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Verve halts enrollment of lead trial after grade 3 side effects, prioritizing next-up PCSK9 editor
2024-04-02
临床结果临床3期临床1期
Verve Therapeutics CEO Sekar Kathiresan, M.D., does not expect the deprioritization of VERVE-101 to affect cash runway or the size of the workforce.
Verve Therapeutics is halting enrollment in a phase 1 study testing its lead gene editiVERVE-101ne as a treatment for serious high cholesterol, pivoting to a follow-up asset that will soon launch into the clinic.
Verve Therapeuticsotech said Tuesday that one of the six patients treated with a 0.45-mg/kg dose of VERVE-101 experienced grade 3 increases in a particular liver enzyme and a grade 3 case of low blood platelets. The patient was hospitalized and observed for two days despite being asymptomatic.
The individual experienced no bleeding, and the lab abnormalities resolved independently within a feVERVE-101he company added.
In consultation with the trialbleedingmonitoring board, Verve has halted enrollment in the trial and notified regulators. The biotech is investigating the lab data and said it will work with health officials to chart a path forward for VERVE-101. In the meantime, the company is turning its attention to VERVE-102, which uses different lipid nanoparticle technology to deliver the same PCSK9 base editor as VERVE-101.
Vervepdated lipid nanoparticle for VERVE-102 uses an ionizable lipid and a GalNAc liver-targeting ligand, which diversifies the range of pathways through which the treatment can enter the liver. Fortifying Verve’s confidence is the fact that Intellia Therapeutics licensed the same ionizable lipid from Novartis, which has yet to raise safety concerns with about 75 patients' worth of data.
Verve expects to launch a clinical VERVE-102 VERVE-102 in a matter of months, if not weeks, recruiting out of the U.K. and Canada. The first patient is expected to be dosed in the second quarter. It’s a similar strategy as VERVE-101, where thIntellia Therapeutics while the company worked through a preeNovartisDA clinical hold.
Ververesan underscored that despite the safetVERVE-102s among the higher dose group, VERVE-101 seemed to validate the editing technology, exemplified by cholesterol lowering. The first five patients treated with the 0.45-mgVERVE-101averaged a 46% drop in low-density lipoprotein cholesterol levels, with the hiFDAst reduction reaching 73%.
“This, to us, suggests that the guide and the editor are working well,” the CEO said.VERVE-101
Kathiresan added that the near-term decision to deprioritize VERVE-101 has no impact on the company's cash runway and should not spur any layoffs.
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