The Readout Damian Garde

Can a new spin on old technology outfox CAR-T?

Is anything too weighty to be explained over the internet? And does that controversial drug even work?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, Dorothy Pomerantz joins us to explain what it’s like to learn devastating medical information from the website of a consumer genetic testing company. Later, Amgen executive Dr. Greg Friberg joins us to explain why the company is betting on BiTEs, an approach to cancer immunotherapy that could succeed where CAR-Ts falter. Then, we dive into why Sarepta Therapeutics is yet to start an important clinical trial it promised the FDA three years ago.

You can listen to the episode here. To listen to future episodes, be sure to sign up on iTunes, Stitcher, Spotify, or wherever you get your podcasts.

Oncology enters its agnostic period

It used to be that if you had a cancer drug, getting FDA approval meant finding a lot of patients with, say, ovarian cancer and proving in a clinical trial your treatment could help. If you thought it might work in lung cancer, too, you had to recruit another study and prove it all over again.

But yesterday’s approval of a drug from Roche illustrates what is becoming the new normal in oncology. The treatment, Rozlytrek, is approved for patients with any cancer type so long as they test positive for a certain genetic mutation. 

It’s the third time the FDA has granted such an approval, and it’s unlikely to be the last. The promise of such precision medicine has lured a host of companies into developing tumor-agnostic cancer therapies, many of which are now in clinical trials.

Read more.

A novel idea in the NASH race

Meet NLRP3, a complex of proteins that plays a role in the inflammatory process and, according to some in biotech, looks like a winning target for treating the prevalent liver disease NASH.

As STAT's Diana Cai explains, NLRP3 has an essential role in protecting the body from foreign invaders, but in the case of inflammatory diseases like NASH, the protein complex can get overactive and lead to tissue damage. That’s why NodThera, a U.K. startup developing NLRP3 inhibitors, managed to raise $40 million in venture cash last year. And it’s why Swiss pharma giant Novartis bought the NLRP3-focused company IFM Tre back in April.

But any drug aimed at the protein complex will need to be precise, scientists warn. A mis-tuned molecule could interfere with NLRP3’s protective function and leave patients susceptible to infection.

Read more.

Of what value is youth?

If you don’t spend a lot of time reading venture capitalist blogs, you might have missed what was less a feud and more a dialectic between two men who make lots of money when startups succeed.

There were many points of polite disagreement between the blog posts of Jared Friedman, from Silicon Valley startup incubator Y Combinator, and Bruce Booth, of the Boston-based biotech VC Atlas Venture. But one particularly interesting facet was this: How much does experience matter?

Friedman’s point was that it is now nearly as easy to start a biotech company as it to start a tech one, and one of the results is that straight-out-of-school founders can build biotech firms from scratch instead of relying on the now-dominant venture creation model employed by the likes of Atlas. Those startups will “look like tech companies,” Friedman wrote, which means “instead of being run by VCs and hired execs, they’ll be run by the founders who care about their ideas, and who will sustain that passion building companies they love and that change the world for the better.”

Perhaps deliberately ignoring the implication that “VCs and hired execs” don’t “care,” Booth’s blog zooms in on one particular trait of straight-out-of-school founders who’ve never worked in biotech: the fact that they’ve never worked in biotech. Experience goes a long way, Booth writes, and “the idea that a recent Ph.D. or post-doc who has studied a narrow piece of biology for five-plus years is ready to run a multi-disciplinary drug R&D organization powered with $10 million in their first job out of academia seems far-fetched.”

More reads

  • Top U.S. medical centers roll out DNA sequencing clinics for healthy (and often wealthy) clients. (STAT)
  • Warning signs were always there for Novartis’s AveXis deal. (EP Vantage)
  • Regeneron's great science led to an important drug. But will it flop commercially? (Forbes)
  • Nuclear pharmacies sue a key supplier of radiology medicines for pursuing an ‘illegal monopoly.’ (STAT Plus)

Thanks for reading! Until next week,


Friday, August 16, 2019


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