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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.
