Phase III study of IONIS-TTRx shows QoL benefits in hereditary ATTR amyloidosis.- Ionis Pharma.

Positive data from a Phase III study of IONIS-TTRx (inotersen), from Ionis Pharmaceuticals, in patients with hereditary ATTR amyloidosis (hATTR) was presented at the 2018 American College of Cardiology (ACC) Annual Meeting. Key quality of life data in the Phase III study was shown in the poster "Inotersen Improves Quality of Life in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy and Cardiomyopathy: Results of the Phase III Study Neuro-TTR" by Matthew S. Maurer, M.D., Medical Director, HCM Center at New York-Presbyterian Hospital/Columbia University Medical Center. The data highlighted the improvement in quality of life (QOL) and functional capacity experienced by 50% of treated with the investigational antisense drug.

Neuro-TTR demonstrated significant benefit compared to placebo in patients with cardiac disease at baseline in both primary endpoints (Norfolk QOL-DN, p=0.036 and mNIS+7, p<0.001) and in the sf-36 health survey endpoint p="0.025)" at 15 months. it was shown that half of patients receiving ionis-ttrx were more capable of completing daily activities than they were at the start of the study and these benefits were significant early and sustained across patient types. during the study 2 safety issues arose: thrombocytopenia and serious renal adverse events aes. these complications aside the most common aes included nausea chills vomiting and anemia.>

Comment: The FDA has set a Prescription Drug User Fee Act (PDUFA) date of July 6, 2018. If inotersen is approved, the company has stated that it is prepared to promptly launch the drug in the U.S. and EU.