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The Readout Damian Garde & Meghana Keshavan

Amgen’s unapproved cancer drug might already have a pricing problem

Last week, investors balked at the news that Amgen had begun studying a lower dose of its cancer treatment already under FDA review, deducing that there might be a safety problem that could stand in the way of approval. But there’s a simpler explanation, one that wouldn’t change the FDA process but that could create a headache for Amgen down the road.

As STAT’s Adam Feuerstein reports, the FDA asked Amgen to run the new trial based on data suggesting that a lower dose of the drug, sotorasib, could be just as effective as a higher dose previously tested but with fewer side effects. The good news, for Amgen, is that there were no new safety concerns that could delay approval of the higher dose.

The potential downside relates to drug pricing. Amgen is expecting to win approval for a 960 mg dose of sotorasib, at which point it will set a list price. Some time after that, the new study on a lower, 240 mg dose of sotorasib will read out. If it turns out that 240 mg works just as well, one bottle of sotorasib would last four months instead of one month — cutting the effective price of the medicine by 75%.

Read more.

This U.S. biotech is helping run an unregulated anti-aging gene therapy trial in Mexico

Last year, six patients with dementia traveled to Mexico to be injected with an investigational anti-aging gene therapy, part of an unregulated clinical trial run by a Seattle company with a dim view of scientific prudence.

As STAT’s Megan Moletni reports, the startup BioViva and its partners recruited patients to Mexico City with the promise of a one-time treatment that could extend their lives and treat Alzheimer’s disease. Missing were the disclosures about trial design, safeguards, and scientific details that accompany regulated clinical trials, which one bioethicist said was misleading and manipulative.

Beyond the safety risks of the study, the project raises the specter of an overseas medical tourism industry that recruits desperate patients into similarly unregulated trials, providing unproven genetic medicines that could have permanent side effects.

Read more.

How do microbiome treatments actually work?

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Scientists have long understood that the trillions of bacteria making up the human microbiome have sizable impacts on health and huge potential to treat disease. And the most practical way to harness that promise is the fecal transplant, which involves taking stool from a healthy person and putting it into a sick person. But how does tinkering with the microbiome actually work?

In the latest episode of “The Facts, STAT,” our video explainer series, Kate Sheridan explains how centuries of medical practice evolved into the modern fecal transplant. The basic idea is that the microbiome is an ecosystem, with bacterial checks and balances that keep everything running smoothly. When something goes awry — like if a person gets infected with C. difficile — doctors can restore that harmony with a fecal transplant, reintroducing helpful bacteria that can bring peace to the microbiome.

But the fecal transplant could become a relic of medical history. Biotech companies are pressing forward with treatments designed to replicate their beneficial effects without the need for donated feces, and some stool banks are already preparing for obsolescence.

Watch the video.

Emergent Bio’s story has evolved

About a month ago, amid reports that Emergent Biosolutions botched the production of Johnson & Johnson’s Covid-19 vaccine by accidentally intermingling it with the AstraZeneca one, CEO Bob Kramer was clear in his denial: “It wasn’t the case where an ingredient from one vaccine contaminated or impacted the other,” he told CNBC on April 1.

But last week, on Emergent’s earnings call, Kramer said that actually, yeah, an ingredient from one vaccine went ahead and contaminated or impacted the other. Asked to explain the change in narrative, Emergent told CNBC that Kramer “simply misspoke on your program.”

This does not help Emergent solve its credibility crisis, which analysts expect will have a negative effect on its contract manufacturing business in the years to come. It also focuses more unwelcome attention on Kramer, whose lucratively timed stock trades caught the eye of Sen. Elizabeth Warren. Kramer sold about $10 million of Emergent stock before the company’s production problems would slash its valuation by half. Those trades were set in motion back in November, but Warren is calling on the SEC to investigate what Kramer knew when, guaranteeing more unwanted headlines on the horizon for Emergent.

More reads

  • U.S. government has invested $6 billion in Moderna’s Covid-19 vaccine. (Boston Globe)
  • White House is split over how to vaccinate the world. (Washington Post)
  • EU aims to cut foreign reliance on pharma materials. (Reuters)
  • HHS to prioritize newborn screening programs’ pipette tip orders. (STAT)

Thanks for reading! Until tomorrow,

Monday, May 3, 2021

STAT

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