Moderna's CMV vaccine flops...

Moderna's highly touted cytomegalovirus vaccine — long billed as its next big win after its Covid-19 shot — failed a Phase 3 trial, the company announced yesterday. It showed just 6% to 23% efficacy in blocking infection, and missed its primary endpoint.

The setback delivers another blow to the biotech, whose Covid sales have cratered and whose RSV shot trails rivals from Pfizer and GSK.

The CMV program was expected to showcase the "power of mRNA technology," per CEO Stéphane Bancel in November 2020, but analysts had since grown skeptical amid what they saw as dwindling odds.

Read more from STAT's Jason Mast and Matthew Herper

THERApeutics

... but the company is pressing ahead in cancer

Setbacks aside, Moderna is pressing forward with mRNA therapeutics for cancer and rare diseases.

At ESMO, Moderna shared early data from mRNA-4359, an off-the-shelf immunotherapy that showed immune responses in PD-L1–positive melanoma patients. The company's head of oncology, Kyle Holen, said its Phase 3 personalized cancer vaccine with Merck is fully enrolled and could yield data by 2026.

He noted Moderna's cost-cutting hasn't slowed its oncology push, and regulatory agencies remain supportive despite broader skepticism about mRNA vaccines. "We're still seeing positive signals from regulators," Holen said.

hiv/aids

CVS presses Gilead over pricey HIV prevention drug

CVS Caremark is declining to cover Gilead's new twice-yearly HIV prevention drug, Yeztugo, citing "clinical, financial, and regulatory" concerns — but an internal email obtained by STAT shows the pharmacy giant may mainly just want a lower price, STAT's Ed Silverman writes.

The $28,000-a-year injectable, hailed for its convenience, remains off CVS formularies even as rivals Express Scripts and UnitedHealth have added it. CVS argued the drug is too expensive compared with existing PrEP options, including $20-a-month generics.

But the companies are under pressure from activists, who argue Yeztugo can both provide a more convenient option for existing PrEP users and also attract people who have faced challenges adhering to a daily pill regimen.

Read more.

artificial intelligence

European oncologists issue first LLM use guidelines

At its annual meeting in Berlin, the European Society for Medical Oncology unveiled its first guidance on using large language models in cancer care — acknowledging both their promise and their risks.

Published in the Annals of Oncology, the framework divides AI use into three categories: patient-facing tools, clinician aids, and institutional systems. The authors warned that chatbots and decision-support tools lack robust evidence and could erode physician skills or blur accountability. Still, they urged pragmatic oversight rather than prohibition.

"Basically, we are acknowledging that people are using this. We are not discouraging them from using this. But of course, there are some limitations," said Jakob Kather, a professor for clinical artificial intelligence from the Dresden University of Technology and one of the authors of the guidance, said at ESMO. "For example, patients should not let a ChatGPT override what their physician recommended without discussing this with the physician, right?"

Read more from STAT's Katherine MacPhail and Brittany Trang.

Chronic disease

Arcturus stock plummets after CF drug disappoints

Arcturus Therapeutics' shares nosedived 55% after interim data from its Phase 2 trial of inhaled mRNA therapy ARCT-032 in cystic fibrosis showed no improvement in lung function after 28 days of treatment.

While the study's main goal is safety — which the drug met — the lack of efficacy rattled investors. The San Diego biotech highlighted reduced mucus buildup in most patients, calling it an encouraging early signal, and plans to test higher doses and longer treatment durations next year.
The analyst William Blair remains "somewhat optimistic," FierceBiotech points out, given that further dosing will be tested, but reduced the probability of success for the program from 40% to 25%.

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