Pharming and Swedish Orphan announce publication of randomized clinical trial results with Pharming's recombinant human C1 inhibitor
Leiden, The Netherland and Stockholm, Sweden, October 5, 2010. Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) and Swedish Orphan Biovitrum (STO: SOBI) announced today the publication of the integrated analysis of Pharming's randomized placebo-controlled clinical trials with recombinant human C1 esterase inhibitor (rhC1INH; conestat alfa; Ruconest(TM) in Europe; Rhucin® in other countries) for treatment of acute angioedema attacks in patients with Hereditary angioedema (HAE) in the October issue of the peer-reviewed Journal of Allergy and Clinical Immunology.
In the publication, Zuraw et al report the results of two similar but independent, randomized, placebo-controlled studies that were conducted in 70 HAE patients in total to evaluate the efficacy and safety of 50 and 100 U/Kg of rhC1INH for the treatment of acute angioedema attacks. In the European study, 32 patients were randomized (16 patients received rhC1INH 100U/kg and 16 patients saline) and 38 patients in the North American study (13 patients received 100 U/kg rhC1INH, 12 patients 50 U/kg and 13 patients saline).
Both doses of rhC1INH (50U/kg and 100U/kg) significantly reduced the time to beginning of relief of symptoms for all anatomical locations studied (abdominal, genitourinary, facial-laryngeal or peripheral) compared to placebo and resulted in high response rate for rhC1INH (>90%). Adverse events occurred less frequently in the rhC1INH arm than in the placebo arm. No antibody responses against rhC1INH or host-related impurities were observed. The authors conclude that rhC1INH constitutes a highly effective alternative to plasma derived C1INH for the treatment of acute angioedema attacks in HAE patients.
Lead author Bruce Zuraw, MD, said: "The publication of the results from these randomized controlled trials is an important step in educating the medical community about recombinant human C1INH for HAE patients with acute angioedema." Dr. Zuraw is a leading authority on HAE and is Professor of Medicine and Chief, Section of Allergy & Immunology in the Department of Medicine, University of California, San Diego and Research Scientist at the San Diego VA Healthcare, La Jolla, CA.
"The publication in this peer-reviewed journal provides a solid background for us to build ahead of the anticipated European market launch in the fourth quarter of this year," said Dr Rienk Pijpstra, Chief Medical Officer at Pharming.
"We are excited about the data and clinical efficacy of Ruconest and are very much looking forward to spearhead the launch of Ruconest in most countries in Europe," said Kennet Rooth, Vice President, Marketing and Sales at Swedish Orphan Biovitrum. "Ruconest will help many patients and make life much easier for them."
About HAE and C1 esterase inhibitor
Pharming's rhC1INH is a human protein developed through Pharming's proprietary technology where the human protein is expressed in milk of transgenic rabbits. HAE is a human genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in an overreaction of the immune system. The disease is characterized by unpredictable and debilitating episodes of intense swelling of the extremities, face, trunk, genitals, abdomen and upper airway, which may last up to five days when untreated. In addition to the life-threatening nature of the disease in case of laryngeal attacks, quality of life for individuals with the disease may be seriously impaired. Approximately one in 30,000 individuals (1:10,000 - 1:50,000) suffers from HAE with an average of approximately eight acute attacks per year.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of genetic disorders, specialty products for surgical indications, and nutritional products. On June 24, the European Medicines Agency adopted a positive opinion for Ruconest(TM) (Rhucin® in non-EU territories) for the treatment of angioedema attacks. Market Authorization in the European Economic Area is therefore expected imminently with an anticipated market launch in the fourth quarter 2010. The product is also under development for follow-on indications, i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF) following kidney transplantation. 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. Additional information is available on the Pharming website, www.pharming.com<http://www.pharming.com>.
About Swedish Orphan Biovitrum
Swedish Orphan Biovitrum is a Swedish based niche specialty pharmaceutical company with an international market presence. The company is focused on providing and developing specialist pharmaceuticals for rare disease patients with high medical needs. The portfolio consists of about 60 marketed products and an emerging late stage clinical development pipe-line. Our focus areas are: hemophilia, inflammation/autoimmune diseases, fat malabsorption, cancer supportive care and inherited metabolic disorders. Swedish Orphan Biovitrum had pro-forma revenues 2009e of about 2 BSEK and approximately 500 employees. The head office is located in Sweden and the share (STO: SOBI) is listed on NASDAQ OMX Stockholm. For more information please visit www.sobi.com<http://www.sobi.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.
Pharming: Marjolein van Helmond, Manager Corporate Communication, T: +31 (0)71 52 47 431 or +31 (0)6 109 299 54
Swedish Orphan Biovitrum Kennet Rooth, VP and Head of Sales & Marketing, T +46 (0) 8 697 20 00
Press release (PDF)<http://hugin.info/132866/R/1449016/391044.pdf>
Posted: October 2010