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Ionis reports positive topline 66-week results of eplontersen Phase 3 study for patients with ATTRv-PN
Eplontersen met co-primary endpoints demonstrating sustained reduction in TTR and benefits in neuropathy and quality of life through 66 weeks35 and 66-week
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"Eplontersen met co-primary endpoints demonstrating sustained reduction in TTR and benefits in neuropathy and quality of life through 66 weeks35 and 66-week data to be presented at the American Academy of Neurology (AAN) Annual Meeting in AprilCARLSBAD, Calif., March 27, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a debilitating and potentially fatal disease that leads to peripheral nerve damage and motor disability.\n\n \n \n \n \n \n \n\n \nAt 66 weeks, patients treated with eplontersen continued to demonstrate a statistically significant and clinically meaningful change from baseline versus an external placebo group on the co-primary endpoints of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). The study also met its third co-primary endpoint demonstrating a statistically significant reduction in serum TTR concentration versus an external placebo group. TTR reductions were consistent with those reported at week 35. Eplontersen continued to demonstrate a safety and tolerability profile consistent with that observed at 35 weeks.\n\"The positive results from the 66-week analysis of the Phase 3 NEURO-TTRansform trial show that eplontersen provided consistent and sustained transthyretin protein reduction and that a substantial number of patients improved in measures of both neuropathy progression and quality of life,\" said Sami Khella, M.D., chief, department of neurology, Penn Presbyterian Medical Center and professor of clinical neurology, University of Pennsylvania School of Medicine. \"This builds on the favorable 35-week results, which first demonstrated eplontersen's potential to significantly improve outcomes in this underserved population.\"\n\"These latest results from our NEURO-TTRansform study represent an important step towards delivering a potential new therapy for ATTRv-PN patients living with this debilitating and fatal disease. We are encouraged by the sustained benefit demonstrated by eplontersen and what a self-administered treatment could mea...