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Buenos dias! Writing from one of many extra distinctive areas in my tenure as a Readout writer…. Hola from a mercado in Mexico Metropolis. On faucet at the moment: Early research present that checkpoint inhibitors could also be more practical if mixed with JAK inhibitors. Additionally, extra promising anti-obesity outcomes and maybe a stronger PrEP for HIV.
Sarepta gene remedy accepted for many Duchenne sufferers
The FDA yesterday dramatically expanded its approval for Elevidys, a gene remedy for Duchenne muscular dystrophy, authorizing entry for almost all sufferers with the deadly muscle-wasting illness.
It’s clear that the therapy, made by Sarepta Therapeutics, gained’t be a panacea — “Nobody pretends it is a remedy,” one affected person’s mom informed STAT’s Jason Mast — however the gene remedy presents not less than incremental profit to sufferers with Duchenne. It’s now accepted for all sufferers over the age 4 who can stroll on their very own. For sufferers who depend on wheelchairs, the company granted accelerated approval, a type of conditional authorization that must be confirmed in a bigger, ongoing study.
It’s value noting the approval resolution was made virtually completely by Peter Marks, the FDA’s director of the Middle for Biologics Analysis and Analysis, who overruled three overview groups and two high lieutenants.
Can JAK inhibitors bolster checkpoint inhibitors?
Checkpoint inhibitors like Keytruda and Opdivo are fickle: At occasions they work fantastically in sufferers, however in others, not a lot. However two small medical trials printed in Science discovered {that a} JAK inhibitor made by Incyte, when mixed with checkpoint inhibitors, led to most sufferers responding to immunotherapy. In a single trial, sufferers had been in a position to overcome resistance to checkpoint inhibitors, STAT’s Angus Chen writes.
In that research, 10 out of 19 members with Hodgkin lymphoma whose illness had already progressed or relapsed responded to the drug — and 6 achieved an entire response. Within the different research, researchers mixed Keytruda with the JAK inhibitor and noticed an improved response.
“It may actually change the panorama when it comes to therapy for some sufferers,” an immunologist not concerned with the work informed STAT. “It opens the door for a complete collection of research for cancers that are handled with immune checkpoint inhibitors which work already, however they’re not good and never sufficient. It could give one other arrow within the armamentarium of clinicians.”
Learn extra.
Gilead’s experimental PrEP for HIV impresses
Gilead’s new antiviral drug lenacapavir utterly protected cisgender girls from contracting HIV in a big Part 3 trial. Not one of the 2,134 girls taking the twice-a-year injection contracted the virus. By comparability, 16 of 1,068 girls taking Truvada and 39 of the two,136 girls taking Descovy contracted HIV.
“To haven’t any infections in over 2,0000 girls… I imply that’s a outstanding end result,” the research’s principal investigator informed STAT’s Jason Mast. The trial was carried out in Uganda and South Africa; if a follow-up research in different international locations confirms the outcomes, lenacapivir might be accepted as a potent pre-exposure prophylaxis, or PrEP, for HIV.
“This presents a very completely different possibility, as a result of it’s simply twice a 12 months, it’s discrete and personal, as a result of it’s not in a bottle that you need to carry dwelling, that somebody may discover,” Gilead’s HIV franchise head informed STAT. “For therefore many individuals on the market in so many various locations, this might present a PrEP possibility that’s workable for them.”
Learn extra.
Zealand Pharma’s anti-obesity drug exhibits promise
One other investigational anti-obesity drug is exhibiting promise in early stage trials. Petrelintide, made by Zealand Pharma, led to eight.6% weight reduction at 16 weeks, in comparison with the 1.7% weight reduction amongst sufferers taking placebo within the Part 1b trial. The injectable drug has a unique mechanism than the wildly common GLP-1 medication sweeping the market: This drug targets the amylin hormone.
Zealand isn’t the one firm with amylin as an anti-obesity goal, STAT’s Elaine Chen writes. Novo Nordisk and Eli Lilly are additionally wanting on the hormone, which is produced in pancreatic beta cells and co-secreted with insulin as a response to meals consumption. Novo has a late-stage candidate in trials referred to as CagriSema that prompts amylin, calcitonin, and GLP-1 hormone receptors.
Learn extra.
Fauci’s memoir & Alnylam’s tenuous trial
Can Alnylam discover success with its newest cardiac therapy after tweaking the trial? And did STAT’s editorial workforce pull off a covert mission to get a sophisticated copy of a brand new e book?
We cowl all that and extra on this week’s episode of “The Readout LOUD.” STAT reporter Jason Mast joins us to debate Anthony Fauci’s memoir, “On Name,” which was printed this week. Hosts Allison DeAngelis and Adam Feuerstein additionally dissect Alnylam’s forthcoming medical trial knowledge and take inventory of how the biotech markets are succeeding and doubtlessly falling flat halfway by the 12 months.
Hear right here.
Extra reads
- Trade teams again drugmakers’ enchantment in Zantac most cancers lawsuits, Reuters
- J&J’s subcutaneous model of Tremfya excels in Crohn’s illness research, FiercePharma
- Vertex reaches a brand new cope with NHS England over its dear cystic fibrosis therapies, STAT
