Legal Proceedings Report • Oct 31, 2012
Legal Proceedings Report
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Leiden, The Netherlands, October 31, 2012. Biotech company Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) announced today that the positive results of a clinical trial with recombinant human C1 inhibitor (rhC1INH; RUCONEST®) to prevent attacks of hereditary angioedema (HAE) have been accepted for publication in the international peer-reviewed journal, 'Allergy'.
This was an open-label study to evaluate the prophylactic effect of once-weekly administration of Ruconest in 25 HAE patients. The patients included in this study had a history of frequent HAE attacks (mean 0.9 attacks/week). During the 8 week Ruconest treatment period, the mean frequency of HAE attacks was reduced by more than 50% to 0.4 attacks/ week. The repeated administrations were generally safe and well-tolerated. The full study results will be published online in November.
Lead author and study principal investigator, Dr. Avner Reshef, Director of the Allergy and Clinical Immunology Unit, and Angioedema Center at The Sheba Medical Center at Tel Hashomer, Israel, commented: "We studied severely affected HAE patients who had fewer angioedema attacks using once weekly Ruconest treatment. I look forward to working with Pharming to further study these encouraging findings."
Dr. Bruno Giannetti, Pharming's COO commented: "Over the last few years, the practice of prophylactic treatment of HAE with a fractionated human plasma C1 inhibitor product became part of the treatment paradigm for some HAE patients. We plan to work with the FDA to understand the regulatory requirements to provide a highly purified recombinant treatment option for the prophylaxis of HAE attacks."
RUCONEST® (INN conestat alfa) is a recombinant version of the human protein C1 inhibitor (C1INH). RUCONEST is produced through Pharming's proprietary technology in milk of transgenic rabbits and is approved in Europe for the treatment of acute angioedema attacks in patients with HAE. RUCONEST® is an investigational drug in the U.S. and has been granted orphan drug designation both for the treatment of acute attacks of HAE and prophylactic treatment of HAE. HAE is a genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in unpredictable and debilitating episodes of intense swelling of 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.
Pharming Group NV is developing innovative products for the treatment of unmet medical needs. RUCONEST® is a recombinant human C1 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 (OMX: SOBI). RUCONEST® is partnered with Santarus, Inc (NASDAQ: SNTS) in North America where the drug is undergoing Phase III clinical development. The advanced technologies of the Company include innovative and validated platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products. A feasibility study, using the validated transgenic rabbit platform, aimed at the development of recombinant Factor VIII for the treatment of Haemophilia A is underway with partner, Renova Life, Inc. Additional information is available on the Pharming website, www.pharming.com. To download the Pharming Group Investor Relations App, click here.
This press release contains forward looking statements that involve known and unknown risks, uncertainties and other
factors, which may cause the actual results, performance or achievements of the Company to be materially different from the results, performance or achievements expressed or implied by these forward looking statements.
Sijmen de Vries, CEO: T: +31 (0)71 524 7400
FTI Consulting
Julia Phillips/ John Dineen, T: +44 (0)207 269 7193
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