Wednesday, November 16, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of all the biotech news. For more, visit STAT. On Twitter: @damiangarde@megkesh, and @statnews.

With great biopower comes great bioresponsibility

New advances in biotechnology pose a sizable threat to national security, as we learn from a sobering new letter from the president’s scientific advisory council.

High throughput DNA synthesis could hasten the creation of viruses. Gene delivery advances could help them spread. The all-powerful CRISPR gene editing tool makes the list of biothreats, too.

The letter urges the White House to build a new biodefense strategy — because protocols developed during the Cold War don’t cut it anymore.

Suggested preparations include a $2 billion public health emergency response fund, since America “still lacks a sustainable, reliable way rapidly to fund a response to epidemic emergencies,” the letter says. The council also urges earmarking $250 million per year to stock up on vaccines, and says we should make it a national priority to develop new classes of broad-spectrum antibiotics and antivirals. 

What will our next president do with this advice? Not clear. Donald Trump has spoken often of bolstering national security, so he may well heed the call to gird against bioterror. But his views on scientific research remain murky. We'll be watching for clues as his administration shapes up.

Schrödinger’s cholesterol drug

The Medicines Company released its much anticipated data on a novel new cholesterol drug, and the world is having trouble deciding just what to make of it. The good news is it looks like the drug, dosed two or three times a year, works about as well current therapies that must taken once or twice a month. The bad news is that might not really matter.

In a mid-stage study involving 500 patients, the Medicines Company’s inclisiran reduced bad cholesterol by more than 50 percent on average and led to few serious side effects, the company said. Management has begun mapping out a late-stage study, hoping to eventually convince the FDA to approve inclisiran.

But does the world need another cholesterol therapy? 

The antibodies on the market now — from Amgen and the partnership of Sanofi and Regeneron — have demonstrated similar effects on cholesterol, and yet they’re not exactly flying off the shelves. That’s in large part because physicians and payers are waiting on long-term data showing that slashing bad cholesterol with those antibodies can actually prevent cardiovascular death. If so, the treatments may blossom into the blockbusters they once seemed destined to become. If not, they’ll remain niche therapies for select patients with severely high cholesterol.

So what of the Medicines Company? In the best-case scenario, inclisiran could win approval by 2020, at which point the antibody treatments will be firmly entrenched. And it’s quite possible those same doctors and payers will want to see years of outcomes data on inclisiran before prescribing it widely, meaning the Medicines Company’s big payoff could be many years down the line, when cheaper biosimilar competition will loom around the corner for its rivals.

These risks played out in miniature with the company’s stock price, which boomed, tanked, and eventually settled at right about even after Tuesday’s announcement.

Sponsor content by ACT for NIH

It’s time for Congress to restore NIH funding

NIH research discovers treatments and cures for diseases like cancer and Alzheimer’s. NIH research also protects us from public health crises, keeps America the world leader in science and medicine, and grows the economies of cities and towns across the country. Urge Congress to protect, restore, and grow NIH funding before the year is out. There are too many patients to be patient.

Beware the ‘short, crappy trial’

The randomized clinical trial, or RCT, has long been the gold standard in science. But a combination of fracture, siloing, and ego has led to the proliferation of the SCT: “short, crappy trial.”

That’s according to FDA Commissioner Dr. Robert Califf (who, to be fair, was quoting CDER Director Dr. Janet Woodcock). Speaking at the annual FasterCures conference in New York on Tuesday, Califf bemoaned the rise of studies that seem to be done for study’s sake, wagering little, discovering less, and featuring “more coauthors than endpoints.”

The problem is that many scientists and academic institutions are territorial by nature, performing vanity-project trials that contribute little to scientific understanding. Ditto for small, cash-light biotech companies, which are often incentivized to show something resembling positive data on the cheap. 

A better way, according to Cancer Moonshot Director Greg Simon, is to be more open and collaborative. In his view, many clinical trials — especially in oncology — should never have been done. If scientists were more willing to share their data, he said, such waste could be avoided.

Yes, California. We know you're important.

The California Life Sciences Association just put out its annual industry report, which includes the standard rah-rah language around its "unique life sciences ecosystem." It's got stats on employment and salaries and grants and such. Here's a table we thought was interesting:


The Parker Institute's shiny new IRB protocol

The IRB can be one of the most frustrating aspects of conducting clinical research. Oh, the bureaucracy! 

So the Parker Institute for Cancer Immunotherapy has kicked off a new IRB process, meant to save time and money.

It’s set up a cooperative arrangement with six institutions — UCLA, UCSF, Stanford, Penn, MD Anderson, and Memorial Sloan Kettering. Trials that are funded by the institute and span multiple sites will only need to go through one IRB — at whichever university has more experience with that subject matter.  Cellular therapy trials, for instance, will most likely be examined by the folks at University of Pennsylvania.

“We thought about actually creating an independent Parker Institute IRB, but thought that’d be reinventing the wheel,” said Dr. Ramy Ibrahim, vice president of clinical development at the Parker Institute.

The idea is modeled after a new system the NIH is working on, called “IRB Reliance” — which whittles down the number of IRB reviews a study might need. Stay tuned for more of this in 2017.

More reads

  • Israeli billionaire Marius Nacht is raising $100 million to invest in biotech startups in his home country. (Reuters)
  • Why are cardiology conferences stocked with such unhealthy foods? (CardioBrief)
  • CRISPR gene editing has been tested in a human now. In China. (Nature)
  • Trump used to rail against drug prices. Now the industry's allies are helping him shape his agenda. (Los Angeles Times)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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