RFK Jr. listening to, prime modifying, Septerna x Novo
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Good morning. We’ve bought quite a bit happening in the present day. A bunch of my colleagues are in San Francisco for our Breakthrough Summit West, which is targeted on the impression of AI on well being innovation. Amongst these taking the stage: Robert Califf and Reed Jobs.
Additionally in the present day, well being secretary RFK Jr. shall 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 generally known as MASH. The deal for the “Section 3 prepared” drug, referred to as efimosfermin, contains one other $800 million if sure milestones are met.
RFK Jr. set to defend HHS cuts on Capitol Hill in the present day
Invoice Cassidy, the Republican chief of the Senate’s well being committee, plans to query well being secretary Robert F. Kennedy Jr. in the present day about his overhaul of HHS, in keeping with excerpts of his opening remarks at a listening to scheduled for later in the present day.
Cassidy plans to encourage reforms on the company — saying “bureaucratic bloat and regulatory hurdles” are impeding the company’s core work — but additionally push for Congress to be included within the plans for reform, in keeping with 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 scale back HHS’ price range by almost 1 / 4 from its present ranges. Kennedy can also 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 drugs
Novo Nordisk mentioned this morning that it’s going to work with the biotech Septerna to develop oral GLP-1 weight problems medicine, because it races to catch as much as Eli Lilly’s efforts to convey a extra handy weight reduction therapy to the market.
Beneath the deal, Septerna, which focuses on making small molecule medicine, 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 growth packages for potential small molecule remedies directed at targets inside a category of receptor referred to as G protein-coupled receptors — together with generally focused receptors like GLP-1, GIP, and glucagon hormone receptors.
First affected person handled with prime modifying
From my colleague Jason Mast: The primary ever affected person has been handled with prime modifying, a robust expertise 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 power granulomatous illness, a really uncommon genetic dysfunction that impairs the immune system and leaves sufferers susceptible to lethal infections. Researchers are trying to insert two lacking DNA letters into sufferers’ blood cells — a genetic surgical procedure that may solely be completed with prime modifying.
Prime, which has been struggling financially, obtained FDA clearance for the trial final August. However the rarity of the illness and the complication of the process – blood cells should be eliminated, edited, after which returned – has seemingly made recruitment gradual. The corporate has promised to launch knowledge this 12 months.
For extra information from the ASGCT assembly, join Jason’s e-newsletter right here.
Trump tucks a refined risk in his drug pricing order
Embedded in President Trump’s government order this week is a sentence that seems to threaten that the Meals and Drug Administration may withdraw drug approvals if pharma corporations don’t decrease their costs.
The order particularly provides 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 international locations pay. Notably, the order says that if “important progress” isn’t made on reducing costs, FDA Commissioner Marty Makary can be empowered to “overview and doubtlessly modify or revoke approvals granted for medicine… 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 knowledge on effectiveness.
Learn extra from STAT’s Ed Silverman.
Can the U.S. get Europe to boost drug costs?
One other side of Trump’s plan pertains to his aim of getting different developed areas like Europe to pay extra for medicine, as a way to slim the discrepancy between drug costs within the U.S. and different international locations.
U.S. officers have advised that they might increase the difficulty of drug costs as a part of ongoing commerce negotiations with the European Union, however it gained’t be simple to get European international locations 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 unhealthy actors. Moreover, European international locations have already been spending extra on medicines over time as costs rise, leaving them little room to pay much more for medicines.
Extra reads
