Verve Not Ready To Pull Plug On Anti-PCSK9 Gene Editing Candidate

But Firm Shifts Focus To Back-Up Candidate

The company told Scrip that the sixth patient with elevated liver enzymes and low platelets recovered after hospital observation and oral steroids, and it may still continue VERVE-101’s development.

• Source: Shutterstock

Verve Therapeutics, Inc. has paused enrollment in its Phase Ib Heart-1 trial of gene-edited cholesterol medicine VERVE-101 after a patient experienced liver enzyme elevations and low platelet counts. But while the company has decided to prioritize its development of a different asset with the same target, VERVE-102, it said VERVE-101 still might have a shot.

The biotech company said on 2 April that it had voluntarily paused enrollment in the study in heterozygous familial hypercholesterolemia (HeFH) after a sixth patient on the 0.45mg/kg dose experienced asymptomatic grade 3 ALT elevation and thrombocytopenia

Key Takeaways
  • Verve paused enrollment in its Phase Ib study of gene-editing candidate VERVE-101 after a sixth patient experienced grade 3 ALT elevations and thrombocytopenia.

  • The company plans to focus more on VERVE-102, which has the same target but uses a different lipid nanoparticle technology

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