Ionis Phase 3 trial for eplontersen in ATTR-CM fails primary endpoint

Filed July 9, 2026 · Period ending July 9, 2026 · ~1 min read

Key Changes

• high

  CARDIO-TTRansform Phase 3 trial (1,432 patients, largest ATTR-CM study to date) failed to show statistically significant benefit on cardiovascular mortality and recurrent CV events versus placebo at Week 140.

  Item 8.01 — Other Events verify on EDGAR →
• medium

  Prespecified subgroup analysis showed nominally significant benefit (hazard ratio 0.71) for eplontersen monotherapy, but no treatment effect in patients already on stabilizer therapy (57% at baseline, 81% total).

  Item 8.01 — Other Events verify on EDGAR →
• medium

  Company reaffirmed cash flow breakeven target by 2028 and highlighted ongoing launches including TRYNGOLZA, its first drug in a prevalent population.

  Exhibit 99.1 verify on EDGAR →