Pharming Group Completes Recruitment of RUCONEST(R) US Pivotal Phase III Study
Published: Jul 05, 2012
Leiden, The Netherlands--(Marketwire - July 05, 2012) - Biotech company Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) today announces that it has reached full recruitment of its ongoing US pivotal Phase III clinical study (Study 1310) evaluating the investigational drug RUCONEST® (recombinant human C1 inhibitor) for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE). 75 randomized patients are in the study and no further patients will be entered.
Study 1310 will now continue until such time that either all of the treated patients have received an open label treatment for a subsequent HAE attack, or until 90 days have lapsed since their randomized attack. This will be followed by the analysis of the results. Positive results of the study will trigger a US$ 10 million milestone payment to Pharming from its US partner Santarus Inc.
Currently 53 of the 75 patients have experienced a repeat attack or achieved a 90 day attack free period.
Bruno Giannetti, COO of Pharming, said, "Completion of recruitment into this clinical study is an important milestone for Pharming. As previously stated, the final step to completion of the trial consists of an additional follow up period of up to 90 days, depending on when patients experience a subsequent attack.
However, it is a generally accepted assumption that approximately one to two attacks occur per month in otherwise untreated HAE patients, suggesting that those individuals in our study may typically experience a subsequent attack significantly sooner than 90 days post their randomized attack."
About RUCONEST® and Hereditary Angioedema
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 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 for the treatment of acute attacks of HAE, 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.
About Pharming Group NV
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 product is also being evaluated for follow-on indications in the areas of transplantation and reperfusion injury. 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.
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