May 16, 2025

The need-to-know this morning

BioMarin is paying $270 million to acquire Inozyme Pharma, developer of a treatment for ENPP-1 deficiency, a rare, inherited disease that affects blood vessels, soft tissue and bone. The deal values Inozyme at $4 per share, or a 182% premium over its closing price on Thursday.

Europe

Novo Nordisk CEO steps down as company seeks turnaround

From STAT's Andrew Joseph: Lars Fruergaard Jørgensen, who led Novo Nordisk as it rode the boom of its obesity and diabetes medications to become one of the biggest players in pharma, only to see that success stall in recent months, is stepping down as CEO, the company said Friday.

In a statement, Novo noted that the company’s sales, profits, and share price have almost tripled during Jørgensen’s tenure, as it became the frontrunner in the race to market GLP-1 diabetes and obesity treatments. But the company acknowledged that the executive changeup is being “made in light of the recent market challenges Novo Nordisk has been facing,” specifically citing the collapse of its share price over the past year, a stretch when it’s fallen by half.

The company’s board and Jørgensen “have jointly concluded that initiating a CEO succession is in the best interest of the company and its shareholders,” the company said.

The search for the next CEO is ongoing.

n-of-1 therapies

First-ever bespoke CRISPR therapy given to baby

In a stunning medical first, researchers have used CRISPR to edit the unique genetic mutation of an infant with a deadly liver disorder, STAT’s Jason Mast writes. KJ, who is now 9.5 months old, was diagnosed with CPS1 deficiency days after birth — a condition that prevents the liver from breaking down ammonia. KJ was given an experimental treatment crafted at record speed by a team of scientists led by researcher at CHOP and University of Pennsylvania. He was given the first dose of the bespoke CRISPR therapy by age six months; the work was published this week in NEJM.

“It is still early days to be able to make definitive statements about how well this worked,” said Rebecca Ahrens-Nicklas, the Children’s Hospital of Philadelphia physician who administered the therapy. “I think we can say this has been shown to be safe and well tolerated, and there's hints that this has been a benefit to him, but we just need more time.”

antitrust

Amgen loses $400 million antitrust battle to Regeneron

A federal jury has ordered Amgen to pay Regeneron more than $400 million after finding it violated U.S. antitrust laws by unfairly promoting its PCSK9 cholesterol Repatha over Regeneron’s rival therapy, Praluent. The verdict stems from a 2022 lawsuit alleging Amgen tied rebates for blockbuster drugs like Enbrel and Otezla to Repatha’s preferential placement, pressuring pharmaceutical benefit managers to exclude Praluent from formularies.

This latest ruling follows years of legal battles between the two over their competing PCSK9 inhibitors and marks another win for Regeneron, which last year prevailed at the Supreme Court in a related patent dispute.

vaccines

FDA signals overhaul of Covid vaccine policy

FDA Commissioner Marty Makary said the agency will soon release guidance reshaping the regulatory path for Covid-19 vaccines — potentially requiring new clinical trials for seasonal updates, which is a major shift. Speaking at the FDA Law Institute conference, Makary said the framework aims to offer transparency and predictability to vaccine makers. But he left key questions unanswered, including whether Pfizer and Moderna will have to face the same demands as Novavax, which was reportedly asked to rerun trials.

Makary said Vinay Prasad, the new director of CBER — the division that oversees vaccines — is meeting with industry and FDA staff to clarify regulatory expectations. Concern has been swirling around this shift in strategy amid the recent HHS layoffs, evolving CDC guidance, and the Trump administration’s aggressive new push to lower drug prices.

podcast

Trump's pricing plan and Theranos 2.0

How likely is it for President Trump's new drug-pricing plan to be implemented? Why have investors seemed unbothered by the proposal? And will we see a return of Elizabeth Holmes' blood-testing ambitions?

We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. We chat about the mixed sentiment in biotech markets these days and a new blood-testing company formed by Holmes' husband.

We also bring on STAT's D.C. correspondent Daniel Payne to talk about Trump's "most-favored nation" drug pricing policy and health secretary Robert F. Kennedy Jr.'s recent congressional hearing.

Listen here.

More around STAT