Oncology
Bringing CAR-T back to kids
The early promise of CAR-T cancer therapy stemmed from its potential to treat dire cases of pediatric cancer. Now, due to scientific hurdles and economic incentives, the majority of CAR-T therapies are used on adults. Brainchild Bio, a newly launched startup, is working to steer the technology back to its founding ideals.
As STAT's Jason Mast reports, the company is working on a series of CAR-T medicines aimed at pediatric tumors of the brain and central nervous system, all based on the work of Mike Jensen, a pediatric oncologist at Seattle Children's.
Unlike most companies, Brainchild will start in children and only later move into the larger and more lucrative adult indications. Jensen, who will serve as chief scientist, said the company can defer its work on adult cancers in part because Seattle Children's is funding its first two years of operation.
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M&A
The half-empty take on a billion-dollar deal
Carmot Therapeutics, a privately held company working on treatments for obesity, was on track for what looked like a bellwether IPO in a troubled biotech market. Then came yesterday's news that Roche will pay nearly $3 billion for it, canceling what could have been a major story in 2024.
There have been just six biotech IPOs so far this year, according to the law firm Goodwin, and the returns haven't been great. Four of those companies have lost more than half of their value since going public, including Turnstone Biologics, which is down about 80%, and Acelyrin, which has fallen 60%. The only bright spots are the autoimmune biotech Apogee Therapeutics, up 20% since its July IPO, and Structure Therapeutics, which has an oral GLP-1 medicine and has more than tripled in value since going public in February.
The latter point makes the Carmot acquisition that much weightier. Obesity is just about the only thing that seems to move biopharmaceutical stocks upward, and now the sector heads into 2024 without what could have been a rare IPO success story.
Regulatory
FDA is doing fewer inspections and finding more problems
That's according to a new analysis, which noted a significant decline in FDA inspections of pharmaceutical manufacturing plants around the world coupled with an increase in the number of citations.
As STAT's Ed Silverman reports, there was a 79% drop in inspections of overseas plants and a 35% decline in domestic facilities from 2019 to 2022. In that same period, the number of plants that needed remediation went up. For foreign facilities, about 17% of all inspections conducted the past two years yielded citations compared with just 5% in 2020.
"It's a problem and will worsen with time," said David Ridley, a Duke University professor who co-authored the analysis. "The longer the lag between inspections, the more likely that unresolved problems arise."
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