LEIDEN, Netherlands and SAN
DIEGO (June 25, 2013) - Pharming Group NV (NYSE Euronext:
PHARM) and Santarus, Inc. (NASDAQ: SNTS), announced that new data
from a pivotal Phase III clinical study with RUCONEST®
(recombinant human C1 esterase inhibitor) for the treatment of
acute angioedema attacks in patients with hereditary
angioedema (HAE) were featured in a poster presentation yesterday
at the European Academy of Allergy and Clinical Immunology (EAACI)
& World Allergy Organization (WAO) World Allergy & Asthma
Congress in Milan, Italy. The data indicate that the time to
beginning of relief of symptoms in patients experiencing an acute
attack of HAE was statistically significantly shorter with RUCONEST
compared with placebo.
"RUCONEST has the potential to be an important
addition to the therapeutic options available for the treatment of
acute attacks of HAE based on the encouraging clinical data
observed to date," said Marc Riedl, M.D., Section Head Department
of Clinical Immunology and Allergy, University of California, Los
Angeles and lead author of the poster.
The poster is titled, Recombinant Human C1 Inhibitor for Treatment of Acute
Attacks of Hereditary Angioedema: A Randomized, Double-Blind,
Placebo-Controlled Clinical Trial. The primary endpoint and
safety data from the study are summarized below:
Time to
Beginning of Relief of Symptoms
|
Median (95% CI), minutes |
p-value* |
|
Placebo (N=31) |
RUCONEST (N=44) |
Based on Treatment Effect Questionnaire
(TEQ) (Primary Endpoint) |
152 (93, -) |
90 (61, 150) |
0.031 |
Based on Visual Analog Scale (VAS)
Decrease >= 20 mm |
303 (81, 720) |
75 (60, 105) |
0.003 |
*Based on log-rank test stratified by primary
attack location; CI, confidence interval: -, not calculable.
Treatment-Emergent Adverse Events Occurring Within 72 Hours
in 5% or More of Patients
|
Placebo
(N=18)
n (%) |
RUCONEST
(N=56)
n (%) |
Treatment-emergent adverse events |
4 (22%) |
4 (7%) |
Sinus congestion |
1 (6%) |
0 |
Vasomotor rhinitis |
1 (6%) |
0 |
Diarrhea |
1 (6%) |
0 |
Dyspepsia |
1 (6%) |
0 |
Note: Patients who received placebo (saline)
followed by RUCONEST as rescue medications are summarized as
placebo up to receipt of rescue medication and as RUCONEST
afterwards.
-
No thromboembolic events, anaphylaxis or
neutralizing antibodies were observed
-
One patient experienced a serious adverse event
(abdominal hernia) approximately 79 days after RUCONEST
administration
-
No patients withdrew due to adverse events
Pharming and Santarus are seeking U.S. marketing
approval of RUCONEST for the treatment of acute angioedema attacks
in patients with HAE. The Biologics License Application (BLA)
filing for RUCONESTis under review by the U.S. Food and Drug
Administration (FDA) with a response expected by April 16, 2014.
RUCONEST is approved in Europe for the treatment of acute
angioedema attacks in patients with HAE and is an investigational
drug in the U.S. that has been granted orphan drug designation by
the FDA.
About RUCONEST and
Hereditary Angioedema
RUCONEST (INN conestat alfa) is a recombinant
version of the human protein C1 esterase inhibitor, and is produced
with Pharming's proprietary transgenic technology. RUCONEST is
approved in Europe for the treatment of acute angioedema attacks in
patients with HAE, a genetic disorder in which the patient is
deficient in or lacks a functional plasma protein C1 esterase
inhibitor, resulting in unpredictable and debilitating episodes of
intense swelling. The swelling may occur in one or more anatomical
areas, including the extremities, face, trunk, genitals, abdomen
and upper airway. The frequency and severity of HAE attacks vary
and are most serious when they involve laryngeal edema, which can
close the upper airway and cause death by asphyxiation. According
to the U.S. Hereditary Angioedema Association, epidemiological
estimates for HAE range from one in 10,000 to one in 50,000
individuals. RUCONEST is an investigational drug in the U.S. and
has been granted orphan drug designation by the FDA both for the
treatment of acute attacks of HAE and for prophylactic treatment of
HAE.
About EAACI
The European Academy of Allergy and Clinical
Immunology, EAACI, is a non-profit organisation active in the field
of allergic and immunologic diseases such as asthma, rhinitis,
eczema, occupational allergy, food and drug allergy and
anaphylaxis. EAACI was founded in 1956 in Florence and has
become the largest medical association in Europe in the field of
allergy and clinical immunology. It includes over 7,800 members
from 121 countries, as well as 42 National Allergy Societies.
About WAO
The World Allergy Organization (WAO) is an
international alliance of 92 regional and national allergy, asthma
and clinical immunology societies. Through collaboration with the
Member Societies, WAO provides a wide range of educational and
outreach programs, symposia, and lectureships to
allergists/immunologists around the globe and conducts initiatives
relating to clinical practice, service provision, and physician
training in order to better understand and address the challenges
facing allergists/immunologists worldwide.
About Pharming Group
NV
Pharming Group NV is developing innovative
products for the treatment of unmet medical needs.
