Thursday, September 1, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, your daily source for all things biotech. Check out the STAT Pharmalot newsletter for more on the drug industry, and follow us on Twitter for more: @damiangarde@megkesh, and @statnews.

CAR-T therapy: Novartis move shakes the faith

Novartis just made a move that could shake faith in CAR-T immunotherapy: It announced yesterday that it will dissolve its cell and gene therapies unit, cutting 120 jobs. Its CAR-T research isn't evaporating, just being absorbed into the company’s main cancer division. Still, stock prices for CAR-T competitors Kite Pharma and Juno Therapeutics tumbled amid increased pressure to prove cell therapies can be commercially viable.

We chatted with Brad Loncar, founder of a cancer immunotherapy exchange-traded fund (and one of STAT’s top BioTweeters!) 

What are your thoughts on Novartis disbanding its cell therapy unit?

It’s not a total surprise, but it’s certainly disappointing. Whenever you have somebody reducing their investment in an emerging field, it’s a setback for that field. What it suggests is that Novartis does not see much utility for cell therapies beyond [its leading pipeline drug for] CD19, and other low hanging fruit indications that it’s already been hugely successful with initially — like acute lymphoblastic leukemia and other blood cancers.

What does Novartis’ move mean for CAR-T therapy?

It’s definitely negative, but I think Kite Pharma’s CAR-T’s news next month is going to be even more consequential for the entire field. Kite is supposed to announce interim results for its DLBCL trial, and while we won’t see the full results until ASH [in December], what we’ll find out from Kite next month is whether they have data that is good enough to be submitted to the FDA for registration. That’s hugely important.

One of these companies has to show that CAR-T technology is commercially viable. If Kite doesn’t do that, it’s going to be a very big setback for CAR-T — even more of a setback than today’s news from Novartis. Kite’s upcoming results are like a playoff game — they must win.

CAR-T seems to work well in blood cancers. Do other targets look promising? 

One target that’s promising is Bluebird Bio’s B-cell maturation antigen (BCMA) approach to multiple myeloma — we’ll see their data this year at ASH... Otherwise, no. There isn’t much data showing it’ll work in solid tumors.

We’re really spoiled because the first [acute lymphoblastic leukemia] trials had 90 percent cure rates, when we first saw them. That’s almost impossible for anyone to recreate. But sadly, nothing has come even close to that, so other than BCMA , I’m not wildly optimistic about anything right now.

You have an immunotherapy fund. What does this mean for you? Are you losing hope in CAR-T?

First of all, I’m not not excited. I’m excited, and a CAR-T believer. But what we have to keep in mind: What Novartis and Kite and Juno have in trials right now truly is version 1.1 — and companies are learning every single day from what they’re seeing. Over time, new versions of this are going to come out. So as a starting point, it’s extremely promising. 

I think the storyline today is, CAR-T is really walking a fine line. A year ago, there was definitely exuberance. But now, we’re on the precipice of people pulling back, just like Novartis did. It’s going to take success and commercialization to keep up CAR-T’s momentum so it can get fully developed and researched.

The convergent evolution of optogenetics

casting a blind eye over the origins of optogenetics? (zheng-hua pan)

Ever heard of Alfred Russel Wallace? Possibly not — but he’s a British naturalist who independently came up with the concept of evolution. Charles Darwin, however, tends to get the bulk of the credit. 

Ever heard of Zhuo-Hua Pan? Likely not. But he’s basically the Alfred Russel Wallace of optogenetics — the field that uses light to control cellular activity, particularly in neurons.

The Darwins — that is, Stanford’s Karl Deisseroth and MIT’s Ed Boyden — may be hailed as the inventors of optogenetics, and are all but certain to win a Nobel prize. But in his quest to cure blindness, Pan seems to have gotten there first. 

Read more.

Sponsor content by hubweek

Boston innovation festival HUBweek to host 90+ events from 9/25–10/1

HUBweek, a festival of the future, will take place from Sept. 25 – Oct. 1, 2016 in Boston, with over 90 events exploring the intersections of art, science, and technology. STAT, MIT, Harvard, and others will host thought-provoking conversations on inclusive innovation, precision medicine, drug regulation, and more. STAT experts Dr. Jennifer Adaeze Okwerekwu, Damian Garde, David Armstrong, and Ed Silverman will lead a series of discussions on Sept. 27. Curious? Visit and register today.

Celgene buys into a blockbuster field rife with competition

Celgene is the latest major drug maker to set its sights on NASH, a fast-spreading liver disease with no approved drugs that could present a blockbuster opportunity for the first company to crack it.

Under a longtime partnership with Massachusetts biotech Forma Therapeutics, Celgene bought the European rights to a NASH drug on the verge of human trials. Forma is on the hook to conduct Phase 1 studies of the drug, after which Celgene will take over global development, the companies said.

Short for non-alcoholic steatohepatitis, NASH is an increasingly prevalent disease in which fat builds up in the liver and leads to a dangerous, tissue-scarring process called fibrosis that can cause organ failure. As it stands, NASH affects about 15 million Americans, but the causes of the disease — poor diet, lack of exercise — are on the rise, and thus doctors believe it may become the leading driver of liver transplants by the end of the decade.

And the drug industry has taken notice. Intercept Pharmaceuticals is the farthest along, with a late-stage drug analysts say could bring in $1.6 billion in peak sales if it wins approval to treat NASH. Gilead Sciences bought into the field through a $400 million deal with Nimbus Therapeutics earlier this year, joining a group that also includes AstraZeneca and Enanta Pharmaceuticals.

Talk about gaming the system

you'll get a surge of adrenaline just from playing this game (epipen tycoon)

This EpiPen sales simulator tasks you, Mylan CEO Heather Bresch, with the precarious role of jacking up the cost of the epinephrine autoinjector while balancing the outrage of both the public and the investor base. Can you make enough money before one, or the other, has you canned? 

Play here. 

Biogen and the difference between the business and science pages

Yesterday, Nature published the results from a small study on Biogen’s all-important Alzheimer’s disease candidate. The data “spark(ed) optimism,” according to the Washington Post. The drug seemed "alleviate memory loss," Nature wrote. The Guardian called the results “tantalizing."

And yet these same data were roundly seen as disappointing by the business press when Biogen disclosed them last year. They even sent Biogen’s stock price down.

What gives?

It's likely a matter of differing expectations.

The backstory: In May of 2015, Biogen shared data showing that two doses of its drug, 3 mg and 10 mg, had a statistically significant effect on cognitive ability for patients with Alzheimer’s. Two months later, the company shared results for a 6 mg dose, which analysts had hoped would be more effective than the mini dose, with fewer side effects than the mega dose. It missed the mark. That was seen as a big setback.

Fast forward to this week's Nature paper, which combined the data on all three doses. The statistically significant improvements stood out as hopeful signs in a field strewn with bad news. The whole thing highlights how biotech coverage often resorts to horse-race journalism  — focusing on what edges stocks up or down  — where mainstream health coverage looks for hope. And then there’s whatever The Times (of London) did here.

More reads

  • Roger Tsien, a Nobel Laureate who found new ways to peer into cells, has died at age 64. (San Diego Union-Tribune)
  • FDA to consider new rules for off-label marketing. (STAT)
  • A $15.4 million industry/academia partnership brings together J&J, Salk, Johns Hopkins to screen for better drugs for schizophrenia and bipolar disorder. (Press release)

Have a news tip or comment you want to send us?

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Thanks for reading! Until tomorrow,

Damian & Meghana

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