Isis Pharmaceuticals just earned a $3.5 million milestone payment from Biogen Idec associated with the dosing of the first patient in a Phase II study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA) as part of a deal inked in January of 2012. ISIS-SMNRx is an antisense drug designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein.
Isis received an up-front payment of $29 million under terms of the January 2012 deal and could receive up to $45 million in milestone payments associated with clinical development of ISIS-SMNRx prior to licensing. If Biogen Idec takes up its option to license ISIS-SMNRx (which it can until completion of the first successful Phase II/III study), Isis could earn another $255 million in license fees and regulatory milestone payments plus double-digit sales royalties. The $3.5 million is just the beginning.
The Phase II study of ISIS-SMNRx is an open-label, multiple-dose, dose-escalation pilot study conducted at centers in the United States and Canada, which will include eight infants who have been diagnosed with SMA. In this study, children will either receive two or three doses of ISIS-SMNRx over the course of the study. To meet enrollment criteria, the firm says, infants must be between the ages of three weeks and seven months, live in close proximity to a study site, and pass screening evaluations conducted at study sites.