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The Readout Damian Garde

Happy Monday! If you're a STAT Plus subscriber and you missed our event last week, you can watch it here.

How Alzheimer's patients are faring after trial blowup

The failure of closely watched Alzheimer’s trials late last month was a blow to Biogen and its partner Eisai. But it’s been downright devastating for some patients who had pinner their hopes on the possibility that the experimental drug aducanumab might allay some symptoms of their neurodegenerative disease.  

Biogen was legally obligated to publicly announce that the trials would be halted. So participants went to bed one night thinking their drug might be working — and woke up to news to the contrary. It felt “like the rug was pulled out from under us,” one patient told STAT's Andrew Joseph. “I had this overwhelming sense of dread, almost like a hopelessness,” said another.

Read more.

Pancreatic promise from the Parker Institute

The Parker Institute for Cancer Immunotherapy, launched in 2016 with $250 million from Silicon Valley billionaire Sean Parker, has unveiled its very first results — and they look promising. 

The Phase 1 trial showed tumor shrinkage in metastatic pancreatic cancer, thanks to a multi-drug regimen administered to patients with the disease. The patients were treated with a combination of gemcitabine and Abraxane — commonly used for pancreatic cancer — and an experimental antibody called APX005M. Half were also given the checkpoint inhibitor Opdivo. 

A little more than half of the 24 evaluable patients showed a partial reduction of their respective tumors — with the best responses observed in patients treated with all four of the drugs. 

Read more.

Ironwood is branching out into rare disease

Ironwood Pharmaceuticals, the Cambridge biotech behemoth known for drugs that treat gastrointestinal disorders, is now spinning off a new startup in the rare disease space. GI drugs may be losing a bit of their lusters, according to analysts, whereas the rare disease space remains lucrative.

The new company, called Cyclerion Therapeutics, has five drug development programs in the works — working on sickle cell disease and other life-threatening, but uncommon, diseases of the central nervous system, liver, and lungs. 

The startup is kicking off with a strong start: About 140 employees will move over to Cyclerion from Ironwood.

Read more. 

Questioning the efficiency of surrogate endpoints

Dr. Vinay Prasad is tackling surrogate endpoints yet again — this time in JAMA Internal Medicine. Surrogate endpoints, of course, are results that show that a drug has some impact — tumor shrinkage, for example. Drugs are often approved on this basis, because it’s generally more efficient. But Prasad’s whole platform has centered around pushing for analysis of overall survival — studying, instead, how patients feel or how long they live. This takes more time. 

But not that much more time, according to the new paper: “Any way you slice it, the time saved by surrogates is not that much,” Prasad and his co-authors argue. It takes about 12 percent longer to conduct a trial that uses overall survival as an endpoint, instead of surrogate endpoints — or about 11 months, when placed in the context of the average development time of cancer drugs, which is 7.3 years.

More reads

  • It's a difficult time for the 857 people in the U.S. named Elizabeth Holmes. (Business Insider)
  • Can genetic testing help doctors better prescribe antidepressants? (Washington Post)
  • New drugs that unleash the immune system on cancers may backfire, fueling tumor growth. (Science)

Thanks for reading! More tomorrow,

Damian

Monday, April 1, 2019

STAT

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