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Hi! Hope you had a nice extended weekend.

Today: Eli Lilly’s gene-editing data seems promising for high cholesterol, an AI drug discovery CEO dispelled some AI drug discovery hype, and the new interim FDA chief is so far well received.

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WASHINGTON — People in the food world didn’t know what to expect when the Trump administration appointed a little-known Florida attorney as the FDA’s top food official in 2025. 

They knew Kyle Diamantas worked at Jones Day representing food, beverage, and tobacco-industry clients. They saw the picture of him and Donald Trump Jr. holding giant, dead wild turkeys after a hunt. He had no experience in public health, in medicine or science, or in government.

The credentials didn’t scream qualified. And Diamantas was stepping into a center rocked by DOGE layoffs and a defiant resignation by former leader Jim Jones. 

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SAN MATEO, Calif. — Peyton Greenside, CEO of BigHat Biosciences, has hot takes. 

Her company designs antibody therapies using machine-learning, but she doesn’t like demos in which people open their computers and show off how fast they can design a drug. “If you want me to design you a protein right now in six hours, I’m happy to do it,” she said.

In fact, she can do it in 20 minutes. But if you’re actually in the business of making drugs, you are still going to have to do all the downstream tests, which take time and lots of money, Greenside said. “That’s where the hard work is, is making the actual drug.” 

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Eli Lilly said Monday that a high dose of its gene-editing therapy reduced cholesterol levels by 62% in participants in a clinical trial, an early but encouraging test of whether a one-time treatment may one day help people seeking to lower their LDL, or “bad,” cholesterol.

Lilly acquired the therapy, VERVE-102, in its $1 billion buyout of Verve Therapeutics last year. Executives tout it as a potential treatment to broadly prevent heart disease, the world’s leading killer, as many patients struggle to stay on existing, more conventional medicines for reducing cholesterol levels.

There were no treatment-related serious adverse events in the Phase 1 study — a notable finding, given that Verve had to shelve its first candidate due to safety concerns. 

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Merck’s bet on an antibody-drug conjugate developed in China has paid off in a major Phase 3 lung cancer study, where sacituzumab tirumotecan paired with Keytruda sharply outperformed Keytruda alone in a subset of lung cancer patients.

Also, the NIH is down another leader, and Sam Altman-backed longevity startup Retro Biosciences raises more funds.

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Retro Biosciences, the longevity startup backed by OpenAI CEO Sam Altman, has raised more money at a $1.8 billion valuation, it announced Friday. 

Retro has a big mission: Add 10 healthy years to the human lifespan. It is seeking to do that by using a variety of technologies, including in vivo gene therapies, cell replacement therapies, and other approaches to spur younger, healthier cells into aging tissues. 

The company is currently running its first clinical trial — testing a pill designed to enhance the body’s ability to better clear out protein aggregates in patients with Alzheimer’s disease. Retro CEO Joe Betts-LaCroix told the audience at STAT’s Breakthrough Summit West on Tuesday that the trial is going “super good” and that researchers haven’t seen any dose-limiting toxicities. He said he anticipates releasing some data from the trial around August. 

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The Food and Drug Administration on Friday rejected — again — an experimental treatment for advanced skin cancer developed by Replimune Group. 

Replimune’s treatment, an engineered virus designed to rev up the immune system against melanoma, has been a flashpoint in a simmering debate over shifting standards at the agency.

The drug was initially rejected in July, just two months after Vinay Prasad was appointed the FDA’s head of biologics. As an academic oncologist, Prasad criticized regulators for approving drugs with limited data, and the Replimune decision was viewed as a possible sign of the stricter stance he might take at the agency.

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CAR-T therapies are continuing to gain traction in autoimmune disease, with a notable new case.

Also, the FDA has withdrawn approval of GSK’s leucovorin for a rare brain disorder tied to autism, closing out an unusual episode shaped in part by political pressure.

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Why is old exon science getting new traction? What’s unsettling biotech VCs? And who will be the next CEO of PhRMA?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s weekly biotech podcast.

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Georg Schett had two things: a young patient deathly ill with lupus, and a couple of mouse studies raising the possibility that special T cells could tame the condition.

The German physician-scientist could produce the cells — chimeric antigen receptors, or CARs — at his institution, which was half the battle. Another hurdle: The patient’s parents. “They were like, ‘Don’t do that. You’re crazy,’” recalled Fabian Müller, Schett’s collaborator at the University of Erlangen-Nuremberg. A widespread fear at the time was that T cells would trigger or worsen autoimmune disease. 

