Monday, July 31, 2023
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has successfully completed the enrollment phase of the Phase 3 CARDIO-TTRansform cardiovascular outcomes study for eplontersen, an investigational drug targeting transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a life-threatening condition associated with a significant risk of cardiovascular events and affects a growing number of patients.
Eplontersen, an antisense medicine, aims to slow the progression of cardiomyopathy in individuals with hereditary or wild-type ATTR-CM by inhibiting transthyretin production.
The CARDIO-TTRansform study, the largest and most comprehensive study of its kind, enrolled over 1,400 patients. It will provide robust analysis of the composite endpoint, including cardiovascular mortality and recurrent cardiovascular events. The study also seeks to gather data from diverse patient subsets, including those with or without stabilizer therapy and those with or without hereditary disease within the evolving ATTR-CM landscape. Results are expected to be reported by the first half of 2025.
Ionis and AstraZeneca are jointly developing and commercializing eplontersen globally, not only for ATTR-CM but also for the treatment of transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The companies have submitted a New Drug Application to the U.S. Food and Drug Administration for eplontersen, with a PDUFA action date set for December 22, 2023. The drug has also received Orphan Drug Designation in the U.S.
Eplontersen is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of TTR protein. Administered as a monthly self-injection, it is intended to treat all types of ATTR. ATTR amyloidosis is a systemic, progressive, and fatal disease caused by the accumulation of TTR amyloid deposits in various tissues and organs, leading to organ failure and ultimately death.
Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is a severe condition caused by misfolded TTR protein accumulating in the heart muscle. It results in ongoing, debilitating heart damage, progressive heart failure, and typically leads to death within three to five years from the onset of the disease. ATTR-CM encompasses both the genetic and wild-type forms, affecting an estimated 300,000 – 500,000 patients worldwide.
