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The Readout Damian Garde & Meghana Keshavan

The architect of a biotech rainmaker is at it again

John Hood started his last company, Impact BioMedicines, in 2016 with $22 million in venture capital. Less than two years later, Celgene bought it for more than $2 billion, creating a roughly 90-fold return for investors. It is perhaps no surprise that investors are listening to his next idea.

As STAT’s Matthew Herper reports, Hood has returned with another company, Endeavor BioMedicines, and raised $62 million to get it started. The plan is similar to the one that made Impact a success. Endeavor has licensed a drug that had fallen by the wayside at a major pharma company, in this case Eli Lilly, and redirected at a deadly and difficult-to-treat lung disease.

“We've identified a best-in-class molecule that's got lots of human use history, and we just think it was in the wrong place,” Hood said. With the money raised, Endeavor plans to start a pair of Phase 2 trials that will help determine whether he’s right.

Read more.

Moncef Slaoui’s not going anywhere

Moncef Slaoui, the former pharma executive whose perch at Operation Warp Speed angered some progressives, will stick around after Inauguration Day.

As STAT’s Nicholas Florko reports, Slaoui will serve as an adviser to President-elect Biden, a role he described as less active than his current job. The news comes as a surprise considering Biden’s recent criticism of Operation Warp Speed's failure to distribute as many Covid-19 vaccines as previously promised.

It’s also sure to be a disappointment to some of Biden’s base. Progressive groups like Public Citizen and lawmakers including Sen. Elizabeth Warren opposed Slaoui’s hiring on the grounds that his industry ties — and millions in stock holdings — created a conflict of interest at Operation Warp Speed.

Read more.

CRISPR cured progeria in mice

Scientists used everyone’s favorite genome editing technology to cure mice of progeria, a fatal disease marked by accelerated aging, a finding outside experts said could be a breakthrough in longevity research.

As STAT’s Sharon Begley reports, researchers used base editing, an evolutionary form of CRISPR, to alter a single pair of misspelled genetic letters in the cells of mice with progeria. The simple edit was so effective that half the animals lived for 510 days, which is old age for mice and twice as long as the lifespans of those untreated. 

This is all taking place in mice, and mice are not people, but the effects of the one-time treatment were so dramatic that “this has the feel of something that could be a true genetic cure with a single injection,” said Leslie Gordon, a physician and founder of the Progeria Research Foundation.

Read more.

Pfizer beats Sarepta to Phase 3 in gene therapy race

Pfizer dosed the first patient a Phase 3 trial of a gene therapy for Duchenne muscular dystrophy, beating rival Sarepta Therapeutics in a race to market. But just which treatment will find its way to patients first remains up in the air.

The news from Pfizer is that it has begun a placebo-controlled, 99-patient pivotal study that will determine whether its one-time therapy can preserve muscular function over the course of a year. Sarepta, which expects data any day now from a smaller study on its similar gene therapy, has yet to start its Phase 3 trial.

What’s unclear is which product is technically in the lead. Investors expect Sarepta to file for FDA approval based on two studies, the placebo-controlled trial and a 10-patient open-label study that will read out in the coming months. If the FDA goes for that, Sarepta could submit its application in 2021. If the agency holds out for Phase 3 results, Pfizer will have outpaced its competitor.

More reads

  • Scientists are monitoring a coronavirus mutation that could affect the strength of vaccines. (STAT)
  • Zai Lab Samantha Du buys China rights to an autoimmune drug in $175 million licensing deal. (Endpoints)
  • CDC reports more allergic reactions to Covid-19 vaccines, but cases remain few. (STAT)

Thanks for reading! Until tomorrow,

Thursday, January 7, 2021

STAT

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