The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies together.
A trend in gene therapy setbacks continues as Vertex Pharmaceuticals gave Verve Therapeutics back the rights to a liver disease gene editing program.
The research and development deal dates back to 2022, when Vertex plunked down $60 million up front with up to $400 million in development and commercial milestones as well as tiered royalties on any potential sales. The plan was for a four-year partnership to study in vivo gene editing to treat a liver disease, though the specific disease and the gene to be targeted were never announced.
News of the separation came Thursday, buried at the bottom of an announcement discussing pipeline updates and the retirement of Verve’s chief medical officer.
“Vertex notified the company of its decision to terminate the research collaboration due to changing priorities within its development portfolio,” Verve’s statement read.
Verve’s lead candidate is, coincidentally, a gene editor aimed at the liver, called VERVE-102. The drug, taken in a single course, targets the PCSK9 gene in liver cells with the aim of reducing low-density lipoprotein cholesterol (LDL-C) to treat heterozygous familial hypercholesterolemia (HeFH), a condition excess cholesterol leads to strokes and heart attacks.
That drug is currently in the early Phase Ib Heart-2 trial, and the company has stated that VERVE-102 has so far been well-tolerated without otherwise releasing any data. That data are expected in the second quarter of 2025.
Losing the Vertex partnership doesn’t close off Verve’s relationships with bigger companies. In 2023 Eli Lilly paid $60 million for the rights to Verve’s PCSK9-targetting program. In the Vertex announcement, Verve’s CEO Sekar Kathiresan said that Verve is delivering an opt-in package to Lilly and expects a decision back in the second half of 2025.
VERVE-102 took on the role of the company’s lead molecule after testing of VERVE-101 in the Phase Ib Heart-1 study was paused in April 2024 when a patient developed signs of liver damage. VERVE-101 is also a gene editor for PCSK9 in HeFH patients; the company has not revealed what the difference is between VERVE-101 and VERVE-102.
The dissolved partnership comes on the heels of some recent good news, but mostly bad, for gene editing. Layoffs have hit companies in the space, including Editas, Intellia and Encoded, in the last few months, and Pfizer recently canned its hemophilia gene editing treatment Beqvez. However, Intellia’s NTLA-2002 treatment showed huge reductions in angioedema events one month before the company’s layoffs were announced, while Regeneron showed off data on its treatment for otoferlin-related deafness just this week.
As noted by Pfizer in its announcement of the shuttering of the Beqvez program, the issue seems to be disinterest from the market, especially given the eye-watering costs gene therapies can have. Beqvez was priced at $3.5 million per dose.
