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The Readout Damian Garde

How Vertex went from the verge to the vanguard

This week, Vertex Pharmaceuticals reached a rarefied milestone, winning approval for its fourth treatment for cystic fibrosis and giving it a medicine to treat nearly 90% of patients with the genetic disease.

The company’s success was hardly assured. Back in 2012, Vertex had leaky offices and a shaky business model, about to get pushed out of the hepatitis C market by curative treatments on the rise. As STAT’s Adam Feuerstein and Matthew Herper report, Vertex’s choice to bet the company on a precision approach to cystic fibrosis was inherently risky, as “there were like 14 things that had to go right,” as Dr. David Altshuler, the company’s chief scientific officer, put it.

Seven years later, Vertex has gone from being able to treat a handful of cystic fibrosis patients to having a product for the vast majority of them. “That’s historic,” CEO Dr. Jeffrey Leiden said. “It’s never been done in the industry before.”

Read more.

We have questions about Biogen’s resurrected Alzheimer’s drug

Two days after Biogen unhyperbolically shocked the world by bringing back a once-shelved therapy for Alzheimer’s disease, there are more than a few lingering questions.

As STAT’s Sharon Begley reports, Biogen’s rehabilitation plan has sweeping implications. Does the in-retrospect promise of the therapy mean we should revisit the graveyard of failed treatments past? And does it mean skeptical scientists should retract their eulogies for the amyloid hypothesis?

Or is this all a waste of time? More than a few experts have spotted some confounding details in Biogen’s data, and they’ll be seeking clarity when the company faces the scientific public in December. Then there’s the matter of whether the FDA will buy into all this and, most important, whether unleashing this drug on the public will actually improve people’s lives.

Read more.

If you’re keeping score in the liquid biopsy race

Guardant Health, owner of one last year's best-performing IPOs, is launching a huge trial it hopes will prove that it can diagnose colon cancer with a blood test.

As STAT’s Matthew Herper reports, Guardant’s 10,000-patient study will determine how well the company’s test compares with colonoscopy when it comes to cancer or pre-cancerous polyps. If it’s positive, Guardant will make its case to the FDA.

The news comes as a host of companies jostle to develop blood tests that might detect cancer at its earliest — and most treatable — stages. The multibillion-dollar allure of that idea has attracted companies including Freenome, Thrive, and a unicorn unsubtly named Grail.

Read more.

What if clinical trial volunteers made drug pricing demands?

Kaylene Sheran has Gorlin syndrome, a disease that leads to scores of skin tumors requiring frequent surgeries. Because the disease is rare, patients like Sheran are enthusiastically recruited by companies trying to develop treatments for Gorlin. But Sheran, presented with the opportunity to join a clinical trial that might improve her condition, wasn’t interested.

In the second part of "The Medicine Hunters," STAT’s Eric Boodman explains Sheran’s position: Unless a drug company can guarantee that its medicine will be affordable once it’s approved, she’s not interested in donating her time or her body.

Sheran’s experience is one facet in the fascinating story of the molecule that might treat Gorlin, a saga that involves a Utah forest, a biotech bubble, and decades of winding research.

Read more.

More reads

  • More new medicines had publicly supported research than you might think. (STAT Plus)
  • The big money in startups comes from investing before the IPO. (Bloomberg)
  • NIH, Gates Foundation aim to bring genetic cures to the poor. (STAT)
  • 'Insta-billionaire': A 24-year-old pharma heir who parties with celebrities became richer than Donald Trump overnight. (Business Insider)

Thanks for reading! Until tomorrow,

Thursday, October 24, 2019

STAT

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