Wednesday, August 17, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, your daily source for all things biotech. Follow us on Twitter for more: @damiangarde@megkesh, and of course, @statnews.

How biotech companies deal with ever-present failure

They can't all be Sovaldi. Or even Afrezza for that matter. (MOLLY FERGUSON/STAT)

Drug companies love to tout how hard it is to be a drug company. The fact that most projects fail is a fundamental datum in biopharma, one that executives use to defend the increasingly high costs of drugs that don't.

But what about the researchers toiling away on those probably not-going-to-work treatments? In order to maintain morale, their managers reward their noble failures, handing out plaques, ceremonial vials, and flutes of champagne to commemorate the many drugs that weren't.

Read more.

Speaking of drugs failing

Robert Plenge, Merck’s head of translational medicine, wrote an authoritative piece in Science Translational Medicine that tries to pull back the curtain on why biopharma R&D is so inefficient. 

In essence, he concludes that drug companies need four things to maximize their odds of success: a validated biological target, a molecule that can actually affect it, a reliable way to test whether it’s working, and a trial design that will get an answer quickly.

That all makes plenty of sense, and it has been put into practice to great success in the rare disease space, as one example. 

But as Derek Lowe notes in a blog post, what of the big unmet needs that don’t fit into that box? Following Plenge’s rubric, no one would endeavor to develop drugs for Alzheimer’s disease, for instance, because few agree on how it even works, and fewer agree on how best to target it. 

To be fair, Plenge is not advocating for a Hammurabian code of clinical trial conduct, and he acknowledges that there are no surefire paths to success. But one wonders: If cash-conscious biopharmas go all out to minimize risk, who will take the home run swings on the big, baffling diseases that have long bedeviled the industry?

In case you needed more proof that pharma's peaked

The biopharma bubble burst, in bar graph form. (EP Vantage)

We’re far from “plunging into the depths of the last recession,” a new report from EP Vantage says, but biopharma and medtech are past their prime, with little chance of returning to last year’s highs anytime soon. Drug pricing, of course, has spooked investors — who are also contending with a slowdown in the Chinese economy and lackluster financial environment in the west. 

And, thanks to a drop in early stage investments for medtech companies, the broader device sector continues to perform staggeringly worse: 

Happy Wednesday.

Taking on socially unacceptable 'underwear issues'

Remember orlistat? That's the weight-loss pill that burst through the gates in the 2000s before being dragged down by a reputation for unpleasant gastrointestinal side effects.

The drug, still sold over the counter as Alli, was back in the news yesterday, courtesy of a new analysis. The upshot: The 1990s-era clinical studies of the drug funded by Roche drastically understated reports of adverse events like diarrhea or incontinence in the resulting journal articles.

But there's new hope for patients holding out on the pill's promise: M Pharmaceutical, a Canadian company, is working on a new formulation of orlistat. They announced yesterday that they'd finished designing a staggered delivery mechanism for the drug's ingredients.

The goal, as the company described it in a phrase you don't see often in press releases: To help patients shed pounds while avoiding the existing pill's "socially unacceptable 'underwear issues.'"

Ketamine-like depression drugs inch closer to market

Johnson & Johnson won a second FDA Breakthrough Therapy Designation for its ketamine-esque treatment for serious depression, giving the company a shot at an accelerated approval and bringing a new class of drugs that much closer to market. 

Ketamine, an oft-abused anesthetic, has for years shown promise in relieving the symptoms of depression, but side effects including psychosis and dependence have stymied its potential. Enter biopharma, which, seeing a potentially lucrative new class of medicines, has endeavored to strip the drug of its undesirable properties and come up with molecules that ward of depression without ill effects.

J&J’s offering is a nasal spray called esketemine, and it has shown promise in treatment-resistant depression and major depressive disorder with imminent risk for suicide. Allergan is moving forward with a similar treatment called rapastinel, an intravenous ketamine derivative that has also secured the FDA’s breakthrough tag. Each is now in late-stage development.

More reads

  • The cost of fighting over the rights to CRISPR gene editing technology is getting expensive, and biotech companies are footing much of the bill. (STAT)
  • Biotech CEOs are more worried about whether they can raise money than whether their drugs work, according to a survey. (Kineticos)
  • OncoGenex is exploring "strategic alternatives" after its latest drug failure. (Reuters)
  • Chiasma, reeling from an FDA rejection, is laying 44 percent of its employees to conserve cash. (Press release)

Correction: Tuesday's Readout misstated how much money Hillary Clinton and the PACs that supporter had raised from the health care industry. It is $3.9 million, not $3.9 billion.

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Thanks for reading! Until tomorrow,

Damian & Meghana

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