Pharming Announces Presentation Of New Ruconest® Clinical Data
Leiden, The Netherlands, June 18, 2012. Biotech company Pharming Group NV (“Pharming” or “the Company”) (NYSE Euronext: PHARM) today presented clinical safety and efficacy data for Ruconest® (recombinant human C1 inhibitor, or rhC1INH) at the annual meeting of the European Academy of Allergy and Clinical Immunology (EAACI), Geneva, Switzerland, 16 to 20 June 2012.
The new data covers a number of aspects that are relevant to the increasing number of physicians that use Ruconest in the day-to-day treatment of HAE patients in the European Union: Efficacy and safety data for Ruconest in the treatment of adolescents suffering from acute attacks of HAE; underpinning a potential extension of the European labeling of Ruconest and “real- life” experience by French physicians; building confidence in Ruconest by successf ul treatment with Ruconest of a HAE patient that previously failed various other treatments.
In addition an analysis of a previously reported open- label study to explore potential benefits of Ruconest in prophylaxis of HAE, potentially an additional indication that could be explored.
The headlines and authors of the poster presentations are:
Toubi et al: “Safety and Efficacy evaluation of rhC1INH for the treatment of HAE attacks in adolescent patients”. This analysis reviewed data from 16 adolescent HAE patients who were treated for a total of 50 angioedema attacks with Ruconest. Patients were treated up to 7 times for HAE attacks at all locations. Median times to onset of symptom relief for successive attacks ranged from 19 to 123 minutes. Median times to minimal symptoms ranged from 120 to 650 minutes. Ninety percent of the attacks responded within 4 hours after treatment, and none of the attacks relapsed. The most frequentl y reported adverse event was headache. No hypersensitivity reactions and no drug-related serious adverse events were observed. No treatment-emergent antibodies developed in these patients.
Bouillet et al: The case of a type III HAE patient who failed several therapies and was successfully treated with Ruconest.
Reshef et al: “Safety and Efficacy of a weekly infusion of Recombinant Human C1 Inhibitor (rhC1INH) for Prophylaxis of Hereditary Angioedema (HAE) attacks”. This was an open label study on the prophylactic effect of once-weekly administration of Ruconest in 25 HAE patients. Patients included in this study had a history of frequent HAE attacks, with a significant impact on their quality of life. The frequency of HAE attacks during the 8 week treatment period was reduced by approximately 50 percent, from a median of 0.6 attacks per week to 0.3. The repeated administrations were generally safe and well-tolerated.
A bout the EAACI Annual Meeting
The EAACI Congress 2012 will attract around 7000 international clinicians, researchers and allied health professionals. This is the most important professional meeting for advances in research, treatment and prevention of allergic and immunologic diseases (such as asthma, rhinitis, eczema and occupational allergy, food and drug allergy, severe anaphylactic reactions, rheumatic and autoimmune diseases, AIDS). More information on the congress can be found on www.eaaci2012.com.
RUCONEST® Phase III Study
Pharming is conducting a Phase III clinical study with RUCONEST® under a Special Protocol Assessment (SPA) that is intended to support the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). RUCONEST is being evaluated for the treatment of acute attacks of angioedema in patients with HAE in an international, multicenter, randomized, placebo-contro lled Phase III study at a dosage of 50 U/kg with a primary endpoint of time to beginning of relief of symptoms. Santarus has licensed certain exclusive rights from Pharming to commercialize RUCONEST in North America for the treatment of acute attacks of HAE and other future indications. Under the terms of the license agreement, a $10 million milestone is payable to Pharming upon successful achievement of the primary endpoint of the Phase III clinical study. The study is expected to be completed in Q3 2012.
