| | | |   | Hello, everyone. Damian here with a reminder that if you've ever endeavored to keep tabs on the maddening pace of clinical trials, you might want to tune in for a live event at 1 p.m. ET today in which my colleague Matthew Herper will show off the capabilities of STAT Trials Pulse, our new tool for tracking medicines in development. Sign up to tune in. | | | 30 months and countless debates later, Biogen published its Aduhelm data Among the lengthy list of complaints about Aduhelm is that despite the drug’s Phase 3 trials concluding in 2019, Biogen never got around to publishing the resulting data in a peer-reviewed journal. Yesterday, that changed. But whether the long-delayed manuscript can change the increasingly heated debate around Aduhelm remains an open question.
As STAT’s Matthew Herper reports, the focus has been less on the data and more on where they appear. Biogen published the Aduhelm results in the Journal of the Prevention of Alzheimer’s Disease, a little-known publication whose impact factor, a measure of scientific relevance, is 4.5. By contrast, the New England Journal of Medicine has an impact factor of 91, and the Journal of the American Medical Association, from which Biogen pulled its study from consideration last year, clocks in at 56.
Meanwhile, Biogen remains at the mercy of the agency that oversees Medicare, which has proposed a policy that would strictly limit coverage of Aduhelm. A final decision is expected next month, and unless it is significantly loosened, Biogen’s Alzheimer’s treatment will likely remain a commercial nonentity.
Read more. | Stocks sometimes go up, too Apellis Pharmaceuticals managed a rare feat in today’s biotech bear market: announce positive clinical trials results that also boost the stock price.
The trigger for the 13% jump in Apellis’ stock price yesterday was an update from two identical clinical trials showing a durable benefit for the company’s experimental treatment for geographic atrophy — a chronic eye disease and a leading cause of blindness in older people.
Last September, a 12-month readout revealed confounded results: One study showed the company’s drug slowed the progression of the disease; the other did not by a narrow margin. Apellis shares sank on investor concerns that mixed data lessened the odds of approval.
But Apellis took another look at the twin studies with 18 months of follow up. The benefit seen in the positive study proved durable, while the formerly negative study switched positive. The statistical rigor of said reversal is debatable, but investors interpreted the updated results as more supportive of approval than previously thought. And that’s how a beaten-down stock goes up, even in a terrible market. | STAT Trials Pulse redefines clinical trials intelligence with a new tool powered by artificial intelligence and STAT’s biotech team STAT and AI company Applied XL have partnered to develop a new clinical trials intelligence platform, STAT Trials Pulse. Combining machine learning and feedback from STAT’s trusted newsroom, the platform allows you to monitor the clinical trials landscape and surface critical insights and trends more easily than ever before. Learn more at statnews.com/trials-pulse, or skip the details and start your four-week free trial at trials.appliedxl.com. | A cell therapy for the brain With the colossal caveat that we’re about to talk about mice, researchers have found a novel use of cell therapy that could replace the brain’s molecular janitors and thereby clean up the messes that cause Alzheimer’s disease and other disorders.
As STAT’s Jonathan Wosen reports, the idea relies on microglia, immune cells that patrol the brain for anything that doesn’t belong and dispose of it accordingly. When microglia are mutated or otherwise defective, cerebral trash piles up and neurodegenerative disease arises, suggesting that putting them back to work could be a way to treat a multitude of disorders.
A team led by Stanford University scientists came up with a way to do it. They gathered some mice genetically engineered to produce mutated microglia, gave them a stem-cell transplant and then administered a chemical to clear out their existing microglia. The stem cells grew into healthy microglia, and the treated mice had better balance, movement, and survival than a control group.
Read more. | On drug pricing, who’s ‘misleading’ whom? Depending on whom you ask, the cost of medicine in the U.S. is ever-escalating despite drug industry rhetoric, or on a gradual decline despite political grandstanding. This week we got the case for both claims — plus dueling accusations that the other side is “misleading” the public.
As STAT’s Ed Silverman reports, a group of senators pointed out that the list prices for some top-selling drugs rose by an average of 5% in January and asked if the global pharmaceutical industry would like to explain why. PhRMA, the trade group, responded that the lawmakers used “flawed data” to draw “fundamentally misleading conclusions” and, actually, net prices are on pace to decrease by as much as 3% through 2025. Lawmakers called that “misleading information” and concluded “there is no good explanation for drug manufacturers’ behavior other than corporate profiteering.”
The backdrop for all this was yesterday’s Senate Finance Committee hearing, which covered all of the familiar arguments and revisited a bunch of familiar statistics. Similarly familiar is the widely held doubt that Congress will pass meaningful laws on the matter any time soon, despite the bipartisan popularity of legislation targeting the price of drugs.
Read more. | More reads - With infections spiking in Europe and a variant on the rise, experts warn the U.S. could face a Covid resurgence. STAT
- Merck pauses Russia investments, to supply essential medicines. Reuters
- A battle is brewing between a senator and a nonprofit over pharma patenting practices. STAT+
- 'Biotech sisterhood': 25 female CEOs find new ways to 'sponsor' the next generation at Arizona retreat. FierceBiotech
| Thanks for reading! Until tomorrow,  | | | |
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