2022-05-12| Trials & Approvals

Verve Cleared to Test Gene Editing Drug for Heart Disease in Humans

by Joy Lin
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Verve Therapeutics, a gene editing company is announcing its transition to a clinical-stage biotech after the clearance of its candidate, VERVE-101, for Phase 1 testing in patients with heterozygous familial hypercholesterolemia (HeFH) in New Zealand. The company also provided an update on its ANGPTL3 program and reported on financial results for Q1 2022. 


UK, US Trials Expected for VERVE-101


A common and potentially life-threatening subtype of atherosclerotic cardiovascular disease (ASCVD), HeFH is characterized by mutations in the PCSK9 gene in the liver that leads to the production of disease-driving low-density lipoprotein cholesterol (LDL-C). 

VERVE-101 is designed to permanently turn off the PCSK9 gene. According to Verve, the drug has proven effective in editing PCSK9 in non-human primates, leading to reduced LDL-C levels sustained out to one year.

Having cleared its first clinical trial application (CTA) for VERVE-101 in New Zealand, the company aims to begin Phase 1 trials in HeFH patients in mid-2022. A CTA in the United Kingdom, as well as an IND application in the US is expected to be filed in the second half of 2022. Verve expects to report initial data from the Phase 1 of VERVE-101 in 2023. 

Related article: New Approaches to Effectively Manage Hemophilia in Pregnant Women


Durable Effects and No Long Term Toxicity for ANGPTL3 Base Editor 


Verve’s treatment for the homozygous counterpart of HeFH (HoFH), like VERVE-101, involves turning off a gene that drives high LDL-C blood levels. The gene in question is ANGPTL3. In a recent presentation, the Verve ANGPTL3 base editor led to a 96% reduction in blood ANGPTL3 protein from baseline in non-human primates. The animals were followed up to 616 days, with no long-term impacts observed on liver toxicity markers. 

“Recent data from our ANGPTL3 program support its continued development toward IND-enabling studies,” said Andrew Berlinger, MD, PhD, chief scientific and medical officer of Verve. These studies are expected to begin in the second half of 2022.  


Expenses Up, but Cash Runway Secure 


Verve looks to be in a good position for advancing clinical development of its drug candidates, with cash, cash equivalents and marketable securities sitting at $323.3 million as of March 31, 2022, compared to $360.4 million as of December 31, 2021. The company expects its war chest to fund operations into 2024. 

R&D expenses were $24.5 million in Q1 2022, up from $11.3 million in the same quarter the previous year. The company’s general and administrative expenses were $7.4 million in Q1 2022, up from $2.7 million in the same quarter last year.

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