Sarepta, Keytruda, Gilead, Alnylam news

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Buenos dias! Writing from one of many extra distinctive places in my tenure as a Readout creator…. Hola from a mercado in Mexico Metropolis. On faucet right now: 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 authorised for many Duchenne sufferers

The FDA yesterday dramatically expanded its approval for Elevidys, a gene remedy for Duchenne muscular dystrophy, authorizing entry for practically all sufferers with the deadly muscle-wasting illness.

It’s clear that the remedy, made by Sarepta Therapeutics, received’t be a panacea — “Nobody pretends it is a treatment,” one affected person’s mom informed STAT’s Jason Mast — however the gene remedy gives at the very least incremental profit to sufferers with Duchenne. It’s now authorised 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 price noting the approval determination was made virtually completely by Peter Marks, the FDA’s director of the Middle for Biologics Analysis and Analysis, who overruled three evaluate groups and two prime lieutenants.

Can JAK inhibitors bolster checkpoint inhibitors?

Checkpoint inhibitors like Keytruda and Opdivo are fickle: At occasions they work superbly in sufferers, however in others, not a lot. However two small scientific trials revealed 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 have been capable of overcome resistance to checkpoint inhibitors, STAT’s Angus Chen writes.

In that examine, 10 out of 19 contributors with Hodgkin lymphoma whose illness had already progressed or relapsed responded to the drug — and 6 achieved a whole response. Within the different examine, researchers mixed Keytruda with the JAK inhibitor and noticed an improved response.

“It may actually change the panorama by way of remedy for some sufferers,” an immunologist not concerned with the work informed STAT. “It opens the door for an entire sequence of research for cancers that are handled with immune checkpoint inhibitors which work already, however they’re not excellent 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 fully protected cisgender girls from contracting HIV in a big Section 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 don’t have any infections in over 2,0000 girls… I imply that’s a exceptional end result,” the examine’s principal investigator informed STAT’s Jason Mast. The trial was performed in Uganda and South Africa; if a follow-up examine in different nations confirms the outcomes, lenacapivir may very well be authorised as a potent pre-exposure prophylaxis, or PrEP, for HIV.

“This gives a extremely totally different choice, as a result of it’s simply twice a yr, it’s discrete and personal, as a result of it’s not in a bottle that it’s important to carry house, 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 choice 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 Section 1b trial. The injectable drug has a unique mechanism than the wildly widespread GLP-1 medicine 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 trying 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 remedy after tweaking the trial? And did STAT’s editorial staff 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 revealed this week. Hosts Allison DeAngelis and Adam Feuerstein additionally dissect Alnylam’s forthcoming scientific trial information and take inventory of how the biotech markets are succeeding and probably falling flat halfway via the yr.

Pay attention right here.

Extra reads

• Business teams again drugmakers’ attraction in Zantac most cancers lawsuits, Reuters
• J&J’s subcutaneous model of Tremfya excels in Crohn’s illness examine, FiercePharma
• Vertex reaches a brand new cope with NHS England over its dear cystic fibrosis remedies, STAT