Need to keep on high of the science and politics driving biotech at the moment? Join to get our biotech e-newsletter in your inbox.
Morning! As we speak we begin with some breaking newss: Novo Nordisk’s CEO is stepping down. We additionally focus on a brand new, tailor-made CRISPR remedy for an toddler with an epidemic, how Amgen owes Regeneron greater than $400 million now, and extra.
The necessity-to-know this morning
- BioMarin is paying $270 million to purchase Inozyme Pharma, developer of a remedy for ENPP-1 deficiency, a uncommon, inherited illness that impacts blood vessels, comfortable tissue and bone. The deal values Inozyme at $4 per share, or a 182% premium over its closing value on Thursday.
Novo Nordisk CEO steps down as firm seeks turnaround
From STAT’s Andrew Joseph: Lars Fruergaard Jørgensen, who led Novo Nordisk because it rode the growth of its weight problems and diabetes medicines to turn into one of many largest gamers in pharma, solely to see that success stall in latest months, is stepping down as CEO, the corporate mentioned Friday.
In an announcement, Novo famous that the corporate’s gross sales, income, and share value have nearly tripled throughout Jørgensen’s tenure, because it grew to become the frontrunner within the race to market GLP-1 diabetes and weight problems therapies. However the firm acknowledged that the chief changeup is being “made in mild of the latest market challenges Novo Nordisk has been going through,” particularly citing the collapse of its share value over the previous 12 months, a stretch when it’s fallen by half.
The corporate’s board and Jørgensen “have collectively concluded that initiating a CEO succession is in the very best curiosity of the corporate and its shareholders,” the corporate mentioned.
The seek for the subsequent CEO is ongoing.
Learn extra.
First-ever bespoke CRISPR remedy given to child
In a shocking medical first, researchers have used CRISPR to edit the distinctive genetic mutation of an toddler with a lethal liver dysfunction, STAT’s Jason Mast writes. KJ, who’s now 9.5 months previous, was recognized with CPS1 deficiency days after start — a situation that stops the liver from breaking down ammonia. KJ was given an experimental remedy crafted at document pace by a staff of scientists led by researcher at CHOP and College of Pennsylvania. He was given the primary dose of the bespoke CRISPR remedy by age six months; the work was revealed this week in NEJM.
“It’s nonetheless early days to have the ability to make definitive statements about how effectively this labored,” mentioned Rebecca Ahrens-Nicklas, the Kids’s Hospital of Philadelphia doctor who administered the remedy. “I feel we will say this has been proven to be protected and effectively tolerated, and there’s hints that this has been a profit to him, however we simply want extra time.”
Amgen loses $400 million antitrust battle to Regeneron
A federal jury has ordered Amgen to pay Regeneron greater than $400 million after discovering it violated U.S. antitrust legal guidelines by unfairly selling its PCSK9 ldl cholesterol Repatha over Regeneron’s rival remedy, Praluent. The decision stems from a 2022 lawsuit alleging Amgen tied rebates for blockbuster medicine like Enbrel and Otezla to Repatha’s preferential placement, pressuring pharmaceutical profit managers to exclude Praluent from formularies.
This newest ruling follows years of authorized battles between the 2 over their competing PCSK9 inhibitors and marks one other win for Regeneron, which final 12 months prevailed on the Supreme Courtroom in a associated patent dispute.
FDA indicators overhaul of Covid vaccine coverage
FDA Commissioner Marty Makary mentioned the company will quickly launch steerage reshaping the regulatory path for Covid-19 vaccines — probably requiring new scientific trials for seasonal updates, which is a serious shift. Talking on the FDA Legislation Institute convention, Makary mentioned the framework goals to supply transparency and predictability to vaccine makers. However he left key questions unanswered, together with whether or not Pfizer and Moderna should face the identical calls for as Novavax, which was reportedly requested to rerun trials.
Makary mentioned Vinay Prasad, the brand new director of CBER — the division that oversees vaccines — is assembly with business and FDA workers to make clear regulatory expectations. Concern has been swirling round this shift in technique amid the latest HHS layoffs, evolving CDC steerage, and the Trump administration’s aggressive new push to decrease drug costs.
Trump’s pricing plan and Theranos 2.0
How seemingly is it for President Trump’s new drug-pricing plan to be carried out? Why have traders appeared unbothered by the proposal? And can we see a return of Elizabeth Holmes’ blood-testing ambitions?
We focus on all that and extra on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. We chat in regards to the combined sentiment in biotech markets lately and a brand new blood-testing firm shaped by Holmes’ husband.
We additionally deliver on STAT’s D.C. correspondent Daniel Payne to speak about Trump’s “most-favored nation” drug pricing coverage and well being secretary Robert F. Kennedy Jr.’s latest congressional listening to.
Extra reads
