Vertex terminates liver disease gene editing collab with Verve

Vertex Pharmaceuticals has bowed out of a research collaboration with Verve Therapeutics focused on an in vivo gene editing program for liver disease.

Verve plans to independently progress the novel, preclinical gene editing program following Vertex’s decision to end the agreement due to what Verve described in its 2024 financial filing as “changing priorities" in Vertex's development portfolio.

The 2022 collaboration was intended to be an exclusive, four-year global research partnership focused on discovering and developing an in vivo gene editing program for a single undisclosed liver disease. Per the deal, Verve was to advance the discovery, research and certain preclinical development, with all program costs funded by Vertex. Vertex would have then been responsible for subsequent development, manufacturing and commercialization of any program stemming from Verve’s research efforts.

Vertex had handed Verve an upfront payment of $60 million, including a $35 million equity investment. Verve will now miss out on up to $66 million in success payments, as well as up to $340 million in development and commercial milestones payouts.

Fortunately, Verve’s tie-up with Eli Lilly is still intact. Through Lilly’s acquisition of Beam Therapeutics in October 2023, the drugmaker picked up 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. Verve’s PCSK9 product candidates, VERVE-101 and VERVE-102, and ANGPTL3 product candidate, VERVE-201, are designed to potently and durably lower low-density lipoprotein cholesterol throughout the lifetime of patients with or at risk for atherosclerotic cardiovascular disease, the most common form of CVD.

For the PCSK9 and ANGPTL3 product candidates, Lilly has the right to opt-in to share 33% of worldwide development expenses and to jointly commercialize and share profits and expenses related to commercialization in the U.S. on a 50/50 basis. In its financial report, Verve said it plans to deliver the opt-in data package for the PCSK9 program and receive a decision from Lilly in the second half of 2025, at which time Verve will initiate the phase 2 clinical trial for the PCSK9 program.