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The Readout

Your guide to decoding clinical trials 

How can something be “double-blind”? Who decides whether a result is “significant”? And what on earth does “intent to treat” mean?

If you’ve tried to decipher clinical trial data, you’ve likely run up against similar questions. Study results can be absurdly (and unnecessarily) difficult to read, and that’s on top of the spin administered by drug companies and short-sellers on Twitter.

That’s why we’ve drafted STAT’s Sharon Begley and Adam Feuerstein, learned hands when it comes to parsing clinical data, for a subscriber-only lesson on how not to get duped. On Sept. 25, at 1:30 p.m. ET, Sharon and Adam will share their recommendations on what to look for — and what not to fall for — when trying to make sense of the data, and they’ll walk through some case studies to help you prepare for the next big readout.

Sign up here.

A pharma exec plans to raise price of an antibiotic by 400 percent 

“I think it is a moral requirement to make money when you can . . . to sell the product for the highest price.”

That might sound like the sort of thing that infamous pharma bro Martin Shkreli might say. Surely, amid a national debate over drug-pricing and growing concern over health care costs, no pharma CEO in his right mind would deign to utter such a remark, right?
Well, no. That observation comes to us from Nirmal Mulye, CEO of Nostrum Laboratories, who told the Financial Times for a story published yesterday that he would quadruple the price of a generic liquid antibiotic used to treat bladder infections.
FDA Commissioner Scott Gottlieb quickly responded on Twitter, saying that the drug, nitrofurantoin, is in a “competitive drug category” and that the agency would be releasing more data “soon.” But beyond shaming Nostrum, the case appears to illustrate the limited means Gottlieb and other administration officials have to get drug makers to avoid dramatic price hikes.
Read more.

What’s going to happen to OPKO?

On Friday, Nasdaq halted trading of the biotech company OPKO Health after the SEC alleged that its founder and CEO, Phillip Frost, played a role in a pump-and-dump stock scheme.

And that pretty much brings us to now. OPKO’s shares remained halted throughout yesterday, and the company said in a statement that it has no idea when that will change. “OPKO is working expeditiously to respond to Nasdaq’s request for information,” the statement reads, but management doesn’t know how long that will take. (OPKO maintains that “once a proper investigation is completed and the facts of the case have been fully disclosed, the matter will be resolved favorably for the company.”)

This leaves investors in a strange position. If you own shares of OPKO and you want to get out, you’re indefinitely stuck holding a stock you don’t want. And if you were suspicious all along and decided to short OPKO, you’re similarly left sitting on your hands with no idea how this is all going to play out. In the worst-case scenario, for OPKO and shorts alike, the company could get delisted and never trade again, which would effectively punish the shorts for being right. It has happened before.

Elsewhere, "SEC Charges Against Phillip Frost Might Just Be the Tip of the Iceberg," reads this headline.

Is data from four patients worth $4 billion?

Yes, according to Wall Street, which sent Amgen's market value up by roughly that much this week on the back of early results from a treatment for blood cancer.

The exuberance is simple: Amgen's antibody treatment has the potential to work just as well as in-development CAR-T therapies but without the costly and time-consuming labor that goes into crafting patient-specific therapies.

But ascribing $1 billion per patient might be a little over the top, especially considering they've only been followed for 10 months. Amgen has a lot of catching up to do with its CAR-T rivals, which are already in Phase 3, and the history of biotech is littered with therapies that showed promise in early studies only to come back to earth when tested in larger patient groups.

"I don't think we've even passed 'go' yet," tweeted Paul Rennert, CSO of the cell-therapy-focused Aleta Biotherapeutics.

Patent judges aren't scientists

And that's OK, writes Jacob Sherkow, a professor of law at New York Law School.

In the long-running debate over just who deserves credit for CRISPR-Cas9 genome editing, the sticking point has been whether editing complex cells, which the Broad Institute patented, is a significant advance over editing bacteria, which the University of California did first.

This week, a federal appeals court sided with the Broad, adopting an interpretation of scientific research that rankled many actual scientific researchers. But, according to Sherkow, it's possible that the court got the science wrong and the patent decision right, illustrating "a classic disconnect between the legal standards of patent law and the realities of scientific research."

Read more.

More reads

  • Pharma chief defends 400 percent drug price rise as a ‘moral requirement.’ (Financial Times)
  • Archived: an online chat discussing Ebola, vaccines, and a new epidemic. (STAT)
  • FDA urged to take stronger action to protect medical devices from hacking. (STAT Plus)
  • Biologists checked out this NBA player’s DNA for clues to his immense height. (MIT Tech Review)

Thanks for reading! Until tomorrow,


Wednesday, September 12, 2018


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