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The Readout Damian Garde

Meet your 2019 best and worst biopharma CEOs

Every year, STAT’s Adam Feuerstein invites readers to vote on the best and worst drug industry CEOs, and the results for 2019 are in.

The public’s choice for best CEO presided over an FDA approval, a pivotal success in the clinic, and the steady transformation of a groundbreaking idea into a series of actual medicines.

And, in the close race for worst, the chosen executive had a year marked with confusion, controversy, and an ever-present sense that all of this could have been avoided with a little creativity.

Read more.

Which drug defined the decade?

And how does gene therapy actually work?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, STAT's Matthew Herper joins us to discuss the most important drugs of the past decade, including treatments for hepatitis C, cystic fibrosis, and cancer. Then, we delve into the holiday tradition that is voting on the best and worst CEOs in biotech. Later, science journalist Karen Weintraub joins us to explain the crucial but little-discussed technology that makes gene therapy work.

You can listen to the episode here. To listen to future episodes, be sure to sign up on Apple Podcasts, Stitcher, Spotify, or wherever you get your podcasts.

How do you get gene therapy to children in need? Novartis is running a lottery

In the U.S., Novartis’s gene therapy for the rare and deadly spinal muscular atrophy can be had for $2.1 million for a one-time dose. But in Europe, where it’s not yet approved, there’s no commercial access. And, because there’s already an approved SMA treatment on the market there, Novartis can’t give it away under compassionate use.

So, as STAT’s Ed Silverman reports, the company has set up a lottery. Starting in 2020, Novartis will set aside as many as 100 doses of its gene therapy, called Zolgensma. Doctors can then nominate babies who would benefit from the treatment. Every two weeks, names will be drawn, and the winners will get the potentially life-saving therapy.

If that rings a little dystopian to you, you’re not alone.

“The fact that the manufacturer is lotterying it strikes me as capturing every injustice about health care globally,” wrote Dr. Steven Joffe, who heads the division of medical ethics at the University of Pennsylvania. “It just fesses up to the reality of access to care in the most honest way I’ve ever seen.”

Read more.

The biotech startup that went nearly 10 years without a VC dollar

FORMA Therapeutics is breaking with tradition. After nearly 10 years of paying the bills with payments from pharma partners, the Massachusetts company has raised $100 million in venture cash and plans to compete in a crowded field.

As STAT’s Kate Sheridan reports, FORMA’s cash comes from a syndicate that includes includes RA Capital, Cormorant Capital, and Wellington Management. The plan is to focus on FT-4202, an early stage drug meant to treat sickle cell disease by boosting an enzyme called PKR.

But FORMA’s not alone. Agios Pharmaceuticals has a similar treatment already in late-stage development, and Global Blood Therapeutics just won FDA approval for a sickle cell treatment that uses a different mechanism. Meanwhile, on the rise are gene therapies that could bring a one-time treatment for the disease.

Read more.

More reads

  • Aducanumab isn’t the simple solution to the complicated Alzheimer’s crisis. (STAT)
  • Gene therapy clinical holds take centre stage. (EP Vantage)
  • Scientists aren’t sure why identical twins differ. Armadillo quads offer an answer. (STAT)

Thanks for reading! Until next week,

Friday, December 20, 2019

STAT

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