GSK to buy RAPT Therapeutics, maker of food allergy treatment, in $2.2B deal

From STAT's Andrew Joseph: GSK said this morning that it would buy RAPT Therapeutics in a deal worth $2.2 billion, picking up an experimental medicine for food allergies. GSK is paying $58 per share for the South San Francisco biotech, a roughly 65% premium on Rapt’s closing price on Friday.

The centerpiece of the deal is ozureprubart, an antibody designed to protect people from food allergens. The medicine is in a Phase 2 trial, with data expected in 2027. It targets an immune molecule known as IgE that is involved in allergic reactions.

Current anti-IgE prophylactic treatments for food allergies have to be injected every two to four weeks, while ozureprubart could be given every 12 weeks.

Inflammation

Corvus pill shows promise in early-stage eczema trial

From STAT's Adam Feuerstein: Corvus Pharmaceuticals reported updated results from an early-stage study of an oral treatment for eczema that has the potential to be more effective than currently approved drugs, including the blockbuster therapy Dupixent.

After eight weeks, 75% of participants treated with the Corvus pill, called soquelitinib, achieved at least a 75% improvement in the extent and severity of skin lesions — an efficacy metric known as EASI 75 — compared to 20% of participants offered a placebo, the company said.

Wall Street analysts were expecting soquelitinib to show improvements in eczema skin and severity scores comparable to Dupixent, the most widely prescribed injectable treatment for eczema, which is marketed by Sanofi and Regeneron Pharmaceuticals. The soquelitinib results were better, albeit from a smaller trial than the trials used by Regeneron and Sanofi to secure Dupixent’s approval.

Cancer

Benefit from Moderna’s skin cancer immunotherapy endures

From STAT's Matthew Herper: Moderna and Merck said that adding the cancer immunotherapy intismeran autogene to Keytruda slowed the rate of relapse or death in patients with metastatic melanoma by 51% after five years.

That is exactly the benefit that was seen in an analysis of the treatment, an mRNA therapy that primes patients’ immune systems to attack tumors, at three years in the same 157-patient clinical trial. The original results of the study were published in the Lancet in 2024. Volunteers were given either the new therapy and Keytruda alone after surgery to remove their cancer.

The durable benefit relative to Keytruda indicates that patients are continuing to benefit. Moderna did not respond to requests to clarify the absolute rate at which patients on the therapy are going without disease recurrence or death. Merck and Moderna have fully enrolled a Phase 3 study of the treatment in melanoma and are enrolling separate Phase 3 studies in lung cancer.

REGULATION

FDA bets on flexibility for riskier gene therapies

The FDA is loosening oversight of cell and gene therapies in the name of “flexibility,” promising faster development but also introducing real uncertainty for patients, sponsors, and regulators alike, STAT contributor Paul Knoepfler writes in a new edition "Lab Dish."

The new approach lowers early manufacturing and data requirements and allows more mid-trial changes, which could help accelerate desperately needed therapies, especially for rare diseases. But, Paul notes, it also raises the odds of noisier data, uneven efficacy, late-emerging safety issues, and even post-approval reversals.

There’s worry that the new FDA policy is vague and legally shaky. And it was rolled out via a brief announcement rather than formal guidance, leaving reviewers to walk a tightrope to speed things up without compromising safety and the agency's authority.

rare disease

U.K. study testing if bespoke drugs make regulatory and financial sense

From my colleague Jason Mast: Nearly eight years ago, a Colorado mom and a Boston researcher announced the creation of the first-ever custom-made drug crafted to patch the unique mutation in a single young girl facing a fatal neurodegenerative disease. The announcement heralded a new era of individualized medicine for patients with rare diseases but also raised thorny questions about how such drugs, which can require sprawling teams and millions of dollars to create, could be scaled to more than a handful of patients.

Now, for the first time, researchers in the U.K. are launching a trial to see if such individualized drugs can be formally approved and paid for. The study, first reported by The Economist, will use a standardized method to treat 11 patients with fatal and life-threatening neurological conditions, in hopes of then obtaining a “process approval” to treat more patients with the same process. The first patient, a teenager with Niemann-Pick disease type C, was treated last week.

The study is supported by EveryONE medicines, a startup co-founded and, as of Jan. 5, run by Julia Vitarello, the Colorado mom. Although her daughter Mila died in 2021 at age 10 — the treatment came too late for her — Vitarello has remained a tireless advocate for individualized therapy.

The trial will test antisense oligonucleotides, short strands of DNA-like molecules that can be used to modulate a gene’s expression. But efforts in the U.S. are also underway to obtain the same type of “process approval” with gene editing. Officials in both countries have recently opened the door for these studies by laying out new regulatory pathways, after years of advocacy by Vitarello and others.

"My mission has been to make individualized medicines accessible to patients at scale to help solve the global health crisis of genetic disease. Milasen and the treatments that followed proved that science isn’t the limiting factor — it’s the system that was never designed for this paradigm," Vitarello told STAT. "EveryONE Medicine’s treatment of Patient A in London last week represents the beginning of a major shift toward Process Approval with the expectation of reimbursement."

legal

Supreme Court takes up 'skinny label' showdown

The Supreme Court has agreed to hear a high-stakes dispute that could determine the future of “skinny labeling,” a long-standing strategy that lets generic drugmakers launch cheaper medicines for some uses while carving out still-patented ones.

At issue is a fight between Amarin and Hikma Pharmaceuticals over a generic version of Vascepa, STAT’s Ed Silverman writes. An appeals court ruled that Hikma’s public statements — not its FDA-approved label — could still improperly induce doctors to prescribe the drug for a patented use.

Generic manufacturers warn the decision threatens a core mechanism of the Hatch-Waxman Act and could chill generic and biosimilar launches, driving up drug prices. Amarin argues the ruling won’t meaningfully slow generics.

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