LEIDEN, Netherlands and SAN DIEGO, Nov. 8, 2013 (GLOBE NEWSWIRE) --
Pharming Group NV (NYSE
Euronext: PHARM) and Santarus, Inc. (NASDAQ: SNTS) announced that new data from
an open-label extension of the pivotal Phase III clinical study with RUCONEST(®)
(recombinant human C1 esterase inhibitor, or rhC1INH) will be featured in a
poster presentation on November 9 & 10, 2013 at the 2013 American College of
Allergy, Asthma & Immunology Annual Scientific Meeting at the Convention Center
in Baltimore, Maryland. The poster is titled, Efficacy and Safety of
Recombinant Human C1 Esterase Inhibitor for Acute Attacks of Hereditary
Angioedema: An Open-Label Study.
RUCONEST was administered for the treatment of 224 repeat acute angioedema
attacks in 44 patients with hereditary angioedema (HAE) following initial
treatment in the pivotal randomized controlled clinical study. RUCONEST is an
investigational drug in the U.S. and has been granted orphan drug designation by
the U.S. Food and Drug Administration (FDA) for the treatment of acute attacks
of HAE.
"The results from this clinical trial are consistent with findings from previous
studies supporting that RUCONEST reduces time to symptom relief when used for
repeat HAE attacks," said H. Henry Li, M.D., Ph.D., Director of Chevy Chase
Clinical Research, Institute for Asthma and Allergy, Chevy Chase, Maryland. "In
addition, it is important clinically to evaluate the safety findings from these
multiple exposures."
The median time in minutes (95% confidence interval [CI]) to onset of symptom
relief following treatment as measured by patient responses to a Treatment
Effect Questionnaire (TEQ) for the first five repeat attacks ranged from 62.5
(48, 90) to 134.0 (75, 150) and across all attacks was a median of 75.0 (69,
89). The median (95% CI) time in minutes to minimal symptoms (first three
attacks per patient) as measured by a TEQ ranged from 243 (76, 1440) to 304
(150, 719) and for all assessed attacks was 303 (211, 367).
Exposure and Safety Information
* Only one dose of RUCONEST was administered for 96% of the 224 attacks.
* 12 of 44 (27%) patients experienced at least one treatment emergent adverse
event (TEAE) within 72 hours of completion of RUCONEST infusion.
* TEAEs occurring in 5% of patients were nasopharyngitis, cough, fibrin D-
dimer increase, and headache
* The percentage of patients experiencing TEAEs did not increase with
RUCONEST treatments for repeat attacks.
* There were no discontinuations due to adverse events, no thrombotic or
anaphylactic events, and no neutralizing anti-C1INH antibodies observed with
repeat RUCONEST treatment.
Santarus and Pharming 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.
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. In July 2013, the Platform was partnered with Shanghai
Institute for Pharmaceutical Industry (SIPI), a Sinopharm Company, for joint
global development of new products. Pre- clinical development and manufacturing
will take place at SIPI and are funded by SIPI. Pharming and SIPI initially plan
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 September
30, 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: PHARMING CONTACTS:
Martha L. Hough, VP Finance & Investor Relations Sijmen de Vries, CEO
+1 (858) 314-5824 Tel:
+31 (0)71 524 7400
Debra P. Crawford, Chief Financial Officer
+1 (858) 314-5708
Westwicke Partners, LLC FTI Consulting
Stefan Loren, Ph.D. (sloren@westwicke.com) Julia Phillips/John Dineen
+1 (858) 356-5930 Tel: +44 (0)207
269 7193
Robert Uhl (robert.uhl@westwicke.com)
+1 (858) 356-5932
[HUG#1741731]