Wednesday, June 21, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.

A lesson in messaging from a biotech lifer

Dr. David Hung, the famed new Axovant Sciences CEO, is in a bit of a strange position.
His company is “keeping all of [its] fingers and toes crossed” that intepirdine, a treatment for Alzheimer’s disease, will succeed in a Phase 3 trial slated to read out in September, he said. It would be a major validation for Axovant and likely make for the first approved Alzheimer’s drug in 15 years.
But intepirdine is not a cure for Alzheimer's, and it’s not a therapy that can reverse the disease’s effects. Hung knows that, and that’s why the plan at Axovant is to eventually pursue “more risky, more futuristic approaches” to Alzheimer’s, he said at BIO yesterday in one of his first public appearances since taking the Axovant job in April.
It's a delicate line to walk. Hung can’t publicly characterize intepirdine as an incremental advance (as some scientists have). But he also has to make clear that Axovant has ambitions to far surpass it in the long term (and is therefore worth a long-term investment).
So he looked to baseball.
“If [the trial] hits, we're going to be swinging for more fences,” Hung said. “We don’t need more base hits. Life’s too short for that. We need more home runs.”

Which would make intepirdine, like, a solid double? Or a whiff. We'll know soon.

What will Trump's FDA look like?

Is the FDA under new Commissioner Scott Gottlieb willing to be flexible with the rules, and maybe even lower regulatory standards, to approve new drugs? It’s a question that will be partially answered in the coming days when the FDA hands down a decision on betrixaban, a new anticoagulant developed by Portola Pharmaceuticals.

Portola is asking the FDA to approve betrixaban to reduce the risk of dangerous blood clots in patients who are leaving the hospital after treatment for a medical (as opposed to a surgical) illness. The betrixaban clinical data, however, are messy. The drug failed to significantly reduce blood clots, when compared with placebo, in its sole phase 3 clinical trial. Normally, a negative study would stall an FDA submission, but Portola pushed ahead anyway.

Adam Feuerstein examines the case for and against betrixaban's approval — and the broader implications.

Read more on STAT Plus.

FDA's speedy new generics guidelines

Speaking of FDA.... The agency just laid out new guidelines to expedite access to generic drugs. Applicants need only notify FDA two months before a submission to obtain a priority review and shave two months off the approval timeline. That is, of course, if the generic in question is deemed worthy of priority status.

Giving state drug pricing reforms a helping hand

Tired of waiting for congressional action that may never come, states from New York to Nevada have been experimenting with ways to hold down drug prices. Now, a new center — funded by the Arnold Foundation — has been launched to help them. 

States are “desperate” to reduce the budget burden of high drug prices, Dr. Rebekah Gee, secretary of the Louisiana Department of Health, told STAT. The Trump administration made noise about stepping in, “but that interest seems to have dwindled, so it’s up to states to drive the conversation,” she said.

Read more on STAT Plus.

Meanwhile, at Mylan

Change could be afoot at America's favorite generics company.

On Thursday, Mylan shareholders will convene to decide the fate of the company's controversial management team, which has presided over headline-grabbing price hikes while handsomely rewarding itself with bonuses and stock grants.

But as STAT's Ed Silverman notes, this is hardly a textbook shareholder revolt. A group of major pension funds has publicly called for a regime change at Mylan, and the influential Institutional Shareholders Service has recommended the same. Mylan however, may hold a winning hand as it, requires a two-thirds majority vote from shareholders to unseat any board member.

Read more.

More reads

  • Pricing pressure and patent expirations force a drop in projected drug sales. (STAT Plus)
  • A private equity firm is buying CRO giant Parexel for $4.5 billion. (Reuters)
  • FDA commissioner vows to eliminate backlog of orphan drug designation requests. (RAPS)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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