Biogen Idec will team up with a joint venture of Italy’s Fondazione Telethon and Ospedale San Raffaele to develop gene therapies for hemophilia A and B—a collaboration that deepens the biotech giant's presence in battling the bleeding disorders.
The joint venture—known as San Raffaele-Telethon Institute for Gene Therapy, or TIGET—has developed a lentiviral gene transfer vector designed through direct administration into the body to target liver cells. The vector gene therapy approach has shown promise in clinical trials for the treatment of some immune-hematologic and neurodegenerative diseases—a promise the collaboration partners hope to show in hemophilia.
TIGET will receive $5 million upfront from Biogen Idec for rights to the gene therapy collaboration’s programs and their development. The joint venture will also receive undisclosed additional payments from Biogen Idec tied to achieving development milestones.
In return, Biogen Idec agreed to pay the research costs for two programs—one for hemophilia A, the other for hemophilia B. Biogen Idec will have the option to exercise worldwide development and commercialization rights for each program after initial clinical proof of concept trials.
“There is hope that gene therapy could one day lead to a single-dose, lasting therapy,” Olivier Danos, Ph.D., Biogen Idec svp of gene therapy, said in a statement. “Collaborating with the gene therapy pioneers at TIGET is an exciting step as we build a world-class platform in an effort to identify new approaches that could fundamentally change the lives of people with life-altering diseases.”
Added TIGET director Luigi Naldini: “We at TIGET have worked for several years to develop a new vector design that upon administration into the blood stringently targets expression of its genetic cargo to the hepatocytes, the main cell type of the liver. This was crucial to establish long-term expression of the therapeutic gene and obtain proof-of-principle of its therapeutic benefit in experimental models of hemophilia B.”
TIGET was formed in 1995 by the San Raffaele Hospital and Scientific Institute and the Telethon Foundation to carry out research on gene transfer and cell transplantation, as well as and translate its results into clinical applications of gene and cell therapies for different genetic diseases.
The collaboration brings Biogen Idec further into development of hemophilia treatments. Last year, the biotech giant joined with Swedish Orphan Biovitrum (Sobi) to launch of the companies’ first product for people with hemophilia, a combination of the coagulation Factor IX (Recombinant) molecule combined via fusion with the protein fragment Fc. Alprolix is designed to treat adults and children with the rare disease hemophilia B, which is estimated to affect about 3,300 Americans, primarily males.
Alprolix won marketing approval from the FDA in March 2014, enabling Biogen Idec and Sobi to enter a market whose largest players to date have included pharma giants like Pfizer and Baxter International.
Two months later, coinciding with the launch of Alprolix, Biogen Idec and Sobi said they will donate 1 billion units of clotting factor to developing nations over the next decade. The companies said at the time that their donation would enable physicians to treat more than 75,000 joint bleeding episodes, more than 2,000 life threatening bleeding episodes, and thousands of elective surgical procedures that require access to clotting factor.
In June 2014, Biogen Idec and Sobi won the FDA’s nod again for a second hemophilia treatment, Eloctate, which combines the coagulation Factor VIII (Recombinant) molecule via fusion with Fc. Eloctate is indicated for adults and children with hemophilia A.
Over the first nine months of 2014, Alprolix generated $35.702 million in product revenues, while Eloctate racked up $21.560 million. Full-year results are set to be reported later today after the close of financial markets.