Breaking Boundaries: Lilly’s Investment in Base Editing for Heart Disease

Pharmaceutical giant Eli Lilly is expanding its efforts in cardiovascular disease research through a new deal with Beam Therapeutics and Verve Therapeutics worth up to $600 million. The deal centers around base editing, an emerging gene editing technology that Beam and Verve are pioneering for new precision genetic medicines.

Under the agreement announced today, Lilly will acquire Beam’s opt-in rights to co-develop and co-commercialize several of Verve’s base editing programs targeting cardiovascular disease. This includes lead programs focused on PCSK9 and ANGPTL3 – two high profile genes involved in cholesterol regulation and metabolism. A third undisclosed target related to liver-mediated cardiovascular disease is also included.

In exchange, Beam will receive a hefty $200 million upfront payment along with a $50 million equity investment from Lilly. Beam is further eligible for up to $350 million in future milestone payments as the programs advance through clinical trials and regulatory approvals.

For Lilly, this deal provides access to a promising new approach to treating cardiovascular disease, an area where the company already has a major presence. Lilly has been a leader in cholesterol drugs like statins for decades, and more recently entered the PCSK9 market through its ownership of Repatha. But despite effective medications, cardiovascular disease remains a top killer globally.

Base editing offers a way to precisely and permanently modify disease-causing genes in order to lower cholesterol and potentially deliver stronger treatment effects than current options. Early human trials have already shown base editing of PCSK9 can lower LDL cholesterol. Verve recently initiated a clinical trial using base editors to target both PCSK9 and ANGPTL3 simultaneously.

Take a moment to take a look at Ocugen (OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines.

As a technology pioneer, Beam is widely considered the leader in base editing. The company has uncovered a series of natural enzymes that can be programmed to make single letter DNA changes at targeted sites without cutting the double strand like traditional CRISPR gene editing. This opens possibilities for more precise control while minimizing unintended effects.

Beam CEO John Evans highlighted base editing as a core strategic priority, and views creative partnerships as a key path to accelerate development. “Our initial collaboration with Verve and this new transaction with Lilly are exemplary of our execution of that strategy,” Evans said. “This deal provides meaningful upfront capital to advance our portfolio of clinical- and research-stage programs, with significant additional value achievable as the Verve programs advance.”

For Beam, the capital influx provides fuel to advance its broader base editing pipeline including programs in sickle cell disease, alpha-1 antitrypsin deficiency, and glycine encephalopathy. The company now expects its cash runway to extend into the second half of 2026.

Lilly’s history with Verve also preceded this acquisition. In 2021, Lilly led Verve’s $105 million Series B financing and took a stake in the company. That marked another early mover deal to tap into base editing. Verve CEO Sekar Kathiresan said “Lilly’s extensive capabilities in drug development and commercialization make them an ideal partner for Verve as we work together to advance base editing programs aimed at reducing CVD risk through genome editing.”

Beam and Verve join a short list of biotechs focused on realizing the promise of base editing. But Lilly’s involvement marks a huge vote of confidence from the pharma world. As base editing advances toward the clinic, deals like this suggest major players view the technology as more than just hype.

Lilly has been aggressively scouting the latest biotech innovations through both in-house R&D and external deals. The past year saw Lilly acquire hot companies like POINT Biopharma and Repare Therapeutics for large sums. Base editing adds a new tool in Lilly’s toolkit for next generation therapeutic approaches.

Cardiovascular disease also represents an attractive area for investment despite already having effective medications. Heart attacks, strokes and other complications remain a top cause of mortality globally. In the US alone, 1 in 4 deaths are attributable to heart disease each year. Even modest improvements in treatment can translate into major public health benefits.

The risk for Lilly and other major pharmaceutical companies is that base editing and gene editing fail to live up to their early promise in the clinic. However, most experts are optimistic the technology will usher in a new wave of therapies over the next decade. For a pharma giant like Lilly with over $28 billion in yearly revenue, the potential reward is well worth the investment risk to stay on the cutting edge.

Lilly Makes $1.4 Billion Bet on Radioactive Cancer Drugs with POINT Biopharma Acquisition

Pharmaceutical giant Eli Lilly is expanding its cancer treatment portfolio into a promising new area by acquiring POINT Biopharma, a company developing radioactive drugs that precisely target tumors, for $1.4 billion.

POINT specializes in radioligand therapies, an emerging approach to cancer treatment that uses radioactive particles linked to molecules that bind to receptors on cancer cells. This enables the radiation to selectively kill tumors while limiting damage to healthy tissue.

Lilly is paying $12.50 per share in cash for POINT, an 87% premium over the stock’s latest closing price. The deal will give Lilly control of POINT’s pipeline of radioligand therapy candidates, which includes two late-stage experimental drugs.

One drug, PNT20021, targets prostate cancer tumors by binding to a protein called PSMA. Study data expected later this year will show whether it extends the lives of men with metastatic castration-resistant prostate cancer.

The other late-stage drug, PNT20031, homes in on neuroendocrine tumor cells via their somatostatin receptors. It may provide a new option for patients with advanced gastroenteropancreatic neuroendocrine tumors.

Take a look at PDS Biotechnology, a clinical stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies.

Beyond these lead programs, POINT has several earlier stage radioligands in development for cancers of the breast, lung, and brain. Lilly gains full access to progress these toward human testing.

The deal also gives Lilly two specialized facilities Point has built to produce and research radioligands. Manufacturing the drugs involves linking medical radioisotopes like actinium-225 to the targeting molecules, which requires nuclear expertise.

Jacob Van Naarden, head of Lilly’s oncology division, touted the promise of radioligands to safely destroy cancer while avoiding the side effects of traditional chemo. “We are excited by the potential of this emerging modality,” he said.

Lilly has been growing its cancer treatment business in recent years through deals for other firms’ drug candidates and technologies. The POINT acquisition similarly expands Lilly’s footprint into an area well-suited for precision medicine.

The U.S. Food and Drug Administration has already approved over a half dozen radioligand therapies from Lilly competitors like Novartis. Their success is driving a surge of investment and deal-making in the radiopharmaceutical field.

But analyst Geoffrey Porges of SVB Securities thinks Lilly overpaid for POINT. “We believe the valuation fails to reflect the very high risks inherent in drug development,” he wrote in a note to investors.

Porges added that Lilly may need to invest over $2 billion more to fully develop POINT’s pipeline over the next 5-7 years, with no certainty the drugs will pan out.

Lilly expects the acquisition to close by the end of 2023 after gaining required antitrust and regulatory approvals. The majority of POINT shareholders also must tender their shares as part of the agreement.

The deal marks Lilly’s second major oncology purchase in 2022. It paid $1.1 billion earlier in the year access to cancer drug candidates from China’s Zymeworks. With POINT, Lilly is now positioned as a leader across multiple next-wave approaches in the high-stakes race to develop better cancer treatments.