Pharming announces agreement with Renova Life targeting factor VIII for the treatment of haemophilia A
Biotech company Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) today announced that it has signed a service agreement with Renova Life, Inc, (RLI) a biotech company based in Maryland, USA. The agreement covers the development and supply of founder transgenic rabbits from RLI to Pharming. The founder rabbits will enable Pharming to start the commercial production breeding process. The first protein to be expressed in the rabbits will be recombinant human Factor VIII (rhFVIII) for the treatment of haemophilia A.
RLI recently announced the birth of rhFVIII transgenic rabbits through their Chinese subsidiary, Lannuo Biotechnologies (Wuxi, China) and have previously had successful functional expression of rhFVIII in mice. Pharming will leverage its proprietary and validated rabbit platform for the production of recombinant human proteins to develop rhFVIII.
The recent European approval of Pharming's rh C1 inhibitor (Ruconest®) has demonstrated Pharming's ability to produce industrial volumes of high quality recombinant human protein through a method which requires significantly lower upfront capital investment and manufacturing costs compared to current cell based technologies.
Haemophilia A is an X chromosome linked hereditary disorder caused by defects in the Factor VIII (FVIII) gene that lead to lower levels of the functional FVIII protein. Lack of functional FVIII diminishes the body's clotting ability, which in turn can lead to damaging or fatal bleeding episodes. The global rhFVIII market was estimated to worth US$3.8 billion in 2009, with 90% of sales in the developed markets and very high unmet medical needs in the developing markets, such as China. In addition, only approximately 50% of the world-wide estimated haemophilia market can currently be supplied with appropriate FVIII therapy. Hence, there is still a high unmet medical need in this field with an estimated total market potential of US$10 billion.
Dr Fuliang Du, President of RLI, commented: "RLI is very excited to combine our recent technological successes of expressing significant quantities of active rhFVIII in mice and the subsequent birth of our first FVIII transgenic rabbits with Pharming's validated and industrialised transgenic platform."
Bruno Giannetti, COO of Pharming, stated: "Pharming is pleased to have taken this first step towards developing recombinant transgenic Factor VIII and addressing the current demands and unmet needs of the global haemophilia market. This agreement is in line with Pharming's stated strategy of leveraging the embedded value of our proprietary transgenic platform through licensing and co- development agreements, to develop protein therapeutics targeting unmet medical needs and commercially attractive markets
