May 29, 2025

The need-to-know this morning

Intellia Therapeutics disclosed a case of severe liver toxicity in a patient treated with its experimental, CRISPR-based therapy for ATTR-CM, a progressive heart condition. The patient did not experience any symptoms and measures of liver toxicity were resolving, the company said. Still, Intellia's stock price fell on concerns that a risk of liver toxicity could hamper enrollment in its ongoing Phase 3 study, or threaten the viability of the one-time treatment given the approval of other, effective drugs for the disease.
Keros Therapeutics, a biotech currently engaged in a public fight with one of its largest shareholders over the future direction of the company, said it was shelving an experimental drug for a lung condition and reducing its workforce by 45% to reduce operating costs.
Merck and Daiichi Sankyo withdrew their FDA marketing application for patritumab deruxtecan seeking accelerated approval as a potential treatment for EGFR-mutated non-small cell lung cancer. The decision to pull the filing was made after the drug failed to prolong survival in a Phase 3 study.

vaccines

HHS drops Moderna pandemic flu vaccine deal

In a politically charged decision that experts say undercuts pandemic preparedness, HHS has canceled a nearly $600 million contract with Moderna to develop mRNA-based vaccines for potential pandemic flu strains like H5N1, citing safety concerns despite a strong immune response in early trials.

"In a rapidly expanding pandemic, time matters. … The sooner the population is protected from a lethal virus the more lives that will be saved. Of its many features, what mRNA technology provided most is speed," said Bruce Gellin, a former director of the National Vaccine Program Office in the Bush and Obama administrations.

Moderna, reeling from its plummeting stock and shifting market prospects, says it will seek other paths forward.

tigit

Post-TIGIT flop, iTeos Therapeutics is shuttering

Immuno-oncology player iTeos Therapeutics is shutting down, just weeks after scrapping its TIGIT program with GSK due to lackluster clinical data. The company ended the first quarter of 2025 with $624.3 million in hand and several assets, including two early-stage oncology programs and a preclinical obesity candidate. Its plan now is to return as much capital as possible to shareholders. Investors are pleased: Its stock jumped 28% on the news yesterday.

TIGIT as an immunotherapy target had been hyped up for years, but it's been largely disappointing. BeiGene, for instance, abandoned its own TIGIT-targeting lung cancer drug ociperlimab just last month. Roche faced a similar TIGIT setback late last year.

exclusive

Vivodyne raises $40M to reinvent preclinical testing

Nearly two decades after helping pioneer organs-on-chips, bioengineer Dan Huh is back with a more ambitious vision: biologically complex, AI-analyzed "lab-grown biopsies" meant to change the framework of drug development. His startup Vivodyne has just raised $40 million to expand its robotic tissue engineering platform, including a new 23,000-square-foot lab in San Francisco, STAT's Allison DeAngelis reports.

Backed by Khosla Ventures, among others, and collaborating with at least six unnamed pharma companies, Vivodyne's tech arrives at a pivotal moment — weeks after the FDA and NIH signaled intent to reduce reliance on animal testing.

fda

Former FDA chief: Loss of generics team jeopardizes progress

Recent layoffs at the FDA threaten to undo a cornerstone of the Trump administration's efforts at improving drug affordability: the fast-tracking of generics. In a new First Opinion in STAT, former FDA commissioner Scott Gottlieb points out that the agency recently dismissed 13 veteran staff from its Division of Policy Development — experts who wrote the scientific playbooks that allowed lower-cost versions of sophisticated drugs like Ozempic and COPD inhalers to reach the market.

"The FDA's political leaders reconsidered cuts initially made elsewhere within the agency's drug programs, even rehiring some key scientists," he writes. "However, the regulatory policy team in the generic drug group was dismissed wholesale, with no plans to restore this uniquely specialized team."

While some generics giants can go it alone, most smaller firms rely on this guidance to compete, he writes. The move, framed as a cost-cutting measure, is short-sighted and could undo years of progress, Gottlieb says.

clinical trials

More biopharma trials are happening only in China

A global oncology report from IQVIA outlines, via cancer trial stats, how China's role in biopharma is ever-increasing. There are fewer studies being conducted across several countries, while more are being conducted in China — which could spell a downtick in trial diversity. In 2015, 37% of trials were multi-country studies, compared to just 20% in 2024. Meanwhile, China-specific studies rose from around 5% in 2015 to around 40% in 2024. Behold:

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