Thursday, August 25, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, your daily source of biotech news and analysis. For more, visit STAT's home page and follow us on Twitter: @damiangarde@megkesh, and @statnews.

Regrowing that severed toe

horror movie, or science experiment? (matthew orr / stat)

The human body has an extraordinary capacity for regeneration: Take STAT’s own Megan Thielking, our Morning Rounds scribe, whose tonsils managed to grow back despite a childhood tonsillectomy. 

The rest of us can self-mend, too: Our broken bones fuse back together, and our paper cuts heal with new skin. What we can’t do? Regrow a severed toe. 


Scientists are studying a species of salamander, called the axolotl, that has astonishing abilities to regenerate bones, muscles, nerves and blood vessels — all in the right places. 

STAT’s Carl Zimmer explores the gene expression of the humble axolotl, speaking with researcher Jessica Whited about the key genes axolotls turn on to build out a new limb from scratch.

Watch his new Science Happens! video here.

Is the FDA going to decide on Sarepta’s drug soon?

Investors seem to think so. The Cambridge company’s share price has risen as much as 14 percent since last week on the expectation that regulators will soon hand down a decision on its treatment for Duchenne muscular dystrophy.

It has been a long, frenetic ride for Sarepta at the FDA, marked by regulatory delay, emotional testimonials from patients, and pressure from elected officials. The company’s treatment has shown glimpses of promise in small studies, but Sarepta has yet to complete the randomized clinical trials usually required for approval. 

In a move widely seen as a compromise, the FDA has asked for interim data showing whether Sarepta’s drug can increase production of the protein dystrophin, which is lacking in patients with DMD. Wall Street analysts expect Sarepta to submit that information by the end of this month, with an FDA decision following shortly thereafter. Just what sort of results will be needed to secure approval remains a mystery, and neither the FDA nor Sarepta is commenting on the matter.

@HillaryClinton, mover of markets

Hillary Clinton's Twitter account is more powerful than any clinical trial failure. (@adamfeuerstein)

It happened again. Hillary Clinton railed against drug price hikes, and biotech stocks took a spill. This time, the Nasdaq biotech index fell 3.5 percent after Clinton called out Mylan and its many price hikes on the life-saving EpiPen in a lengthy statement and a pithy tweet.

It’s an ominous echo of September, when Turing Pharmaceuticals and Valeant Pharmaceuticals were the industry’s bete noirs, and a tweet from Clinton sent the same index down about 5 percent. That one-day drop signaled the beginning of tough times for biotech stocks, as investors panicked that the industry’s profitability might come under attack.

The market has slowly rallied since then, but does Clinton’s latest tweet signal that biotech is headed back for the trough?

Taking the grassroots approach to drug development

Looks like a nonprofit is proving that sky-high drug prices could be… unnecessary. 

The Switzerland-based Drugs for Neglected Diseases Initiative has earned regulatory approval for six treatments, including sleeping sickness, malaria, and Chagas disease, and has another 26 in development. And it’s gotten to this point with just $290 million. That's a far cry from the $1 billion figure typically batted around as the cost of developing a drug stateside.

DNDi is, of course, developing drugs for diseases that impact the world’s poorest people and are, in turn, generally neglected by the global biopharmaceutical industry — so, naturally, there’s less competitive pressure. These kinds of drugs can also have easier approval pathways. Still, it’s an impressive model that’s worth taking a hard look at — especially in these days of Shkrelis and Valeants and monopolizing EpiPens

“We can't match our financial figures one to one,” executive director Bernard Pécoul told Nature. “But we believe that DNDi can demonstrate that a different model is possible for R&D.”

Read more.

Pfizer's newest blood thinner play

Move over, Warfarin: A Pfizer-UCSF effort has developed an antibody that could stop blood clots from forming — and a partner drug that could reverse its effects in case dangerous bleeding occurs, which, of course, is one of the risks of taking a blood thinner. Testing is still preclinical, but it’s interesting:

The compound targets FXIa, a protein that’s key to the formation of blood clots. Tests didn’t show that the drug ran the risk of causing excess bleeding, according to a new paper in Science Translational Medicine, but the research team developed an antibody to reverse the action of the medication if the problem does crop up. 

More reads

  • FDA rejects Amgen's hyperparathyroidism drug. (Endpoints)
  • Martin Shkreli is long gone, and Turing Pharmaceuticals is still working to make a better treatment for toxoplasmosis. (Forbes)
  • Jump starting the brain: Experimental device suggests new path for rousing patients from comas. (STAT)
  • Five blunders that many biotech startups make on the road to clinical trials. (STAT)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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