Raptor Pharmaceutical Corp. Signs Collaborative Research and Development Agreement With NIDDK on Phase 2b Clinical Trial of RP104 (DR Cysteamine Tablets) in Non-Alcoholic Steatohepatitis ('NASH')
Raptor Pharmaceutical Corp.
19.12.2011 12:30
---------------------------------------------------------------------------
Phase 2b Trial Anticipated to Start in Q1 2012
Company to Host NASH Event at JP Morgan Healthcare Conference
NOVATO, Calif., Dec. 19, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp.
('Raptor' or the 'Company') (Nasdaq:RPTP), today announced the signing of a
cooperative research and development agreement ('CRADA') with the National
Institute of Diabetes and Digestive and Kidney Diseases ('NIDDK') part of the
National Institutes of Health ('NIH') to conduct a Phase 2b clinical trial. The
clinical trial will evaluate the safety and potential efficacy of RP104,
Raptor's proprietary delayed-release tablet formulation of cysteamine
bitartrate, as a potential treatment of non-alcoholic steatohepatitis ('NASH'),
an advanced form of non-alcoholic fatty liver disease ('NAFLD'), in children.
The clinical trial is expected to begin in the first calendar quarter of 2012
with NIDDK and Raptor sharing the costs to conduct the clinical trial.
The Cysteamine Bitartrate Delayed-Release for the Treatment of Non-alcoholic
Fatty Liver Disease in Children ('CyNCh') trial is expected to enroll a total
of 160 pediatric participants at ten U.S. centers in the NIDDK-sponsored NASH
Clinical Research Network ('NASH CRN'). Enrolled participants will range in age
from 8 to17 years and have biopsy-confirmed moderate to severe NAFLD. The
primary objective of this randomized, multicenter, double-blind,
placebo-controlled clinical trial is to evaluate whether 52 weeks of treatment
with RP104 in children reverses damage caused by NASH as measured by changes in
NAFLD Activity Score ('NAS'), a histological rating scale of disease activity.
Secondary endpoints will include blood markers for liver health including
alanine transaminase ('ALT') and aspartate transaminase ('AST'), as well as
safety and tolerability.
'NAFLD now affects millions of children in the United States', said Jeffrey
Schwimmer, M.D., the Director of the Fatty Liver Clinic at Rady Children's
Hospital San Diego and the Principal Investigator for the NASH CRN Pediatric
Clinical Center at the University of California, San Diego ('UC, San Diego').
'In the absence of an effective therapy, we are increasingly seeing adolescents
with advanced liver disease. Therefore we are excited about the opportunity to
evaluate RP104 as a potential treatment for pediatric NAFLD in the NASH
Clinical Research Network.'
Raptor estimates the total cost of the CyNCh trial to be in the range of
$14-$16 million. Under the CRADA agreement, Raptor will fund a total of $6
million of the cost of the trial, in addition to providing clinical trial
materials and drug manufacturing/quality support estimated at approximately $1
million. The remainder of the funding will come from NIDDK. Raptor holds
worldwide, exclusive licenses from UC San Diego to patents relating to use of
cysteamine in NAFLD and NASH. Under this CRADA collaboration, Raptor will
retain exclusive development and commercial rights to the clinical data
resulting from the CyNCh trial.
'This agreement is an important step in advancing our RP104 program for NASH.
In addition to the substantial funding, this collaboration enables us to work
with an already established network of many of the leading NASH clinics in the
U.S.,' said Ted Daley, President of Raptor. 'We believe that this will enable
Raptor to commence and complete the Phase 2b clinical trial far sooner than if
we were to conduct the clinical trial entirely on our own. We are excited to
work with NIDDK and these top investigators to advance our program.'
This planned CyNCh trial follows positive results of an open-label Phase 2a
clinical trial which was conducted under a collaboration agreement between
Raptor and UC San Diego at UC San Diego's General Clinical Research Center. The
Phase 2a clinical trial involved children with biopsy-confirmed diagnosis of
moderate to severe NAFLD and baseline ALT and AST measurements at least twice
that of normal levels. These patients received a prototype formulation of RP104
twice daily for six months, followed by a six-month post-treatment monitoring
period. All patients showed marked decline in ALT and AST levels during the
treatment period with 7 of 11 patients achieving a greater than 50 percent
reduction and 6 of 11 patients with reductions to within normal range. The
reductions in ALT and AST were largely sustained during the 6 month
post-treatment phase. Other important liver function markers showed positive
trends with cytokeratin levels decreasing by an average of 45 percent and
adiponectin increasing by 35 percent during the treatment period.
