Novartis Shares Fall 3.3% After Pelacarsen Trial Misses Primary Endpoint
Key Facts
Novartis shares fell 3.3% in Monday morning trading after the company said its late-stage Lp(a)HORIZON trial of pelacarsen missed its primary endpoint.
The primary endpoint compared pelacarsen with placebo using a composite of 4 outcomes: cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and urgent coronary revascularization requiring hospitalization. The announcement establishes that the composite endpoint was missed, not that the data proved failure on every component individually.
Pelacarsen lowered lipoprotein(a), or Lp(a), levels, but Novartis said that did not translate into reduced cardiovascular risk in the overall study population. Ionis said the drug demonstrated an acceptable safety profile.
The randomized, double-blind, placebo-controlled trial enrolled 8,323 patients with elevated Lp(a) and established cardiovascular disease. Participants were receiving guideline-directed treatments, including lipid-lowering and antihypertensive therapies.
Pelacarsen is an investigational antisense oligonucleotide designed to inhibit Lp(a) production. Ionis discovered the drug and conducted its early development, while Novartis holds development, manufacturing and commercialization rights under a license and collaboration agreement.
The companies said full Lp(a)HORIZON data will be presented at an upcoming medical congress. Until those data are released, effect-size estimates and subgroup results will remain central to assessing the program's future after the primary-endpoint miss.
Latest Updates · 2
- Notable·
Update: The company faced an additional setback after announcing that a late-stage study for del-desiran, a drug targeting myotonic dystrophy, failed to meet its primary metric. Despite this further pipeline disappointment, Novartis reaffirmed its sales guidance through 2030, seeking to reassure investors regarding its long-term growth stability.
- Notable·
Update: The fallout from the Novartis trial failure has extended to the broader sector, raising concerns about the efficacy of rival cholesterol drugs currently in development. According to reports, investors are now cautiously monitoring the prospects of similar treatments from Amgen and Eli Lilly, increasing uncertainty surrounding the success of the Lp(a) targeting drug class.