Akcea Therapeutics (NASDAQ:AKCA) and Ionis Pharmaceuticals (NASDAQ:IONS) have initiated the NEURO-TTRansform Phase 3 clinical trial for AKCEA-TTR-LRx in patients with polyneuropathy caused by h856ereditary TTR amyloidosis, or 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.
The co-primary efficacy endpoints at week 66 are:
Percent change from baseline in serum TTR concentration;
Change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7); and
Change from baseline in Norfolk Quality of Life Questionnaire-Diabetic Neuropathy.
Ionis shares are up 2% premarket.
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