Biogen Idec and Isis Pharmaceuticals announce neurological disease collaboration

WESTON, Mass. and CARLSBAD, Calif.—Marking their fourthcollaboration in the last two years, Biogen Idec and Isis Pharmaceuticals Inc. announcedthis week that they have entered into a broad, multiyear collaboration toleverage antisense technology to advance the treatment of neurologicaldiseases. Previously, in 2012, Isis and Biogen Idec established collaborationsto develop antisense drugs to treat spinal muscular atrophy, myotonic dystrophytype 1 and three novel targets for neurological disorders.

The latest agreement combines Biogen Idec'sexpertise in neurology with Isis' leadership in antisense technology, with aneye toward developing novel therapies to treat neurological disorders. As partof the six-year research collaboration, Biogen Idec will gain exclusive rightsto the use of Isis' antisense technology to develop therapies for neurologicaldiseases, and it is paying $100 up front as part of that.

Isis is eligible to receive milestone payments,license fees and royalty payments for all treatments developed through thiscollaboration, with the specific amount dependent upon the modality of themolecule advanced by Biogen Idec. In the case of antisense molecules, themilestone payments could be as much as $220 million, plus additional amountsrelated to the cost of clinical trials conducted by Isis under thecollaboration.

"This strategic alliance with Isis builds onour existing relationship and combines the unique strengths of each partner tosignificantly advance the treatment of serious neurological diseases,"said Dr. Douglas E. Williams, Biogen Idec's executive vice president ofresearch and development. "Antisense is a validated technology that webelieve will help build our pipeline of antisense, small molecule and biologicsprograms, strengthening our leadership in neurology."

Antisense technology is designed to alter a gene'sfunction, silencing a mutation or activating a gene to compensate for anunderlying genetic defect. When the genetic sequence of a gene is known tocause 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 bythat gene and effectively turn that gene "on" or "off."

Biogen Idec and Isis will work together to selectand validate neurological disease targets and conduct drug discovery activitieswith the goal of identifying clinical development candidates. Isis will beprimarily responsible for drug discovery and early development of antisensetherapies, while Biogen Idec will be responsible for the creation anddevelopment of small molecule treatments and biologics. In each case, BiogenIdec will determine the molecular modality best suited for advancement throughclinical development. Biogen Idec will also be responsible for later stagedevelopment and commercialization of all drugs arising from the collaboration.

"We are pleased that Biogen Idec, a worldleader in neurological diseases, has selected antisense drug technology to fuelits neurology drug pipeline and are pleased to expand our already successfulrelationship," said B. Lynne Parshall, chief operating officer at IsisPharmaceuticals. "Biogen Idec's access to novel targets and contribution toresearch and development efforts should enable us to identify, validate andadvance a number of promising new therapies. Combining our antisense drugdiscovery with Biogen Idec's expertise in severe neurological diseases hasalready significantly enhanced the development of our spinal muscular atrophyand myotonic dystrophy programs."