Under its licenses with UC San Diego, Raptor is developing proprietary
formulations of delayed-release ('DR') cysteamine bitartrate for a number of
therapeutic indications, including RP103 (DR cysteamine bitartrate microbeads
in capsules) for the potential treatment of nephropathic cystinosis and
Huntington's Disease, and RP104 for NASH. Cysteamine is a precursor of the
potent liver anti-oxidant glutathione ('GSH') and increasing GSH has the
potential to reverse NASH-related liver damage. GSH itself does not enter
easily into cells, even when given in large amounts. However, GSH precursors,
such as cysteamine, enter into cells and have been shown to be effective in the
treatment of certain conditions by preventing significant GSH depletion.
Raptor intends to host an event providing an overview of its NASH program at
the upcoming JP Morgan Healthcare Conference in San Francisco. This event will
include Raptor's management and NASH experts on Tuesday, January 10, 2012 at
4:30pm PST. The Company will provide a teleconference feed for those not
attending the conference. To attend the event, please contact Lauren Glaser at
lglaser@troutgroup.com.
About NASH
NASH is a more aggressive form of NAFLD that affects 2 to 5 percent of
Americans. It occurs in patients who drink little or no alcohol, and is the
most common cause of chronic liver disease in North America. Although most
patients are asymptomatic and feel healthy in early phases of the disease, NASH
causes decreased liver function and can lead to cirrhosis, liver failure and
end-stage liver disease. While NASH is most common in insulin-resistant obese
adults with diabetes and abnormal serum lipid profiles, its prevalence is
increasing among juveniles as obesity rates rise within this patient
population. There are no currently approved drug therapies for NASH; patients
are limited to lifestyle changes such as diet, exercise and weight reduction to
manage the disease.
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
Phase 2b CyNCh trial in juveniles with NASH will begin in the first calendar
quarter of 2012, if at all; that Raptor/NIH will enroll a total of 160
pediatric participants at nine U.S. centers in the NIDDK-sponsored NASH
Clinical Research Network, if at all; that RP104 will reverse damage caused by
NASH; that the total cost of the CyNCh trial will be in the range of $14-$16
million; that Raptor will be able to adequately supply clinical trial materials
to sustain the CyNCh trial; that the NIH will be able to commence and complete
the CyNCh trial far sooner than if Raptor conducted the clinical trial on its
own; that the CyNCh trial will produce results similar to the Phase 2a clinical
trial at UC San Diego; and that Raptor will be able to successfully develop
RP104, RP103 or DR Cysteamine 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 filed with the SEC on November 14, 2011;
and Raptor's Amendment No. 1 to its Annual Report on Form 10-K filed with the
SEC on 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
19.12.2011 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.
19.12.2011 12:30
---------------------------------------------------------------------------
Phase 2b Trial Anticipated to Start in Q1 2012
Company to Host NASH Event at JP Morgan Healthcare Conference
NOVATO, Calif., Dec. 19, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp.
('Raptor' or the 'Company') (Nasdaq:RPTP), today announced the signing of a
cooperative research and development agreement ('CRADA') with the National
Institute of Diabetes and Digestive and Kidney Diseases ('NIDDK') part of the
National Institutes of Health ('NIH') to conduct a Phase 2b clinical trial. The
clinical trial will evaluate the safety and potential efficacy of RP104,
Raptor's proprietary delayed-release tablet formulation of cysteamine
bitartrate, as a potential treatment of non-alcoholic steatohepatitis ('NASH'),
an advanced form of non-alcoholic fatty liver disease ('NAFLD'), in children.
The clinical trial is expected to begin in the first calendar quarter of 2012
with NIDDK and Raptor sharing the costs to conduct the clinical trial.
The Cysteamine Bitartrate Delayed-Release for the Treatment of Non-alcoholic
Fatty Liver Disease in Children ('CyNCh') trial is expected to enroll a total
of 160 pediatric participants at ten U.S. centers in the NIDDK-sponsored NASH
Clinical Research Network ('NASH CRN'). Enrolled participants will range in age
from 8 to17 years and have biopsy-confirmed moderate to severe NAFLD. The
primary objective of this randomized, multicenter, double-blind,
placebo-controlled clinical trial is to evaluate whether 52 weeks of treatment
with RP104 in children reverses damage caused by NASH as measured by changes in
NAFLD Activity Score ('NAS'), a histological rating scale of disease activity.
Secondary endpoints will include blood markers for liver health including
alanine transaminase ('ALT') and aspartate transaminase ('AST'), as well as
safety and tolerability.
'NAFLD now affects millions of children in the United States', said Jeffrey
Schwimmer, M.D., the Director of the Fatty Liver Clinic at Rady Children's
Hospital San Diego and the Principal Investigator for the NASH CRN Pediatric
Clinical Center at the University of California, San Diego ('UC, San Diego').
