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FDA halts Verve plans to test gene editing therapy for heart disease in US.

Verve didn’t say what led to the FDA’s decision, but claimed it hasn’t observed any safety issues in an ongoing trial in New Zealand and the U.K, where enrollment will continue.

The Food and Drug Administration has halted plans by Verve Therapeutics to test a new gene editing treatment for heart disease in a Phase 1 trial in the U.S.


Alongside its quarterly earnings statement on Monday, Verve said the Food and Drug Administration placed a clinical hold on the treatment, VERVE-101, disrupting its plans to begin the trial in the U.S. Verve didn’t say what triggered the FDA’s decision. It submitted data to regulators in October and expects to receive an official letter with the FDA’s questions within a month. The company said it will work with the agency to resolve the hold “as promptly as possible.”


The clinical hold is the first notable setback for Verve since it was co-founded by cardiologist and geneticist Sek Kathiresan in 2018. Kathiresan formed the company to develop a treatment that’s meant to prevent heart attacks for life with a single infusion. Since then, it raised $267 million in one of 2021’s larger biotech initial public offerings and became the first company to test “base” editing — a more precise form of CRISPR gene editing — in a human clinical trial.


That trial, known as heart-1, is underway in New Zealand and the U.K. and is testing VERVE-101 in patients with a rare and inherited form of high cholesterol. Three patients have been treated to date, and Verve hasn’t observed any treatment-related adverse events. All side effects have been mild, and a trial monitoring board has cleared the company to begin evaluating a higher dose, Verve said Monday. Initial results will be disclosed at a medical meeting next year.


“The safety profile observed in the first dose cohort with VERVE-101 is encouraging, and in-line with safety data generated with VERVE-101 in our preclinical studies,” said Andrew Bellinger, Verve’s chief medical and scientific officer, in a statement.


Enrollment will continue in New Zealand and the U.K. But Verve’s plans have now been slowed in the U.S., where regulators are closely scrutinizing gene editing medicines.


Regulators previously paused applications of gene editing drugs from partners Vertex and CRISPR Therapeutics, as well as Editas Medicine, before allowing their initial trials to begin. Earlier this year, the FDA halted a request from Beam Therapeutics, Verve’s longtime partner, to start testing a gene editing cancer treatment and has since asked for more technical details.


Those events, combined with Verve’s setback, may lead investors to “see this as a broad read through” of the FDA’s feelings about gene editing, wrote Stifel analyst Dae Gon Ha in a research note. But while “it’s tough to know what exactly contributed and culminated in VERVE-101’s IND clinical hold … we suspect it is more case-by-case and look for additional clarity.”


“Overall, we are not jumping to conclusions given the profound clinical promise the technologies hold,” he added.


Verve’s treatment uses base editing to permanently inactivate a gene called PCSK9. Turning off PCSK9 is meant to help lower levels of LDL, or “bad,” cholesterol, and thereby reduce heart disease risk. Other drugs targeting PCSK9 are currently available, but must be taken chronically.


With $550 million in the bank, Verve has enough cash to fund operations through the second half of 2025.


Verve shares fell 25% in pre-market trading on Monday.


Published Nov. 7, 2022 Ben Fidler, Senior Editor

https://www.biopharmadive.com/news/verve-therapeutics-fda-clinical-hold-heart-disease/635888/?utm_source=Sailthru&utm_medium=email&utm_campaign=Newsletter%20Weekly%20Roundup:%20BioPharma%20Dive:%20Daily%20Dive%2011-12-2022&utm_term=BioPharma%20Dive%20Weekender

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