ALS TDI was founded in 1999 as a nonprofit biotech by theJohn Haywood family, whose son Stephen had been diagnosed with ALS (he lost hisbattle with the disease in 2006). Over time, the research group moved from theHaywood's basement to the garage, then occupied a couple of small labs, Perrinrecounts. Today, ALS TDI occupies a 15,000 square-foot facility and has plans to move up to 26,000 square feet inthe near future. Focused on meeting this urgent unmet medical need, ALS TDIexecutes a robust target discovery program, while simultaneously operating theworld's largest efforts to preclinically validate potential therapeutics,including a pipeline of dozens of small molecules, protein biologics, genetherapies and cell-based constructs. Believed to be the world's first nonprofitbiotech, ALS TDI has developed an industrial-scale platform that allows for thedevelopment and testing of dozens of potential therapeutics each year. Withmore than 30 professional scientists, the research Institute collaborates withleaders in both academia and industry to accelerate ALS therapeutic development.
Biogen Idec and IsisPharmaceuticals partner on antisense program targeting SMA
WESTON, Mass.—Biogen Idec also announced in early Januarythat it has entered into an exclusive, worldwide option and collaborationagreement with
Isis Pharmaceuticals Inc. under which the companies will developand commercialize Isis' antisense investigational drug, ISIS-SMNRx, for thetreatment of spinal muscular atrophy (SMA).
Under the terms of the agreement, Isis will receive anupfront payment of $29 million and is eligible to receive up to $45 million inmilestone payments associated with the clinical development of ISIS-SMNRx priorto licensing. Biogen Idec has the option to license ISIS-SMNRx until completionof the first successful Phase II/III trial. Isis could receive up to another$225 million in a license fee and regulatory milestone payments. In addition,Isis will receive double-digit royalties on sales of ISIS-SMNRx.
Isis will be responsible for global development ofISIS-SMNRx through the completion of Phase II/III clinical trials, with BiogenIdec providing advice on the clinical trial design and regulatory strategy. IfBiogen Idec exercises its option, it will assume global development, regulatoryand commercialization responsibilities.