GTC Biotherapeutics, Inc. said it has entered into a $1 million definitive agreement on an exclusive license to develop and commercialise a blood coagulant developed by Virginia, Blacksburg-based ProGenetics, LLC.
The licence includes ProGenetic's recombinant human factor IX, factor VIII, and human fibrinogen in North America, Europe and Japan.
ProGenetics is developing production of the human coagulation factors IX and VIII in the milk of transgenic pigs and has patents that cover the transgenic production of human fibrinogen. ProGenetics will receive a non-exclusive license to GTC's patent in the United States for the transgenic expression of therapeutic proteins in milk to enable the commercial development of these products outside of North America, Europe and Japan.
ProGenetics received $500,000 from GTC upon signing of the agreement and will receive approximately $500,000 from GTC in the second quarter of 2008. GTC will also receive a small equity interest in ProGenetics.
"We are very pleased to work with ProGenetics to add factor IX, factor VIII, and fibrinogen to our portfolio of recombinant coagulation factors," said Geoffrey F. Cox, Ph.D., chairman and chief executive officer, GTC. "Together with our program for recombinant human factor VIIa, GTC has a leading position in developing recombinant versions of all the major coagulation factors used to treat haemophilia and potentially other bleeding conditions. Our strategic portfolio of recombinant plasma proteins also includes ATryn, our recombinant form of human antithrombin, and recombinant human alpha-1 antitrypsin, establishing a broad franchise in hematology and associated genetic disorders."
ProGenetics will be responsible for the production of the proteins in the milk of their transgenic pigs. GTC will be responsible for manufacturing, clinical development, regulatory affairs, and commercialisation activities in its territories.
Haemophilia is caused by genetic conditions in which the patients' failure to express enough coagulation factors may lead to excessive bleeding. Type A haemophilia is due to the lack of factor VIII. Type B haemophilia is due to the lack of factor IX. The preferred treatment strategy is to provide supplemental coagulation factors prophylactically to avoid episodes of excessive bleeding. The price and availability of current recombinant coagulation factors often allows for use in only limited indications and markets. The aggregate market for plasma-derived and recombinant factor IX, factor VIII, and fibrinogen products in North America, Europe and Japan is currently estimated to be greater than $3 billion annually.
GTC Biotherapeutics develops, produces, and commercializes therapeutic proteins through transgenic animal technology. GTC obtained the first approval anywhere in the world for a transgenically produced protein when ATryn, a recombinant form of human antithrombin, was approved by the European Commission for use in patients with hereditary antithrombin deficiency undergoing surgical procedures. GTC's intellectual property includes a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal.