December 15, 2025

The need-to-know this morning

Kyverna Therapeutics said its patient-specific CAR-T therapy improved walking ability in patients with stiff person syndrome, an autoimmune disease. The results achieved the primary goal of the pivotal clinical trial and will support a marketing submission to the FDA next year.
Argenx discontinued a Phase 3 study of Vyvgart Hytrulo in thyroid eye disease after independent data monitors conducted an interim analysis and concluded the drug would not achieve the study's primary goal.
A drug developed by Immunome reduced the risk of disease worsening or death by 84% compared to placebo in patients with advanced desmoid tumors. The results achieved the primary goal of the Phase 3 study. Immunome plans to seek FDA approval next year.
Milestone Pharmaceuticals won FDA approval for a new nasal-spray medicine to treat episodes of paroxysmal supraventricular tachycardia, a a common type of abnormal heart rhythm.

artificial intelligence

Brain-targeted gene therapy's sobering reckoning

A long-anticipated breakthrough in gene therapy for brain diseases has been jolted by tragedy after the first patient dosed with a new, brain-penetrating virus — developed by Capsida Biotherapeutics — died days later from cerebral edema. This outcome wasn't predicted by the extensive animal testing that led up to the study, STAT's Jason Mast writes.

"This is an outlier that to me is the most material event that I've seen in the field of genetic medicine for 10 years, where there was no suggestion that this was going to happen," Jim Wilson, a prominent gene therapy researcher, told a rare disease conference in late October. "And this is scary, I'm sure, for all those involved."

So the death has rattled researchers, companies, regulators, and rare disease families alike. It has forced a reckoning over whether these next-generation viruses carry previously unseen risks, how to detect them before humans are dosed, and whether the field can balance extraordinary therapeutic promise against newly exposed uncertainty, as scientists race to understand what went wrong without freezing progress for patients with devastating, otherwise untreatable neurological diseases.

cancer

Mukherjee: Oncology's next frontier is via the metabolic pathway

In an opinion piece for STAT, physician-scientist and author Siddhartha Mukherjee argues that cancer medicine has overlooked one of its most basic levers: what tumors eat. Drawing on a century of metabolic biology and newer clinical insights, he makes the case for "tumor-informed metabolism," in which diet is treated not as generic lifestyle advice but as a precision co-therapy, tailored to a tumor's biology, the drugs a patient receives, and the body's metabolic responses.

Mukherjee contends that cancers routinely evade treatment by rewiring their fuel use — such as exploiting insulin spikes caused by PI3K inhibitors — and that carefully designed, time-bound nutritional interventions could close those escape routes, deepen responses, and extend remissions. The approach, he writes, demands rigorous trials, mechanistic pairing of food and drugs, and equitable delivery, but could ultimately place metabolism alongside genetics and immunology as a first-class target in oncology.

"We will still sequence tumors. We will still give the best drugs we have. We will still sit with our patients on the hard days. But we can also do something elemental that does not subtract from strength or dignity," he writes. "We can feed the person and starve the cancer, on purpose. That is an old idea made new by the precision of our time."

sexually transmitted infection

Two oral drugs finally offer new ways to treat gonorrhea

The FDA has approved zoliflodacin, to be marketed as Nuzolvence, giving clinicians the first new, uniquely targeted treatment for gonorrhea in decades. The drug, made by Entasis Therapeutics, is now the second newly licensed option in as many days, after GSK's gepotidacin — or Blujepa — received a supplemental approval, STAT's Helen Branswell writes.

The single-dose oral drug arrives as resistance to the injectable antibiotic ceftriaxone — the last reliably effective standard treatment — continues to spread worldwide. In fact, fears were growing that gonorrhea could again become untreatable.

Nuzolvence was cleared for uncomplicated urogenital gonorrhea in patients 12 and older, without the usage restrictions placed on Blujepa, and is expected to expand access globally. While resistance could still emerge, experts say the approvals buy critical time against a fast-evolving pathogen and mark a rare bright spot in antibiotic development.

gout

Sobi bets big on next-gen gout drug

Swedish Orphan Biovitrum said it will acquire San Diego-based Arthrosi Therapeutics for up to $1.5 billion, snapping up a late-stage gout drug as it looks to strengthen its inflammation pipeline and drive growth into the next decade. The deal includes $950 million upfront and up to $550 million in milestones.

It brings Sobi control of pozdeutinurad, a once-daily oral URAT1 inhibitor now in two fully enrolled Phase 3 trials expected to read out in 2026. Arthrosi's drug has shown sustained uric acid lowering and a favorable safety profile in midstage studies, positioning it as a potential option for patients inadequately treated with existing therapies.

Just this past October, Arthrosi raiesd $153 million in a Series E. Pozdeutinurad inhibitis URAT1, a transporter that has been a target for gout drugs for decades.

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