rare disease resulting from an inborn metabolic
error characterized by the abnormal transport of cystine, an amino acid, out of
lysosomes. Poor compliance with current treatments for nephropathic cystinosis
can cause serious health consequences, including: renal failure and resultant
need for a kidney transplant; growth failure; rickets and fractures; and
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 Cysteamine and RP103
RP103 is Raptor's proprietary enteric-coated, delayed-release, microbead oral
formulation of cysteamine bitartrate designed to potentially reduce dosing
frequency and reduce gastrointestinal side effects associated with
immediate-release cysteamine bitartrate, which is approved for sale by the FDA
and EMA to treat nephropathic cystinosis. Raptor has been granted orphan
product designation for RP103 by both regulatory agencies.
In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diego for the development RP103 for nephropathic
cystinosis and cysteamine for other potential indications including
Huntington's Disease, and Non-alcoholic Steatohepatitis ('NASH').
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 file six months safety data from the extension study with the FDA
and EMA; that Raptor will be able to enroll new patients in its extension
study; that Raptor will file its NDA and MAA in the first calendar quarter of
2012, if at all; that Raptor will use additional extension study data in its
NDA and MAA updates; that the data from these additional extension study
patients will be helpful for physicians in determining optimal treatments in a
broader population of patients with nephropathic cystinosis; that Raptor will
file for 'Priority Review' in the US and 'Accelerated Approval' in the EU; that
Raptor will be granted Priority Review in the US and/or Accelerated Approval in
the EU; that Raptor will receive marketing approval in 2012, if at all; that
Raptor will successfully commercialize RP103, if approved; that Raptor will be
able to build its US and European commercial infrastructure and pre-commercial
programs to market RP103; 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 filed with the SEC on November 14, 2011 and December 19, 2011; 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
21.12.2011 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.
21.12.2011 12:30
---------------------------------------------------------------------------
On Track to File Marketing Applications in US and EU in the First Quarter of
2012
NOVATO, Calif., 2011-12-21 12:30 CET (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP) announced
today a program update for its lead investigational compound, RP103 for the
potential treatment of nephropathic cystinosis. RP103 is Raptor's proprietary,
enteric-coated, microbead delayed-release oral formulation of cysteamine
bitartrate designed to potentially reduce dosing frequency and reduce
gastrointestinal side effects associated with the currently approved
immediate-release cysteamine bitartrate.
Following the successful completion of Raptor's pivotal Phase 3 clinical trial
of RP103, which was announced in July 2011, the Company has been conducting a
planned voluntary extension study to monitor white blood cell ('WBC') cystine
levels and collect long-term safety and quality of life data. Of the 40
patients who entered the safety study after completing the Phase 3 clinical
trial, 38 are currently still enrolled. All of these 38 patients have now been
taking RP103 in the extension study for at least 6 months, with some patients
having been in the extension study for as long as 15 months. Raptor intends to
include at least 6 months of safety data for all Phase 3 completers who remain
in the extension study, with its New Drug Application ('NDA') and Marketing
Authorization Application ('MAA') filings that are expected to be filed in the
first calendar quarter of 2012. The Company plans to keep the extension study
open to all enrolled patients until RP103 becomes locally commercially
available.
Based on the positive results of Raptor's Phase 3 clinical trial and on the
findings of its bioequivalence study, which demonstrated similar drug exposure
whether administered in whole capsule or sprinkled onto applesauce, US and
European regulatory agencies approved the Company's expanded enrollment in the
extension study to include patients who did not qualify for the Phase 3
clinical trial. These patients include children 1-6 years old and patients who
have undergone a kidney transplant. Ten of an anticipated 18 additional
patients have already enrolled in the expanded extension study. The Company
also plans to study RP103 in cystinosis patients who have either stopped taking
or have not been adequately controlled with the currently-marketed,
immediate-release cysteamine bitartrate. While the data for these additional
patient groups are not required to be included in Raptor's NDA and MAA filings,
the Company expects to include them in subsequent updates to these marketing
applications.
'The data from these additional study patients will be helpful for physicians
in determining optimal treatments in a broader population of patients with
nephropathic cystinosis than we were able to study in our Phase 3 clinical
trial,' said Patrice P. Rioux, M.D. Ph.D., Raptor's Chief Medical Officer.
Raptor has completed pre-submission meetings with the US Food and Drug
Administration ('FDA') and the European Medicines Agency ('EMA') and is on
track to submit marketing applications in both markets in the first calendar
quarter of 2012. Raptor plans to file for 'Priority Review' in the US and
'Accelerated Approval' in the EU. If granted, the Company could potentially
receive approval for the sale of RP103 for the treatment of nephropathic
cystinosis in both the US and the EU in the fourth calendar quarter of 2012. In
anticipation of the potential approval in 2012, Raptor is actively building its
US and European commercial infrastructure and pre-commercial programs to market
RP103.
About Nephropathic Cystinosis
Nephropathic cystinosis is a rare disease resulting from an inborn metabolic
error characterized by the abnormal transport of cystine, an amino acid, out of
lysosomes. Poor compliance with current treatments for nephropathic cystinosis
can cause serious health consequences, including: renal failure and resultant
need for a kidney transplant; growth failure; rickets and fractures; and
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 Cysteamine and RP103
RP103 is Raptor's proprietary enteric-coated, delayed-release, microbead oral
formulation of cysteamine bitartrate designed to potentially reduce dosing
frequency and reduce gastrointestinal side effects associated with
immediate-release cysteamine bitartrate, which is approved for sale by the FDA
and EMA to treat nephropathic cystinosis. Raptor has been granted orphan
product designation for RP103 by both regulatory agencies.
In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diego for the development RP103 for nephropathic
cystinosis and cysteamine for other potential indications including
Huntington's Disease, and Non-alcoholic Steatohepatitis ('NASH').
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 file six months safety data from the extension study with the FDA
and EMA; that Raptor will be able to enroll new patients in its extension
study; that Raptor will file its NDA and MAA in the first calendar quarter of
2012, if at all; that Raptor will use additional extension study data in its
NDA and MAA updates; that the data from these additional extension study
patients will be helpful for physicians in determining optimal treatments in a
broader population of patients with nephropathic cystinosis; that Raptor will
file for 'Priority Review' in the US and 'Accelerated Approval' in the EU; that
Raptor will be granted Priority Review in the US and/or Accelerated Approval in
the EU; that Raptor will receive marketing approval in 2012, if at all; that
Raptor will successfully commercialize RP103, if approved; that Raptor will be
able to build its US and European commercial infrastructure and pre-commercial
programs to market RP103; 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 filed with the SEC on November 14, 2011 and December 19, 2011; 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
21.12.2011 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
---------------------------------------------------------------------------