Ionis reports updated Phase III data for inotersen in FAP

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) reported updated data from the Phase III NEURO-TTR trial of inotersen (IONIS-TTRRx) in 172 patients with familial amyloidotic polyneuropathy (FAP), a form of transthyretin (TTR) amyloidosis.

On the first co-primary endpoint, 300 mg subcutaneous inotersen given 3 times every other day for the first week and then once-weekly thereafter led to a significant mean improvement in the modified Neuropathy Impairment Score +7 (mNIS+7) of 8.69 and 19.73 points from baseline to 8 and 15 months, respectively, compared to placebo (p=0.0005 and p=0.00000004, respectively). On the second co-primary endpoint, inotersen significantly improved mean Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (QOL-DN) score by 6.14 and 11.68 points from baseline to 8 and 15 months, respectively, compared to placebo (p=0.032 and p=0.0006, respectively)...