Wednesday, March 29, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, your source for biotech news. For more on the industry, check out STAT Plus. Subscribe to the Pharmalot newsletter. And, of course, follow STAT on Facebook and Twitter

Whither the biotech blockbuster?

In biotech, as in rocketeering, it helps to be optimistic. (MIKE REDDY/STAT)

Repatha, Praluent, Entresto — each hit the market with stratospheric sales projections, and each ran into atmospheric problems with payers and PBMs.

Will the same fate befall Dupixent, the new eczema drug from Sanofi and Regeneron that arrived yesterday with similar peak-sales fanfare? And what do all these sluggish launches mean for biotech's ability to crank out blockbuster drugs?

These and other rhetorical questions will be addressed in the following story about the changing calculus of drug development.

Read on.

Ocrelizumab's approval: A 40-year research effort realized

Dr. Joseph Hauser has focused his life's work on multiple sclerosis — and after four decades of grit, tenacity, and seemingly insurmountable setbacks, his research has come to fruition:  The FDA just approved Roche's ocrelizumab, a drug based on Hauser's research that "stops MS in its tracks." 

Ocrelizumab, which will be priced at $65,000 per year, is the only drug to have shown efficacy in the most aggressive form of multiple sclerosis.

"It's personally incredibly rewarding," Hauser told STAT's Ron Winslow. "This is a big deal for people with MS." 

Read more. 

The CRISPR patent clash isn’t going away

Just when the dispute over who invented what in CRISPR seemed to be closing in on closure, another front in the fight opened up.

The European Patent Office moved to hand a broad range of patents over to the University of California and its CRISPR compatriots. Now come the so-called opposition proceedings, in which third parties can challenge those patents. And the Broad Institute, arguing for its own gene editing provenance, will almost certainly mount such a challenge, thereby extending the process further.

If there is an upshot to this contentious, plodding, and now transatlantic fight, it is perhaps this: The more tangled this all gets, perhaps the more likely it becomes that the parties strike up a licensing agreement that will allow us all to move on.

More consumers feeling the prescription drug pinch

There are three ways that people pay for their medications: Through copays, coinsurance, and deductibles. 

And folks using the latter two options are seeing their out-of-pocket drug expenses rise, because health plans and PBMs aren't negotiating down costs as they do with the copays, according to a new report from PhRMA. 

We've got the breakdown exclusively for STAT Plus subscribers. One notable finding: About 34 percent of patients pay for high-cost, specialty drugs with coinsurance and deductibles — and thus are getting a bit of a raw deal.

Read more.

A reproductive system, in miniature

(hyacinth empinado / stat)

It’s called the “Evatar” — a portmanteau of “Eve” and “avatar.” Which makes sense: the hand-sized device is a working facsimile of the female reproductive system.

The Evatar’s basically a five-organ organ-on-a-chip, holding bits of ovarian, fallopian tube, uterine, cervical, and liver tissue, and can simulate a 28-day menstrual cycle.

Read more.

More reads

  • Vertex is heading to the FDA with its next-generation cystic fibrosis therapy on the strength of new clinical data. (TheStreet)
  • FDA nominee plans recusals from decisions on many drug firms. (Wall Street Journal)
  • At the same time, Vertex is consolidating its R&D footprint, shuttering a research site in Canada. (Endpoints)
  • Senator Joe Manchin, father of embattled Mylan CEO Heather Bresch, says drug companies like hers are "getting the living crap beat out of them" for high prices. Not that he's defending the prices. (STAT Plus)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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