Thursday, August 31, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.

CAR-T happened

As you probably heard, Novartis won that pioneering FDA approval everyone saw coming, and more than a month early. More interesting: The Swiss drug maker plans to charge $475,000 for its therapy, a number that is unassailably quite large but also indisputably smaller than what many assumed — and what Novartis could have gotten away with.

That puts Gilead Sciences in a tough spot, says STAT's Adam Feuerstein. Sitting on a mint thanks to their remarkable success in hepatitis C, the leaders of Gilead chose cell therapy as their next target territory, agreeing to pay about $12 billion for Novartis competitor Kite Pharma. Now that Novartis has put a price tag on CAR-T, Gilead has a tough choice: Price its product accordingly and make less money than possible, or barrel into the market with a costly infusion and face public backlash.

But then, the public backlash tends to come no matter what. Patients For Affordable Drugs says the price is "excessive," adding that "Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more.”

For another take on the price, read this from health economist Anna Kaltenboeck and Dr. Peter Bach, the director of the Drug Pricing Lab at Memorial Sloan Kettering.

Back to school with Mr. Feuerstein



Sharpen your pencils and shine up those apples: Summer vacation’s over, and STAT’s Adam Feuerstein is ready to school you. On large cap biopharma, that is.

He grades some of the most-followed biotech stocks, such as Biogen, Regeneron, and the woebegone Alexion. Who’s got the most potential for the coming year? 

Read more on STAT Plus.

Why inflammation is a compelling, but complex, drug target

This week’s canakinumab news cast an exciting new light on inflammation and the role it can play in many different diseases. The CANTOS study showed that the drug could reduce the recurrence of cardiovascular complications by 15 percent and also cut down on incidence of lung cancer. 

But that study had some drawbacks: As predicted, canakinumab increased the users’ risk for serious infection — because anti-inflammatory drugs, by definition, put a damper on the immune response. 

So although inflammation is implicated in myriad other diseases — from autism to Alzheimer’s disease to AIDS — it’s difficult to target any of those conditions with a simple anti-inflammatory approach.

“The problem is, if you block inflammation, you’re blocking a primordial mechanism by which we are protected from the organisms that share the planet with us,” one researcher told us. 

Read more.

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Embryo editing claims draw skepticism

Dr. Shoukhrat Mitalipov of Oregon Health and Sciences University drew overnight fame after reporting that he used CRISPR to gene-edit human embryos. But prominent geneticists are now calling his conclusions into question. 

Mitalipov’s claim that he could edit out a disease-causing gene from an embryo came with a caveat: The edited DNA behaved in an unexpected manner. While the disease-causing gene — which was from the father’s sperm — was successfully excised, it didn't get replaced with a synthesized stretch of DNA. Instead, the embryo supposedly swapped in a healthy strand of genetic material from the mother’s egg. 

The skeptics, led by Maria Jasin of Memorial Sloan Kettering, say this isn’t consistent with the way they’ve seen genome repair occur — and are doubtful that the embryos behaved as Mitalopov reported. 

Read more on STAT Plus.

Repatriation lives!

Moving on from the strange and arguably damaging saga of the CEO councils, President Trump has pivoted to an issue biopharma actually likes: tax reform.

And perhaps most alluring, the president revisited a campaign promise to let companies with cash stashed overseas to move it back home at a decreased rate. As it stands, repatriating cash comes with a levy as high as 35 percent. Trump has suggested a one-time holiday that would lower that rate to 10 percent.

This is particularly important for biopharma, whose biggest players have about $175 billion parked offshore, according to Leerink Partners. That includes roughly $35 billion from Amgen, $29 billion from Gilead Sciences, $19 billion from Merck, and $14 billion from Pfizer.

But it’s unclear whether repatriation will, as Trump promised Wednesday, “spur billions of dollars of new investments in our struggling communities and throughout our nation.” Last time the government tried this, in 2004, companies mostly used the money to buy their own stock and take out competitors.

More reads

  • Incyte and Merck are out with new data on their combo melanoma immunotherapy, and it looks promising. (STAT Plus)
  • Parents hoping to cure their child's rare disease confront the high cost of research. (Wall Street Journal)
  • FDA invites Eli Lilly to refile its rheumatoid arthritis drug without additional study. (Endpoints)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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