U.S. officials halted Verve Therapeutics' plan to begin testing in people a gene-editing approach to high cholesterol on Monday, leading VERV stock to crash.
Verve had asked the Food and Drug Administration for permission to test its treatment in 40 patients with high cholesterol due to a genetic defect. The company hopes to use a next-generation form of CRISPR gene editing to permanently turn off a problematic gene in these patients.
Instead, the FDA placed Verve's application on hold. Verve, which has already begun human tests in New Zealand and the U.K., says it will likely receive the FDA's questions within a month.
On today's stock market, VERV stock collapsed 30.5% to 21.75. Shares of Beam Therapeutics stock also took a hit, plunging 11.4% to 37.91. Verve is licensing Beam's technology.
VERV Stock: A New Angle On CRISPR
Verve is using a next-generation form of CRISPR known as base editing.
Traditional CRISPR makes its genetic edits by cutting both strands of DNA's double helix. Then, the DNA sews itself back up. Critics say this is where unplanned problems could occur — though that hasn't happened so far. Base editing aims to avoid that debate by simply cutting one strand of the double helix.
In the case of Verve, the company hopes to permanently shut off a gene called PCSK9. This gene makes a protein that can lead to elevated levels of "bad" LDL cholesterol. Long-term high cholesterol can lead to a host of cardiovascular problems including heart attack, stroke or death.
Verve asked the FDA for permission to begin testing its technology in people back in October. It received notice the FDA is holding its application on Friday. The news Monday sent VERV stock into a tailspin. Shares hit their lowest point since August. Verve stock fell below the low end of a consolidation with a buy point at 43.10, according to MarketSmith.com.
Initial Data Expected In 2023
Though Verve has already tested its high cholesterol treatment in patients in New Zealand and the U.K., the earliest results were not included in the request to the FDA, Verve said in a news release. So far, there have been no serious side effects. A team of independent data monitors said Verve could go ahead and test a larger dose in patients. The company expects to have initial data in the second half of 2023.
"We anticipate receiving details from the FDA in the next month, and are committed to working closely with the agency to address their questions, so that we may open enrollment for patients with (genetically high cholesterol) in the U.S.," Verve Chief Executive Sekar Kathiresan said in a written statement.
VERV stock is highly rated, according to IBD Digital. Shares have a Relative Strength Rating of 96 out of a best-possible 99. This means Verve stock ranks in the top 4% of all stocks in terms of 12-month performance.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
