DGAP-News: Raptor Pharmaceutical Corp. Reports Fiscal Year 2012 Financial Highlights and Program Updates
(firmenpresse) - Raptor Pharmaceutical Corp.
01.11.2012 11:30
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NOVATO, Calif., 2012-11-01 11:30 CET (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the Company') (Nasdaq:RPTP) today
announced its fiscal year 2012 ended August 31, 2012 financial highlights, and
updates on its clinical programs including the ongoing extension study of
PROCYSBI(tm) (cysteamine bitartrate) delayed-release capsules for the potential
treatment of patients with nephropathic cystinosis ('cystinosis').
Financial Highlights
-- As of October 19, 2012, Raptor had approximately $41 million in cash, cash
equivalents and short-term investments, taking into account approximately
$13.4 million of net proceeds the Company raised under its At-The-Market
sales issuance agreement ('ATM').
-- Based on current financial projections, the Company believes that this cash
balance should provide the Company with sufficient capital to fund
operations into the third quarter of calendar 2013.
Cystinosis Updates
- 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, will present 'Extended Treatment with
RP103 (PROCYSBI) in Patients with Nephropathic Cystinosis' on Saturday,
November 3rd at Kidney Week 2012, which is being held October 30-November 4,
2012 in San Diego, California. This presentation will provide new data on
cystinosis patients treated with PROCYSBI for up to 24 months.
- In anticipation of potential launch, Raptor has hired sales and medical
affairs field launch teams in the US.
- In Europe, the Company has hired Country Managers in key markets and staffed
medical affairs and other commercial functions. In keeping with plans for a
rolling European Union launch, Raptor is establishing necessary foundational
legal and operational structures.
- Raptor recently launched RaptorCares(tm), (www.raptorcares.com), a dedicated
education, access and support program for the cystinosis community. RaptorCares
provides access to education and support materials developed in partnership
with the cystinosis community.
- In the US, Raptor has partnered with Accredo Health Group, Inc., also an
Express Scripts Company, as its specialty pharmacy and distributor. If PROCYSBI
receives marketing approval, this dedicated specialty pharmacy will provide
individualized caseworker support and counseling to each patient (or caregiver)
as they transition to PROCYSBI.
- The final Process Validation batches of PROCYSBI necessary for approval have
been initiated and the Company is ramping up production for potential launch
upon drug approval.
Huntington's Disease ('HD')
Raptor announced enrollment completion in its Phase 2/3 study using
delayed-release cysteamine (RP103) in 96 HD patients in June 2012. The Phase
2/3 clinical trial is being conducted under a previously announced
collaboration agreement with The Centre Hospitalier Universitaire d'Angers
('CHU d'Angers') of Angers, France.
The Phase 2/3 clinical trial enrolled 96 patients in an 18-month,
placebo-controlled study to be followed by an open-label extension study with
all patients on the placebo capsules rolling onto RP103 and all other patients
continuing on RP103 for up toan additional 18 months. The primary endpoint of
the clinical trial is based upon the Unified Huntington's Disease Rating Scale
('UHDRS'). Under the collaboration agreement with CHU d'Angers, Raptor will
supply RP103 and placebo capsules for the clinical trial and open-label
extension study in exchange for commercial rights to the clinical trial
results. Clinical expenses of the study are covered by a grant from the French
government (PHRC 2004-03bis CYST-HD).
This Phase 2/3 clinical trial was contemplated based on encouraging preclinical
results reported by Institut Curie, CNRS and INSERM scientists that cysteamine
prevents death of neurons and increases levels of a brain growth factor called
brain-derived neurotrophic factor ('BDNF') in HD preclinical models. Addressing
reduced levels of BDNF in the brain have been widely reported to be an
important factor in the development of therapeutics to treat HD.
Raptor holds exclusive worldwide licenses to intellectual property related to
the potential treatment of HD from the Weizmann Institute of Science in Israel
and Japan's Niigata University. In 2008, Raptor received FDA orphan drug
designation for cysteamine formulations, including RP103, for the potential
treatment of HD.
