Tuesday, November 15, 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.

The big asterisk on all that Medicare spending data

The Centers for Medicare and Medicaid Services just released a trove of useful data on how much Medicare and Medicaid spent on some key drugs last year. But keep in mind: The figures don’t include those murky manufacturer rebates we hear so much about.

It’s not that CMS is trying to mislead anyone; federal law prohibits the agency from disclosing exact rebates. What CMS will say is that in 2014, Medicare Part D collected an average rebate of 17.5 percent. That’s well below the 30 percent figure drug companies often cite, a gap that exists in part because “Medicare cannot harness its full purchasing power to negotiate for rebates across all Part D plans,” the agency wrote.

That, of course, could change. President-elect Donald Trump has in the past promised to let CMS negotiate with drug companies, bucking Republican dogma. If he presses forward with that plan, the numbers CMS just released could become a political football.

Navigating Trumpcare: Top Twitter accounts to follow

Zealous tweeter though he may be, Donald Trump simply can’t serve as your one-stop source on all the forthcoming changes in health care. 

So we’ve compiled a list of the top Twitter accounts worth following to get steady info on what’s next in health and medicine under our new president. 

Policy wonk Dr. Scott Gottlieb, a former CMS official and FDA deputy commissioner, makes the list — as does Dr. Ben Carson, the neurosurgeon-turned-presidential contender-turned possible-education-secretary, and journalist Charlie Ornstein, a health care watchdog at ProPublica.

Another way to stay informed: Subscribe to our new Trump-tastic newsletter: STAT's Trump in 30 Seconds.

Read more.

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.

Epigenetics: More tools needed

Epigenetic targets are growing increasingly druggable. Take Merck’s $515 million foray into the field this past January, which was all about exploring how the enzyme PRMT5 regulates genes involved in cancer-causing mutations and blood development.  It’s compelling stuff.

But there’s a gaping hole when it comes to the tools it takes to explore the field further, as a new piece in Genetic Engineering & Biotechnology News points out. It’s still tough to validate epigenetic targets.

“If drug developers are to identify and engage epigenetic targets, they will need biological assays capable of interrogating epigenetic alterations,” the article says. “There is an acute need for such assays in the field.” 

GEN goes on to point out some compelling assays that might help broaden the understanding of how gene expression is modified in disease.

All eyes on the Medicines Company

The Medicines Company, owner of biotech’s most on-the-nose name, is scheduled to present some all-important Phase 2 data on a drug that could shift the balance of power in the cholesterol space.

The Medicines Company’s treatment, inclisiran, is designed to help the body clear out bad cholesterol by getting rid of a protein called PCSK9. Current therapies on the market — from Amgen and the partnership of Sanofi and Regeneron — are antibodies that block PCSK9 molecules in the blood. The Medicines Company’s drug, invented by Alnylam, halts PCSK9 at the source by meddling with the gene that regulates it.

At least in theory. Last month, the company said inclisiran had a “significant and durable” effect on bad cholesterol in a 500-patient trial. Later this morning, we’ll find out just what that means. 

If the results are very good — equal to or better than the antibodies — the Medicines Company could wind up “in the cat-bird's seat,” as analysts from Leerink wrote. Inclisiran is dosed as few as two times per year, making it more convenient than the monthly or bi-monthly treatments on the market now.

But even in a best-case scenario, inclisiran probably wouldn’t hit the market until 2020, according to Cowen. And, considering the tepid commercial performance of the first-generation PCSK9 treatments, any predictions about peak sales should be served with ample salt.

Introducing vronc

Yesterday, we asked for your help in renaming Valeant Pharmaceuticals, a one-time Wall Street darling now synonymous with expensive drugs and Congressional inquiries.

Your choice: vronc, which picked up 35 percent of the vote to overcome a strong challenge from Gut Guy & Co. and outpace Ouroboros Therapeutics.

Besides being fun to say (Try it. “Vronc.”) the name might also help big-time Valeant backer Bill Ackman convince Dr. Patrick Soon-Shiong, another billionaire prone to bold pronouncements, to buy into the company’s future.

Unfortunately we did not think of Nanteant Pharmaceuticals until just now.

More reads

  • Prisons are fighting opioids with Vivitrol, a $1,000 injection. But does it work? (Associated Press)
  • Their brains had the telltale signs of Alzheimer’s. So why did they still have nimble minds? (STAT)
  • SEC Chairwoman Mary Jo White plans to step down once President Barack Obama leaves office, nearly three years before the end of her term. (Bloomberg)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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