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Monday, October 10, 2016

The Readout by Damian Garde & Meghana Keshavan

Good morning, and welcome to your daily roundup of biotech news and analysis. Check out the rest of STAT's newsletters, too.

Cancer drug showdowns at ESMO

The European Society for Medical Oncology conference this past weekend in Denmark proved quite newsy. 

First, we had a showdown on PARP inhibitors for ovarian cancer, featuring data form biotechs Tesaro and Clovis Oncology.

Both companies’ treatments aim to stop cancerous cells from repairing themselves after they’ve been hit by chemotherapy — and Tesaro proved the clear winner. Its drug did well across the board but was most effective for women with the BRCA mutation:  Their tumors didn’t start to grow again for 21 months, on average, compared to 5.5 months for a control group that received a placebo. More on the findings here.

On another front, Merck and Bristol-Myers Squibb went head-to-head on their immunotherapy approaches to lung cancer. Merck’s Keytruda was shown to delay tumor growth substantially in newly diagnosed non-small cell lung cancer, when compared to chemo. BMS’s Opdivo, by contrast, was “a total failure in newly diagnosed lung cancer,” as Adam Feuerstein points out

A flexible new approach to RNA drug delivery 

The dream of using RNA-based therapies to shut down genes that cause disease took a heavy blow last week when the leader in the field, Alnylam Pharmaceuticals, shut down its top drug contender after several patients in a late-stage clinical trial died.

But Alnylam is still expressing optimism about its other experimental drugs. And biotechs elsewhere are also forging ahead with RNA research.

In San Diego, startup Arcturus Therapeutics has developed a new technology for delivering RNA-based therapies into cells. It involves creating strands of nucleic acids in the lab, and then tacking them onto therapeutic RNA. The immune system doesn’t recognize the strands, known as UNAs, so it doesn’t attack them. They also persist in the body longer than natural RNA, so any drug they help deliver has a longer half-life.

The technology is still very early, in the pre-clinical stage. But CEO Joseph Payne told STAT that his synthetic delivery method is “safer than nature,” in part because the UNAs his team creates in the lab are so flexible. “This flexibility translates into improved safety — because rigidity promotes an immune response,” Payne said.

Payne said he’s “definitely rooting for Alnylam and all the other companies working on RNA” but couldn’t help but add that the bad news for Alnylam has stirred new interest in his company’s method for delivering RNA drugs. (It's already working with Johnson & Johnson on a hepatitis B therapy.) In that way, he said, “the news last week impacted Arcturus in a positive way.”

Which CRISPR company is worth the most?

CRISPR Therapeutics, one of three gene-editing companies launched in recent years, is looking to join its rivals on the public markets. And if things go according to plan, it’ll be the most valuable.

In updated documents filed with the SEC, CRISPR Therapeutics said it’s planning to raise about $75 million at the mid-range of its IPO, which would value the company at roughly $636 million. That’s more than rivals Intellia, which has a market value of $540 million, and Editas Medicine, worth about $497 million.

Investor exuberance for the space has significantly cooled since early this year, with Editas losing more than 75 percent of its value since April and Intellia falling by about 50 percent since May. Each is now trading below its IPO price.

But gene editing still has a hold on the public imagination. Check out this video ode to the power of CRISPR-Cas9 from A Capella Science, set to the tune of "Mr. Sandman" and featuring lyrics like: "CRISPR-Cas9, bring me a gene, encoding for a specific protein. Make a few snips at this coded locus...you work so well inside a streptococcus."

Trust us, it's worth a listen.

Parsing the Theranos legacy

Now that Theranos has fully and embarrassingly retreated from its founding business model, everyone's looking for tidy lessons learned from whole debacle.

The "real victims of Theranos' lies," tweeted The New York Times' Nicole Perlroth, "are biotech/heath entrepreneurs with life-changing ideas who now have to reckon with her aftermath."

But Ethan Perlstein, the 36-year-old founder of biotech startup Perlara, doesn't think there'll be a chilling effect for young entrepreneurs seeking venture dollars. "Investor demand — especially on the crossover tech side — for hyper-ambitious, science-driven [startups] is nowhere near peaking," Perlstein said in an email.

If there is a lesson from the Theranos saga, it's "live by stealth, die by stealth," he said.

"Going forward, if any founder thinks they can don a turtleneck, evade peer review or any kind of scientific transparency, good luck to them."

More reads

  • Investors including Starbucks CEO Howard Schultz have pumped $50 million into an Irish startup working on treatments for Alzheimer's and multiple sclerosis. (The Times)
  • Hoping to keep out an inspector from the FDA, employees at one drug manufacturer formed a human barricade in front of their lab. (STAT)
  • Just what is a "down round," and why do startups go to such lengths to avoid them? (Wall Street Journal)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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