Not ‘immune’ to each other’s charm

WESTON, Mass.—Biogen Idec and South San Francisco-basedPortola Pharmaceuticals Inc. joined forces recently in an exclusive, worldwidecollaboration and license agreement for the development and commercializationof highly selective, novel oral Syk inhibitors. The two companies will focus onvarious autoimmune and inflammatory diseases, including rheumatoid arthritis(RA) and systemic lupus erythematosus (SLE).

Per the terms of the agreement, Portola will gain $45million from Biogen Idec, who will provide Portola with $36 million in anupfront cash payment and will also purchase $9 million of Portola equity.Portola could stand to receive additional payments of up to $508.5 million ifcertain development and regulatory milestones are achieved. Biogen Idec will beresponsible for global development and commercialization efforts for the Sykinhibitor program in major indications, such as RA and lupus, and Portola willhandle U.S. development and commercialization for select smaller indications,in addition to handling discovery efforts for follow-on Syk inhibitors. Portolawill retain an option to co-promote major indications in the United States withBiogen Idec. The worldwide costs and profits will be split between thecompanies, with Biogen Idec fielding 75 percent and Portola fielding 25percent.

"Biogen Idec is a world-class R&D organization with asignificant global footprint and track record of success in developing andcommercializing innovative autoimmune disease therapies," William Lis, CEO of Portola,said in a press release. "For Portola, the terms of this collaboration reflectthe scientific advances we've made in the discovery of orally available kinaseinhibitors and our vision to commercialize products with clear and meaningfulvalue. Partnering with Biogen Idec provides us with the resources and addedexpertise to pursue the full potential of our Syk inhibitor program."

Lis says the collaboration will include Portola's leadmolecule, PRT062607, and "a number of Syk backup molecules." PRT062607 iscurrently in Phase I trials, and has proven to be both highly potent andselective as an oral inhibitor of Syk in a panel of in-vitro kinase and cellular assays. The studies suggestPRT062607 is well tolerated and suitable for once-daily dosing. Portola willwork with Biogen Idec on development in major indications, Lis notes, and willhave "lead-discovery responsibilities on minor indications such as idiopathicthrombocytopenic purpura and hemolytic anemia." The agreement, he says, is "agood cultural and scientific fit between two biotech companies."

"Biogen shares Portola's vision that there is broadtherapeutic potential for Syk inhibitors, and has the focus and expertise inautoimmune therapies including development and commercialization in rheumatoidarthritis," says Lis.

"A significant portion of people with rheumatoid arthritisdo not respond to currently approved treatments or have only modest responses,and treatment options for lupus are limited," Dr. Doug Williams, executive vicepresident of research and development at Biogen Idec, said in a press release."Inhibition of Syk has the potential to provide effective, well-toleratedtherapies for patients with these and other autoimmune diseases. We areencouraged by the preclinical and clinical data to date and see an opportunityto develop a best-in-class, highly selective oral treatment for thesedevastating diseases."

Naomi Aoki, director of public affairs and a spokeswoman forBiogen Idec, says the company considers Syk inhibitors a "really interestingapproach," noting that when it was looking at various Syk inhibitors, Portola'sstood out due to its high selectivity.

"We think that it has the potential to be a best-in-classoral 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 thescientists and management at Portola very well, and there is "a very high levelof respect for the quality of the science that they do."

In terms of the potential of Syk inhibitors, Lis saysPortola feels novel oral therapies are "set to transform multipleautoimmune/inflammation markets," which he says hardly lack for demand.

"By 2020, the market is expected to exceed $40 billion," Lisnotes. "In the G6 nations, there are more than 20 million patients acrossindications (RA, lupus, psoriasis, severe allergic asthma, organ transplant,IBD and multiple sclerosis)."

The World Health Organization sets the number of RAsufferers at approximately 1.3 million within the United States, and over 23million worldwide. Approximately 1.5 million Americans have lupus, and anestimated 5 million are stricken with the disease worldwide, according to the LupusFoundation of America.