The rest of the story is the rare scientific fairy tale: The patient got better. Five years on, she is still in remission, and working in the very clinic where she was treated. Her case upended the world of autoimmune disease, driving a flood of experimentation and investment and offering new hope to millions of patients. 

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PhRMA CEO Steve Ubl is stepping down. Biotech investors are grappling with pressure from AI and rising competition from Chinese labs, even though firms sit on large cash reserves.

Also, the Trump administration is stepping back from the “indirect cost” fight at the NIH, and new data suggest genetics may modestly influence responses to GLP-1 drugs.

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For some advanced cancers, sequencing the tumor genome should be one of the first steps patients and physicians take. But a new study finds that many patients never receive genomic testing and so never get the chance to know if they might have benefitted from newer, more targeted therapies.

The study, published on Tuesday in JAMA Network Open, examined how many patients diagnosed with one of five different metastatic cancers received genetic sequencing for the cancers. For most cancers in the study, roughly half of patients in the cohort received genetic sequencing. Patients with low income, Medicare or Medicaid coverage, and Black or Hispanic race or ethnicity were also less likely to receive sequencing.

Cancer medicine and research have made enormous progress over the last few decades. The overall five-year survival rate has pushed up to 70% as of 2026, and the five-year survival rate for metastatic cancer has doubled since the 1960s. That’s in large part thanks to advances in medicines and technologies that can help treat cancer, like targeted therapies that work by exploiting key cancer mutations.

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In a move that will grow its oncology pipeline, Gilead Sciences is spending more than $3 billion to buy the German biotech Tubulis, the companies said Tuesday.

The deal includes an upfront payment of $3.15 billion and up to $1.85 billion more if certain milestones are reached. It also highlights both the therapeutic and commercial promise of antibody-drug conjugates, the next-generation chemotherapy treatments that the privately held Tubulis is developing.

Gilead’s latest move comes just months after it said it would acquire Arcellx in a deal worth $7.8 billion. The two companies had already been working together on a multiple myeloma CAR-T therapy that could be approved later this year. Gilead last month also announced that it was buying Ouro Therapeutics, which is focused on drugs for autoimmune disease, for up to $2.18 billion.

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An abdominal aortic aneurysm is a life-threatening vascular condition with limited treatment options. 

Now, researchers from the University of Michigan Frankel Cardiovascular Center have identified a driving force behind the condition, opening up a potential target for new therapies. Their paper uncovering the causal link between triglycerides and abdominal aortic aneurysms won the STAT Madness 2026 popular vote. 

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WASHINGTON — The Food and Drug Administration used the president’s budget to propose policies aimed at encouraging domestic development and manufacturing of drugs.  

FDA Commissioner Marty Makary has said the agency needs “giant, big ideas” to counter China’s dominance in early-stage clinical development of drugs. Among the FDA’s ideas are proposals to make it easier to run early-stage trials in the U.S. and to hand an advantage to U.S.-based generics manufacturers.

The Trump administration has been using a variety of policy levers to try and bring drug manufacturing to the U.S. For example, many of the brand drugmakers that struck deals to lower U.S. prices also promised to increase domestic manufacturing, under the threat of tariffs.

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For the second time in two years, a bill is moving through the Colorado legislature that would exempt orphan drugs, which are used to treat rare diseases, from pricing caps that might be pursued by the state’s Prescription Drug Affordability Board — a panel whose work is being closely watched elsewhere in the country.

The effort reflects concerns that patients may lose access to these drugs if pharmaceutical companies halt sales of such treatments in the state. But opponents argue exemptions would unnecessarily extend to numerous big-selling medicines for common conditions that — thanks to regulatory endorsements — also happen to have an orphan designation.

As a result, consumer advocates complain the maneuver would only increase the risk that countless patients could have trouble paying for a wide variety of medicines. They further argue that the legislation would preserve profits for drug companies at the expense of the state government — and its taxpayers — as it tries to cope with budgetary strains.

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A four-month FDA delay prompted a cash-strapped biotech company to shutter. The White House’s proposed NIH cuts are drawing bipartisan backlash. Peptide hype is outpacing evidence. And more!

The need-to-know this morning

• Neurocrine Biosciences said this morning that it would buy Soleno Therapeutics and its treatment for Prader-Willi syndrome for $2.9 billion. Neurocrine is paying $53 a share for Soleno, a 34% premium to its closing price on Thursday.
  

A four-month FDA delay derailed a small biotech. Is it a sign of the times?

FDA delays can happen. For large drugmakers, they can be frustrating; for small drugmakers, they can be existential.

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