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The Readout Damian Garde

‘Real world evidence’ put to the test

Joe Raedle/Getty Images

Millions of people take amphetamine medicines such as Adderall or Vyvanes to tame their ADHD. A fraction of them — 1 in 660, according to a new study in the New England Journal of Medicine — have a psychotic event.

That’s the headline, but the way researchers arrived at the conclusion is also newsworthy, STAT’s Matthew Herper reports. The researchers gathered “real-world evidence” from medical practices, not the gold-standard of randomized, controlled clinical trials, to arrive at their conclusions. They worked with Aetion, a New York City health data startup. Another New York startup, Flatiron Health, was acquired last year by Roche — for $1.9 billion — for its business mining patient electronic medical records to replace more traditional clinical trial data. Flatiron is collaborating with the FDA, so real-world evidence is looking real.

Read more.

Labor’s speaking up on drug pricing

Biotech leaders, take note.

Richard Trumka, the president of the nation’s largest labor federation, the AFL-CIO, will brief reporters Thursday afternoon on a new platform supporting policies to lower the price of prescription drugs. The yet-to-be-unveiled “statement of principles for prescription drug policy” has been endorsed by The Coalition for Fair Drug Prices, an alliance of liberal groups including the AFL, the labor union SEIU, Families USA, Public Citizen, and the Center for American Progress.

While the labor movement has dipped its toe into the debate over drug pricing in the past — the AFL endorsed a number of policies in 2016 — Thursday’s event may be the clearest sign yet that the labor movement is becoming more active on the drug-pricing issue.

Pfizer partners again in gene therapy

Pfizer will pay up to $636 million to win the rights to gene therapies being developed by French biotech Vivet Therapeutics. This follows similar deals it struck with Spark Therapeutics — before Roche bought Spark — for its hemophilia A treatment and with Sangamo Therapeutics for its hemophilia B therapy.

While Pfizer invests, Novartis and Biogen have, like Roche, gone the acquisition route, scooping up gene therapy developers AveXis and Nightstar Therapeutics. For Pfizer, the gene therapy furthest along in Vivet’s pipeline would reverse Wilson disease, a rare, inherited liver disorder that causes copper to accumulate in the body, requiring lifetime treatment and sometimes liver transplant. Vivet’s therapy, which would replace the single gene at fault, will be tested in humans starting next year.

Pfizer is also developing gene therapies in-house, including one for Duchenne muscular dystrophy, which began human trials last year. Sarepta Therapeutics and Solid Biosciences are also in that mix.

Flagship pulls in its biggest VC round

Flagship Pioneering, the money and minds behind Moderna Therapeutics and more than 75 other life sciences businesses, has raised $824 million in venture capital to bankroll startups, the company’s largest single fundraising effort.

Not that Moderna fits that description anymore. The former unicorn’s IPO set a record late last year, when its valuation tipped the scales at roughly $7.5 billion as it sought to raise $500 million. But Moderna is the type of company that would have gotten money from the new “special opportunities” pool for startups, Noubar Afeyan, founder and chief executive of Flagship, told the Boston Globe’s Jonathan Saltzman.

More reads

  • Sage’s depression therapy arrives with big spread in sales forecasts (Vantage)
  • Peering inside life's microworld: MIT's 2019 image award winners (STAT)
  • Protein-slaying drugs could be the next blockbuster therapies (Nature)
  • America's best weapon in the opioid epidemic just got cheaper (Bloomberg)

Thanks for reading! Liz Cooney signing off for the vacationing Damian Garde. Until tomorrow, when the baton is passed to our colleague Kate Sheridan.

Thursday, March 21, 2019


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