Side effects from delivery system force Verve to scrap lead gene editing asset

02 Apr 2024
firstwordpharma.com
Clinical ResultPhase 1Gene TherapysiRNA
Verve Therapeutics is switching to a different lipid nanoparticle (LNP) delivery system to target PCSK9 after side effects scuppered development of its lead gene editing medicine. CEO Sekar Kathiresan noted that at therapeutic dose levels of VERVE-101, “we have observed certain asymptomatic laboratory abnormalities, which we believe are attributable to the LNP delivery system.”
VERVE-101 was designed to be a single course treatment that permanently turns off the PCSK9 gene in the liver to reduce low-density lipoprotein cholesterol (LDL-C). Verve noted that 13 patients have been dosed in the Heart-1 Phase Ib trial, including six at the 0.45mg/kg dose level.
The company indicated that in the first five participants at this dose level, VERVE-101 demonstrated time-averaged LDL-C reductions ranging from 21% to 73%. However, the sixth person treated in the 0.45mg/kg cohort experienced a Grade 3 drug-induced transient increase in serum alanine aminotransferase (ALT) as well as a serious adverse event of Grade 3 drug-induced thrombocytopenia within the first four days after dosing.
Verve said that it will now pivot to focus on development of VERVE-102, which uses the same base editor and guide RNA for PCSK9 but a different LNP delivery system than VERVE-101. The company has received clearance in the UK and Canada to start the Heart-2 study in the second quarter.
More to follow.
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