Health care CEOs cash in despite weak stock growth

The biggest companies in health care underwhelmed Wall Street last year, but their CEOs still raked in record pay, an analysis of corporate filings from STAT's Bob Herman and J. Emory Parker shows. Leaders of 275 firms collected a combined $3.6 billion, with average compensation topping $13 million. And 91 executives earned at least $10 million, the most since 2021, even as the S&P 500 health care index barely budged and biotech stocks slipped.

Long tenures and lucrative stock awards fueled the windfalls, with BioNTech's Uğur Şahin topping the list at more than $300 million, followed by DaVita's Javier Rodriguez at $148 million. Notably, the stock price of Regeneron Pharmaceuticals plummeted almost 20% in 2024, but co-founder and CEO Len Schleifer — one of the richest men in health care — made more than $124 million.

gene editing

What David Liu is up to

From my colleague Jason Mast: An approach he calls "disease-agnostic" genome editing. The strategy, presented for the first time at the annual Cold Spring Harbor CRISPR meeting last week, is an attempt to tackle one of the field's biggest bottlenecks. "I know it sounds crazy," the storied Harvard University and Broad Institute molecular biologist said, comparing it to a famous South Park episode involving a questionable business plan hatched by underpants-thieving gnomes.

Modern genome editors are pretty good at making small changes to DNA: Swap one letter here, insert two letters there. But the human genome, famously, contains a great number of letters, many of them consequential. It's too costly and time-consuming to, one by one, make gene-editing treatments for each. So companies have almost exclusively focused on the tiny handful of mutations common enough to justify investment.

Researchers have proposed a few solutions, including regulatory changes and new systems for inserting whole genes. Liu, who previously designed two powerful and widely used gene-editing tools, is also developing a spin on a field called transfer RNA (tRNA) therapy, named after a molecule critical in building proteins.

Many diseases are caused by so-called nonsense mutations that tell the cell to prematurely stop making a protein. These tRNA molecules are responsible for reading that mutation and halting production. Researchers have long proposed providing new, lab-built tRNAs designed to read the "stop" signal as a "go" and keep building the protein. In principle, the approach could work across nonsense mutations, not just for a particular misspelling or disease. But implementing the idea has been challenging.

Liu's approach, demonstrated with some early lab and animal data, would use prime editing to permanently edit a tRNA in patients to perform that function.

rare disease

Stealth BioTherapeutics makes push for FDA approval

Stealth BioTherapeutics is mounting an unusually public campaign to win FDA approval for its experimental Barth syndrome drug, elamipretide, after years of regulatory back-and-forth and a recent rejection. Now the company is taking the rare step of sharing its FDA rejection letter with select outlets, arguing it has resolved concerns and resubmitted its application under the accelerated approval pathway, STAT's Ed Silverman writes.

The saga underscores tensions over how much flexibility regulators should apply to drugs for ultra-rare diseases. Physicians and families say Stealth's medicine is keeping patients in a so-called expanded access program alive, while delays threaten access — and perhaps the company's survival.

"As we have money, I'm not taking babies off the drug," Stealth CEO Reenie McCarthy told STAT. "There is a path forward … That's what's really come into sharper focus over the ensuing months and leading to resubmission… All the deficiencies [cited by the FDA in the rejection letter] have been addressed. We've crossed the gateway."

glp-1 drugs

FDA clears Wegovy for liver disease MASH

The FDA late Friday granted accelerated approval for Novo Nordisk's obesity drug Wegovy to treat MASH with moderate to advanced fibrosis, expanding the blockbuster drug's reach beyond obesity and heart risks.

The decision, backed by Phase 3 data showing improvements in fibrosis and symptom resolution, positions GLP-1 therapies as contenders in the fraught race to develop treatments for MASH.

While Madrigal's liver-targeted therapy was first to market, STAT's Elaine Chen writes, doctors expect GLP-1s like Wegovy to complement rather than replace liver-directed drugs — likely benefiting early-stage MASH patients more than those with more advanced cases.