Pharming Group N.V. ("Pharming" or "the Company") (Euronext: PHARM) presented today an update on recombinant human C1 inhibitor ("rhC1INH") development for the treatment of hereditary angioedema at its investigator meeting in Budapest. The meeting was attended by investigators from six European markets to present results from on-going clinical trials with rhC1INH and discuss follow-on new indications for the product.
The patients treated with rhC1INH showed rapid time to beginning of relief (15 mins to 4hrs) and time to complete resolution (1hr to 48 hrs) as well as favourable evaluation of treatment by both patients and physicians. The cases presented include the use of rhC1INH for frequent attacks of HAE as well as its use for HAE attacks occurring concurrently in multiple locations in the body.
"We are pleased to report positive results on rhC1INH from these markets as a potential treatment for acute attacks of HAE," said Dr. Francis J. Pinto, CEO of Pharming. "Pharming appreciates the support of the HAE investigators and patients with the clinical trials, which will help bring rhC1INH to market for HAE patients worldwide as quickly as possible."
The results of treatment with rhC1INH are consistent with those previously reported by the Company. Treatments with rhC1INH did not elicit any allergic or clinically relevant immune responses. None of the patients treated with rhC1INH in the clinical trials experienced a relapse of the initial HAE attack. After treatment, patients are monitored for a period of 90 days to evaluate the safety of rhC1INH.
Pharming is conducting a randomized placebo controlled double blind Phase III clinical trial for rhC1INH across Europe and remains on track to file for approval of the product with the European Agency for the Evaluation of Medical Products (EMEA) in 2005.
Background on Hereditary Angioedema ("HAE")
HAE is a genetic disorder caused by C1 inhibitor deficiency. The disease is characterized by acute attacks of swelling of soft tissues (edema), including regions of the skin, the guts, and the mouth and throat. If the soft tissue of the throat is involved, an attack of angioedema can be fatal. In addition to the life-threatening nature of the disease, quality of life may be seriously impaired. HAE attacks that are untreated typically last for 2 to 5 days. Current treatment of HAE consists of prophylaxis and management of acute attacks. Attacks of angioedema can be effectively treated with intravenous administration of C1 inhibitor purified from human blood. However, C1 inhibitor preparations obtained from human blood have only been approved for use in some European countries. Although blood derived material is shown to be effective for HAE, treatment with such plasma-derived preparations has potential drawbacks in terms of safety and product supply.
Background on Pharming Group N.V.
Pharming Group N.V. is developing innovative protein therapeutics for unmet medical needs. The Company’s products include potential treatments for genetic disorders and specialty products for surgical indications. Pharming’s lead product for hereditary angioedema is in Phase III of clinical development. The advanced technologies of the Company include novel platforms for the production of protein therapeutics, as well as technology and processes for the purification and formulation of these products. 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.
