Ionis announced positive results from the Phase 3 study of inotersen (IONIS-TTRRx) for the treatment of familial amyloid polyneuropathy (FAP), now referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

The NEURO-TTR study is a 15-month, randomized, double-blind, placebo-controlled, international Phase 3 trial evaluating the effects of inotersen on neurological dysfunction and quality-of-life in 172 patients with hATTR-PN. The co-primary endpoints were change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score.

The study met both primary endpoints, showing that patients treated with inotersen achieved statistically significant benefit compared to placebo in the mNIS+7 and the Norfolk QoL-DN scores (P<0.0001 and P=0.0006, respectively). Statistically significant differences were also observed for both endpoints at eight months. Treatment-related adverse events were more commonly seen in the inotersen group, including cases of thrombocytopenia and renal dysfunction, which led to the implementation of enhanced monitoring to support early detection and management. 

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Long-term safety and efficacy data for inotersen are ongoing. Detailed results of the NEURO-TTR study will be presented at an upcoming medical meeting and will be submitted for publication in a peer-reviewed medical journal. The company is currently preparing for the regulatory marketing applications of inotersen in patients with hATTR-PN. 

Inotersen (IONIS-TTRRx) is a generation 2.0+ antisense drug administered once weekly as a single 300mg subcutaneous injection.

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