Annual Report 2002 - BIRD Foundation

Life SciencesDevelopment of a Pharmaceutical Composition Containing Recombinant Factor VIII and LiposomesThe CompaniesOMRI Laboratories Ltd. — Bayer Corporation, Biological ProductsThe ProjectOMRI Laboratories Ltd. is a biopharmaceutical companylocated in the Weizmann Science Park, Nes-Ziona, Israel.The company focuses on the development of liposomes asa delivery vehicle for Factor VIII (FVIII) and other short half-lifeproteins. OMRI developed a formulation of FVIII and liposomesthat was evaluated pre-clinically in a hemophilic mousemodel. Results indicated an extension of half-life and enhancedstability of recombinant FVIII (rFVIII), as well as an increasein efficacy when FVIII is administrated in a liposome formulation.Further animal studies showed that the formulation is nontoxic.Bayer Biological Products is devoted to the discovery,manufacturing and marketing of plasma – derived therapiesand their recombinant (genetically engineered) and/or genetherapy replacements. Biological Products is a businessunit within Bayer's global Pharmaceutical Business Group,which is a part of Bayer Worldwide Group, a $32 billion chemicaland pharmaceutical company based in Leverkusen, Germany.The market for Factor VIII, a lifesaving drug for treatment ofhemophilia A patients, is rapidly growing from $2.5 billion/yearto $5 billion/year. Annual treatment costs of severe hemophilia(typically three injections/week) are very high, exceeding$150,000 per patient. This is due to the enormous cost ofmanufacturing of this very complex protein (the price of rFVIIIis about $5 million/gram).Advances in the treatment of hemophilia have been focusedon reducing safety risks (HIV, hepatitis, etc.), but there hasbeen no success in reducing dosage frequency.This project aims to develop a pharmaceutical compoundcontaining recombinant factor VIII and liposomes for thetreatment of hemophilia A patients in a formulation that willenable weekly injections instead of the current three timesper week. The commercial vial of freeze-dried FVIII isreconstituted in a mixture of proprietary liposomes and injected,as is currently done with FVIII.The project is now in a clinical trial with severe hemophilia Apatients. To date, four patients have received the newformulation; the drug has been well tolerated, and preventionof bleeding for over one week was observed in patients whotypically bleed every two to four days.10