Ionis and Royalty Pharma Both Absorb Financial Impact From Novartis Pelacarsen Phase 3 Miss; Program Future Under Review
Ionis Pharmaceuticals (NASDAQ: IONS) and Royalty Pharma (NASDAQ: RPRX) both issued statements Friday September 4, 2026 following the Novartis pelacarsen Lp(a)HORIZON Phase 3 primary endpoint miss. Ionis, the original developer that licensed pelacarsen to Novartis in 2019, is entitled to potential low-double-digit tiered royalties on future net sales that now appear unlikely absent a resubmission based on a different endpoint or population. Royalty Pharma acquired a portion of Ionis's royalty rights on pelacarsen in a 2019 transaction and had modeled multi-hundred-million-dollar revenue on approval. Both companies emphasized the substantial Lp(a) reduction achieved in the trial and the value of the dataset for the broader Lp(a) research field. The miss lands within days of Ionis's September 3 FDA approval of Zanvastro (zilganersen) for Alexander disease, which provided commercial and pipeline offset. Analyst commentary noted that the Ionis antisense franchise remains strong despite the pelacarsen setback, with Wainua (eplontersen), Zanvastro, and the deep pipeline anchoring the platform.