Roche and Ionis report Phase 3 success for RNA therapy sefaxersen in IgA nephropathy

A late‑stage Phase 3 trial of the Roche‑Ionis RNA‑targeted drug sefaxersen met its primary endpoint, showing a statistically significant reduction in proteinuria for patients with primary IgA nephropathy.

The reduction in 24‑hour urine protein‑to‑creatinine ratio suggests the therapy could preserve long‑term kidney function and potentially delay the need for dialysis or transplant.

Roche did not release the exact magnitude of the change but said the interim data support a path toward accelerated FDA approval. The result follows Ionis’s recent positive ALS trial, underscoring the strength of its antisense platform.