April 14, 2025

The need-to-know this morning

Third Harmonic Bio, a company that has been developing treatments for inflammatory diseases, announced a plan to liquidate and dissolve itself.

obesity

Pfizer discontinues its experimental GLP-1 pill

From STAT's Matthew Herper: Pfizer said this morning that it would stop development of danuglipron, its experimental oral GLP-1 medicine to treat obesity, focusing its efforts on another medicine with a different mechanism of action.

The company said in a press release that an asymptomatic volunteer in the company's studies experienced "potential drug-induced liver injury," which resolved after stopping the medication. After reviewing all clinical data for the medicine and consulting with regulators, Pfizer decided to halt research on the medicine.

"While we are disappointed to discontinue the development of danuglipron, we remain committed to evaluating and advancing promising programs in an effort to bring innovative new medicines to patients," said Chris Boshoff, Pfizer's chief scientific officer and head of R&D, in a statement.

In the release, Boshoff emphasized that Pfizer would continue to develop another oral obesity drug, called a GIP antagonist, and other earlier obesity programs.

gene editing

Verve treatment shows promise for high cholesterol

Verve Therapeutics is inching closer to making its one-and-done cholesterol treatment a reality. New Phase 1 data released this morning show that Verve-102, its updated gene-editing therapy, lowered LDL cholesterol by 53% in patients on the highest dose without triggering serious side effects.

This is a win for Verve, which had previously paused its first-generation therapy over safety concerns tied to its lipid nanoparticle delivery system.

"This is an impressive result and suggests that editing of the PCSK9 gene can yield large and durable reductions in LDL cholesterol with good tolerability," one Cleveland Clinic cardiologist not involved in the study told STAT.

RESEARCH

NIH grant bottleneck loosens, at least a little

After months of paralyzing silence from the NIH, advisory councils are finally starting to meet again — a cautious step forward after the Trump administration's sweeping January freeze on health agency communications effectively halted billions in biomedical research funding. That said, the meetings that took place were shortened, opaque, and leave open questions about the continuity of grantmaking.

In the wake of cuts at the NIH, labs across the country are experiencing their own layoffs.

"This is a positive step, but certainly things are not 'back to normal,'" one NIH advisory council member told STAT's Megan Molteni.

drug pricing

The case against importing foreign drug prices

The U.S. shouldn't tie drug prices to artificially low overseas rates via the "most-favored nation" policy, opine two health policy experts at USC. They call the notion of such a policy a a blunt tool that would cede pricing power to foreign governments, threaten global access, and ultimately hobble innovation.

Instead, the U.S. should take charge with pricing reform by setting rational, value-based prices and then spreading that model abroad. This means rewarding drugs that actually work, they write, with success-based pricing.

"We often recoup these additional costs in the form of longer and healthier lives — cancer is a case in point," they write. "Only American price leadership can both help consumers and sustain innovation into the future."

crispr

Biotech investors: Real progress isn't always flashy

It's time for biotech investors to learn an important lesson: CRISPR isn't a miracle cure, opine two gene-editing leaders at the ChristianaCare hospital system.

But CRISPR is steadily becoming a powerful tool against diseases like oral cancer, especially in underserved communities. By targeting chemo resistance directly within tumors, researchers are opening new doors for patients who've run out of options.

But biotech hype cycles threaten to eclipse real, incremental wins: Companies like Tome Biosciences raised massive sums and then lost steam after trying to chase systemic cures that science hasn't quite figured out yet. Meanwhile, less glamorous forms of research get sidelined, they say.

"The market gyrations are an indictment of how venture capital tends to look for the next really big thing instead of focusing on starting small and slowly building on victories to support patients and their health," the authors write.

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