Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol

Verve Therapeutics Inc VERV announced an update from the Heart-1 Phase 1b trial of VERVE-101.

VERVE-101 is being evaluated in patients with a genetic condition that leads to high cholesterol levels, which raises the risk of heart disease. The treatment is supposed to permanently turn off a protein called PCSK9, which helps reduce blood cholesterol levels.

The company said 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 thrombocytopenia (low blood platelets). 

The participant did not experience any bleeding or other symptoms related to the laboratory abnormalities, which resolved fully within a few days.

Related: Verve Therapeutic Provides First Look At Gene-Editing Drug For Inherited High ‘Bad’ Cholesterol, Shares Fall.

In consultation with the study’s independent data and safety monitoring board (DSMB), Verve has decided to pause enrollment in the Heart-1 clinical trial. 

Verve is investigating the laboratory abnormalities and expects to work with regulatory authorities to define a path forward for VERVE-101 based on those results. 

Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial. 

VERVE-102 uses the same base editor and guide RNA for PCSK9 but a different lipid nanoparticle (LNP) delivery system than VERVE-101. 

Verve has received regulatory clearances for the Heart-2 clinical trial in the U.K. and Canada. It plans to initiate the Heart-2 clinical trial in patients with HeFH or premature coronary artery disease in the second quarter of 2024.

VERVE-101 is a novel, investigational gene editing medicine designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver to reduce disease-driving low-density lipoprotein cholesterol.

Verve and Beam Therapeutics Inc BEAM entered into a collaboration and license agreement in April 2019, under which Verve received exclusive access to Beam’s base editing, gene editing, and delivery technologies for human therapeutic applications against certain cardiovascular targets.

In October 2023, Verve announced the expansion of its relationship with Eli Lilly And Co LLY, which has acquired from Beam Therapeutics certain product rights to Verve’s cardiovascular in vivo gene editing programs targeting PCSK9 and ANGPTL3, as well as a third undisclosed cardiovascular disease target.

Price Action: VERV stock is down 36.0% at $8.18, and BEAM shares are down 7.37% at $30.29 on the last check Tuesday.

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