Verve Temporarily Halts Lead Base Editing Study Following Adverse Event, Prioritizes Alternative Therapy

- Verve Therapeutics has paused enrollment in their Phase Ib Heart-1 study evaluating VERVE-101, a lead gene editing program, due to a serious adverse event involving a patient receiving a 0.45-mg/kg dose.
- The patient experienced a grade 3 elevation in serum alanine aminotransferase (ALT) levels and grade 3 thrombocytopenia, both without clinical symptoms and fully resolving within a few days.
- Verve's stock price dropped 31% in premarket trading on April 2nd, 2024.
- The company remains focused on the development of VERVE-102, another investigational gene editor, which uses a different lipid nanoparticle (LNP) delivery system, with an ionizable lipid previously tested in external clinical trials.
- VERVE-102 targets the PCSK9 gene like VERVE-101, intending to lower low-density lipoprotein cholesterol (LDL-C).
- The Heart-2 clinical trial of VERVE-102 is scheduled to commence in Q2 2024, focusing on safety, tolerability, pharmacokinetics, PCSK9, and LDL-C concentrations.
- The VERVE-102 Investigational New Drug Application remains active with the FDA and other regulatory authorities worldwide.
- Clinical Trial Applications for Heart-2 have been cleared by the UK MHRA and Health Canada.