Friday, September 23, 2016

The Readout by Damian Garde & Meghana Keshavan

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

The race for new migraine therapies

The millions of Americans with frequent migraines have few viable treatment options, left to pick between drugs with middling efficacy and repurposed therapies with heavy side effects.

But a new crop of injected treatments offers some hope — and could jump start a multibillion-dollar market. Four companies are racing into late-stage trials with novel therapies that aim to disrupt a protein called CGRP, which plays a role both in pain signaling and in the dilation of blood vessels in the brain. If all goes well, the first CGRP therapy could come to market by 2018.

Read more.

(Arguably) bad news for biotech’s favorite millennial

Axovant Sciences CEO Vivek Ramaswamy, seeking to build “the Berkshire Hathaway of drug development,” is facing some fresh skepticism. (Roivant)

Axovant Sciences and its 30-year-old CEO became the talk of biotech last year, turning a twice-failed treatment for Alzheimer’s disease into a company worth nearly $3 billion upon its IPO. But the failure of a drug that looks an awful lot like Axovant’s has spooked investors.

Yesterday, Danish drug company Lundbeck revealed that its Alzheimer’s candidate showed “weak efficacy” in a late-stage trial, failing to meaningfully beat out a placebo in improving cognition and function.

Here’s where Axovant comes into play: Lundbeck’s drug is designed to block the brain’s 5-HT6 receptors and release a chemical called acetylcholine, which is believed to improve mental function. Axovant is chasing the same target, and thus Lundbeck’s failure wouldn’t seem to bode well for Axovant’s highly anticipated late-stage results, expected in the middle of next year. The company’s shares fell as much as 26 percent on Wednesday.

But Axovant fans (Axofans?) don’t see much to worry about. Heading into Phase 3, Lundbeck had to lower the dosage of its drug to avoid liver toxicities, a move that might have set it up for failure. Axovant, by contrast, hasn’t had that problem. And analysts from Baird, RBC, and Evercore/ISI remain bullish on its future.

Like astronaut ice cream, but drugs

mr. wizard would have a field day with this (wyss institute / harvard university)

It’s called the Freezemobile — and it’s not some Batman arch villain mode of transport. Rather, it’s a portable contraption that allows for delicate, shelf-unstable biological drugs to be transported into the remotest corners of the developing world.

Problem is, constant drug refrigeration via the Freezemobile isn’t all that practical. Instead, researchers at Harvard, MIT, and University of Toronto have proposed a possible solution: Freeze-drying the ingredients it takes to make a drug — so that when they reach their destination, all folks have to make their own drugs is just add water.

“It’s essentially how you make ramen,” Peter Nguyen, who co-authored a paper on the subject, told STAT.

Read more. 

About those rah-rah press releases...

Biotech companies always seem to have such good news. But trial results are often a lot less rosy if you know what to look for.
Enter Adam Feuerstein, journalist and STAT-proclaimed most feared man in biotech, who offered a master class in puncturing hype yesterday in an illuminating column for The Street. His case study: a misleading press release from Maryland biotech Northwest Bio highlighting new trial data on its experimental personalized cancer vaccine. Feuerstein points out a few things that set off his BS detector:

  • Statistics that group together a bunch of patients with different cancer types
  • No context on which and how many treatments patients have exhausted before entering the trial
  • An arbitrarily-defined group of top-faring patients — without any info about how their peers did

To Feuerstein's excellent list, we'll add a few more things that set off alarms for us when we read press releases: Analysis of how a narrow subset of patients fared. No mention of whether a trial achieved its pre-determined goal.

And the phrase "trend toward efficacy."

Time to get mildly annoyed, SF readers

Lest the overwrought biotech rivalry between Boston and San Francisco begin to wane, we’ve decided to fan the flames just a bit. The life sciences outlook report from real estate services firm JLL is out, and for the fifth year running, Boston’s the top biotech cluster in the country. San Francisco takes second place.

Of course, JLL’s looking at all of this from a real estate perspective — but its metrics do include life sciences employment concentration and growth, venture funding, and NIH support, along with the cost and availability of space.

According to those metrics, Boston’s still walloping San Francisco — though rent’s on the rise in both. North Carolina, San Diego, and Seattle were cited as the next biggest hubs. Here’s a nice little infograph of biotech venture funding, by region: 

Boston biotech is pretty cash rich, that's for sure. But that $3 billion from Mark Zuckerberg this week might help even things out a bit... 

More reads

  • After Allergan's head-turning acquisition of Tobira, it's worth taking a look at how other big-premium deals have fared. (EPVantage)
  • How the FDA manipulates the media. (Scientific American)
  • Uniqure's CEO resigned after just nine months at the company, a decision "due solely to personal family reasons," he said. (Press release)

Have a news tip or comment you want to send us?

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Thanks for reading! Until tomorrow,

Damian & Meghana

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