Santarus and Pharming Announce Poster Presentation of Pivotal Clinical Data for RUCONEST (Recombinan
Santarus and Pharming Announce Poster Presentation of Pivotal Clinical Data for RUCONEST (Recombinant Human C1 Esterase Inhibitor)
SAN DIEGO & LEIDEN, Netherlands--(BUSINESS WIRE)-- Santarus, Inc. (NAS: SNTS) and Pharming Group NV (NYSE Euronext: PHARM) 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
|n (%)||n (%)|
|Treatment-emergent adverse events||4 (22%)||4 (7%)|
|Sinus congestion||1 (6%)||0|
|Vasomotor rhinitis||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
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. 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.
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.
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. (NAS: 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.
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.
Martha L. Hough, VP Finance & Investor Relations
Debra P. Crawford, Chief Financial Officer
Westwicke Partners, LLC
Stefan Loren, Ph.D., +1-858-356-5930
Robert Uhl, +1-858-356-5932
Pharming Group NV
Sijmen de Vries, CEO
Tel: +31 (0)71 524 7400
Julia Phillips/John Dineen
Tel: +44 (0)207 269 7193
KEYWORDS: United States Europe North America Netherlands California Italy
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