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Ionis announces eplontersen met co-primary and secondary endpoints in interim analysis of the Phase 3 NEURO-TTRansform study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
- Eplontersen showed statistically significant and clinically meaningful improvements in mNIS+7 and Norfolk QoL-DN - Eplontersen demonstrated a favorable
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"- Eplontersen showed statistically significant and clinically meaningful improvements in mNIS+7 and Norfolk QoL-DN \n- Eplontersen demonstrated a favorable safety profile\n- Ionis and AstraZeneca expect to file a New Drug Application this year\nCARLSBAD, Calif., June 21, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from a 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for the co-primary endpoints of percent change in serum transthyretin (TTR) concentration and the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, versus the historical placebo group. Eplontersen also met its key secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), showing treatment with eplontersen significantly improved patient-reported quality of life versus the historical placebo group.\n\n \n \n \n \n \n \n\n \nIn the study, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns. The study data are consistent with the clinical profile seen across Ionis' other LICA programs, further validating how advancements in the company's LIgand-Conjugated Antisense technology position Ionis to deliver potentially transformative treatments for a range of unmet medical needs. \nBased on these study results, the companies will seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.\n\"These encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide much needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy,\" said Teresa Coelho, M.D., a neurologist and neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the NEURO-TTRansform study.\n\"We are pleased that the data in the NEURO-TTRansform study demonstrate eplontersen had a positive impact on disease progr...