Study Phase 3 to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in patients with Polyneuropathy

Akcea and Ionis Announce Initiation of NEURO-TTRansform Phase 3 Clinical Trial for AKCEA-TTR-LRx in Patients with Polyneuropathy.

NEURO-TTRansform Phase 3 Study Design

NEURO-TTRansform is a global, open-label, randomized study evaluating the efficacy and safety of AKCEA-TTR-LRx in patients with polyneuropathy due to hATTR amyloidosis. The study will enroll 140 adult patients with stage 1 or stage 2 polyneuropathy and will be compared to the historical placebo arm from the TEGSEDI® (inotersen) NEURO-TTR Phase 3 study that was completed in 2017. Of the total 140 participants, 20 patients will begin on TEGSEDI and move to AKCEA-TTR-LRx after week 35. The primary endpoint analysis will be completed at week 66 and all patients will be followed until week 85 when they will have the option to transition into the open label extension study.

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