The trial failed. Novartis's drug pelacarsen, studied in patients with high levels of a blood protein linked to heart disease, did not reduce cardiovascular events compared to placebo. The investor that had put $150 million on it says the deal will still make money.
That investor is Royalty Pharma, which in January 2023 put $500 million into a funding deal with Ionis Pharmaceuticals. The structure of that deal is why Royalty Pharma is not calling it a disaster. Only $150 million rode on pelacarsen. The remaining $350 million bought royalties on Spinraza, a treatment for spinal muscular atrophy — and the Spinraza money keeps flowing regardless of what happened in the HORIZON cardiovascular trial.
Under the terms of the deal, Royalty Pharma holds 25% of Ionis' Spinraza royalty stream through 2027, rising to 45% from 2028. The arrangement ends once royalty payments reach $550 million, which Royalty Pharma says represents a 1.1x return on the total amount funded. Because the trial failed, the company also confirmed it will make no further milestone payments to Ionis.
Novartis shares closed 1.90% lower at $159.99 on September 5, on dollar volume of about $203 million.
Novartis said it plans to present the full HORIZON data at an upcoming medical congress, according to its September 4 press release. That release noted pelacarsen did lower lipoprotein(a) — the blood protein the drug was designed to target — among participants already receiving standard heart disease treatments. The drug lowered the protein. It just could not turn that into fewer deaths or hospitalisations.