July 9, 2026
BIOINVEST NEWS: IONIS (IONS)
Ionis (IONS) – IONS’ Wainua Fails in ATTR, Focus on Tryngolza Launch in SHTG
IONS and their partner AZN are both down on negative trial results from CARDIO-TTRansform. The trial failed to meet its primary endpoint of the composite outcome of cardiovascular (CV) mortality and recurrent CV clinical events in ATTR-CM patients through Week-140. Although the drug was well tolerated in study participants, adding Wainua to the standard of care did not demonstrate a statistically significant benefit compared to placebo. The CARDIO-TTRansform negative results were a surprise, as we/most investors were expecting positive results. Wainua is already approved for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN), a condition in which misfolded transthyretin (TTR) protein deposits damage peripheral nerves. While this bad news was almost completely unexpected, it may serve as a clearing event for potential acquirers who have been kicking the tires. In our view, today’s selloff creates a buying opportunity considering the strength of IONS’s wholly-owned portfolio supporting our long-term bullish outlook. We continue to expect a strong launch for Tryngolza in SHTG as the ability to both lower severely high triglycerides and prevent severe pancreatitis makes the drug a game changer.
Although Wainua failed to meet the primary endpoint of cardiovascular mortality and recurrent cardiovascular events in ATTR-CM through Week-140, we note the study was conducted in a contemporary treatment landscape where most patients received stabilizer therapy. Importantly, a pre-specified monotherapy subgroup demonstrated a nominally significant hazard ratio of 0.71, while secondary endpoints, imaging, and biomarker measures generally favored Wainua.
We continue to expect a strong launch for Tryngolza in SHTG as the ability to both lower severely high triglycerides and prevent severe pancreatitis makes the drug a game changer. IONS has multiple late-stage drug development opportunities advancing forward, and management has also reiterating expectations for cash flow breakeven by 2028. While this bad news was almost completely unexpected, it may serve as a clearing event for potential acquirers who have been kicking the tires. In our view, today’s selloff creates a buying opportunity considering the strength of IONS‘s wholly-owned portfolio supporting our long-term bullish outlook.