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The Readout

Biogen's Alzheimer's data look promising. Now what?


The much-anticipated data from a large clinical trial in Alzheimer's disease were unveiled at a scientific meeting in Chicago yesterday, and they did not disappoint: Patients who got the highest dose of an experimental drug, called BAN2401, performed 30 percent better on a cognitive test than those getting placebo after 18 months. That could set the stage for the drug's makers, Biogen and Eisai, to try to seek an early approval, which would transform a field littered with failure after failure.

Keep in mind, though, that Biogen's data come with caveats. The Phase 2 trial failed its main goal. And the metric that researchers used to measure mental acuity was developed internally and has never before been used to win FDA approval. Those factors raise some big questions: Will BAN2401 be subject to another larger study? And will the FDA be willing to lower its traditionally high bar for approval in the field?

Read more.

And for still more, STAT Plus subscribers can dial into a call at 10 a.m. today. Dr. Howard Fillit, chief science officer of the Alzheimer’s Drug Discovery Foundation, will answer questions about the results, their implications, and what to expect next.

Christmas in July for at least one small biotech 


What do you call it when the drugs you're developing for a rare form of some disease suddenly look like they might help every form of the disease? A 30-fold increase in the size of your potential market, the lead scientist in a new study of Parkinson's disease told STAT.

About 3 percent of Parkinson's patients have an inherited form, caused by a mutation in the LRRK2 gene that results in a mutant, hyper-activated kinase of the same name (pronounced "lark 2"). But research published on yesterday suggests that many, and maybe all, cases of Parkinson's (1 million in the U.S.) have hyper-activated LRRK2, even when the gene isn't mutated.

Denali Therapeutics has two LRRK2 inhibitors in Phase 1 clinical trials. If either works, the company might have not a niche drug but a blockbuster.

Read more.

Sure, call it a positive study


Throw enough darts and you’re likely to hit the bullseye at least once. That doesn’t make you a barroom shark, it only makes you lucky.

ResTORbio was yesterday's dart thrower. As explained by STAT's Adam Feuerstein, the small biotech claimed victory in a mid-stage clinical trial of a drug for respiratory tract infections. But a deeper read into the details of the study reveals more luck than skill.

A low dose of the ResTORbio drug, called RTB101, did not work. The highest dose of the drug did not work. A combination of RTB101 with another drug did not work. The middle dose of RTB101 was found to be effective but only after ResTORbio pooled together patients from two separate parts of the study and used a lower statistical bar that would not pass muster with regulators.

But sure, call the study positive.

Read more.

Health care bigwigs find a landing pad in Google


What do a former FDA commissioner, a former director of the National Institute of Mental Health, and former heads of the Cleveland Clinic and the University of Utah health system have in common?

They've all recently moved out of power and into gigs at Google companies — which are emerging as the landing pad of choice for big names in health care. That's a marked shift from not so long ago, when a cozy perch at a university was the usual post-power destination. STAT's Rebecca Robbins explains why

Sarepta's DMD trial on hold 

 
A trial for Sarepta Therapeutics’  otherwise promising, early-stage gene therapy for Duchenne muscular dystrophy has been put on a clinical hold, the company announced. The FDA told Nationwide Children’s Research Institute, where the trial is being conducted, to pause the trial.
 
Though the lot of plasmids that was tested wasn’t used in patients, four people did get a dose from a prior lot from the same supplier. However, no side effects have been reported and no trace of the DNA fragment was found in biopsies. 
 
To fix the problem, Sarepta CEO Ingram said the company would be switching from research-grade to cGMP-grade plasmids. If the FDA approves of the company’s plan to address the issue, Ingram said Sarepta’s plans to continue to a pivotal clinical trial later this year might still hold.

Read more.

More reads

  • Gilead’s golden era ends with the executive departures of ‘The Johns’ (STAT)
  • GlaxoSmithKline’s big R&D idea? Focusing on the immune system (FierceBiotech)
  • Making personalized cancer vaccines takes an army — of robots (Wired)

Thanks for reading! Until tomorrow,

Thursday, July 26, 2018

STAT

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