January 28, 2025

The need-to-know this morning

Daiichi Sankyo and AstraZeneca said the FDA approved the expanded use of Enhertu, their blockbuster antibody-drug conjugate drug, to treat patients with metastatic, hormone-positive breast cancer that expresses so-called "low" and "ultra low" levels of HER2, following hormone therapy. This is the most common type of breast cancer.

oncology

Radiopharma company passes Phase 3, but stays mum on specifics

From my colleague Allison DeAngelis: A new cancer treatment developed by ITM Isotope Technologies Munich SE has succeeded in a Phase 3 trial, setting the stage for a competitor to Novartis' Lutathera.

ITM announced today that its experimental radiopharmaceutical therapy ITM-11 led to a statistically significant progression-free survival in patients with neuroendocrine tumors that are inoperable. The company didn't give many more details than that, though, and declined to provide more specific data on patient response. The company plans to present more of the trial results at a medical meeting, and potentially submit them to the FDA for drug approval later this year.

The drug is a targeted therapy that delivers radioactive isotopes to a marker found on neuroendocrine tumors called SSTR. These types of treatments have boomed in recent years, in large part due to the success of Novartis' drugs Pluvicto — for prostate cancer — and Lutathera, which is also for neuroendocrine tumors.

ITM, which is based in Germany, largely operated under the radar. But the privately held company has strong roots in the radiopharma field: Outside of developing drugs, ITM produces and sells radioactive isotopes to companies like Novartis.

obesity

New startup follows Amgen's obesity approach

A new startup called Helicore Biopharma launched today with a focus on developing obesity drugs that block the GIP hormone, an approach similar to that of Amgen's experimental treatment MariTide.

The biotech raised $65 million in a Series A round, led by Versant Ventures and OrbiMed, and with participation from Longitude Capital and Wellington Management.

Helicore plans to start Phase 1 testing of its lead asset, a monoclonal antibody designed to bind to GIP, in the first quarter. The company is aiming for the treatment be dosed less frequently than currently available drugs, possibly monthly or even quarterly, CEO Gerrit Klaerner said in an interview. And unlike other GIP antagonists, Helicore's drug does not bind to GIP receptors, but instead binds to circulating GIP ligands, an approach that the Klaerner said will more effectively block the effects of GIP.

The company also plans to combine its GIP antibody with peptides that target other hormones, such as GLP-1.

There's been debate about whether obesity drugs should activate or block GIP. There are genetic data suggesting that loss of function of the GIP receptor is associated with lower body weight, but at the same time, drugs such as Eli Lilly's Zepbound — which activates both GLP-1 and GIP — have been shown to lead to substantial weight loss.

politics

Clarification on one aspect of Trump's freeze, but confusion about another

Last week, the Trump administration instituted a freeze on communications made by health agencies that sowed broad chaos and confusion.

In a recent memo to staff, NIH acting director Matthew Memoli clarified what staff can and cannot do. He wrote that employees can start new work on mission-critical research and continue working on ongoing studies, but cannot publicly communicate about them until the new Trump administration lifts the communications freeze. The agency also expects "additional guidance" this week on the freeze from HHS. Read more.

Meanwhile, the White House late yesterday released a separate order — telling government agencies to pause grants and loans on a host of federal programs.

The internal memo explicitly targets gender-affirming care and global financial assistance, but its broad language about "grants and loans" could ensnare a number of other federal health and assistance programs, ranging from medical research to food assistance and potentially even Medicaid, health care and legal experts speculated. Read more.

public health

CDC ordered to stop working with WHO immediately

All CDC staff who work with the WHO were told on Sunday night to immediately stop their collaborations and "await further guidance," the Associated Press reported.

President Trump, in one of his first executive orders, began the process of withdrawing the U.S. from WHO, but the order did not take immediate effect, since leaving the global group requires the approval of Congress and the U.S. is still meeting its financial obligations for the current fiscal year.

Experts were expecting a slow withdrawal, so the sudden stoppage came as a surprise. It comes as health authorities around the world are monitoring bird flu outbreaks among U.S. livestock and, in relatively rare cases, people.

As STAT's Helen Branswell has reported, a U.S. withdrawal from the WHO could have wide-ranging implications for global health and for domestic U.S. health programs. For example, the WHO runs the process through which the strains that annual flu shots and Covid vaccines should target are selected.

politics

A deep dive into RFK Jr.'s plans for vaccine regulation

As RFK Jr. faces confirmation hearings this week on his nomination to lead HHS, my D.C. colleagues closely analyzed his plans for regulating vaccines.

Though RFK Jr. has insisted that he doesn't want to take away vaccines and rather just wants to make sure they're safe, he has already sketched out a blueprint that could subject recommended vaccines to renewed scrutiny, redirect research, strip legal protections for vaccine makers, and change how vaccines are advertised.

This analysis offers a window into how RFK Jr. could use the levers of power as the top U.S. health official to sow doubt about vaccines at a time when rates of childhood immunizations are already slipping.

Read more from STAT's Rachel Cohrs Zhang and Sarah Owermohle.

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