Tuesday, May 30, 2017

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.

This is why we biotech

Why does immunotherapy work in some patients and not in others? We're beginning to get a clearer picture, as demonstrated by the recent approval of Merck's Keytruda for solid tumor cancers with a specific genetic signature known as MMR deficiency.

The importance of learning how to better target these targeted therapies is beautifully highlighted in a Washington Post piece that shows Keytruda's impact on a 20-something with colon cancer. It's worth a full read.

Survey says: Alexion is in trouble

Last week, we asked readers a simple question: In light of all the revelations and revolutions at Alexion these days, will its share price be lower or higher one year from now?

Most of you were less than optimistic, with 62 percent of respondents betting that the rare disease juggernaut will be in worse shape in the future than it is now. Soliris, the company’s banner drug, has only a few more years of patent exclusivity, and its heir, a drug called ALXN1210, will only be blockbuster success if Alexion can keep up a commercial pace that might be unsustainable.

The future rests on the shoulders of Dr. Ludwig Hantson, Alexion’s new CEO, who began last week with farewells to four of the company’s top managers and is now poised to remake the firm to his liking. We’ll be sure to check in a year from now to see if he can prove his doubters wrong.

Insert pun about root cause for hair loss

Wherefore do our luxurious tresses fall? UCSF scientists have found a tantalizing clue in a set of immune cells called Tregs. These regulatory T cells appear to help stem cells in the skin morph into hair follicles — and when they’re removed, baldness results. At least in mice.

Could this be a new pathway for hair loss therapies? 

Read more on STAT Plus.

What should the Army do about its Sanofi problem?

The whole world wants a Zika vaccine, and Sanofi could be in a position to deliver one. But a life-saving vaccine is of little use if no one can afford it. The US Army holds patents that could be key to the vaccine, and thus it's in a position to hold Sanofi’s feet to the fire when it comes to pricing.

Should it?

STAT’s Ed Silverman unpacks the thorny issue in his latest Pharmalot View column. 

Uncle Sam wants you... for the Precision Medicine Initiative

A beta test of the Precision Medicine Initiative is finally ready to launch this week, according to Politico. The effort will be led by the University of Pittsburgh, and will start out slow — collecting samples like blood and urine from 10,000 to 15,000 volunteers. The PMI will ultimately expand to do complete genetic profiling of participants.

The private sector, meanwhile, is launching a parallel project, as Verily begins enrollment of some 10,000 volunteers for its Baseline study.

More reads

  • CRISPR gene editing can cause hundreds of unintended mutations. (
  • High-priced drugs raise out-of-pocket costs for seniors on Medicare. (Wall Street Journal)
  • What if the disease you're targeting isn't economically viable? (MedCity News)

Have a news tip or comment you want to send us?

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Thanks for reading! Until tomorrow,

Damian & Meghana

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