Verve reveals letter from FDA that lays out conditions to lift base editing trial hold
We now know why Verve’s lead candidate was placed on hold last month by US regulators.
In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.
The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.
The goal of the therapy is to permanently turn off the PCSK9 gene in patients with an inherited disease that causes extremely high cholesterol levels.
Verve said in the filing that it plans to “submit a response as expeditiously as possible” the FDA. The biotech also said that it is continuing to enroll patients for the trial in the UK and in New Zealand — and plans to report initial data from the dose-escalation part of the trial later this year.
Verve declined to comment to Endpoints News outside of the filing.
The revelations of the FDA’s asks are the newest development that Verve announced since its base editing trial got put on hold just last month. A Phase Ib trial investigating VERVE-101 kicked off in July as the biotech started dosing patients in New Zealand and the UK.
The lead candidate, a single base editor, is being tested in patients with heterozygous familial hypercholesterolemia (HeFH). The trial is the first time that base editing has been done in humans. Base editing, a genome editing technology, works differently than traditional CRISPR, using a chemical process to change a specific letter in the target gene — rather than making a double-stranded cut in DNA.
Investors did not appear happy with the results, as $VERV took an 18% dive on the Nasdaq Monday morning.
Verve is the third biotech to have gotten regulatory permission to edit genes in humans directly, thanks to New Zealand regulatory authorities in May. Intellia and Editas Medicines were the first two companies to have gotten the go-ahead.