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Running interference

Alnylam’s patisiran data settle the question of whether RNAi can be used safely

Editor’s Note: An earlier version of this story was published in BioCentury on Sept. 25. It has been updated to include reporting about safety and efficacy data that was not included in the original. The update also corrects information about Alnylam’s delivery technologies and the timeline for ESC+ GalNAc to enter the clinic.

Phase III data for Alnylam Pharmaceuticals Inc.’s patisiran resolve one big question: it is now clear that safety signals that have led the company to discontinue or halt previous programs are not intrinsic to its RNAi platform.

The company, which plans to proceed to registration based on the data, says patisiran demonstrates it is possible to build safer, more effective molecules on the back of its growing clinical trial experience.
On Sept. 20, Alnylam reported top-line data from the APOLLO study of patisiran in 225 patients with TTR familial amyloid polyneuropathy (TTR-FAP). Patisiran met the primary endpoint of change in mean modified Neuropathy Impairment Score +7 (mNIS+7) from baseline to 18 months (p<0.00001 vs. placebo). Patisiran also met all secondary endpoints.

Detailed results will come at the European ATTR Amyloidosis

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