March 10, 2025

The need-to-know this morning

Indian drugmaker Sun Pharma said it will acquire Checkpoint Therapeutics, maker of an approved cancer immunotherapy, for $355 million. The deal values Checkpoint at $4.10 per share, a 66% premium to Friday's closing price.
Novo Nordisk reported results from the REDEFINE 2 clinical trial, showing its experimental amylin/GLP-1 drug Cagrisema induced 12.6% placebo-adjusted weight loss after 68 weeks in patients who are obese or overweight with Type 2 diabetes.
Mineralys Therapeutics reported results from two placebo-controlled studies, showing its experimental drug called lorundrostat significantly reduced systolic blood pressure in patients with high blood pressure not controlled by current medications.
Cytokinetics said the FDA completed a mid-cycle review of its heart drug aficamten, and that the company maintains its "expectation for a differentiated label and risk mitigation profile for aficamten, if approved by FDA."

CRISPR

Beam reports first evidence of in-vivo gene editing for lung disease

Beam Therapeutics said this morning that it used a form of CRISPR called base editing to correct, in several patients, the mutation that drives a debilitating lung condition that may affect tens of thousands of Americans.

The company touted the data as evidence of the first successful use of CRISPR gene editing to fix a disease-causing mutation. Most previous CRISPR trials have focused on knocking out genes that are either deleterious or that, when removed, can have profound impacts in particular diseases, like sickle cell.

Patients with the condition Bean targeted, alpha-1 antitrypsin deficiency, or AATD, generally have a single misspelled letter in the gene that codes for AAT, a protein that helps protect the lungs.

politics

HHS offers buyouts to workers as Collins warns of Trump cuts

Most of the 800,000 workers at HHS were emailed a buyout offer to leave their job for as much as $25,000, as part of President Trump's government cuts.

The workers have until 5 p.m. Friday to submit a response, the Associated Press reports. The email was sent across the agencies within HHS, which include the CDC, the NIH, and the FDA. It landed in inboxes days before agency heads are due to offer plans for cutting their workforces. Read more.

The news came as Francis Collins, former director of the NIH, warned that two signature programs — an initiative to decipher how the brain works, and another to build a massive and diverse genomics database — are threatened by Trump administration actions.

He was speaking at one of dozens of rallies held around the U.S. on Friday to protest Trump administration policies that are affecting scientific research. Collins also stressed NIH's role in bankrolling the basic science that fuels clinical research, and called out the pharma industry's silence on the recent funding cuts.

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SOURCE: ERIN CLARK/THE BOSTON GLOBE

The industry "also has much to lose if the federal investment is damaged," Collins said. "By the way, it would be good to hear more about that from pharmaceutical company leaders, just saying."

Read more from STAT's Anil Oza and Katherine MacPhail.

regulation

Could the FDA introduce a new category for biologics?

Currently, the FDA classifies biologics as either being drugs, or not. Biologics that are categorized as drugs require huge sums of money and time to be proven out in clinical trials and eventually brought to market.

But it seems possible that, under the Trump administration and the oversight of health secretary Robert F. Kennedy Jr., the FDA may introduce a third category, writes Paul Knoepfler, a professor of cell biology at UC Davis, in a new First Opinion column on regenerative medicine. That was his takeaway from attending a recent FDA workshop on cell therapies and tissue-based products.

The third category could speed up the approval of promising therapies and save biotechs and other sponsors millions of dollars, Knoepfler writes. But at the same time, it could also be"ripe for abuse by some sponsors such as those running clinics marketing unproven biologics"

exclusive

Google scrubs health equity language on websites

Google has revised its websites to downplay its commitment to health equity, the latest example of a company changing language following President Trump's attack on DEI initiatives.

Since January, Google has been removing language that referred to addressing social, economic, and other factors that contribute to disparities in health outcomes for different population groups, according to a STAT review. The company's "health equity" page has been retitled "health optimization," and the company's global head of health equity, Heather Cole-Lewis, has a new title: global head of health optimization.

research

Scientists file antitrust lawsuit against research publishers

Four researchers have sued six of the world's biggest publishers, alleging that they violated antitrust law by colluding not to pay researchers for peer reviewing manuscripts, preventing them from submitting papers to more than one journal at a time, and blocking authors from publicly discussing or sharing work once they've submitted it to a journal.

It's a stark sign of scientists' escalating frustration with how academic journals operate. "The scheme has been remarkably profitable for the publisher defendants, while doing tremendous damage to science and the public interest," the plaintiffs claim.

The case could have sweeping implications for the research community. While it's unclear whether this particular lawsuit will succeed, the rising discontent threatens to topple the system tasked with vetting, recording, and communicating biomedical breakthroughs, experts said.