Ionis Touts Inotersen's Convenience As Phase III Safety Data Disappoints

Although inotersen met efficacy endpoints in Phase III familial amyloidosis study, adverse event rates raised a red flag and give Alnylam's competing patisiran an edge on safety, at least for now.

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As the market absorbs news of some serious safety issues in a Phase III study of Ionis Pharmaceuticals Inc.'s familial amyloidosis drug inotersen, the company is stressing the drug's convenience advantages, including subcutaneous delivery with fewer injection reactions than the competing infused patisiran from Alnylam Pharmaceuticals Inc. and no need for high-dose steroids.

Designed with Ionis's antisense platform, inotersen (IONIS-TTRRx) is a once-weekly subcutaneously-delivered therapy that inhibits the production of transthyretin (TTR). The...

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