Wednesday, January 31, 2018

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.

Here comes pharma's next attempt to supplant opioids

They're called NGF inhibitors, and in clinical trials, they've been able to tamp down chronic pain without the risk of addiction that sparked a nationwide crisis.

Thing is, they might not be safe. Back in 2010, when NGF was ringing up dollar signs in the eyes of sell-side analysts, the FDA shut down the whole show after seeing worrying signs of joint deterioration in patients who took the drug. Five years of hand-wringing and data collection ensued, and now a few major drug makers think they've figured out how to underline NGF's benefits while minimizing its risks.

“The real question is going to be, at the end of all these phase 3 studies, is the result going to be positive?” said Dr. David Weinreich, who leads clinical development at Regeneron Pharmaceuticals, which is working on an NGF drug.

Read more.

There could be trouble in biotech's enchanted forest

Yesterday, we asked readers a simple question: If — again, if — biotech's current boom goes bust, what will be the thing that sets off the inflammatory cascade?

The largest group, 36 percent, said it would be the death of a unicorn, meaning, we assume, some quote-unquote startup the size of Moderna Therapeutics or Intarcia proving to be a great big flop and thus undermining the whole virtuous cycle that has put biotech VCs in increasingly fancy cars since 2013. (Here's a story about that.)

Another 28 percent said doom will come after Incyte's much-anticipated immuno-oncology trial turns out to be a dud. About 22 percent said a disappointment for Regeneron Pharmaceuticals in cholesterol will tip the scales, while just 14 percent believe President Trump is going to go full-bore on the drug industry and scare away all the generalist investors in the process.

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A third of therapies approved in 2017 were personalized



The FDA in 2017 broke a recent record for most new drug approvals — and, new data show, a big and growing chunk of those approvals were for personalized medicines. The chart above, which tracks personalized medicine approvals as a percentage of all FDA approvals, comes from a new report from the Personalized Medicine Coalition, a trade group that's partly funded by drug and diagnostics makers. (PMC is counting personalized medicines as any products with labels referencing specific biological markers identified with diagnostics.)

The approvals data reflect what you already knew: These are boom times for precision medicine. But, of course, it's another challenge entirely for drug makers to generate this kind of growth in adoption among payers and doctors.

Gene therapy pioneer has doubts on gene therapy

Dr. James Wilson, a University of Pennsylvania scientist who has become synonymous with gene therapy, has been something of a fixture in biotech news for the past week, and it hasn't been particularly good for sentiment around gene therapy.

First, he semi-mysteriously resigned from the board of Solid Biosciences — which had an interesting week — due to what the company called "emerging concerns" over the safety of the virus used to deliver gene therapy.

Then, this week, he published a paper about how a high dose of a gene therapy from AveXis killed four animals in a preclinical study, sounding not entirely confident about the future of the field.

It's not an immediate scare for AveXis, which has already dosed human beings with the therapy in question. But it does slightly shift the accepted narrative — that safety scares are a thing of the past in gene therapy — to have one of the field's pioneers suddenly seem so wary.

The smartest take on AmazonBerkshireJPMorganHealthcareNewCo


Jeff, Warren, and Jamie haven't fixed America's health care system quite yet, but they sure have wreaked havoc on the investment portfolios across the country. The 10 largest insurers and health care services companies lost a collective $25 billion in market value on Tuesday.

More reads

  • A biopharma innovator’s 5 wishes for Trump’s State of the Union address. (STAT)
  • Nasdaq exec 'even more bullish' on biotech IPOs as new entries soar. (Forbes)
  • Pfizer's new tax rate higher than investors hoped, shares fall. (Reuters)
  • Sunovion Parkinson’s drug heads to FDA, setting up battle with Acorda. (Xconomy)

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