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Tuesday, July 5, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, your daily glimpse at what's happening in biotech. Today, we look at an emerging target in the world of pain management, the second act for a post-Shkreli company, and when 'survival' doesn't actually mean 'survival.' Need more? Visit statnews.com and follow us on Twitter.

A molecular 'on-off switch for agony' sets off a biopharma scramble

A new class of drugs aims to treat pain by targeting its molecular roots. (James King-Holmes/Science Source)

The opioid epidemic has researchers scrambling to find new solutions for the roughly 100 million Americans with chronic pain. Now, taking clues from genetic mutants who either feel no pain or cannot escape it, scientists have found a novel target that could provide side effect-free relief. And, seeing a multibillion-dollar opportunity, the biopharma industry has crowded in.

Read more.

How global warming could help Martin Shkreli's old company stay out of bankruptcy

After years of work on monoclonal antibodies, KaloBios announced last fall it was winding down operations. A white knight came in the unlikely form of Martin Shkreli, who took over the company with a $3.2 million investment and stepped up as its CEO — until he was arrested on securities fraud charges. The company looked again on the verge of insolvency.
 
It’s managed to right its course a bit, however, announcing last week that it has emerged from bankruptcy and raised new funding. STAT chatted with new CEO Dr. Cameron Durrant, who joined KaloBios in March (with a salary of $50,000 per month):
 
On top of developing monoclonal antibodies, you’re now pursuing a drug that treats Chagas disease. Is it because fears of global warming are leading to the prospect of a rise in this tropical disease?
Benznidazole is considered the gold standard treatment for Chagas disease, which was added to the FDA list of neglected diseases in August last year. But the drug’s only available on a spotty basis from the CDC. The majority of the disease pops up among people who have migrated from Latin America, but there’s growing evidence that there are some de novo cases of Chagas in the US now and we expect there will be more. The initial discussions to acquire the drug were between Savant Neglected Disease and Martin Shkreli in December.
 
So did Shkreli help KaloBios get back up off the ground?
There’s no doubt that people like to watch a bit of a circus — and maybe that benefited the company in terms of garnering some of the attention, which it perhaps wouldn’t have gotten otherwise. But we’re also moving past that.
 
With all the trouble surrounding the company, what made you want to join KaloBios?
Why join? I like the challenge. I’ve never had to deal with a company that's in Chapter 11 bankruptcy, being delisted by Nasdaq, have the CEO arrested, the clinical trials suspended, have lots of people leave, have the board resign, and have investors ask for their money back — never mind having all that stuff happen all at once. So, from a personal development point of view, I thought this would be an interesting job.

Takeda bets on stem cell therapy for Crohn's disease

Takeda is betting on stem cells in a deal announced early this morning with TiGenix, a biotech based in Belgium. Takeda has committed more than $400 million (starting with an upfront payment of nearly $28 million) to license, develop, and market Cx601, which uses stem cells derived from fat as an injected treatment for complex perianal fistulas in patients with Crohn's disease. The therapy has already been submitted to the European Medicines Agency for approval.

Stem cells are hot in the US, too, where some scientists are pushing for Congress to pass a bill that would let the FDA give stem cell treatments conditional approval before getting phase 3 trial data on their effectiveness.

Happy Dollyversary!

the world's most famous sheep  was euthanized on Feb. 14, 2003 after contracting a lung disease. she's been stuffed and put on display in an Edinburgh museum. (ALEX HOGAN/STAT, GETTY IMAGES)

Twenty years ago today, Dolly the sheep was born. The first animal ever cloned from an adult of its species, Dolly had a powerful impact on the development of stem cell science — and pet cloning and animal husbandry.

 "There are only a handful of truly monumental scientific discoveries that actually become household knowledge, and Dolly is definitely one of those few," said  Paul Tesar, an associate professor of genetics at Case Western Reserve University.

Tesar was a sophomore at CWRU at the time the paper describing Dolly's conception came out, and it's informed his career path since. He's now working on drug discovery that could help the body's own brain stem cells work more effectively to reverse disability in diseases like multiple sclerosis — which he'll be discussing at a fall symposium, "Coming of Age: The Legacy of Dolly at 20."

The Biotech Devil's Dictionary

There’s a lot of jargon, coded language, and outright nonsense in biotech, and we want to clear up — and celebrate — as much of it as we can through this glossary. Have a phrase to contribute? Email it on over.

Progression-free survival (n.): The amount of time after treatment that a patient — often a cancer patient — is able to live with the disease without it getting noticeably worse. Clinical trials often measure PFS.

Reader Vinay Prasad came up with this analogy to describe progression-free survival as it relates to ovarian cancer — as a means of correcting STAT’s ungraceful definition of PFS in a recent story:

“Ovarian cancer is like a long run, and we want drugs that make you run farther. In this study, after the first water break, one group carries a water bottle, the other does not. (Maintenance water = drug.) The group that carries the water lasts longer before they need a second water break. That’s PFS. But the question is, do you run farther?"

That fundamental question — do patients on this treatment live longer?  — often isn't addressed in clinical trials, including the one we wrote about last week.

More reads

  • House Democrats want answers from Elizabeth Holmes and Theranos. (Morning Consult) 
  • Have research papers gotten too complicated? (STAT)
  • Janssen has exercised a license on FR104, a monoclonal antibody therapy developed by OSE Immunotherapeutics for autoimmune disease and organ transplantation. (Press release)
  • AstraZeneca has cut ties with an auto-immune treatment that ran into issues of suicidal ideation in its late-stage trials. (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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