Interim analysis confirms immunosafety of Rhucin
Leiden, The Netherlands, June 1, 2009. Biotech company Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) announced today that it has presented an abstract describing the interim analysis of the immunosafety of Rhucin® (recombinant human C1 inhibitor) in patients with Hereditary Angioedema (HAE) at the 6th C1-Inhibitor Deficiency Workshop in Budapest. Rhucin® immuno-safety review is an important part of both the EMEA (European Medicines Agency) and the FDA (US Food and Drug Administration) upcoming regulatory submissions. Results of the presented interim-analysis confirm the absence of immunological side-effects of Rhucin®.
A detailed analysis of possible immunological side-effects of Rhucin® was requested by the EMEA as part of its evaluation of Rhucin® to treat patients suffering from acute attacks of HAE. This request was made since therapeutic recombinant proteins in general often feature some side-effects that are related to immune responses of the patients. This is especially the case when the therapeutic product is used repeatedly.
Currently 140 HAE patients, undergoing more than 350 treatments, have been systematically followed-up for up to three months post-treatment. With the exception of one healthy volunteer that participated in the very first Phase I investigation and had pre-existing rabbit allergy, no clinical immunological side effects of Rhucin® were identified. Blood samples were collected from every person that received Rhucin®, before and after exposure, and tested for several types of antibodies against: C1-inhibitor and against possible impurities. An interim analysis of the immunological data of Rhucin® of more than 200 administrations to symptomatic HAE patients presented at the Budapest meeting supports the absence of antibody responses in all these patients, including patients who were treated with Rhucin® at multiple occasions.
"We are extremely pleased with the results of this extensive immuno-safety analysis. It, again, confirms the overwhelmingly positive benefit-risk ratio of Rhucin®. A full immuno-safety analysis will form an important element of our submissions to the EMEA and FDA to obtain marketing approval for Rhucin®. It also forms a very strong basis from which we can develop C1 inhibitor for follow-on indications, for instance in the important field of rejection of organ transplants," said Pharming's COO, Dr. Bruno Giannetti.
In the meantime, Rhucin® clinical trials are continuing and data from them will be included in the dossiers for the FDA and EMEA to demonstrate yet further increased numbers of patients and treatments. In addition, Pharming is preparing filings for Rhucin® in territories outside the USA and the European Union. These activities will, from time to time, result in new clinical studies being initiated to meet approval requirements in these specific countries. These trials do not affect the submission of the Rhucin® dossier to the EMEA and the submission to the FDA.
A more detailed overview of the interim results as presented at the Budapest meeting can be found in the investor information section on our website http://www.pharming.com.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of genetic disorders, ageing diseases, specialty products for surgical indications, intermediates for various applications and nutritional products. Pharming has two products in late stage development - Rhucin® for Hereditary Angioedema and human Lactoferrin for use in food products and one product in early stage clinical development - Prodarsan® for Cockayne Syndrome. The advanced technologies of the Company include innovative platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products, as well as technology in the field of DNA repair (via DNage). Additional information is available on the Pharming website, http://www.pharming.com.
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, Pharming Group NV, T: +31 (0)71 52 47 400 or +31(0)6 519 17 162.
Posted: June 2009