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Ionis completes enrollment in landmark Phase 3 CARDIO-TTRansform study in patients with TTR-mediated amyloid cardiomyopathy
CARDIO-TTRansform is the largest, most comprehensive ATTR-CM study with more than 1,400 patients enrolledEplontersen is currently under U.S. FDA review for
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"CARDIO-TTRansform is the largest, most comprehensive ATTR-CM study with more than 1,400 patients enrolledEplontersen is currently under U.S. FDA review for ATTRv-polyneuropathy, with ATTR-CM representing a second, larger potential patient populationCARLSBAD, Calif., July 31, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the completion of enrollment in the Phase 3 CARDIO-TTRansform cardiovascular outcomes study of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a large and growing patient population at risk of life-threatening cardiovascular (CV) events. The study enrolled more than 1,400 patients.\n\n \n \n \n \n \n \n\n \nEplontersen is an investigational antisense medicine designed to inhibit the production of transthyretin and slow the progression of cardiomyopathy for people living with hereditary or wild-type ATTR-CM.\n\"CARDIO-TTRansform is the largest and most comprehensive ATTR-CM study ever conducted. The evaluation of eplontersen in this broad, diverse patient population will enable robust and clinically meaningful analysis of the composite endpoint of CV mortality and recurrent CV events,\" said Eugene Schneider, M.D., executive vice president and chief clinical development officer for Ionis. \"It will also allow us to gather data from important patient subsets, including patients with or without stabilizer therapy and those with or without hereditary disease within the evolving ATTR-CM landscape. We look forward to reporting results as early as the first half of 2025.\"\nAs part of a global development and commercialization agreement, Ionis and AstraZeneca are seeking regulatory approval for eplontersen for the treatment of transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) in the U.S. and plan to seek regulatory approval in Europe and other parts of the world. The U.S. Food and Drug Administration accepted the New Drug Application for eplontersen for the treatment of ATTRv-PN with a PDUFA action date of Dec. 22, 2023. Eplontersen was granted Orphan Drug Designation in the U.S.\nFor more information on the CARDIO-TTRansform study, please visit www.clinicaltrials.gov (NCT04136171).\nAbout the CARDIO-TTRansform Study\nCARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase 3 cardiovascular outcome study in more than ...