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Good morning. STAT printed an investigation this morning on the untold story of the Human Genome Mission and ethics issues surrounding the formidable challenge. Test it out right here.
The necessity-to-know this morning
- Pfizer Chief Scientific Officer and R&D Chief Mikael Dolsten is leaving the company after 15 years. A seek for his successor is anticipated to final till early subsequent yr, the pharma large mentioned.
- UniQure mentioned its experimental gene remedy known as AMT-130 slowed the progression of Huntington’s disease by 80% in comparison with an exterior management group in a mid-stage medical trial.
Interius to begin first ever in-vivo CAR-T medical trial
Interius Biotherapeutics will quickly start the world’s first medical trial of in-vivo CAR-T remedy. Sufferers will obtain an IV infusion designed to rework their immune cells into most cancers killers inside their our bodies.
Conventional CAR-T remedy is “ex-vivo,” which means the immune cells are manipulated exterior the physique. It’s a sophisticated and costly course of that includes extracting the cells from the affected person, delivery them to a specialised facility to be labored on, after which reinfusing the cells again into the affected person.
Conventional CAR-T is out of attain for a lot of sufferers, and researchers hope that the brand new in-vivo strategy might present a less expensive and extra scalable possibility.
Learn extra from STAT’s Jason Mast.
Pharma is shedding mates on Capitol Hill
Pharma’s place in Washington has already been weakening over the previous few years, as underscored by the passage of the Inflation Discount Act, which allowed Medicare to barter drug costs. The trade’s affect is ready to wane much more as a few of its closest allies depart Capitol Hill.
My colleague Rachel Cohrs Zhang reviews that at the very least six key pharma-friendly lawmakers are anticipated to have left their seats by the start of subsequent yr, and their possible replacements are much less pleasant to the trade and fewer taken with well being care.
One instance is Sen. Bob Menendez of New Jersey, residence of pharma giants Johnson & Johnson, Merck, and Bristol Myers Squibb. Menendez is at the moment on trial for bribery costs, and the Democrat vying to exchange him, Rep. Andy Kim, helps the get together’s extra formidable drug pricing plans.
Learn extra on the opposite allies pharma will likely be shedding.
Lilly’s weight problems drug seems stronger than Novo’s in observational examine
In its pivotal Part 3 trial, Eli Lilly’s tirzepatide (bought as Mounjaro/Zepbound) led to extra weight reduction than what was seen within the trial of Novo Nordisk’s semaglutide (bought as Ozempic/Wegovy). It’s been exhausting to instantly examine the 2 medicine, although, since there haven’t but been outcomes from any head-to-head trials, however a brand new observational examine suggests Lilly’s drug might certainly result in better weight reduction.
The examine, published yesterday in JAMA Internal Medicine, analyzed the well being information of over 18,000 individuals and located that these on tirzepatide had about 15% weight reduction at one yr, whereas these on semaglutide had about 8%. Moreover, 42% of sufferers on tirzepatide achieved greater than 15% weight reduction, in contrast with 18% of sufferers on semaglutide.
Because it’s a retrospective observational examine, there are lots of limitations. For instance, sufferers might have encountered shortages and should not have been capable of constantly take their drugs. Sufferers might have additionally been on completely different diets and train regimens.
We’ll be waiting for outcomes of a randomized head-to-head trial that Lilly is working that’s anticipated to finish in November this yr.
A personal fairness strategy to investing in neuro medicine
Buyers have lengthy been reluctant to put money into drug applications for neurological issues. There’s been a historical past of failed research and lots of trial endpoints are subjective and troublesome to measure.
Bruce Leuchter, CEO of Neurvati Neurosciences, a Blackstone Life Sciences portfolio firm, argues in a brand new opinion piece that buyers ought to undertake a a “personal fairness mannequin” to put money into neurology drug candidates.
This implies molecules later within the growth lifecycle slightly than at early-stage medicine that the basic enterprise mannequin focuses on. Whereas the prices to accumulate a later-stage drug will likely be larger, buyers would be capable to higher vet this system and make sure the rationale behind the drug’s mechanism, Leuchter writes.
Learn extra.
Extra reads
- When can pharma firms appropriate on-line misinformation? FDA explains, Endpoints
- AI leads the best way as well being tech funding inches towards a post-pandemic stability, STAT
- Trump’s 2024 platform abandons calls to chop Medicare, broadly prohibit abortion, STAT
- New examine sparks debate about whether or not H5N1 virus in cows is customized to raised infect people, STAT
