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Dollars and antisense
October 2013
by Ilene Schneider  |  Email the author


WESTON, Mass.Biogen Idec and Isis Pharmaceuticals Inc., have announced a multiyear collaboration to leverage antisense technology to advance the treatment of neurological diseases. Over the next six years, teams at the two companies will work closely together to help build a neurological disease franchise aimed at producing many novel medicines to populate the Biogen Idec and Isis pipelines.   
Founded in 1978, Biogen Idec, the world's oldest biotechnology company, discovers, develops and delivers therapies for the treatment of neurodegenerative diseases, hemophilia and autoimmune disorders, including multiple sclerosis therapies. Isis uses its antisense technology to discover and develop novel drugs for its product pipeline, which consists of 30 drugs 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 upfront with potential future milestone payments and license fees, plus royalties. The agreement is the fourth collaboration between the two companies in the last two years.
"These agreements represent a broad, strategic commitment shared by the two companies," says Dr. Stan Crooke, Isis' chairman and CEO. "We believe that together, we can enhance our understanding of neurological diseases and make important advances in their treatment. Coupling our antisense technology with Biogen Idec's internal and external network of disease knowledge from basic science through complex, cutting-edge clinical development holds great promise for patients."
According to Dr. Douglas E. Williams, Biogen Idec's executive vice president of research and development, "this strategic alliance with Isis builds on our existing relationship and combines the unique strengths of each partner to significantly advance the treatment of serious neurological diseases."
"Antisense is a validated technology that we believe will help build our pipeline of antisense, small-molecule and biologics programs, strengthening our 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 underlying genetic defect. When the genetic sequence of a gene is known to cause a disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that binds to the messenger RNA (mRNA) produced by that gene and effectively turn that gene on or off.  
As part of the research collaboration, Biogen Idec will gain exclusive rights to the use of Isis' antisense technology to develop therapies for neurological diseases. Biogen Idec will make an upfront payment of $100 million to Isis, which is eligible to receive milestone payments, license fees and royalty payments for all treatments developed through this collaboration, with the specific amount dependent upon the modality of the molecule advanced by Biogen Idec. For antisense molecules, the milestone payments could be as much as $220 million, plus additional amounts related to the cost of clinical trials conducted by Isis under the collaboration.
The two companies will select and validate neurological disease targets and conduct drug discovery activities with the goal of identifying clinical development candidates. Isis will be primarily responsible for drug discovery and early development of antisense therapies, while Biogen Idec will be responsible for the creation and development of small-molecule treatments and biologics. Biogen Idec will determine the molecular modality best suited for advancement through clinical development and be responsible for later-stage development and commercialization of all drugs arising from the collaboration.  
Three previous collaborations between Biogen Idec and Isis were established to develop antisense drugs to treat spinal muscular atrophy and myotonic dystrophy type 1 and to discover and develop antisense drugs against three novel targets for neurological disorders. For the present collaboration, Isis is combining its expertise in neurological diseases with the extensive internal and external knowledge and networks that Biogen Idec has developed to conduct a disease research program intended to explore multiple different targets for neurological diseases. This rigorous exploration of targets is something that antisense technology facilitates, according to the companies.
"We believe the power of this disease research component of the relationship is very significant," says Lynne Parshall, Isis' chief operating officer. "We're hopeful that we'll be successful on validating two or more targets a year as very exciting and potentially novel targets for neurological diseases."
Code: E101325



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