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The Readout Damian Garde

Quick note: The Readout won't be publishing from Dec. 24 to Jan. 1. The newsletter will be back on Jan. 2!

How do you build a biotech cluster?


Officials in Massachusetts believe they’ve got a shot at growing a new Kendall Square about 130 miles west of Cambridge, in a woodsy clime better known as a vacation spot than a startup hub.

That might sound far-fetched, but, as STAT’s Kate Sheridan reports, stranger things have happened. Biotech history is littered with would-be clusters that never quite took off, but there are also counter-intuitive success stories, like the biotech hub in Madison, Wisc.

Whether the Western Mass project has a bright future is an open question, said Charles Wessner, a professor at Georgetown University who focuses on innovation policy in the U.S. But “at first look, this is a wonderful initiative,” he said.

Read more.

Pharma is spending more to get less

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The green cubes are average returns on R&D; the blue cubes are number of late-stage drugs in the pipeline; and the overall outlook is bleak. (Deloitte)

That’s according to Deloitte, whose annual look at drug industry productivity has lots of lines going in the wrong direction.

Studying 12 major pharma companies, Deloitte reported that projected returns on research dollars have tanked since 2010, from about 10 percent back then to under 2 percent in 2018. And, over the same period, the cost of getting a drug from the lab to the pharmacy has increased by 82 percent, according to Deloitte.

But things aren’t so bad if you go down an order of magnitude. Deloitte picked four mid-size biotech companies for comparison and found a much healthier ecosystem. Projected R&D returns are down since 2010, but not nearly as sharply, and peak sales expectations for the biotech group are almost as high as ever.

A glimmer of promise against a wily target


Despite its dramatic success for certain patients, oncology’s immunotherapy revolution has offered little benefit for many tumor types, perhaps none less so than glioblastoma, a cancer of the brain.

But a pair of clinical trials mark a small but rare — and therefore notable — step forward in the disease. As STAT’s Andrew Joseph reports, scientists developed personalized cancer vaccines for glioblastoma, hoping to stoke an immune response that might shrink tumors. None of the patients saw the course of their disease change, but there were signs that the immune system took notice of the malignancies.

“These two studies provide proof of concept that using this type of personalized vaccine, we can induce an immunological response,” said Dr. Neeha Zaidi, an oncologist at Johns Hopkins University’s Sidney Kimmel Comprehensive Cancer Center, who was not involved in the research. “The challenge is, how do we get a clinical response?”

Read more.

Today’s a big day for Novartis


Novartis spent $8.7 billion on the belief that a gene therapy for spinal muscular atrophy could be a life-changing product for patients. So far, the science has worked out fine. Now the question is: How much is such a revolutionary drug worth?

Later today, the Institute for Clinical & Economic Review is expected to issue a report on the cost-effectiveness of that gene therapy, called AVXS-101. The group, abbreviated ICER, doesn’t have any broad authority or regulatory power, but its conclusions have a way of becoming cudgels in negotiations between manufacturers and payers over just how much a new therapy should cost.

And AVXS-101 is no average drug. In a small trial, 100 percent of patients were alive after 24 months, a stunning result in a disease that is most often fatal within two years of birth. Novartis, which hasn’t set a price for the therapy, believes it would be cost-effective at $4 million for a one-time dose. Today, ICER will begin a long, public conversation about what the world should pay for AVXS-101, with implications for the scores of companies — and billions of dollars — invested in gene therapy.

More reads

  • Judge dismisses lawsuit accusing Craig Venter of stealing trade secrets. (STAT)
  • Glaxo and Pfizer raise the question: Should drug companies sell toothpaste and Chapstick? (Forbes)
  • The ethics of genome editing babies. (New York Times)
  • Generic drug firms seek gag order in price-fixing case. (Washington Post)

Thanks for reading! Until tomorrow,

Megan

Thursday, December 20, 2018

STAT

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