Phase 3 failure of AstraZeneca/Ionis antisense drug Wainua shakes ATTR-CM market, opens space for other developers

AstraZeneca and Ionis Pharmaceuticals reported that their antisense therapy Wainua did not achieve a significant cardiovascular benefit in a Phase 3 trial for transthyretin amyloid cardiomyopathy (ATTR-CM). The late‑stage study result was announced last month and has sent ripples through the ATTR field.

Wainua is already approved for treating transthyretin amyloid polyneuropathy (ATTR-PN). ATTR affects roughly six in a million people in the United States, with the disease manifesting in two main clinical forms: ATTR‑CM and ATTR‑PN.

The unexpected failure has created an overhang for other developers of similar antisense approaches while boosting companies pursuing alternative therapies. BioSpace highlighted five firms that continue to advance ATTR assets as the market readjusts.