Wednesday, November 30, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of all the biotech news. For more, visit STAT. On Twitter: @damiangarde@megkesh, and @statnews.

Arrowhead won’t be a trillion-dollar company after all

Arrowhead Pharmaceuticals, which once measured the market for its hepatitis B treatment at more than $1 trillion, is a little more conservative these days.

Last night, the company halted all of its human trials and dumped its entire clinical pipeline, which uses synthetic RNAs to fight disease. Arrowhead’s lead drug was already on an FDA-mandated hold due to some deaths in a primate study, and the company now figures those safety concerns are too great to warrant further development.

The news sapped more than 60 percent of Arrowhead’s market value and piled onto a bad year for RNA-based drugs. Alynlam Pharmaceuticals, Ionis Pharmaceuticals, and Mirna Therapeutics have each run into serious safety issues with RNA therapies over the past few months.

Arrowhead is now regrouping around some preclinical projects that don’t use the offending technology and laying off 30 percent of its staff to conserve cash. The $1 trillion dream may be that much further away, but we’ll always have the presentation slide:

If Bob Langer were a Trump adviser...

Researchers and biotech execs looking for signs of what a President Donald Trump will mean for medical research got their first clear bellwether yesterday with the announcement of Trump's pick for HHS secretary. Representative Tom Price has said he's not willing to give the government a blank check on medical research, and he has been a vocal opponent of stem cell research.

More clarity may come when Trump turns to the NIH. It's not clear whether he'll ask Dr. Francis Collins to stay on, but he sure doesn't seem too happy with the status quo; during the campaign, Trump told a radio host that he hears "so much about the NIH, and it's terrible."

How should we read all these tea leaves? STAT's Rebecca Robbins talked to the prolific MIT inventor and entrepreneur Bob Langer, who is most definitely not a Trump adviser — but knows what he'd say if he were.

What qualities do you want to see in Trump's NIH director?
Somebody who valued basic research, and science and engineering as it relates to health. Someone who would fight to make sure there's as high a budget as possible to support these things. Somebody who's a visionary in thinking about future areas of research... The thing that's going to change the future probably hasn't been invented yet. That, to me, is why it's so important that we have as many grants for really good basic research as possible.

Should researchers be worried that Trump doesn't prioritize these values?
I think we should be worried, until we see what happens. I think we're all concerned about it.

Stem cell researchers are optimistic about 21st Century Cures

Or at least cautiously so. 

The bill, due for a vote today, contains provisions that would allow — but not require — the FDA to approve regenerative medicines on an accelerated basis, judging their merits based on biomarkers rather than hard clinical outcomes. 

As STAT’s Andrew Joseph and Sheila Kaplan report, scientists consider that a welcome retreat from the Regrow Act, proposed in the Senate earlier this year. That bill would have allowed the FDA to approve stem cell therapies based on “preliminary clinical evidence of safety and a reasonable expectation of effectiveness.” (In other words, without Phase 3 trials.)

It’s a nuanced difference, and stem cell experts are still parsing what 21st Century Cures might mean for the future of their field. But at first blush, “it strikes the right balance,” Alliance for Regenerative Medicine co-founder Michael Werner said.

Read more.

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Glow-in-the-dark nucleii for sale

you can watch a video of the cells dividing here. in real life, they're green. (allen institute)

How does a nucleus contort as a stem cell transmutes into a neuron? You can now see that happen in real time, courtesy of a new line of glow-in-the-dark induced pluripotent stem cells from the Allen Institute.

The stem cells are CRISPRed to carry a little tag that codes for fluorescent protein — so when a researcher looks at it under a microscope, the nucleus or the microtubules glow green. 

“You can actually watch how the nucleus changes from an undifferentiated stem cell to a kidney cell, or to a neuron,” said Ruwanthi Gunawardane, director of stem cells and gene editing at the Allen Institute. 

The idea is to gain a basic understanding of how a cell works, and predict its behavior, she said. She said the project should complement the new Zuckerberg-Chan initiative to draw up an intricate “cell atlas”  that maps out how cells function.

The Allen Institute plans to sell its glow-in-the-dark cell line to researchers, though it doesn't aim to make a profit — the price will cover distribution costs only.

Drugging the leaky gut to treat autism

The root cause of autism has long been enigmatic, but new research is starting to link it — and other nervous system disorders — with the gut microbiome. 

With a $19.15 million Series A, a new startup plans to push beyond probiotics, and develop a small molecule approach to treat autism and Parkinson’s disease by way of the gut.

The underlying research for Axial Biotherapeutics comes from a 2013 paper in Cell by CalTech researcher Sarkis Mazmanian. Axial CEO David Donabedian broke down some of the science in an interview with STAT:

Certain simple sugars aren’t being metabolized correctly in mouse models of autism — and kids with autism show similar gut leakiness, he said.

What's more, mice induced to display autism traits have high concentrations of certain bacterial metabolites that aren’t present in control mice. Those metabolites could potentially provide a biomarker for autism spectrum disorder, Donabedian said.

Other startups have begun to plumb the link between gut and brain, like New York-based Kallyope, which last December raised $44 million to develop drugs for obesity and other central nervous system metabolic disorders.

More reads

  • China's Innovent Biologics raised $260 million in venture funding to advance its pipeline of antibody therapies. (Press release)
  • Junk bond trader-turned-philanthropist Michael Milken has some ideas on how the federal government can speed up drug development. (Wall Street Journal)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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