Dollars and antisense

Biogen Idec and Isis Pharmaceuticals collaborate to advance treatment of neurological disorders

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WESTON, Mass.—Biogen Idec and Isis Pharmaceuticals Inc.,have announced a multiyear collaboration to leverage antisense technology toadvance the treatment of neurological diseases. Over the next six years, teamsat the two companies will work closely together to help build a neurologicaldisease franchise aimed at producing many novel medicines to populate theBiogen Idec and Isis pipelines. 
Founded in 1978, Biogen Idec, the world's oldestbiotechnology company, discovers, develops and delivers therapies for thetreatment of neurodegenerative diseases, hemophilia and autoimmune disorders,including multiple sclerosis therapies. Isis uses its antisense technology todiscover and develop novel drugs for its product pipeline, which consists of 30drugs to treat a wide variety of diseases. Its emphasis is on cardiovascular,metabolic, severe and rare diseases and cancer.
Under the agreement, Isis will receive $100 million upfrontwith potential future milestone payments and license fees, plus royalties. Theagreement is the fourth collaboration between the two companies in the last twoyears.
"These agreements represent a broad, strategic commitmentshared by the two companies," says Dr. Stan Crooke, Isis' chairman and CEO. "Webelieve that together, we can enhance our understanding of neurologicaldiseases and make important advances in their treatment. Coupling our antisensetechnology with Biogen Idec's internal and external network of diseaseknowledge from basic science through complex, cutting-edge clinical developmentholds great promise for patients."
According to Dr. Douglas E. Williams, Biogen Idec'sexecutive vice president of research and development, "this strategic alliancewith Isis builds on our existing relationship and combines the unique strengthsof each partner to significantly advance the treatment of serious neurologicaldiseases."
"Antisense is a validated technology that we believe willhelp build our pipeline of antisense, small-molecule and biologics programs, strengtheningour leadership in neurology," he says.
Antisense technology is designed to alter a gene's function,silencing a mutation or activating a gene to compensate for an underlyinggenetic defect. When the genetic sequence of a gene is known to cause adisease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or achemical analogue) that binds to the messenger RNA (mRNA) produced by that geneand effectively turn that gene on or off.
As part of the research collaboration, Biogen Idec will gainexclusive rights to the use of Isis' antisense technology to develop therapiesfor neurological diseases. Biogen Idec will make an upfront payment of $100million to Isis, which is eligible to receive milestone payments, license feesand royalty payments for all treatments developed through this collaboration,with the specific amount dependent upon the modality of the molecule advancedby Biogen Idec. For antisense molecules, the milestone payments could be asmuch as $220 million, plus additional amounts related to the cost of clinicaltrials conducted by Isis under the collaboration.
The two companies will select and validate neurologicaldisease targets and conduct drug discovery activities with the goal ofidentifying clinical development candidates. Isis will be primarily responsiblefor drug discovery and early development of antisense therapies, while BiogenIdec will be responsible for the creation and development of small-moleculetreatments and biologics. Biogen Idec will determine the molecular modalitybest suited for advancement through clinical development and be responsible forlater-stage development and commercialization of all drugs arising from thecollaboration.
Three previous collaborations between Biogen Idec and Isiswere established to develop antisense drugs to treat spinal muscular atrophyand myotonic dystrophy type 1 and to discover and develop antisense drugsagainst three novel targets for neurological disorders. For the presentcollaboration, Isis is combining its expertise in neurological diseases withthe extensive internal and external knowledge and networks that Biogen Idec hasdeveloped to conduct a disease research program intended to explore multipledifferent targets for neurological diseases. This rigorous exploration oftargets is something that antisense technology facilitates, according to thecompanies.
"We believe the power of this disease research component ofthe relationship is very significant," says Lynne Parshall, Isis' chiefoperating officer. "We're hopeful that we'll be successful on validating two ormore targets a year as very exciting and potentially novel targets forneurological diseases."

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