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Good morning, everyone. Damian here with a special thanks to everyone who joined us for STAT’s live event in New York City last night. Today we have a sober look at the future of mRNA, a plot to bring CAR-T back to its founding ideals, and the downside of a billion-dollar buyout.
The need-to-know this morning
• Amicus Therapeutics Executive Chairman John Crowley is stepping down from the board to become CEO of the trade group BIO.
CRISPR Therapeutics is discontinuing the development of two, off-the-shelf CAR-T treatments for blood cancer, shifting to “next generation” versions of the products that have the potential to be more effective. The company also plans develop a CAR-T treatment for the autoimmune disease lupus.
• In a business review with investors and analysts, Johnson & Johnson provided financial guidance for 2024 and long-term operational sales outlook through 2030. STAT’s Matt Herper spoke to J&J’s new R&D chief John Reed about his drug-development strategy.
• BridgeBio completed the filing of a marketing application to the FDA for its heart disease drug acoramidis.
Are we sure mRNA can disrupt the flu?
The Covid-19 pandemic was a historic proving ground for mRNA technology, making billions of dollars for the handful of drugmakers that rapidly developed vaccines. Now, with demand for booster doses waning, the biggest names in the mRNA business have shifted their attention to a perennial market: annual influenza vaccinations.
But, as STAT’s Helen Branswell reports, there’s no guarantee that what worked for Covid will translate to the flu. On the plus side, the speed at which mRNA vaccines can be made could shorten the lead time of each year’s race to predict the most worrisome flu strains. But the side-effect profile of mRNA vaccines could dampen demand, and there are unresolved questions about how much the companies will charge — and how much the market is willing to pay.
Pfizer, Moderna, GSK, and Sanofi are all betting mRNA can prove itself in the flu. It’s of existential importance to Moderna, whose declining Covid-19 revenues have cut the company’s share price nearly in half in 2023. GSK and Sanofi are already major producers of approved flu vaccines might be looking to solidify their places in the market.
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.
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.
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.”
• Official: FDA needs staff influx to meet gene therapy needs, STAT
• WuXi Biologics plunges 24% as Covid boost, biotech boom fade, Bloomberg
Sweeping bill to fight opioid addiction will be considered by Senate health committee, STAT
• Blackstone explores sale of Anthos Therapeutics, Reuters