The company’s treatment appeared to safely lower cholesterol levels in a small trial, giving Verve a chance to rebound from an earlier setback.

An experimental gene editing treatment from Verve Therapeutics appeared to safely lower cholesterol levels in a small clinical trial, giving the biotechnology company a chance to rebound from an earlier setback.

Verve has been working on a one-time treatment that can prevent heart attacks for life by making a precise edit to a liver gene called PCSK9. However, the company scrapped its first attempt after laboratory test results showed signs of potential liver damage and low platelet counts in one study participant.

Verve claimed the side effects may have been related to the lipid nanoparticle “shell” used to deliver the treatment. In response, it paused testing of that first therapy and pivoted to a successor prospect, Verve-102, that uses a different lipid nanoparticle the company believes to be safer.

The results disclosed Monday are the first since making that switch. According to the company, no serious side effects related to treatment were observed among the 14 study participants with at least 28 days of follow-up in an early-stage clinical trial. No “clinically significant” changes in liver enzyme counts, bilirubin or platelets were seen either. One infusion-related reaction occurred, but involved fleeting symptoms, the company said.

The therapy also showed early signs of effectiveness in study participants, all of whom have either a hereditary form of high cholesterol called HeFH or premature coronary artery disease. After a minimum of 28 days of follow-up, patients who received the highest of three doses tested saw their levels of LDL, or “bad,” cholesterol lowered by an average of 53%. Levels of the PCSK9 protein Verve-102 is designed to knock out fell by an average of 60% among those patients, the company said.

While early and from a small number of people, the results suggest Verve has “resolved [the] safety concerns” associated with its original program, wrote William Blair analyst Myles Minter, in a Monday note to clients. The data “reads as a clear win,” Minter added.

Verve aims to join a market with several effective options. Two PCSK9-blocking antibody drugs from Amgen and Regeneron are available for HeFH and other forms of high cholesterol, as is a longer-lasting RNA-based therapy sold by Novartis. Multiple pharmaceutical companies, including Merck & Co. and AstraZeneca, are advancing PCSK9-blocking pills.

Genetic medicine developers in other disease areas, meanwhile, have largely struggled to sell their products.

Verve claims its case is different, though. On a conference call with analysts, the company noted that an estimated 20% of people taking Novartis’ drug, Leqvio, and about half of those on Amgen’s or Regeneron’s medicines stop treatment after one year. CEO Sek Kathiresan said these discontinuation rates lower the “real-world” effectiveness of PCSK9 therapies and create a need for a durable, one-time treatment for many people.

“This is a new story,” he said. “The old commercial analogs for gene therapy and gene editing just simply don’t apply to this kind of situation.”

Jefferies analyst Roger Song added, in a separate research note, that the LDL-C reductions Verve reported were “comparable” to Amgen and Regeneron’s drugs in HeFH patients and exceeded what’s been seen in testing with Leqvio and other therapies. Verve-102 is showing “best-in-class potential,” Song wrote.

Verve is enrolling people in a fourth study cohort testing an even higher dose and plans to report data in the second half of the year. It also intends to start later this year a Phase 2 trial that will include study sites in the U.S. following a recent clearance by the Food and Drug Administration. The company has enough cash to operate through the middle of 2027, it said Monday.

Eli Lilly is collaborating with Verve on Verve-102 and will decide by the end of 2025 whether to opt in to share development costs and profits. Lilly found the data “very encouraging” and is “eager to receive the opt-in package from us” later this year, Kathiresan told analysts.

Verve shares climbed by more than 30% in early trading Monday, but are still down substantially from the company’s public debut in 2021.