September 24, 2026

The need-to-know this morning:

Acadia Pharmaceuticals said the higher of two doses of an experimental treatment for Alzheimer's disease psychosis reduced symptoms compared to a placebo, but the result missed statistical significance by a small margin. Based on the mixed results from the Phase 2 study, Acadia said it will continue to enroll participants into two Phase 3 studies, but with the higher dose of the drug only.

cancer

FDA advisory panel backs Grail's cancer blood test

An FDA advisory panel backed Grail’s Galleri blood test yesterday, finding that its benefits outweigh its risks despite lingering questions about how well the test actually detects cancer. The test is designed to find multiple cancers, STAT’s Matthew Herper writes — including some for which other screening tests do not exist.

But FDA officials noted that a third to half of positive results are false positives, and that Galleri may miss two-thirds of cancers. Panelists also grappled with a large U.K. study that failed to show Galleri led to significantly earlier cancer diagnoses, arguing the test shouldn’t be marketed as an “early” detection test. The FDA doesn’t have to follow the panel’s recommendation, though it often does. An approval could pave the way for broader insurance coverage of Galleri.

“We can't run away from the fact that there was a randomized study that then didn't show a benefit or the reduction in stage that they wanted to,” one panelist said.

“I see this as absolutely not reliably an early detection test,” said another.

drug pricing

Some states not yet sold on Medicaid initiative, despite Trump claims

The Trump administration declared Friday that all 50 states had agreed to participate in its GENEROUS drug pricing program. But officials in several states told STAT's Daniel Payne they haven’t actually decided whether to take part.

“Applying and signing a participation agreement does not commit the state to actually participate in the GENEROUS model in terms of entering into rebate agreements with manufacturers,” the spokesperson for California’s health department said.

States including California, Idaho, New York, and New Mexico said they signed non-binding agreements or applied so they could evaluate confidential pricing information. Others are still determining whether the program’s rebates beat deals they already receive from drugmakers. CMS itself later said only 40 states had signed participation agreements, though all 50 had applied.

“It is too soon for states to know if the MFN rates are more favorable than our current net costs,” a spokesperson for the Minnesota Department of Human Services said.

washington

Trump's FDA nominee set to face the Senate

Following a rapid ascent through medicine and Republican health policy, Heidi Overton has become the youngest-ever nominee to be FDA chief. Her Senate confirmation hearing is today. Notably, in her opening statement, which was obtained by STAT, she said: "We can protect America’s global leadership in biotechnology, but we have to act fast."

Overton grew up in a small town in New Mexico but trained at Johns Hopkins in preventative medicine before serving as a White House fellow during Trump’s first term, STAT’s Margaret Manto writes. She worked at the America First Policy Institute, then returned to the White House in 2025, where she became closely involved with the administration’s MAHA agenda.

People who have worked with Overton describe her as unusually calm, humble, and effective behind the scenes. But she has little managerial experience and such close ties to the White House that there’s some concern in her ability to steer an FDA that lost roughly 20% of its workforce last year.

Read more. And stay tuned here for live-blogging of Overton's confirmation hearing.

cell therapy

Kyverna's cell therapy shows lasting autoimmune benefit

From STAT’s Allison DeAngelis: Kyverna Therapeutics is one step closer to launching the first cell therapy for an autoimmune disease, with new data in hand showing many patients remained off of other therapies a year after treatment. In fact, patients continued to improve months after the single-shot treatment.

The startup knew its treatment for stiff person syndrome worked. In April, Kyverna reported it planned to seek approval after its clinical trial hit the mark. But the new data released today offers insight into just how well the therapy functioned in the long term. Patients who had received one dose of miv-cel a year prior showed a median 49% improvement in a common assessment of leg function, a timed 25-foot walk test. Those same patients had shown a 46% improvement at the 16-week mark.

The data show that the treatment’s effects can not only continue, but can also deepen over time, Kyverna CEO Warner Biddle said. His company plans to submit the data as part of a rolling drug application it started in the spring and anticipates completing it by the end of the year.

Kyverna had taken a different tactic than many other autoimmune drug developers. The company started off treating stiff person syndrome, a rare neurological disease that causes muscle stiffness and spasms, leading to a progressive launch of mobility and function. It affects an estimated 5,000 people in the United States.

In comparison, many of Kyverna’s contemporaries have chosen to start off with more prevalent autoimmune conditions with lupus or myasthenia gravis. Kyverna is also developing miv-cel as a treatment for myasthenia gravis, anticipating that it can tack that on to miv-cel’s label, should it get approved by the FDA.

The new data came through as one of the most prominent faces of stiff person syndrome — legendary singer Celine Dion — kicked off a tour in Paris, her first since announcing her diagnosis publicly. Through her foundation, Dion established an endowed chair at the University of Colorado. The inaugural chair holder, Amanda Piquet, served as the lead investigator on Kyverna’s trial.

When asked about Dion’s connection to the trial and whether she had tried miv-cel, Biddle said he could not comment on the chanteur's personal health, but was very grateful for the awareness she’s raised about the disease.

autoimmune disease

Gilead is betting big on autoimmune drug data

Gilead is talking up its years-long push into inflammation and immunology ahead of a key pressure test later this year, STAT’s Adam Feuerstein writes in his Biotech Scorecard newsletter. It’s buzzing about its Phase 2 data for emvistegrast, an oral drug for ulcerative colitis that works similarly to Takeda’s blockbuster Entyvio.

“It’s one of the three scientific areas that are our core focus,” Gilead CFO Andy Dickinson told investors at the Cantor Global Healthcare Conference earlier this month. “We’re the largest virology company. We’ve built a robust oncology business. The third leg of the stool, so to speak, is the I&I [inflammation and immunology] business that we’re growing.”

Results from the closely watched 229-patient study could help determine how aggressively Gilead builds out that “third leg.”

Thanks for reading! Until next time,