Question · Q3 2025
Jason Gerbery asked how Ionis plans to maximize the benefit of CARDIO-TTRansform data for Eplontersen in combination with tafamidis, ensuring disproportionate benefit and avoiding a 'free-riding effect' for competitors. He also inquired about the contextual importance of NNT data for olzarsen in both all-comer and high-risk sHTG groups for payers.
Answer
Brett Monia, CEO, Ionis, highlighted CARDIO-TTRansform as the largest TTR cardiomyopathy study, with combination usage as a key secondary endpoint. He expressed confidence in the trial design and drug, focusing on Eplontersen's value. Kyle Jenne, Chief Global Product Strategy Officer, Ionis, emphasized that the totality of olzarsen data, including significant triglyceride reductions and 85% AP event reduction, along with hospitalization and NNT data, would drive payer engagement and reimbursement, aiming to prevent first AP attacks.