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The Readout Damian Garde

A plan to ‘eradicate’ heart disease with CRISPR

As ambitious mission statements go, Verve Therapeutics’ is right up there: The just-formed company wants to use genome editing to permanently protect patients from coronary artery disease, upending decades of medical practice and rendering standard treatments obsolete.

As STAT’s Sharon Begley reports, Verve isn’t yet close to running a clinical trial, let alone revolutionizing cardiology. But the idea was enough to convince Dr. Sekar Kathiresan, a well-regarded cardiologist, to quit his job running the Center for Genomic Medicine at Massachusetts General Hospital and become Verve’s CEO. And a group of investors led by GV bought into Verve’s $58.5 million seed round.

Now come the hurdles. First, Verve will have to prove that its approach is safe enough to supplant decades-old medicines with strong track records. Then there’s the small matter of actually editing enough cells to have an effect. And, even if everything goes to plan, the company would have to compete in a cardiovascular market where costly therapies, even when they work, struggle to make a dent commercially.

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When is a trial 'negative,' exactly?

Cytokinetics invented a drug to treat ALS, the rare and devastating muscle-wasting disease. It entered that drug in a 485-patient trial, testing whether it could improve ALS symptoms on three measures. In the end, the drug did not significantly beat placebo on any of them, which is commonly considered a failure.

But, as STAT’s Adam Feuerstein reports, Cytokinetics doesn’t see it that way. In an interview, CEO Robert Blum said there was “a consistent and dose-dependent effect that is clinically meaningful for ALS patients.” The trial’s lead investigator said the data were “among the most impressive” he’d ever seen in an ALS trial, and now Cytokinetics wants to start a Phase 3 study that would support FDA approval.

That might seem ill-advised considering that, again, the drug didn’t meet any of its goals, but Cytokinetics’ share price rose about 6% yesterday, so at least some people think it’s a good idea.

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An international drug price index could save Medicare billions

The idea of tying Medicare payments to drug prices in other countries, endorsed by President Trump and Sen. Bernie Sanders alike, is meant to save taxpayer money. But just how much?

According to a new study, it would add up to about $73 billion a year. 

The authors looked at prices in Japan, the U.K., and the Canadian province of Ontario, finding that brand-name drugs were between three and four times more expensive in the U.S. on average. Which is why tying Medicare payments to foreign prices provides such a savings.

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The Masters of the Universe vs. darts

A biotech company called Reata Pharmaceuticals saw its share price tick upward yesterday thanks to an on-stage endorsement at an event that bills itself as the Super Bowl of investing conferences.

Bihua Chen, who runs Cormorant Asset Management, told the audience of the Sohn Conference that Reata’s kidney drug has “striking efficacy,” and that the company could be “a 10-bagger,” meaning it could offer a tenfold return.

And that could be true: Reata is up about 50% this year on the back of optimism that its drug, bardoxolone, can treat a genetic kidney disease called Alport syndrome. The company expects to have data by the end of this year.

But before you take anything said at Sohn too seriously, consider the track records of its headlining guests. Last year, the cast of the Wall Street Journal’s Heard on the Street tacked stock listings to the wall and threw darts at them. Over the ensuing year, the dart-selected portfolio outperformed what Sohn’s experts recommended by 27%.

More reads

  • Customer complaints show $600 million health startup uBiome has been surprising patients and insurers with bills for years. (Business Insider)
  • Study highlights challenges in using artificial intelligence to personalize cancer treatment. (STAT Plus)
  • Search for buyout targets leads public investors to biotech. (Wall Street Journal)
  • In a crafty move, FDA may have found a way to dampen controversy over a $375,000 rare-disease drug (STAT)

Thanks for reading! Until tomorrow,


Tuesday, May 7, 2019


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