Eli Lilly signaled its growing interest in genetic medicines for cardiovascular conditions when it partnered with Verve Therapeutics earlier this year, committing to share in development of a preclinical gene-editing therapy addressing a key heart target. The pharmaceutical giant is now adding more cardiovascular disease drug prospects, pledging $250 million to secure the right to opt into development and commercialization of three additional Verve gene-editing therapies for other key targets.
This time, however, the cash isn’t going to Verve. Lilly is paying Beam Therapeutics, whose base-editing technology is used in Verve’s experimental genetic medicines. Their original alliance, started in 2019, gave Beam the option to share in the development and commercialization of Verve base-editing therapies for cardiovascular diseases driven by three targets. In exchange, Beam took an equity a stake in Verve and stood to receive milestone payments tied to the progress of the therapies.
Lilly is now buying Beam’s opt-in rights to share 33% of the worldwide development expenses and 50% of the expenses and profits from commercialization of the partnered Verve therapies in the U.S. Verve still holds rights to those therapies in the rest of the world. The Lilly payment to Beam breaks down to $200 million up front and a $50 million equity investment. According to terms of the agreement announced Tuesday, Lilly could pay Beam up to $350 million more depending on the progress of the cardiovascular therapies.
The Lilly cardiovascular therapies pipeline already has experimental therapies for reducing levels of lipoprotein (a), or Lp(a). Produced by the liver, high levels of this protein raise the risk of atherosclerosis, coronary heart disease, and stroke. The two Lilly therapies, a small molecule called muvalaplin and a small interfering RNA drug called lopodisiran, each take different approaches to reducing Lp(a). But they both require chronic dosing.
The partnership Lilly and Verve began over the summer covers an in vivo gene-editing therapy that offers the potential for a one-time treatment for reducing Lp(a). By securing Beam’s opt-in rights to the other Verve programs, Lilly expands its genetic medicines pipeline to the validated cardiovascular targets PCSK9 and ANGPTL3. A third program covered by the agreement addresses an undisclosed cardiovascular target mediated by the liver.
William Blair analysts Sami Corwin and Myles Minter spoke with Beam executives who told them that Lilly initiated the transaction, adding that the deal terms “had been in discussion for some time.” By William Blair’s count, this deal is the third largest—measured by size of upfront payment—in the gene-editing space. The largest was Vertex Pharmaceuticals’ amended collaboration with CRISPR Therapeutics in 2021, which came with $900 million up front, followed by Pfizer’s $300 million upfront payment to Beam to start a collaboration in 2022.
The Lilly transaction follows Beam’s restructuring announcement nearly two weeks ago. The biotech said that at the time that focusing on certain programs and laying off 20% of its staff would extend its cash runway into 2026. With the Lilly agreement, Beam now says it expects its cash to last into the second half of 2026 as it aims to achieve key data milestones for programs across its pipeline. Leerink Partners analyst Mani Foroohar said in a research note that while preliminary data for a Beam sickle cell disease program is on track to post Phase 1 data in 2024, showing differentiation versus the Vertex and Bluebird Bio sickle cell therapy candidates will likely take more time. Both therapies are expected to receive FDA decisions in December.
In a prepared statement, Ruth Gimeno, Lilly’s group vice president, diabetes, obesity and cardiometabolic research, said base editing represents an important new therapeutic approach for a wide range of diseases.
“This agreement expands the scope of Lilly’s ongoing relationship with Verve and gives us exposure to the full breadth of potential with Beam’s base editing platform,” she said. “We believe that single-course gene editing treatments could be a compelling new therapeutic option for patients at risk of cardiovascular disease, and we look forward to working with Verve toward that goal.”
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