J&J's big bet on protein degraders for cancer

As we noted briefly yesterday, Johnson & Johnson is buying Halda Therapeutics for $3.05 billion. In doing so, it is wagering on a class of drugs that may be scientifically lucrative but haven't yielded any approvals yet.

The biopharma industry has invested heavily over the past decade in targeted protein degradation — molecules that can grab cancer or disease-causing proteins and dispose of them, STAT's Jason Mast writes. Halda's RIPTACs, a twist on Yale chemist Craig Crews' PROTAC degraders, work by tethering mutant cancer proteins to their normal counterparts, selectively disabling cancer cells while sparing healthy tissue.

In a small Phase 1 study, over half the treated prostate cancer patients saw PSA levels drop by at least 50%, with all evaluable tumors shrinking. J&J says the approach could overcome resistance and broaden precision oncology. In addition to prostate cancer, Halda is also exploring breast cancer and non-oncology targets.

public health

Vaccine makers have reason for alarm in proposals to reformulate shots

The Trump administration is weighing radical changes to the childhood vaccine program — including banning aluminum adjuvants and dismantling combination shots like the MMR — alarming manufacturers that warn the proposals could gut access to essential immunizations for years.

Under health secretary Robert F. Kennedy Jr., federal officials are scrutinizing ingredients long proven safe, despite industry estimates that reformulating these vaccines could take a decade and cost more than $1 billion per shot, STAT's Daniel Payne reports.

Companies say they're also being shut out of policymaking since Kennedy stacked ACIP with vaccine skeptics, raising fears of shortages, supply-chain collapse, and other challenges if the administration implements its policy ideas.

"There's no way to do it and there's no reason to do it," said Paul Offit, director of the Vaccine Education Center at the Children's Hospital of Philadelphia. "It would delay or halt vaccines for America's children … which is [Kennedy's] goal, by the way."

ACCESS

FDA-tested drugs rarely reach trial countries

A new analysis finds that most medicines tested abroad before FDA approval never become accessible in the countries where they were studied, STAT's Ed Silverman writes. The data raise fresh ethical concerns for an industry that relies heavily on global trial populations.

Of 144 drugs tested outside the U.S. between 2015 and 2018, just 24% were physically available in those countries within five years, the analysis published in JAMA Internal Medicine shows. And only 14% of countries with public authorization records had access to all the drugs tested within their borders.

Access gaps were stark: high-income countries saw 32% availability, while lower-middle-income nations saw just 13%, with African countries waiting a median of 40 months for drugs that reached Western Europe far sooner.

clinical trials

NIH trial cuts leave thousands of patients in limbo

More than 74,000 clinical trial participants have been swept up in the National Institutes of Health's sweeping funding cuts, which halted 383 studies between February and August and derailed research into cancer, heart disease, brain disorders, and especially infectious diseases like flu and Covid-19.

The abrupt shutdowns left some patients without promised trials, others without medications or monitoring, and still others contributing data that may never see daylight — a disruption that could undermine medical progress and public trust. Scientists have long warned that billions of dollars in these lost projects threaten the agency's mission and future participation in U.S. research.

But federal health officials, now realigning priorities under the Trump administration, defended the terminations. They say the axed trials "prioritized ideological agendas over scientific rigor and meaningful outcomes for the American people."

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