February 28, 2025

The need-to-know this morning

Praxis Precision Medicine said independent data monitors recommended the discontinuation of a Phase 3 study involving its experimental treatment for essential tremors. The futility analysis concluded the Praxis drug was unlikely to show a benefit compared to a placebo. Praxis, however, has decided to complete the study and a companion study also underway.

regulatory

BIO CEO: FDA reform needs to be underpinned by strategy, not 'indiscriminate' cuts

The Trump administration could save the lives of countless patients with rare diseases by modernizing and streamlining the FDA's approval process, John Crowley, the CEO and president of the trade group BIO, opines this morning for STAT. Specifically, he said that excising redundancies and bureaucracies and focusing on patients would help accelerate cures.

But Crowley didn't mince words when addressing the massive cuts playing out across government health agencies, arguing that an experienced and properly staffed FDA is critical.

"Simply slashing personnel at federal health agencies… does not equate to removing outdated restraints and modernizing an important regulatory body," Crowley wrote, taking a harder line than much of the industry so far. He added that the "aggressive and often indiscriminate personnel cuts have lacked the strategic insights necessary to modernize and reform our nation's health care agencies, especially the FDA."

DEI

Pfizer shifts DEI focus to approach based on 'merit'

Pfizer has revised its DEI webpage to emphasize merit-based principles, STAT's Elaine Chen and J. Emory Parker report. The page is now renamed "Merit-Based Diversity, Equity, and Inclusion," and has dropped references to a 2023 racial equity assessment and partnerships dedicated to increasing diversity in its talent pipeline.

"We constantly update and evolve the content and design of our website to reflect our current programs, initiatives, and commitment," a Pfizer spokesperson told STAT in regards to the changes.

Similar shifts are happening at other drugmakers like Bristol Myers Squibb and Johnson & Johnson. Pfizer CEO Albert Bourla has met with Trump multiple times, but the company continues to face criticism from Trump's supporters — including Robert F. Kennedy Jr.

manufacturing

Lilly expansion pragmatic, and probably costly

Eli Lilly's $27 billion investment in four U.S. manufacturing sites is a strategic play amid the Trump administration's push for domestic production, potential pharma tariffs, and corporate tax incentives. And the plan may indeed strengthen national security and cut Chinese ingredients out of the supply chain a bit more, STAT's Ed Silverman writes.

But whether it'll lower drug prices is a different question entirely, given how high the cost of labor and construction are stateside.

"I don't think that locating production here will bring costs down," a health care economist at Northwestern University told STAT's Ed Silverman. "I think it may make costs go up and someone will have to find a way to make that palatable… If anything, it may make drugs more expensive, particularly for generics that migth eventually be made here (and many of which are made in India, a lower-cost market). You know, if you want nice things, you have to pay for them."

podcast

Turbulence at the FDA & bluebird Bio's sale

What's it like reporting on the FDA during the early and turbulent weeks of the Trump administration? Why did Bluebird Bio fail and are there broader lessons for the gene therapy field? And who are these young Turks making waves in the biotech VC world?

We talk about all that and more on this week's episode of the "The Readout LOUD," STAT's biotech podcast. It's a STAT reporter two-fer guest episode. First, Lizzy Lawrence joins from D.C. to dish on the upheaval and drama taking place at the FDA. Then, we'll chat with Jason Mast about Bluebird Bio, its fire sale to private equity and what lies ahead for the unsettled world of genetic medicines.

Listen here.

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