RUCONEST® is a
recombinant human C1 esterase inhibitor approved for the treatment
of angioedema attacks in patients with HAE in all 27 EU countries
plus Norway, Iceland and Liechtenstein, and is distributed in the
EU by Swedish Orphan Biovitrum. RUCONEST® is partnered
with Santarus, Inc. (NASDAQ: SNTS) in North America and a Biologics
License Application for RUCONEST is under review by the U.S. Food
and Drug Administration. The product is also being evaluated for
various follow-on indications. Pharming has a unique GMP
compliant, validated platform for the production of recombinant
human proteins that has proven capable of producing industrial
volumes of high quality recombinant human protein in a more
economical way compared to current cell based technologies.
Pharming now plans to utilise this platform for the development of
rhFVIII for the treatment of Haemophilia A. Additional
information is available on the Pharming website,
www.pharming.com.
About Santarus
Santarus, Inc. is a specialty biopharmaceutical
company focused on acquiring, developing and commercializing
proprietary products that address the needs of patients treated by
physician specialists. The company's current commercial efforts are
focused on five products. UCERIS®(budesonide)
extended release tablets for the induction of remission in patients
with active, mild to moderate ulcerative colitis and
ZEGERID®
(omeprazole/sodium bicarbonate) for the treatment of certain upper
gastrointestinal disorders are promoted to gastroenterologists.
GLUMETZA® (metformin
hydrochloride extended release tablets) and CYCLOSET®
(bromocriptine mesylate) tablets, which are indicated as adjuncts
to diet and exercise to improve glycemic control in adults with
type 2 diabetes, and FENOGLIDE® (fenofibrate)
tablets, which is indicated as an adjunct to diet to reduce high
cholesterol, are promoted to endocrinologists and other physicians
who treat patients with type 2 diabetes. Full prescribing and
safety information for Santarus' products is available at
www.santarus.com or by contacting Santarus at 1-888-778-0887.
Santarus' product development pipeline includes
the investigational drug RUCONEST® (recombinant
human C1 esterase inhibitor). A Biologics License Application
for RUCONEST for the treatment of acute angioedema attacks in
patients with hereditary angioedema is under review by the U.S.
Food and Drug Administration with a response expected in April
2014. Santarus is also developing rifamycin SV MMX®, which is in
Phase III clinical testing for the treatment of travelers'
diarrhea. In addition, the company has completed a Phase I
clinical program with SAN-300, an investigational monoclonal
antibody. More information about Santarus is available at
www.santarus.com.
Santarus and Pharming caution
you that statements included in this press release that are not a
description of historical facts are forward-looking statements.
The inclusion of forward-looking statements should not be
regarded as a representation by Santarus or Pharming that any of
its plans or objectives will be achieved. Actual results may differ
materially from those set forth in this release due to the risks
and uncertainties inherent in Santarus and Pharming's businesses,
including, without limitation: whether the FDA will approve
the RUCONEST BLA in a timely manner or at all; whether the FDA will
concur with the clinical interpretation of the Phase III study
results or the conduct of the study; whether the FDA ultimately
will require additional clinical studies or other development
programs before approving RUCONEST; risks related to Santarus'
dependence on Pharming for many functions related to RUCONEST, and
Pharming's ability to continue to perform these functions based on
its limited financial resources; risks related to the license and
supply arrangements between Santarus and Pharming, including the
potential for termination of the arrangements; other difficulties
or delays in development, testing, manufacturing and marketing of,
and obtaining and maintaining regulatory approvals for, Santarus
and Pharming's products; and other risks detailed in prior press
releases as well as in public periodic filings with the Securities
and Exchange Commission, including Santarus' Quarterly Report on
Form 10-Q for the quarter ended March 31, 2013.
You are cautioned not to place
undue reliance on these forward-looking statements, which speak
only as of the date hereof. All forward-looking statements are
qualified in their entirety by this cautionary statement and
neither Santarus nor Pharming undertakes any obligation to revise
or update this news release to reflect events or circumstances
after the date hereof, except as may be required by law. This
caution is made under the safe harbor provisions of Section 21E of
the Private Securities Litigation Reform Act of 1995.
Santarus®, FENOGLIDE®, UCERIS®, and ZEGERID® are registered trademarks of
Santarus, Inc. GLUMETZA® is a trademark of Biovail
Laboratories International S.r.l. licensed exclusively in the
United States to Depomed, Inc. CYCLOSET® is a trademark of
VeroScience LLC. MMX® is a trademark of Cosmo
Technologies Limited. RUCONEST® is a trademark of Pharming
Group N.V.
# # #
SANTARUS CONTACTS:
Martha L. Hough, VP Finance & Investor
Relations
+1 (858) 314-5824
Debra P. Crawford, Chief Financial Officer
+1 (858) 314-5708
|
PHARMING CONTACTS:
Sijmen de Vries, CEO
Tel: +31 (0)71 524 7400 |
Westwicke Partners, LLC
Stefan Loren, Ph.D. (sloren@westwicke.com)
+1 (858) 356-5930
Robert Uhl (robert.uhl@westwicke.com)
+1 (858) 356-5932 |
FTI Consulting
Julia Phillips/John Dineen
Tel: +44 (0)207 269 7193 |
Press release (PDF)
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Source: Pharming Group N.V. via Thomson Reuters ONE
HUG#1711721