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The Readout

Once more into the amyloid hypothesis
 

Everyone's favorite controversial explanation for Alzheimer's disease is back in the news again, as Biogen and Eisai reported a sliver of positivity from a previously failed Phase 2 trial.

The drug is BAN2401, and it targets the free-flowing little protofibrils that eventually curl up and become amyloid plaques in the brain. The study, whose initial failure was disclosed in December, turned up a bit of good news on further examination: One group of patients getting the highest tested dose saw their cognition and amyloid scans improve compared with placebo.

But there are, as you might imagine, some outstanding questions.

Read more.

It’s hot and the year’s half over
 

Why is everyone so down on biotech's household names? How do you become a day trader? And if a stock gets downgraded in an analyst note, should anyone care?

We discuss all that and more in the latest episode of “The Readout LOUD,” STAT’s biotech podcast. Tune in for a conversation with biotech investor and philanthropist Sheff Station about how he got into biotech investing. Then we'll take a look back at the biggest stories and surprises from the first half of 2018, followed by a look ahead to the events that will shape the rest of the year for the drug industry. And then we'll talk about some soul-searching analyst research that questions the very utility of analyst research.

You can listen to it here. And you can expect another episode next Thursday evening — and every Thursday evening — so be sure to sign up on iTunes, Stitcher, Google Play, or wherever you get your podcasts.

When is it OK to reject a drug?
 

You probably have better things to do than relitigate the FDA’s 2016 decision to approve Sarepta Therapeutics’ treatment for Duchenne muscular dystrophy. But we don’t and neither does the Wall Street Journal editorial board, which made a worth-considering point in a new piece.

Last month, Sarepta presented early but promising data from a gene therapy that could benefit DMD patients far beyond the oft-debated effects of its controversial first drug, called Exondys 51. The FDA was pilloried for approving Exondys 51 on scant evidence, but had the agency rejected it, “Sarepta would have lacked the resources to continue its research and testing to treat Duchenne and develop what may be an even better drug,” the Journal argues, referring to the gene therapy.

On its face, that’s true. A rejection back in 2016 could have sent Sarepta to the brink of insolvency. (FDA brass took the company’s cash balance and stock price into account at the time.) 

But the editorial ignores a pretty key fact: Sarepta didn’t invent that gene therapy; Nationwide Children’s Hospital did. So while an FDA rejection could perhaps have put Sarepta out of business before it could license that gene therapy, there’s no reason to assume some other company wouldn’t have come along and picked it up instead.

Do you know what’s in that DNA you bought?
 

Because we live in the future, you can just up and buy customized genetic material and use it to produce perfume, cheese, or fertilizer. But because so much DNA remains mysterious despite decades of dogged study, there remains a slight risk that your dream of a microscopic fromagerie will turn into a bioterror nightmare.

Which is why Ginkgo Bioworks, a company that specializes in genetic engineering, is training computer programs to ferret out potential biosecurity threats. As STAT’s Justin Chen reports, biotech companies don’t sell DNA with pathogenic ties, but because there are so many genes with murky functions, management is concerned that they might unwittingly put some dangerous strands out into the world.

To stop this, Gingko is using the same technology that helps it predict which synthetic sequences will have beneficial effects to spot the ones that might cause unintended consequences.

“We’ve built up these tools to say, ‘Hey, can I predict what that gene in nature that no one has ever tested before is going to do?‘” Ginkgo CEO Jason Kelly said. “It just so happens that’s a tool that can be reapplied on the biosecurity front [to ask], “Hey, is this gene likely to do something that we don’t want?’”

Read more.

You’re skeptical of Hong Kong’s biotech exuberance
 

Now that the Hong Kong Stock Exchange is liberalizing its rules to allow loss-making biotech companies to go public there, a steady stream of hopefuls has darkened its doorstep. Yesterday, we asked readers a simple question: A year from now, will the first generation of Hong Kong-listed biotech companies trade above or below its IPO valuations?

Most people were doubtful. About 62 percent of respondents said they’ll trade down, while the remaining 38 percent figured things will brighten up after 12 months.

But entrepreneurs are more sanguine. Since we asked that question, two more biotech companies — and the first ones based in the U.S. — have applied to go public in Hong Kong. The first, Stealth BioTherapeutics, is targeting rare diseases, while the second, AOBiome, is working in dermatology.

More reads

  • Vertex and U.K. health officials reach an impasse over pricey cystic fibrosis drug. (STAT Plus)
  • Japan's Takeda to sell Osaka headquarters, could raise 60 billion yen. (Reuters)
  • The scientists who advise the FDA are often later paid by the drug makers they help regulate, creating potential conflicts of interest. (Science)
  • THC therapy tanks in Phase 1, sending Zynerba’s stock tumbling yet again. (Endpoints)

Thanks for reading! Until next week,

Megan

Friday, July 6, 2018

STAT

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