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Wednesday, October 4, 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.

Nobel honors an imaging tool that shows proteins in 3D

The Nobel Prize in Chemistry this morning went to Jacques Dubochet of Switzerland’s University of Lausanne, Joachim Frank of Columbia University, and Richard Henderson of the MRC Laboratory of Molecular Biology in England for developing cryo-electron microscopy.

The imaging tool makes it possible to generate crisp, 3D pictures of the molecular machinery that powers life. It has become an essential technology for peering at biomolecules, capturing everything from proteins that cause antibiotic resistance to the surface of the Zika virus.

As the Nobel committee put it, the tool has proved “decisive for both the basic understanding of life’s chemistry and for the development of pharmaceuticals.” 

Read more.

Axovant's guiding guru exits (sort of)

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vivek ramaswamy's right hand man. As in, lawrence friedhoff's on the right. (stat)

The downward tumble of Axovant commenceth: Per a new SEC filing, Dr. Lawrence Friedhoff will no longer continue on as its chief development officer. He's sticking around, however, in his role as Roivant R&D chief.

Friedhoff is a bit of a celebrity in the Alzheimer’s circuit: He led the development of the now-generic Aricept. The pharma lifer joined Axovant CEO Vivek Ramaswamy back in 2014, as they played with the idea of rejiggering a shelved GSK drug, interpidine, for Alzheimer’s disease. 

“When you’ve spent 30-some odd years doing this, you see very dramatically what works and what doesn’t,” Friedhoff told STAT last year. “And I’m rarely wrong.”

A few hundred million dollars and a flopped Phase 3 trial later, Friedhoff was proven — well, wrong.

He'll be hoping that's an anomaly as he helps guide the other drugs being developed under Roivant's umbrella — for conditions ranging from female infertility to atopic dermatitis — through clinical trials. 

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Amicus delivers strong Pompe disease data 

John Crowley’s family is best known for a cameo during President Trump’s address to Congress earlier this year. Trump promised that slashing regulations at the FDA would bring forth more medical miracles — such as the drug taken by Crowley’s daughter, Megan, a college student with Pompe disease.

Now, Crowley's back in the spotlight, as his Amicus Therapeutics reports data from a mid-stage study of a brand-new therapy for Pompe disease. The interim results suggest it improves both muscle and lung function in patients.

The Amicus therapy has two working parts: An infused, replacement enzyme meant to penetrate muscle tissue, and a “chaperone” pill that binds to the replacement enzyme while in the bloodstream, keeping it stable and preserving its activity.

Read more on STAT Plus.

Fido's the new guinea pig for cancer

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must love dogs. (stat)

Jerry Seinfeld famously joshed that if aliens observed us from above, the scatalogical cleanup ritual between humans and dogs would convince them that the four-legged species is the alpha.

That’s reflected, too, in the way we treat our pets: Many owners will go to great medical lengths to save their furry companions. Now, veterinarians — frustrated at the lack of cancer treatment options for cats and dogs — are taking that trend to the next level. They're conducting research on pets to study the efficacy of stem cell infusions, CAR-T therapies, and other such cutting-edge medicines.

Countless drugs, of course, are tested in animals — but rarely in pets. These veterinarians are hoping that testing compelling new drugs on our beagles and retrievers might help ultimately shape human treatments. 

“For a long time, we’ve looked at humans to see how to treat dogs,” one veterinary radiation oncologist told STAT. “We’re starting to do a little bit of the reverse now.”

Read more.

A CRISPR caution from Nature's editors

Shoukrat Mitalipov raised a ton of hype — and eyebrows — this past August when his lab announced it had successfully edited away a disease-causing gene in a viable human embryo. But another aspect of his research — the finding that maternal genes self-duplicated and replaced the portion of DNA that had been CRISPRed out — has drawn a whole lot of ire

That work, published in Nature, this week earned an editor’s addendum. It goes: “Readers are alerted that some of the conclusions of this paper are subject to critiques that are being considered by editors. Some of these critiques have been publicly deposited in preprint form. A further editorial response will follow the resolution of these issues.”

More reads

  • Scott Gottlieb rocketed to the top of FDA. He may keep rising. (STAT)
  • Gene-edited skin could be its own blood-sugar sensor. (MIT Tech Review)
  • Arie Belldegrun's Kite Pharma farewell. (Endpoints)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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