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Santarus And Pharming Announce Poster Presentation Of Pivotal Clinical Data For RUCONEST (Recombinant Human C1 Esterase Inhibitor)

Santarus, Inc. (NASDAQ: 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
        Placebo         RUCONEST
(N=18) (N=56)
          n (%)         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

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 RUCONEST is 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.

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