Non-Alcoholic Steatohepatitis ('NASH')
In June 2012, Raptor announced the initiation of its Phase 2b trial of RP103
for the potential treatment of NASH. The trial is being conducted within a
cooperative research and development agreement ('CRADA') with the National
Institutes of Diabetes and Digestive Disease ('NIDDK') for the potential
treatment of non-alcoholic fatty liver disease in children, known as the CyNCh
trial. The trial will eventually enroll a total of 160 pediatric participants
at ten US centers in the NIDDK-sponsored NASH clinical research network. NIDDK
and Raptor are sharing the costs to conduct the CyNCh clinical trial. Raptor
will retain exclusive development and commercial rights to the clinical trial
data. Complete enrollment is expected in the first quarter of calendar 2013 and
the data report from the clinical trial is anticipated to be released in the
first half of calendar 2014. The CyNCh clinical trial follows previously
reported positive results of an open-label Phase 2a clinical trial conducted at
the University of California, San Diego ('UCSD').
The primary objective of this randomized, multicenter, double-blind,
placebo-controlled Phase 2b CyNCh clinical trial is to evaluate whether 52
weeks of treatment with RP103 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 include blood markers for liver
health including alanine transaminase ('ALT') and aspartate transaminase
('AST'), as well as safety and tolerability.
About Cystinosis, RP103 and PROCYSBI(tm)
Cystinosis is a rare, life-threatening metabolic disorder that causes systemic
toxic cystine accumulation. Toxic cystine accumulation causes progressive and
irreversible tissue damage and multi-organ failure including renal failure,
blindness, CNS damage, respiratory deficiencies, muscle wasting, and premature
death. Cystinosis is usually diagnosed in the first years of life and requires
lifelong therapy. Left untreated, the disease is fatal by the end of the first
decade of life. Renal transplantation and cystine reduction are the primary
treatment strategies for cystinosis.
RP103 is an oral, delayed and extended-release medication containing
enteric-coated spheronized micro-beads of cysteamine bitartrate. PROCYSBI is
the branded name of RP103 for the potential treatment of cystinosis. RP103 is
in clinical development for the potential treatment of HD and NASH. In
cystinosis patients, PROCYSBI may reduce the cellular toxicity by continuously
removing cystine from the lysosome. PROCYSBI, which can also cross the
blood-brain barrier, was engineered specifically to allow release of cysteamine
bitartrate micro-spheres in the duodenum for optimal absorption while
simultaneously enabling administration every 12-hours for the potential
treatment of cystinosis.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. ('Raptor') (Nasdaq:RPTP) seeks to research,
develop, and provide access to medicines that improve life for patients with
severe and rare disorders. Raptor currently has product candidates in clinical
development designed to potentially treat nephropathic cystinosis,
Non-alcoholic Steatohepatitis, Huntington's Disease, and aldehyde dehydrogenase
deficiency.
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's cash balance will provide the Company with sufficient capital to fund
operations into the third quarter of calendar 2013; that the FDA and EMA will
deliver a decision regarding marketing approval of PROCYSBI for the potential
treatment of cystinosis on January 30, 2013 or the first half of calendar 2013,
respectively; that Raptor's Phase 2/3 clinical trial in Huntington's Disease
will replicate preclinical study results; that there will be clinical data on
the Huntington's Disease Phase 2/3 clinical trial in the first half of calendar
2014; that the Phase 2b NASH clinical trial will be fully enrolled by the first
quarter of calendar 2013 and that the clinical data report will be announced in
the first half of calendar 2014; that Raptor's Phase 2b clinical trial in NASH
will replicate the Company's Phase 2a clinical trial results; and that Raptor
will be able to successfully develop PROCYSBI or 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
July 10, 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.
Trout Group (investors)
Lauren Glaser
(646) 378-2972
lglaser(at)troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo(at)evcgroup.com
News Source: NASDAQ OMX
01.11.2012 Dissemination of a Corporate News, transmitted by DGAP -
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Language: English
Company: Raptor Pharmaceutical Corp.
United States
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ISIN: US75382F1066
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