Peptide News Digest

#Pelacarsen

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Clinical Trials · View digest

Novartis Pelacarsen Misses Primary Cardiovascular Endpoint in Phase 3 Lp(a)HORIZON Trial Despite Substantial Lp(a) Reduction; Reshapes Lp(a)-Targeting Landscape

Novartis (NYSE: NVS) announced Friday September 4, 2026 that the pelacarsen Phase 3 Lp(a)HORIZON trial did not meet its primary endpoint of reducing major adverse cardiovascular events (a composite of cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization) versus placebo in patients with elevated lipoprotein(a) plus established cardiovascular disease on guideline-directed background therapy including lipid-lowering and antihypertensive medications. Pelacarsen (an antisense oligonucleotide targeting hepatic APO(a) production, licensed from Ionis Pharmaceuticals in 2019) did achieve substantial lower Lp(a) levels versus placebo. The trial enrolled roughly 8,000 patients globally over 6 years and represents the largest and most consequential test to date of the Lp(a)-as-causal-cardiovascular-risk-factor hypothesis. The miss substantially raises the bar for Amgen's olpasiran (RNAi, Phase 3 OCEAN(a) ongoing), Silence Therapeutics's zerlasiran (RNAi), and Lilly's lepodisiran (RNAi) — all of which are testing similar Lp(a)-lowering-plus-cardiovascular-outcome frameworks. Ionis and Royalty Pharma both hold economic interests in pelacarsen.

Industry · View digest

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.