Verve stock collapsed to a record low Tuesday after a patient who received its cholesterol-lowering gene-editing treatment experienced side effects.
The company says it's pausing enrollment in the study of its treatment, VERVE-101. Patients enrolled in the study experienced up to a 73% reduction in "bad" LDL cholesterol. But the sixth patient who received a specific dose had elevated liver enzymes and low blood platelets. Both effects were temporary.
Now, Verve Therapeutics says it will focus on VERVE-102, which uses a different delivery mechanism and also aims to cut LDL cholesterol. Regulators in the U.K. and Canada have already given Verve the go-ahead to start testing the new treatment in the second quarter.
Verve stock crashed 35% to 8.32. That put Verve stock at its lowest-ever point after going public in mid-2021. Shares of Eli Lilly, who is partnered on the drug, dipped a fraction to 763.96.
Verve Stock: Changing Genes Inside The Body
Verve is testing its gene-editing treatments in patients with heterozygous familial hypercholesterolemia, a genetic disease that causes uncontrollably high LDL cholesterol. These patients also have atherosclerotic cardiovascular disease.
A total of 13 patients have received VERVE-101. But the sixth patient to receive a specific dose had severely elevated liver enzymes and low blood platelets within four days of receiving the drug. The patient didn't experience any bleeding or other symptoms related to the lab abnormalities. The events resolved within a few days.
But Verve and its independent data and safety monitoring board decided to pause enrollment in the study to investigate the side effects. Verve stock, which was trading at a 50-day line ceiling, dropped far below that mark on Tuesday.
Chief Executive Sekar Kathiresan says the study supports the proof-of-concept for the company's base-editing efforts. Base editing is a form of gene editing. In this case, Verve hopes to turn off the PCSK9 gene, which provides the instructions for making a protein tied to cholesterol in the blood. Notably, this is an in vivo gene-editing drug, meaning the genetic edit takes place inside the body.
"However, at potentially therapeutic dose levels of VERV-101, we have observed certain asymptomatic laboratory abnormalities, which we believe are attributable to the (lipid nanoparticle) delivery system," Kathiresan said in a statement.
86 Million People In The U.S. With High Cholesterol
Eventually, Verve could expand its cholesterol-lowering efforts to people with less severe forms of high cholesterol — those whose LDL could be controlled by statins or other injectable drugs that block the PCSK9 protein.
The news Tuesday is a blow to Verve's argument for eventual widespread use of gene editing to lower LDL cholesterol.
Critics say it makes little sense to use a permanent technology to lower cholesterol in a broad group of people. The Centers for Disease Control and Prevention estimates about 86 million people age 20 and older in the U.S. have high cholesterol.
Supporters note compliance is generally poor for cholesterol drugs. People who can benefit from statins don't always take them, or even know they have high cholesterol. High cholesterol is asymptomatic, but carries the risk of heart attack, strokes and other cardiovascular problems.
Verve stock has been under pressure over the last month. After running up to a recent high at 19.34 in late February, Verve shares have tumbled almost 57%, as of Tuesday's close.
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