| |   | Good morning, all. Damian here with the latest from a closely watched Alzheimer's disease conference, news on the solvency of a gene therapy pioneer, and why drug pricing data is getting harder to learn. | | | Roche’s Alzheimer’s drug had an amyloid problem Specifically, it didn’t remove enough of it.
That was the major takeaway from last night’s post-mortem on gantenerumab, an Alzheimer’s disease treatment from Roche that failed to beat placebo in a pair of pivotal trials. In a detailed presentation, Roche explained that gantenerumab led to lower-than-expected reductions in amyloid, the toxic brain plaque thought to drive disease progression.
The release of the data came one day after a presentation about a different medicine, Eisai and Biogen’s lecanemab, which showed dramatic effects on amyloid levels and led to a modest but statistically significant delay in Alzheimer’s progression. Viewing the studies side by side, some researchers concluded that investigational medicines might need to clear out substantial amounts of amyloid in order to show any benefits for patients.
“We see that if you move enough amyloid, you get some kind of clinical effect,” said Rachelle Doody, a neurologist who leads Alzheimer’s research at Roche. “If all the stars line up, you may get the effects you hypothesized and have a successful trial. But you can also have a kind of success in executing your experiment well.”
Read more. | Bluebird gets some much-needed cash Bluebird’s bid to fight another day got a boost Wednesday, as the company announced it sold a priority review voucher to the biotech Argenx for $102 million. The voucher, which allows a company to get expedited review of any drug at the FDA, was one of two Bluebird won for getting two gene therapies approved for rare diseases that affect children. The company had already telegraphed it would look to sell the vouchers in an effort to patch up a bleeding balance sheet, as the biotech once valued at several billion dollars has faced cuts and layoffs.
The infusion will nearly double the amount of cash Bluebird has in reserves, but it’s not clear yet exactly how executives plan to spend it or what the company’s future holds. Although a third gene therapy, for sickle cell disease, could be approved as soon as next year, the company has only one other disclosed program and it’s not clear if any of its three gene therapies will generate significant revenue. | STAT in NYC: A Look Ahead at Biotech in 2023 Join STAT’s biotech team for our upcoming in-person event on December 5. They’ll sit down with major players in biotech and take a look at the coming year, and take the temperature of the big regulatory and financial issues facing the pharmaceutical industry. Stay once the conversation is complete for a networking and cocktail reception with STAT and your fellow attendees. Get your ticket now — STAT+ subscribers can join for free! | Stanford's scientific scandal touches biotech, too This week brought science news from the academic world, with Stanford announcing it had opened a research integrity investigation into its own president, Marc Tessier-Lavigne, over allegations of manipulated images in a number of papers. But biotech insiders probably recognized Tessier-Lavigne's name from his long ties to the industry.
Most notably, Tessier-Lavigne was a top researcher at Genentech starting in 2003, eventually becoming chief scientific officer. Tessier-Lavigne, a onetime Stanford and UCSF professor, returned to academia in 2011, but he's stayed connected to the industry as well. A neuroscientist, Tessier-Lavigne co-founded Denali Therapeutics, which is developing medicines for neurodegenerative disorders, and serves on the board both at Denali and at Regeneron Pharmaceuticals. He also was formerly on the boards of Pfizer, Agios Pharmaceuticals, and Juno Therapeutics.
In a statement, Tessier-Lavigne said: “Scientific integrity is of the utmost importance both to the university and to me personally. I support this process and will fully cooperate with it, and I appreciate the oversight by the Board of Trustees.”
Read more. | A little-known, oft-cited data broker is making drug prices harder to access For years, a division of IBM provided journalists with highly sought data on drug price increases, seeding news stories that stoked backlash against certain companies. Now, concerned that providing such information might alienate its pharmaceutical clients, that data provider is shielding pricing data from the public.
As STAT’s Ed Silverman and Casey Ross report, the company is called Merative, formed from the ashes of IBM’s Watson Health division. Breaking with its predecessor, Merative will no longer provide the media with pricing changes for specific medicines. This includes details such as separate doses and packaging on both current and historical prices.
“Ultimately, they are able to provide whatever information they would like, so (we) can’t fault them if they’re making a business decision,” said Walid Gellad, who heads the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh. “But it’s been stories about large drug price increases that have driven the national conversation around drug pricing, so loss of this information is a net loss for the public.”
Read more. | More reads - Elon Musk says Neuralink’s brain-machine interface could enter human studies next year, STAT
- Covid evolution wipes out another antibody treatment, threatening the country’s medicine cabinet, STAT
- Japanese biotech firm uses tiny worms in test for pancreatic cancer, Reuters
| Thanks for reading! Until tomorrow,  | | | |
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