BioWorld International Correspondent
Pharming Group NV moved its lead drug candidate, recombinant human C1 inhibitor, into a pivotal Phase III clinical trial for treatment of hereditary angioedema (HAE).
Chief Business Officer Rein Strijker told BioWorld International dosing will commence this quarter. "We hope to finish the clinical trial this year," he said.
The multicenter, randomized, placebo-controlled, double-blind study will recruit 50 patients with HAE, a genetic condition resulting from a deficiency of the C1 inhibitor protein, which is important for controlling the complement cascade during inflammation. It is characterized by acute attacks - up to seven or eight per year in some cases - during which soft tissues found in the skin, gut, throat and mouth swell up.
The Leiden, Netherlands-based company aims to launch rhC1INH in Europe during 2005, and last month took a step toward that goal by signing a letter of intent to enter a development and marketing agreement with Laboratorios del Dr. Esteve SA, of Madrid, Spain. The accord, which is subject to due diligence, would cover Spain, Portugal and Greece. The company is in discussions with other potential partners, Strijker said, and it may opt to repeat in certain territories the regional strategy it has adopted in the southern part of the continent.
"These countries are sometimes better served by good local companies than by large multinational companies," he said. However, Pharming does not aim to have more than five partners in total. Its partnering discussions extend to the U.S., where it also aims to commence clinical studies of rhC1INH in the near term.
HAE is an autosomal-dominant disorder, affecting approximately 1 in 30,000 people in the Western world, according to Pharming. "Untreated attacks last several days - up to five days - and in many cases people have to go to hospital," Strijker said. Previous clinical studies showed that rhC1INH can alleviate symptoms in minutes to hours. Pricing, therefore, could reflect the potential cost savings.
Pharming obtains rhC1INH from the milk of transgenic rabbits, and establishing a cell culture production platform for the protein has proved difficult, Strijker said. It currently has sufficient capacity to meet its needs through 2005, and the company has plans to double capacity thereafter. "At some point, if we are going to look at other indications, we might have to look at other production systems," he said. Transgenic cattle would enable the company to produce greater quantities, he said.