By Bill Alpert

A single infusion of a Verve Therapeutics gene-editing treatment cut dangerously high levels of cholesterol in a group of patients by more than half. The company reported Monday that cholesterol levels fell by 59% in the first month, with no significant side effects, for patients getting the high dose.

There were 14 patients in Verve's Phase 1 trail, which it called Heart-2. Higher doses of its genetic medicine led to deeper cuts in the harmful form of cholesterol known as LDL-C.

"These initial Heart-2 data are promising with respect to both safety and efficacy and suggest the potential for a new era of cardiovascular disease treatment where a single dose might lead to lifelong control of LDL-C," said Harvard Medical School professor Eugene Braunwald, in Verve's press release.

Verve stock is up 11% from Monday's open, to $3.63, on 14 times its recent average volume. The shares topped $70 in the biotech heyday of 2021. Then they faded as investors lost patience with very early-stage drug developers. Verve took another drop in 2023, when two of its first 10 patients suffered heart attacks; the group was chosen from those already sick from heart disease and the outcomes were deemed unrelated to Verve's treatment.

Verve has shrunk to a microcap valuation of $330 million, as have many genetic medicine companies that came public in the market's Covid-era enthusiasm for biotech. Backing out Verve's roughly $500 million in cash, its modest debt leaves it with a negative enterprise value. But that cash should tide it over through 2027.

Moreover, drug giant Eli Lilly might chip in before then. Lilly could sign on when the final data from Verve's Phase 1 study come out in this year's second half. If Lilly opts in, it will contribute to the drug's testing and could be a 50/50 partner in U.S. commercialization.

So Verve's tiny capitalization makes it a small wager on a treatment that could find wide use as a lifetime fix for cardiovascular disease -- the world's number one killer.

"If LDL levels are maintained really low, lifelong, it's very hard to suffer a heart attack," said CEO Sekar Kathiresan on a Monday morning conference call.

Verve is one of the half dozen companies applying the Nobel Prize-winning technique for gene editing, called Crispr. The technology can zero in on a disease-causing stretch of DNA in our cells and then snip DNA's double strands to inactivate the troublesome gene.

While other companies target relatively uncommon genetic disorders, such as sickle cell disease, Verve is targeting a common killer: high levels of LDL cholesterol. Patients in its Phase 1 study come from families with genetically high levels of LDL that are caused by an overactive gene called PCSK9. This condition can cause heart attacks and strokes before the age of 30.

As with many firms developing therapies, Verve is testing its PCSK9 editor on the sickest patients first. But in theory, a lifetime reduction in PCSK9 activity -- and therefore LDL-C levels -- would benefit just about everyone alive. One shot and your lifetime risk of heart attack and stroke drops.

There are already PCSK9 drugs on the market that don't effectuate a permanent change, including the fast-selling twice-yearly injection Leqvio, from Novartis, and the twice-monthly shots Repatha, from Amgen, and Praluent, from Regeneron Pharmaceuticals. Merck and AstraZeneca are testing PCSK9-regulating pills.

So far, Verve's LDL reduction is on a par with those drugs.

The main debate on Wall Street is how much demand there will be for Verve's permanent fix in a market with daily pills or twice-yearly injections. Patients don't consistently stay on their PCSK9 doses. So a one-time treatment for life, such as Verve's, is intriguing for fighting an illness such as cardiovascular disease, whose risk rises with cumulative exposure to high levels of LDL cholesterol.

There are hints that Verve's treatment will fulfill its lifetime promise. On Monday's call, Verve said that one patient had maintained a 58% average reduction in LDL-C for the two years since his first infusion.

Verve's infusion now uses specially-crafted nanoparticles that contain the gene-editor. The nanoparticle is less irritating than the formulation in Verve's first Phase 1 study. And the gene editor is a second-generation form of Crispr, called base-editing, that rewrites a faulty gene more gently than first-generation Crispr approaches do.

In the second Phase 1 study discussed in Monday's call, LDL-C reduction increased with higher doses. So the company is currently trying an even higher dose in a few more patients.

The FDA has also authorized Verve to start recruiting patients for the treatment's Phase 2 study, which will examine whether the monthlong reductions seen so far are as long-lasting as expected. It should report the results of that phase by 2027.

Lilly will make its mind up about Verve's treatment this year. Maybe investors will re-evaluate this forgotten stock, too.

Write to Bill Alpert at william.alpert@barrons.com

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April 14, 2025 13:27 ET (17:27 GMT)

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