December 8, 2025

The need-to-know this morning

Agios Pharma said the FDA has delayed a decision on the expanded approval of its drug Pyrukynd to treat thalassemia, an inherited blood disorder. The agency missed its Dec. 7 review deadline.
Dyne Therapeutics said its experimental drug for Duchenne muscular dystrophy — a potential next-generation version of Sarepta Therapeutics' much-debated Exondys 51 — hit the mark in a late-stage study, setting up a potential approval.
Cogent Biosciences said a Phase 3 study of bezuclastinib in advanced systemic mastocytosis achieved its primary goal.
Structure Therapeutics reported results from mid-stage study of its oral GLP-1 drug candidate.

obesity

Early update shows Wave's RNA drug cut visceral fat

From my colleague Elaine Chen: Wave Life Sciences said today that interim data show its investigational siRNA therapy cut visceral fat in a small Phase 1 study, though it's not yet clear how meaningful the reduction is.

Three months after taking a low, single dose of the drug, patients experienced a 9.4% reduction in visceral fat, the most harmful type of fat that's stored deep around the organs, while those on placebo experienced a 0.2% reduction.

Treated patients also experienced a 3.2% increase in lean mass, while those on placebo also experienced an increase of 2.3%.

When looking at overall weight loss, treated patients experienced just a 0.9% placebo-adjusted decrease. Wave executives said in an interview that the rate of overall weight loss will be slower than the rate seen with GLP-1s, as patients on GLP-1s lose both fat and lean mass.

Wave will report six-month follow up data from this cohort, as well as results from cohorts taking higher doses, next year. The company aims to explore the drug as a treatment on its own, as an add-on to GLP-1 treatment, or as a maintenance therapy after patients start on GLP-1s.

While GLP-1s like Wegovy and Zepbound are highly popular, people have to take them chronically every week to maintain their weight loss. Wave is testing whether its therapy could lead to durable weight loss when dosed twice or potentially even once a year.

Some doctors have been concerned that patients on GLP-1s may lose too much lean mass on the drugs, though this is a highly debated issue. Wave's drug aims to silence INHBE mRNA, which regulates fat. The idea is that the therapy blocks fat storage, working differently than GLP-1 drugs, which curb people's appetite.

Wave said there were no discontinuations or serious adverse events. Side effects included mild irritation at the injection site, executives said.

ash 2025

Experimental Incyte myelofibrosis drug shows early promise

Incyte reported early but encouraging data at this year's American Society of Hematology meeting for its experimental drug INCA033989, STAT's Adam Feuerstein writes. The drug showed spleen volume reductions in 33% of evaluable patients. The drug, which targets the calcium-binding protein calreticulin, improved symptoms in 39% of patients after 24 weeks.

This is a meaningful finding in a population largely no longer benefiting from Jakafi, a blockbuster JAK inhibitor also made by Incyte that has a patent cliff approaching.

The pill, designed for the roughly 35% of patients whose cancer is driven by calreticulin mutations, offered a clean safety profile with mostly mild liver enzyme bumps and anemia, which supports higher dosing and a path to Phase 3 trials next year. Although the findings are early, they suggest that the treatment could ultimately serve as a monotherapy.

ash 2025

Gilead's next CAR-T shows clean, strong data

Facing slumping sales of its current CAR-T portfolio, Gilead is banking on anito-cel — and new data from ASH suggest the bet may pay off, STAT's Adam Feuerstein writes. In a pivotal-stage trial of 117 multiple myeloma patients, the therapy delivered a 96% response rate and 74% complete remissions, with 67% progression-free at 18 months and 88% still alive.

Crucially, investigators reported no delayed neurotoxicity or enterocolitis, safety issues that have dogged other products in the class.

"What we're seeing here, with this one time treatment, is that we're able to achieve really promising efficacy in patients who have high-risk disease — and we're able to do that without the risk of delayed or cumulative neurotoxicity," Cindy Perettie, executive vice president for Kite Pharma, the cell therapy division of Gilead, told STAT.

protein degraders

Kymera's oral eczema drug shows Dupixent-esque promise

Kymera Therapeutics reported strong early data for KT-621, a pill designed to challenge the blockbuster biologic Dupixent. The small molecule, a protein degrader, showed a 63% average reduction in eczema severity after four weeks in its 22-patient Phase 1 trial — slightly better than analysts had forecast, STAT's Jonathan Wosen writes. The drug targets the STAT6 protein transcription factor.

And with near-complete STAT6 degradation in skin and blood, Kymera is positioning KT-621 as the first oral alternative capable of mimicking biologics' efficacy. The pill also damped biomarkers tied to asthma and allergic disease, including sharp drops in TARC and exhaled nitric oxide, and was generally well tolerated with no serious safety issues.

The next proving ground will be larger, placebo-controlled studies in eczema and asthma, with pivotal data expected in 2027 — a timeline analysts say could make or break the company's trajectory.

vaccines

ACIP's revolt against science alarms experts

In a chaotic, combative two-day meeting, a newly remade ACIP broke with decades of scientific consensus by reversing a recommendation that infants born to hepatitis B–negative mothers receive the vaccine at birth — despite no new evidence suggesting any risk.

The move, driven by members who echoed long-debunked anti-vaccine talking points and at other points amplified unsubstantiated worries about aluminum and multiple shots, set the stage for a sweeping review of the entire childhood vaccine schedule, just as President Trump ordered federal officials to reconsider it.

Infectious-disease leaders warned the committee is now operating outside evidence-based norms, risking real harm to children and further eroding public trust in immunization — even as frontline physicians say they'll continue following established science.

"That damage has already been happening," Sean O'Leary, a doctor who chairs the American Academy of Pediatrics' Committee on Infectious Diseases, told reporters last Tuesday, ahead of the ACIP meeting. "I think it's only going to happen more."

liquid biopsy

Freenome's $330 million SPAC to fund cancer screens

Freenome is heading to the public markets via a $330 million SPAC merger with Perceptive Capital Solutions Corp. The deal is backed by major biotech investors and designed to accelerate its push into blood-based cancer screening. The Bay Area company plans to use the funds to launch multiple single-cancer tests in 2026 and advance a multi-cancer early detection assay powered by AI, STAT's Jonathan Wosen writes.

The move signals Freenome's belief that public capital will be essential as competition intensifies among liquid biopsy players like Grail, Guardant Health, Natera, and Exact Sciences, even as questions linger about how much multi-cancer tests will ultimately improve patient outcomes.

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