'In the absence of an effective therapy, we are increasingly seeing adolescents
with advanced liver disease. Therefore we are excited about the opportunity to
evaluate RP104 as a potential treatment for pediatric NAFLD in the NASH
Clinical Research Network.'
Raptor estimates the total cost of the CyNCh trial to be in the range of
$14-$16 million. Under the CRADA agreement, Raptor will fund a total of $6
million of the cost of the trial, in addition to providing clinical trial
materials and drug manufacturing/quality support estimated at approximately $1
million. The remainder of the funding will come from NIDDK. Raptor holds
worldwide, exclusive licenses from UC San Diego to patents relating to use of
cysteamine in NAFLD and NASH. Under this CRADA collaboration, Raptor will
retain exclusive development and commercial rights to the clinical data
resulting from the CyNCh trial.
'This agreement is an important step in advancing our RP104 program for NASH.
In addition to the substantial funding, this collaboration enables us to work
with an already established network of many of the leading NASH clinics in the
U.S.,' said Ted Daley, President of Raptor. 'We believe that this will enable
Raptor to commence and complete the Phase 2b clinical trial far sooner than if
we were to conduct the clinical trial entirely on our own. We are excited to
work with NIDDK and these top investigators to advance our program.'
This planned CyNCh trial follows positive results of an open-label Phase 2a
clinical trial which was conducted under a collaboration agreement between
Raptor and UC San Diego at UC San Diego's General Clinical Research Center. The
Phase 2a clinical trial involved children with biopsy-confirmed diagnosis of
moderate to severe NAFLD and baseline ALT and AST measurements at least twice
that of normal levels. These patients received a prototype formulation of RP104
twice daily for six months, followed by a six-month post-treatment monitoring
period. All patients showed marked decline in ALT and AST levels during the
treatment period with 7 of 11 patients achieving a greater than 50 percent
reduction and 6 of 11 patients with reductions to within normal range. The
reductions in ALT and AST were largely sustained during the 6 month
post-treatment phase. Other important liver function markers showed positive
trends with cytokeratin levels decreasing by an average of 45 percent and
adiponectin increasing by 35 percent during the treatment period.
Under its licenses with UC San Diego, Raptor is developing proprietary
formulations of delayed-release ('DR') cysteamine bitartrate for a number of
therapeutic indications, including RP103 (DR cysteamine bitartrate microbeads
in capsules) for the potential treatment of nephropathic cystinosis and
Huntington's Disease, and RP104 for NASH. Cysteamine is a precursor of the
potent liver anti-oxidant glutathione ('GSH') and increasing GSH has the
potential to reverse NASH-related liver damage. GSH itself does not enter
easily into cells, even when given in large amounts. However, GSH precursors,
such as cysteamine, enter into cells and have been shown to be effective in the
treatment of certain conditions by preventing significant GSH depletion.
Raptor intends to host an event providing an overview of its NASH program at
the upcoming JP Morgan Healthcare Conference in San Francisco. This event will
include Raptor's management and NASH experts on Tuesday, January 10, 2012 at
4:30pm PST. The Company will provide a teleconference feed for those not
attending the conference. To attend the event, please contact Lauren Glaser at
lglaser@troutgroup.com.
About NASH
NASH is a more aggressive form of NAFLD that affects 2 to 5 percent of
Americans. It occurs in patients who drink little or no alcohol, and is the
most common cause of chronic liver disease in North America. Although most
patients are asymptomatic and feel healthy in early phases of the disease, NASH
causes decreased liver function and can lead to cirrhosis, liver failure and
end-stage liver disease. While NASH is most common in insulin-resistant obese
adults with diabetes and abnormal serum lipid profiles, its prevalence is
increasing among juveniles as obesity rates rise within this patient
population. There are no currently approved drug therapies for NASH; patients
are limited to lifestyle changes such as diet, exercise and weight reduction to
manage the disease.
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
Phase 2b CyNCh trial in juveniles with NASH will begin in the first calendar
quarter of 2012, if at all; that Raptor/NIH will enroll a total of 160
pediatric participants at nine U.S. centers in the NIDDK-sponsored NASH
Clinical Research Network, if at all; that RP104 will reverse damage caused by
NASH; that the total cost of the CyNCh trial will be in the range of $14-$16
million; that Raptor will be able to adequately supply clinical trial materials
to sustain the CyNCh trial; that the NIH will be able to commence and complete
the CyNCh trial far sooner than if Raptor conducted the clinical trial on its
own; that the CyNCh trial will produce results similar to the Phase 2a clinical
trial at UC San Diego; and that Raptor will be able to successfully develop
RP104, RP103 or DR Cysteamine 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 filed with the SEC on November 14, 2011;
and Raptor's Amendment No. 1 to its Annual Report on Form 10-K filed with the
SEC on 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
19.12.2011 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
---------------------------------------------------------------------------