Biogen Idec, Isis announce SMA collaboration

WESTON, Mass.— A new partnership has been announced thisweek, with Biogen Idec and Isis Pharmaceuticals, Inc. entering into anexclusive, worldwide option and collaboration agreement, under which the twocompanies will develop and commercialize ISIS-SMNRx, Isis' antisenseinvestigational drug indicated for the treatment of spinal muscular atrophy(SMA).

Per the terms of the agreement, Biogen Idec will pay Isis$29 million upfront and up to an additional $45 million in milestone paymentsassociated with the clinical development of the compound prior to licensing.Isis will handle global development through the completion of Phase II/IIIregistrational clinical trials, while Biogen Idec will provide advice on clinicaltrial design and regulatory strategy. In return, Biogen Idec will have theoption to license ISIS-SMNRx until completion of the first successful PhaseII/III trial. If the option is exercised, Biogen Idec will be responsible forglobal development, regulation and commercialization. Isis also stands toreceive up to another $225 million in a license fee and milestone paymentsrelated to the exercise of the option, and will receive double-digit royaltieson sales of the drug.

"Biogen Idec's expertise in the global development andcommercialization of innovative new therapies for neurologic diseases is agreat strategic fit to advance ISIS-SMNRx," Stanley T. Crooke, M.D., Ph.D.,Chairman of the Board and Chief Executive Officer of Isis, said in a press release."This alliance is consistent with our business strategy to develop antisensedrugs to proof-of-concept with a knowledgeable partner that is committed tosupporting the rapid development of the drug. Given the severity of the unmetneed in SMA, our proof-of-concept studies should also serve as theregistrational trials for ISIS-SMNRx. We believe that, together with BiogenIdec, we will be able to expeditiously develop this investigational drug inhopes of bringing to market an effective and desperately needed treatment toimprove the lives of children with SMA."

SMA, a genetic neuromuscular disease that causes muscleatrophy and weakness, is the most common genetic cause of infant mortality,with one child out of every 10,000 births worldwide born with the disease.Infants with SMA appear normal at birth, but symptoms can start developing asearly as a few months after birth. At its most severe, SMA can lead to asignificantly shortened lifespan for children.

ISIS-SMNRx is designed to compensate for the underlyinggenetic defect that leads to SMA. SMA is caused by a loss of or defect in thesurvival motor neuron 1 (SMN1) gene, which leads to a decrease in the proteinsurvival motor neuron (SMN), a protein vital to the health and survival ofnerve cells in the spinal cord responsible for neuromuscular growth andfunction. ISIS-SMNRx, as described on the company's website, "is designed totreat all types of childhood SMA by altering the splicing of a closely relatedgene (SMN2) that leads to the increased production of fully functional SMNprotein." The drug has received Orphan Drug Designation and Fast Track statusfrom the U.S. Food and Drug Administration, and is currently in a Phase I studyof children ages 2-14 with SMA to determine its safety, tolerability andpharmacokinetic profile.

"SMA is a heartbreaking disease – it can kill childrenbefore their second birthday, and there are currently no therapies to treat thedisease," George A. Scangos, Ph.D., CEO of Biogen Idec, said in a pressrelease. "It is exactly the kind of disease and program that we are focused onat Biogen Idec. The unmet need could not be any greater, the program fits withour mission to bring innovative therapies to patients with serious neurologicdiseases and Isis' antisense compound has the potential to be a highlyeffective, first-to-market therapy for this deadly disease. We have the utmostrespect for Isis' scientific leadership and expertise in antisense technology,and we have crafted a collaboration that brings together our two companies'strengths toward a common goal."