Eli Lilly is joining Amgen and Novartis in the pack of drugmakers aiming to treat cardiovascular disease by targeting lipoprotein(a). The Big Pharma landed a spot at the back of the bunch by handing Verve Therapeutics $60 million for rights to its once-and-done gene editing approach to the problem protein.
High levels of Lp(a), a liver-derived protein that circulates in the blood, are associated with a higher risk of heart attack, stroke and other cardiovascular events. There is little overlap between the estimated 11 million patients in the U.S. and Europe who have high levels of Lp(a) and the people who have high levels of LDL cholesterol, another lipoprotein targeted by Verve. And there is a lack of ways to reduce Lp(a), with levels largely determined by genetics, not lifestyle, and statins having little effect.
Those factors, coupled with evidence that people with Lp(a) deficiency are healthy and at low risk of heart attack and stroke, led Verve to identify the lipoprotein as a good fit for its once-and-done gene editing technology. A gene editor tailored to target Lp(a) could safely and permanently reduce a person’s risk of heart attack and stroke, in theory.
Lilly sees promise in the theory. The Big Pharma is handing Verve $60 million, split between an upfront payment and equity investment, and committing up to $465 million in milestones, plus royalties, for the rights to the Lp(a) program. Lilly will fund work up to the end of phase 1 even though this stage of the work will be undertaken by Verve. After that point, the biotech will have the option to share in the costs and profits of the program rather than receive milestones and royalties.
The structure of the deal gives Verve an immediate cash boost—which extends its runway out to an envy-inducing 2026—frees it of the cost of taking the Lp(a) program to an early value inflection point and still leaves it with the chance to snag a share of the profits. Lilly will be responsible for the development of the program after phase 1 as well as for manufacturing and commercialization.
Inking the deal moves Lilly into a space targeted by some of its peers, albeit typically using different drug modalities than gene editing. Novartis paid $150 million to exercise its option on Ionis Pharmaceuticals’ pelacarsen in 2019. The antisense oligonucleotide, which is scheduled to deliver phase 3 data in 2025, is designed to reduce Lp(a) levels by inhibiting the production of apolipoprotein(a).
Amgen is following close behind, with a phase 3 trial of its Arrowhead-partnered siRNA candidate set to wrap up in 2026. Silence Therapeutics has a rival siRNA candidate, SLN360, in midphase development, and CRISPR Therapeutics could provide a more like-for-like threat to Lilly with its research-stage, in vivo gene editing program CTX320 (PDF).