March 14, 2024

The need-to-know this morning

AstraZeneca said it would purchase the French firm Amolyt Pharma in a deal worth up to $1 billion, picking up a rare disease drug in a pivotal trial.
The U.K.'s cost-effectiveness watchdog said it is not yet recommending Vertex Pharmaceutical's CRISPR-based therapy for sickle cell disease, saying in draft guidance it has questions about the medicine's lasting benefits for patients.

Oncology

Small studies offer hope for treating brain cancer

Data from a handful of patients with a deadly, incurable form of brain cancer suggest cell therapy could become a powerful treatment.

As STAT's Matthew Herper and Jason Mast report, one patient treated with a CAR-T therapy in a three-person trial saw his tumor shrink 18.5% in just two days and then decrease further over the next two months until it was 60% smaller than when treatment began. That's a dramatic result in glioblastoma multiforme, or GBM, an aggressive brain cancer that has eluded effective treatments for decades.

The data, published in the New England Journal of Medicine yesterday, are too scant and too early to establish CAR-T as a treatment for GBM. But the results point to an exciting path forward, researchers said. "That's really sort of unprecedented," said Marcella Maus, a Massachusetts General Hospital oncologist and senior author of the paper. "As far as I know, there's really not any other drugs that do anything like that in glioblastoma."

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Liquid biopsy

Guardant's blood test detects colon cancer — with a catch

Guardant Health, among the leaders in the nascent field of liquid biopsy, said its colorectal cancer screening test succeeded in a large study. But the product, which relies on only a blood draw, was considerably less effective at detecting early-stage disease and precancerous polyps.

As STAT's Angus Chen reports, Guardant's study, which recruited nearly 8,000 patients, detected colorectal cancer at stages 2 or later with 100% sensitivity. The test's sensitivity for stage 1 colorectal cancer was 65%, and the number for advanced precancerous lesions was 13.5%.

The results, published in the New England Journal of Medicine, are likely strong enough to merit FDA approval, a major hurdle to insurance coverage. But whether it the data win over clinicians remains to be seen.

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Biotech

Argenx's quest to build a blockbuster

The Belgian biotech firm Argenx made more than $1 billion last year from Vyvgart, a treatment approved for the autoimmune disorder myasthenia gravis. Now the company is nearing an inflection point that would affirm its drug as one of those vaunted pipelines in a product.

As STAT's Adam Feuerstein reports, Argenx is awaiting an FDA approval that would expand Vyvgart's use to include chronic inflammatory demyelinating polyneuropathy, or CIDP, an autoimmune nerve disorder that causes people to lose feeling and muscle strength in the arms and legs.

"CIDP is a brutal disease," Argenx CEO Tim Van Hauwermeiren said. "I was looking at some data the other day — we had quite a few people coming onto our study in wheelchairs. The bulk of them left the wheelchair in six weeks. That is transformational."

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FDA

Is CAR-T safe for early-stage patients?

FDA reviewers have some concerns, according to documents related to a pair of cancer medicines from Johnson & Johnson and Bristol Myers Squibb.

Each is seeking to expand its approval to include patients with early-stage multiple myeloma, a form of blood cancer. But it's unclear, according to FDA reviewers, whether the benefits of CAR-T treatment outweigh its risks. In trials of both treatments, more patients treated with CAR-T died in the early months of study than those receiving standard of care. That trend reversed over the course of each trial, but the signal was enough to raise alarm at the FDA.

The documents are a prelude to Friday's meeting of independent agency advisers, who will discuss the data and hear out each company's case before making a recommendation on expanding the medicines' approval. The outcome could have significant implications for the business of CAR-T, which depends on broadening the use of treatment to include patients with earlier stage forms of cancer.

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