The company claims Isis’ ISIS-SMNRx is developed to compensate for the underlying genetic defect that causes SMA.
As per the collaboration, Isis will receive an upfront payment of $29m and is eligible to receive up to $45m in milestone payments associated with the clinical development of ISIS-SMNRx prior to licensing.
Isis could also receive up to another $225 million in a license fee and regulatory milestone payments besides being entitled to double-digit royalties on sales of ISIS-SMNRx.
Biogen Idec on the other hand has the option to license ISIS-SMNRx until completion of the first successful Phase 2/3 trial.
Isis will take charge of the global development of ISIS-SMNRx through the completion of Phase 2/3 registrational clinical trials, with Biogen Idec providing advice on the clinical trial design and regulatory strategy.