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The Readout Damian Garde

Trying to make heads or tails of all the new therapies for sickle cell disease? On Friday, STAT’s Adam Feuerstein will interview a physician expert on the merits of treatments from Novartis, Global Blood Therapeutics, and others. You can sign up to listen in here.

The cures of the future still rely on a toxic drug from the '50s

The advent of gene therapy brings the promise of a one-time treatment for serious diseases, but clearing the way for curative treatment requires a decades-old chemotherapy whose side effects can ward off potential patients.

As STAT’s Adam Feuerstein reports, scientists use busulfan, a chemotherapy first developed in the 1950s, to eliminate disease-causing cells before administering gene therapy. It’s a necessary step, but one that comes with risks, including organ damage, severe infections, and, in rare cases, death.

That’s why a host of researchers are investigating ways to replicate busulfan’s much-needed benefits while leaving out the toxicity. And there’s some promising data, albeit from animal studies, suggesting they might be on the right track.

Read more.

Illumina’s $1.2 billion merger might be dead

Late yesterday, the FTC reached the same conclusion as the U.K.’s antitrust authority: Illumina’s planned merger with Pacific Biosciences would create an illegal monopoly in the DNA sequencing business.

As STAT’s Matthew Herper reports, the U.S. agency filed a complaint aiming to block the deal, arguing that Pacific Biosciences has evolved into a rival of the dominant Illumina, and that a merger of the two would be anticompetitive. The U.K.’s Competition and Markets Authority said pretty much the same thing in November, which led the analysts at Cowen to declare the merger pretty much dead.

If that’s the case, Illumina, which has struggled to grow at its previous pace, will need to look elsewhere to expand its sales. In July, the company said annual revenue growth would be about half of what analysts previously expected. Shares are down about 15% since.

Read more.

Congress hands pharma a surprise holiday gift

By slipping 17 words into a 1,173-page bill, Congress provided an incremental victory to a drug industry otherwise unpopular in Washington.

As STAT’s Nick Florko reports, the massive spending bill passed by the House yesterday contains a provision that tweaks the definition of a “biologic.” Where that definition was previously limited to lab-grown products like antibodies, it would now cover peptide treatments including Novo Nordisk’s diabetes drug Victoza and Eli Lilly’s osteoporosis drug Forteo.

It’s a minor change that could have major implications. Under current law, peptide treatments are considered small molecules, which means they get five years of market exclusivity after approval. If the House bill is clears the Senate and becomes law, they’ll get the 12-year monopoly granted to biologics.

Read more.

Alnylam goes 3-for-3 in rare disease trials

Alnylam Pharmaceuticals said yesterday that its treatment for a rare liver disease met its goals in a pivotal trial, marking the company’s third straight clinical success and setting the stage for FDA approval.

As STAT’s Kate Sheridan reports, Alnylam’s lumasiran, an RNAi therapy, is meant to treat primary hyperoxaluria type 1, or PH1, which results from an excess of a chemical called oxalate. In a clinical study, the drug reduced the amount of oxalate in patients’ urine, a result the company believes will translate to relief from PH1’s painful symptoms.

But competition is on the way from Dicerna Pharmaceuticals, Alnylam’s former courtroom enemy. DCR-PHXC, a treatment that targets PH1 through a different pathway, is between nine and 12 months behind lumasiran.

Read more.

More reads

  • Purdue, maker of OxyContin, quietly splits ways with PhRMA as it pulls back from lobbying. (STAT)
  • Takeaways from a biotech CEO on the House’s passage of H.R. 3. (STAT)
  • Manufacturing: the next breakthrough in gene therapy. (STAT)
  • STAT Summit: The hypebusters who spoke truth to power. (STAT Plus)

Thanks for reading! Until tomorrow,

Wednesday, December 18, 2019

STAT

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