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The need-to-know this morning
What biopharma leaders think of the new Trump era
My colleagues have gathered reactions from leaders across the biopharma industry about what a second Donald Trump presidency could mean. While Trump has positioned himself as business-friendly, he’s also criticized high drug costs. And in the late stages of his campaign, he embraced Robert F. Kennedy Jr., who’s signaled he wants to shake up to the FDA and other health agencies.
Many said they’d take a wait-and-see approach, but others took a more definitive stance. “I’m optimistic,” Bausch + Lomb CEO Brent Saunders said. Meanwhile, Zach Weinberg, CEO of Curie.Bio, said that while Trump’s policies on health care have been known to shift, “R.F.K. is scary and unqualified to lead anything but a trip to the supermarket.”
And one biotech CEO who spoke on the condition of anonymity was even more blunt. “If you have [RFK Jr.] in there, outlawing vaccines and doing other sorts of absolutely stupid s—, it doesn’t really matter whether the price negotiation starts at nine or 13 years,” he said, referring to disputes over when Medicare should start negotiating prices under the IRA. “Also, if you destabilize the global economy, we’re not gonna have the capital to invest in those drugs regardless. It’s just a completely asinine way to think about the future.”
AstraZeneca senior executive detained in China
AstraZeneca said that Leon Wang, the executive in charge of the company’s China subsidiary, is under detention by Chinese authorities who are investigating employees there.
The disclosure comes after AstraZeneca last week acknowledged that Wang, who also oversees international operations for the drugmaker, was being investigated along with a “small number” of employees. By then, police in China had arrested five current and former AstraZeneca employees for questioning about potential illegal activities.
Read more from STAT’s Ed Silverman.
Sarepta discontinues next-generation Duchenne drugs
From my colleague Jason Mast: Sarepta announced yesterday that it’s discontinuing its next generation of exon-skipping drugs for Duchenne muscular dystrophy over safety concerns.
In addition to its much-debated gene therapy and other approved exon-skipping drugs for particular Duchenne mutations, Sarepta had been developing follow-on treatments designed to be more targeted toward muscle cells.
Seven severe treatment-related adverse events arose in a recent clinical trial for the successor, though, and while Sarepta previously argued the data were still positive, executives said yesterday that the FDA told them they would not be able to file for accelerated approval. Sarepta opted to scrap all its experimental drugs that use the same chemistry after doing its own risk-benefit calculation, Sarepta CEO Doug Ingram said.
The setback could leave room for other competitors with targeted exon skippers to beat out Sarepta in a lucrative market, but trials are still early and they have also faced some safety concerns. “I wish these folks well,” Ingram told investors. “I think they have a high bar and a long road to get to the right place.”
Trump could target Medicaid for spending cuts
Trump’s return to the White House also means that the Medicaid program, which covers medical care for people with low incomes, could face cuts.
Republicans are likely to go looking for some major cuts to spending to help fund a plan to extend Trump’s 2017 tax cuts. The president-elect has already promised to not touch Medicare or Social Security, and has called for increasing the defense budget. Medicaid is just about the only large government program left, and Trump has made no similar promise to preserve it.
However, any plans for cuts would likely get pushback from industry constituents such as private insurers, who are are increasingly running Medicaid programs, and rural hospitals, which rely on Medicaid to keep their doors open.
Read more from STAT’s John Wilkerson.
The companies using AI to streamline clinical trials
Many pharma companies are developing internal approaches of using AI to help design and run clinical trials, but there’s also a growing crop of companies filling gaps in partnership with drugmakers. My colleague Katie Palmer has compiled a list of key companies to watch in the space.
The ultimate manifestation of AI in trials would be total simulation: no human participants, just predicted clinical and safety outcomes based on huge databases. Unsurprisingly, we’re not even close to that yet.
But streamlining processes like trial site selection, as well as predicting trial outcomes to make choices about study design, can make a meaningful difference in timelines.