IISIS-SMNRxis designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein.
As per the terms of January 2012 agreement, Isis is responsible for the global development and Biogen Idec has the option to license the compound ISIS-SMNRx until completion of the first successful Phase 2/3 study.
The US Food and Drug Administration (FDA) has granted orphan drug status and fast track designation to ISIS-SMN for the treatment of patients with SMA.
The second phase study of ISIS-SMNRx is an open-label, multiple-dose, dose-escalation pilot study, which will include eight infants who are between the ages of three weeks and seven months and diagnosed with SMA.
The study will be conducted at centers in the US and Canada.