Positive results in Phase III study of ISIS TTRRx in FAP-Isis Pharma

Isis Pharmaceuticals has announced positive data from an ongoing open-label extension (OLE) study of ISIS TTRRx in patients with familial amyloid polyneuropathy (FAP). FAP patients completing the ongoing Phase III study are eligible to enroll in this OLE study in which all patients receive ISIS TTRRx.

In the first thirteen patients to enter the OLE study, reductions of up to 92 percent in TTR protein after thirteen weeks of treatment with ISIS TTRRx were observed. Across all patients enrolled in the study, 27 TTR mutations were represented to date, including the Val30Met mutation, which is the most common mutation found in patients with FAP. Patients continue to be enrolled as they complete dosing in the study. These data will be presented at the American Academy of Neurology meeting in Washington, DC.