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The Readout Damian Garde

What’s going on at Editas?


Editas Medicine has a license to the most exciting biotechnology in a generation, more than $300 million in cash, and plans to start its first clinical trial in just a few months. So why are leaders stepping down?

Yesterday, Editas CEO Katrine Bosley said she’d resign from the genome-editing company March 1. The company’s chief financial officer will quit the same day, a decision he disclosed last month, and Editas’s chief medical officer just resigned at the end of 2018.

On the one hand, perhaps it’s a natural transition. Bosley was in charge for more than four years, a time that saw Editas go from a private firm with a promising but unproven technology to a publicly traded one on the verge of dosing actual humans. CRISPR Therapeutics, an Editas rival, changed CEOs back in 2017, and there was no apparent upheaval.

On the other hand, Editas’s share price fell about 20 percent yesterday, suggesting the market does not perceive this to be business as usual.

Poundation Medicine

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(JUSTIN SULLIVAN/GETTY IMAGES)

Introducing FidoCure, a genomic service that, for a couple thousand dollars, will sequence your dog’s cancer and recommend a targeted therapy. It’s a real thing, and the company that invented it just raised $5 million from Silicon Valley venture capitalists.

As STAT’s Rebecca Robbins reports, FidoCure is a sort of canine analog to the tumor tests offered by the likes of Foundation Medicine. One Health Company, the manufacturer, said its service churns out a report of a tumor’s genetic signature and the drugs — dog-approved or otherwise — that might stop the cancer from spreading.

But what about the optics of giving dogs the sort of high-cost — and potentially life-saving — genetic testing that many humans struggle to afford? And is extending the life of a cancer-stricken dog only prolonging its pain?

Read more.

Sliding doors in mRNA


In the race to turn mRNA into an actual drug, Translate Bio notched a minor victory when it beat the wealthier Moderna Therapeutics to start a clinical trial. But a pair of diverging updates from the FDA — a bad one for Translate and a good one for Moderna — illustrate just how fickle novel technologies can be.

On Tuesday, Translate disclosed that the FDA has put one of the company’s early-stage therapies on hold because the agency has “additional clinical and nonclinical questions.” That’s bad news, and it delays indefinitely the start of a trial. The hold doesn’t affect Translate’s lead program, which is in the midst of a study in cystic fibrosis, but it still took more than 10 percent off the company's stock price.

Meanwhile, over at Moderna, the FDA gave the company permission to start its first rare-disease trial, which will test an mRNA therapy against methylmalonic acidemia. Such permission is usually a dog-bites-man milestone in biotech, but Moderna’s efforts in rare disease have been rocky and heavily scrutinized, so the FDA’s decision, however incremental, is a check in the credibility column for a much-debated company.

What do parachutes and the gold standard have to do with biotech?


Is there a way around the placebo effect? How many patients have to a take drug before we know it works? And what does "work" even mean?

We spent about an hour yesterday discussing those very concepts in an attempt to explain why randomized clinical trials are so vital, where they can be less than ideal, and how the drug industry is trying to find novel ways to determine whether a new therapy can actually be a product.

It took the form of a webinar, and, if you missed it (or just really like webinars), you can watch the whole thing here.

More reads

  • PhRMA spent a record-breaking $27.5 million on lobbying in 2018, new filings show (STAT)
  • Gene therapy: how much will it cost patients? (EP Vantage)
  • J&J forecasts disappointing 2019 sales. (Reuters)
  • Aurinia’s flop in dry-eye trial brings an unexpected victory — and a shot at challenging Allergan. (STAT Plus)
  • Biosimilars and interchangeability: The FDA's science-based standards will ensure safety (STAT Plus)

Thanks for reading! Until tomorrow,

Megan

Wednesday, January 23, 2019

STAT

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