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Good morning. We’ve bought so much occurring at this time. A bunch of my colleagues are in San Francisco for our Breakthrough Summit West, which is concentrated on the influence of AI on well being innovation. Amongst these taking the stage: Robert Califf and Reed Jobs.
Additionally at this time, well being secretary RFK Jr. will likely be testifying in entrance of lawmakers about cuts to HHS. Extra on that beneath.
The necessity-to-know this morning
- GSK is paying $1.2 billion upfront to Boston Prescription drugs for an experimental drug to deal with the liver illness often known as MASH. The deal for the “Section 3 prepared” drug, known as efimosfermin, consists of one other $800 million if sure milestones are met.
RFK Jr. set to defend HHS cuts on Capitol Hill at this time
Invoice Cassidy, the Republican chief of the Senate’s well being committee, plans to query well being secretary Robert F. Kennedy Jr. at this time about his overhaul of HHS, in response to excerpts of his opening remarks at a listening to scheduled for later at this time.
Cassidy plans to encourage reforms on the company — saying “bureaucratic bloat and regulatory hurdles” are impeding the company’s core work — but in addition push for Congress to be included within the plans for reform, in response to the remarks.
Kennedy goes earlier than the well being committee to debate the Trump administration’s price range request, which — if authorized by Congress — would cut back HHS’ price range by almost 1 / 4 from its present ranges. Kennedy can be scheduled to testify earlier than the Home Appropriations Committee.
Learn extra from STAT’s Daniel Payne.
Novo indicators take care of Septerna to develop weight problems tablets
Novo Nordisk mentioned this morning that it’ll work with the biotech Septerna to develop oral GLP-1 weight problems medication, because it races to catch as much as Eli Lilly’s efforts to convey a extra handy weight reduction remedy to the market.
Beneath the deal, Septerna, which focuses on making small molecule medication, is eligible to obtain about $2.2 billion from Novo, together with greater than $200 million in upfront and near-term milestone funds.
The businesses mentioned they’ll begin with 4 improvement applications for potential small molecule remedies directed at targets inside a category of receptor known as G protein-coupled receptors — together with generally focused receptors like GLP-1, GIP, and glucagon hormone receptors.
First affected person handled with prime enhancing
From my colleague Jason Mast: The primary ever affected person has been handled with prime enhancing, a strong know-how for making exact adjustments to DNA.
Harry Malech, an NIH researcher and investigator on the research, introduced the information yesterday throughout an award speech on the American Society of Gene & Cell Remedy assembly, apparently in opposition to the needs of Prime Medication, the corporate that devised the experimental remedy. “They’ll in all probability throw bricks at me,” he joked.
The remedy is for persistent granulomatous illness, a really uncommon genetic dysfunction that impairs the immune system and leaves sufferers susceptible to lethal infections. Researchers try to insert two lacking DNA letters into sufferers’ blood cells — a genetic surgical procedure that may solely be executed with prime enhancing.
Prime, which has been struggling financially, acquired FDA clearance for the trial final August. However the rarity of the illness and the complication of the process – blood cells must be eliminated, edited, after which returned – has probably made recruitment gradual. The corporate has promised to launch knowledge this yr.
For extra information from the ASGCT assembly, join Jason’s publication right here.
Trump tucks a refined menace in his drug pricing order
Embedded in President Trump’s govt order this week is a sentence that seems to threaten that the Meals and Drug Administration might withdraw drug approvals if pharma firms don’t decrease their costs.
The order particularly offers drugmakers 30 days to agree on decrease costs with the federal government, with the purpose of bringing costs all the way down to what different nations pay. Notably, the order says that if “vital progress” isn’t made on decreasing costs, FDA Commissioner Marty Makary could be empowered to “assessment and probably modify or revoke approvals granted for medication… that [may be] unsafe, ineffective, or improperly marketed.”
Regulatory and authorized consultants query the legality of that provision, although, because the FDA can solely withdraw approval of a drugs if sure statutory standards are met, like if the drug is unsafe or if there’s a lack of information on effectiveness.
Learn extra from STAT’s Ed Silverman.
Can the U.S. get Europe to lift drug costs?
One other facet of Trump’s plan pertains to his objective of getting different developed areas like Europe to pay extra for medication, as a way to slim the discrepancy between drug costs within the U.S. and different nations.
U.S. officers have instructed that they might elevate the difficulty of drug costs as a part of ongoing commerce negotiations with the European Union, however it received’t be straightforward to get European nations to budge, my colleague Drew Joseph reviews.
In Europe, drug costs are negotiated and decided nation by nation, making it tougher for the U.S. to exert affect on areas it deems to be dangerous actors. Moreover, European nations have already been spending extra on medicines over time as costs rise, leaving them little room to pay much more for medicines.
Extra reads
