Raptor Pharmaceutical Corp. Provides Program Update on RP103 (DR Cysteamine) for Nephropathic Cystinosis
On Track to File Marketing Applications in US and EU in the First Quarter of 2012
NOVATO, Calif., Dec. 21, 2011 (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
Image: company logo
Raptor Pharmaceutical Corp.
Posted: December 2011
