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The Readout Damian Garde

When clinical trials try to charge patients


The mainstream research community has long been wary of clinical trials that ask patients to foot the bill. Now, there's a perception that these studies, which charge four- to six-figure fees, may be on the rise — and they've caught the attention of the FDA and NIH.

A federal advisory panel is now drafting recommendations to help institutional review boards, researchers, and patients think about such studies. As STAT's Rebecca Robbins reports, they're trying to strike a balance between protecting patients and the integrity of research — while not ruling out the possibility that such studies could be valuable in some circumstances.

But that comes with its own risks. “If you open the door to these trials, you inevitably are going to get … exploitative trials, and bad trials, and trials that don’t lead to any useful information,” said Dr. Steven Joffe, a pediatric oncologist and bioethicist at the University of Pennsylvania.

Read more.

The unkillable vampire approach to Alzheimer’s disease


It has endured skepticism, controversy, and outright mockery. And now the idea that the blood of the young can benefit the aged has, according to one company, demonstrated a benefit for Alzheimer’s disease.

According to Alkahest, an experimental protein cocktail derived from young blood plasma helped patients with Alzheimer’s maintain their performance on tests of cognition and function. For now, you’ll have to take Alkahest’s word for it: The company isn’t presenting full data from the Phase 2 study until the CTAD conference in San Diego in December.

That presentation will be closely watched in the nascent field. Alkahest is taking a traditional biotech approach to an idea that has captured the imaginations of screenwriters, questionable startups, and concerned regulators alike.

Read more.

How will Regeneron grow?


Later this morning, Regeneron Pharmaceuticals will disclose its second-quarter earnings, and, because of the company’s partnerships with a pair of European pharma companies, we already know a bit about sales of its two biggest products.

What we don’t know is what the narrative will be. The Wall Street consensus is that Regeneron will report earnings of roughly $5 per share, which happens to be nearly identical to what it made in the second quarter of 2018.

Dupixent, the company’s treatment for a growing number of immunological diseases, brings in more money each quarter, but Regeneron, storied for its investment in research, spends more and more on science. If the company ends up reporting flat year-over-year earnings, investors are likely going to want to hear what the plan is for turning that commitment to R&D into actual profits.

A reminder of why biotech investing is difficult


Going into yesterday, the biotech company Allakos had a short interest of about 37%, which means 37% of its outstanding shares had been borrowed and sold short by people betting the company’s stock would fall. For reference, the average short interest among S&P 500 companies is generally around 2%.

The skepticism stemmed from AK002, Allakos’s in-development treatment for a rare stomach disease. An earlier drug, focused on the same biological target, failed in a small trial. Furthermore, AK002 is meant to reduce certain white blood cells, but prior studies with other treatments found no relationship between doing so and improving symptoms. This, to a great many people, was reason enough to bet AK002 would fail in a mid-stage trial.

And then it didn’t. The drug met all of its efficacy endpoints in a 65-patient trial, Allakos said yesterday, including the key metric of patient-reported symptoms. Allakos’s stock price more than doubled on the news, which was an unpleasant development for those who’d shorted the stock. 

More reads

  • Senate bill’s drug pricing provision raises industry alarms. (Roll Call)
  • Close to a decade after Eli Lilly killed it, a 'breakthrough' drug is back and being steered to the FDA. (Endpoints)

Thanks for reading! Until tomorrow,

Megan

Tuesday, August 6, 2019

STAT

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