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

 

“Biogen Idec’s expertise in the global development and commercialization of innovative new therapies for neurologic diseases is a great 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 antisense drugs to proof-of-concept with a knowledgeable partner that is committed to supporting the rapid development of the drug. Given the severity of the unmet need in SMA, our proof-of-concept studies should also serve as the registrational trials for ISIS-SMNRx. We believe that, together with Biogen Idec, we will be able to expeditiously develop this investigational drug in hopes of bringing to market an effective and desperately needed treatment to improve the lives of children with SMA.”  

 

SMA, a genetic neuromuscular disease that causes muscle atrophy 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 as early as a few months after birth. At its most severe, SMA can lead to a significantly shortened lifespan for children.  

 

ISIS-SMNRx is designed to compensate for the underlying genetic defect that leads to SMA. SMA is caused by a loss of or defect in the survival motor neuron 1 (SMN1) gene, which leads to a decrease in the protein survival motor neuron (SMN), a protein vital to the health and survival of nerve cells in the spinal cord responsible for neuromuscular growth and function. ISIS-SMNRx, as described on the company’s website, “is designed to treat all types of childhood SMA by altering the splicing of a closely related gene (SMN2) that leads to the increased production of fully functional SMN protein.” The drug has received Orphan Drug Designation and Fast Track status from the U.S. Food and Drug Administration, and is currently in a Phase I study of children ages 2-14 with SMA to determine its safety, tolerability and pharmacokinetic profile.

 

“SMA is a heartbreaking disease – it can kill children before their second birthday, and there are currently no therapies to treat the disease,” George A. Scangos, Ph.D., CEO of Biogen Idec, said in a press release. “It is exactly the kind of disease and program that we are focused on at Biogen Idec. The unmet need could not be any greater, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases and Isis’ antisense compound has the potential to be a highly effective, first-to-market therapy for this deadly disease. We have the utmost respect 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.”