IONIS NEURO-TTR Phase 3 Clinical Trial Highlighted at American College of Cardiology (ACC) Annual Meeting

Each year, the American College of Cardiology (ACC) Annual Meeting brings together the world’s leaders in cardiology and cardiovascular science to discuss the latest developments in the field with the goal of transforming cardiovascular care and improving heart health. The ACC strives to achieve its purpose of improving cardiovascular health through education, research, quality care and health policy. The 2018 ACC annual meeting was held in Orlando, Florida on March 10-12, 2018.

At this year’s meeting, key quality of life data from the successful Phase 3 study of inotersen for the treatment of hereditary ATTR amyloidosis (hATTR), NEURO-TTR, was presented in the poster ‘Inotersen Improves Quality of Life in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy and Cardiomyopathy: Results of the Phase 3 Study Neuro-TTR’ by Matthew S. Maurer, M.D., Medical Director, The HCM Center at New York-Presbyterian Hospital/Columbia University Medical Center.

The presentation highlighted the improvement in quality of life and functional capacity experienced by 50 percent of patients treated with inotersen. Both quality of life instruments used in the study demonstrated that half of patients receiving inotersen were better able to participate in the activities of their daily life than they were at the start of the study. The observed benefits in the study with inotersen were significant, early and sustained across patient types, providing renewed hope for those suffering from this fatal disease. hATTR is a progressive, systemic, fatal rare disease which robs people of their quality of life and ability to function. Patients with hATTR experience multiple overlapping symptoms in different parts of the body, including peripheral nerves and the heart.

In addition, ACC highlighted two additional posters on inotersen and Ionis antisense programs, including:

  • ‘Potential Reversal of Transthyretin Amyloid Cardiomyopathy with TTR Specific Antisense Oligonucleotide Therapy, Inotersen’ poster presentation by Noel Rakhi Dasgupta, Indiana University
  • ‘Inhibition of Apolipoprotein C-III with GalNAc Conjugated Antisense Drug Potentially Lowers Fasting Serum Apolipoprotein C-III and Trigylceride Levels in Healthy Volunteers with Elevated Triglycerides’ poster presentation by Veronica J. Alexander, Ionis Pharmaceuticals

The complete abstract for Dr. Matthew Maurer’s poster presentation, as well as the other presentations, can be accessed in an upcoming online issue of the Journal of the American College of Cardiology.