The Readout Damian Garde & Meghana Keshavan

Experimental sickle cell therapies aplenty

There is no shortage of data on sickle cell therapies at this year’s meeting of the European Hematology Association. Thirty-two research abstracts in all.

Luckily, STAT’s Adam Feuerstein did a lot of reading. And what he found was a host of companies conducting early-stage studies of experimental drugs. From Imara to Forma Therapeutics and Bluebird Bio — among others — the hope is that there are novel ways to target different disease pathways.

“Like with HIV or cancer, we’re learning that in sickle cell disease, it’s not enough to focus on one target,” one clinician said. “We have to attack the disease in multiple ways. We’re starting to see this approach take shape in drug development right now.”

Read more.

Agios bided its time — and has data to show for it

Agios Pharmaceuticals has been picked on by some investors for helping small numbers of patients while ignoring more profitable business opportunities. New data released early today should help quiet some of that criticism.

Data on Agios’ lead pipeline drug — one targeted at patients with two different types of thalassemia — reported positive results from its first clinical trial at the European Hematology Association meeting. While preliminary, they could set up a pivotal Phase 3 studies starting next year — one that, if successful, turn the drug, called mitapivat, into a blockbuster.

The data “show that mitapivat can address larger patient populations that still have unmet medical needs even with other therapies coming in,” said Chris Bowden, Agios’ chief medical officer.

Read more.

BIO has a ‘frank conversation’ about systemic racism in biotech

Protests around the world have cast a light on the systemic racism at play in health care, education, housing, and policing. Biotech is no exception. And BIO, the group behind the sector’s largest annual conference, didn’t let the moment pass without what organization CEO Michelle McMurry-Heath billed as a “frank conversation” about racism in the industry.

Tony Coles, CEO of Cerevel Therapeutics and a panelist in the discussion, likened racism to “a mutation” in the DNA of society. Legislative efforts like the Civil Rights Act were designed to be “targeted therapies” that would root it out, Coles said, but “what we really needed to do was CRISPR that mutation right out of our social DNA.”

The hour-long discussion, added at the last minute to BIO’s schedule, touched on how executives are addressing the death of George Floyd within their companies, what it means to be a Black CEO in biotech, and the importance of having uncomfortable conversations about race with friends and colleagues.

Read more.

Can a virtual conference feel real?

Do scientists have a say in their benefactors’ business? And what does it take to get remdesivir in the U.S.?

We discuss all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. First, STAT’s Kate Sheridan joins us for a dispatch from this week’s BIO conference, a huge annual event that has been forced online by the coronavirus pandemic.

Then, we discuss the strange situation brewing at the Chan Zuckerberg Initiative, where a bunch of scientists have signed an open letter decrying their benefactor’s other business, Facebook. Finally, STAT’s Eric Boodman calls in to talk about the lengths patients have to go to get remdesivir, Gilead Sciences’ treatment for Covid-19.

Listen here.

More reads

  • Watch: How some cells serve as unlikely heroes to defend the brain from viral invaders. (STAT)
  • Verve, raising another $63 million, seeks to advance gene-editing treatment for heart disease. (Boston Globe)
  • Moderna to start final testing stage of coronavirus vaccine in July. (Reuters)
  • Regeneron begins testing coronavirus antibody cocktail therapy. (Wall Street Journal)

Thanks for reading! Until tomorrow,

Friday, June 12, 2020


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