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Not ‘immune’ to each other’s charm
December 2011
by Kelsey Kaustinen  |  Email the author


WESTON, Mass.—Biogen Idec and South San Francisco-based Portola Pharmaceuticals Inc. joined forces recently in an exclusive, worldwide collaboration and license agreement for the development and commercialization of highly selective, novel oral Syk inhibitors. The two companies will focus on various autoimmune and inflammatory diseases, including rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE).
Per the terms of the agreement, Portola will gain $45 million from Biogen Idec, who will provide Portola with $36 million in an upfront cash payment and will also purchase $9 million of Portola equity. Portola could stand to receive additional payments of up to $508.5 million if certain development and regulatory milestones are achieved. Biogen Idec will be responsible for global development and commercialization efforts for the Syk inhibitor program in major indications, such as RA and lupus, and Portola will handle U.S. development and commercialization for select smaller indications, in addition to handling discovery efforts for follow-on Syk inhibitors. Portola will retain an option to co-promote major indications in the United States with Biogen Idec. The worldwide costs and profits will be split between the companies, with Biogen Idec fielding 75 percent and Portola fielding 25 percent.
"Biogen Idec is a world-class R&D organization with a significant global footprint and track record of success in developing and commercializing innovative autoimmune disease therapies," William Lis, CEO of Portola, said in a press release. "For Portola, the terms of this collaboration reflect the scientific advances we 've made in the discovery of orally available kinase inhibitors and our vision to commercialize products with clear and meaningful value. Partnering with Biogen Idec provides us with the resources and added expertise to pursue the full potential of our Syk inhibitor program."  
Lis says the collaboration will include Portola's lead molecule, PRT062607, and "a number of Syk backup molecules." PRT062607 is currently in Phase I trials, and has proven to be both highly potent and selective as an oral inhibitor of Syk in a panel of in- vitro kinase and cellular assays. The studies suggest PRT062607 is well tolerated and suitable for once-daily dosing. Portola will work with Biogen Idec on development in major indications, Lis notes, and will have "lead-discovery responsibilities on minor indications such as idiopathic thrombocytopenic purpura and hemolytic anemia." The agreement, he says, is "a good cultural and scientific fit between two biotech companies."
"Biogen shares Portola's vision that there is broad therapeutic potential for Syk inhibitors, and has the focus and expertise in autoimmune therapies including development and commercialization in rheumatoid arthritis," says Lis.
"A significant portion of people with rheumatoid arthritis do not respond to currently approved treatments or have only modest responses, and treatment options for lupus are limited," Dr. Doug Williams, executive vice president of research and development at Biogen Idec, said in a press release. "Inhibition of Syk has the potential to provide effective, well-tolerated therapies for patients with these and other autoimmune diseases. We are encouraged by the preclinical and clinical data to date and see an opportunity to develop a best-in-class, highly selective oral treatment for these devastating diseases."  
Naomi Aoki, director of public affairs and a spokeswoman for Biogen Idec, says the company considers Syk inhibitors a "really interesting approach," noting that when it was looking at various Syk inhibitors, Portola's stood out due to its high selectivity.
"We think that it has the potential to be a best-in-class oral therapy for rheumatoid arthritis and lupus and other diseases," says Aoki, adding that people on Biogen Idec's R&D team and executive team know the scientists and management at Portola very well, and there is "a very high level of respect for the quality of the science that they do."  
In terms of the potential of Syk inhibitors, Lis says Portola feels novel oral therapies are "set to transform multiple autoimmune/inflammation markets," which he says hardly lack for demand.  
"By 2020, the market is expected to exceed $40 billion," Lis notes. "In the G6 nations, there are more than 20 million patients across indications (RA, lupus, psoriasis, severe allergic asthma, organ transplant, IBD and multiple sclerosis)."  
The World Health Organization sets the number of RA sufferers at approximately 1.3 million within the United States, and over 23 million worldwide. Approximately 1.5 million Americans have lupus, and an estimated 5 million are stricken with the disease worldwide, according to the Lupus Foundation of America.
Code: E121107



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