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Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.
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The drugmaker’s dominance of the obesity market is fueling predictions that years of growth lie ahead.
Novo Nordisk goes “on the offensive” following Trump deal that also included rival Eli Lilly, putting an exclamation point on rapidly declining GLP-1 drug prices. Experts say the unusual situation makes it hard to predict what’s next.
Drug candidates don’t usually move among Big Pharma, but these five biotechs helped facilitate such hand-offs, scooping up assets from one pharma on the cheap before being bought out for billions by another.
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Although U.S. full-time employees worked an average of 42.9 hours weekly last year, according to Gallup, that wasn’t true for most BioSpace LinkedIn poll respondents. A Karius HR executive discusses a few potential reasons for the longer workweeks.
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Sage Therapeutics discontinued development of its lead candidate dalzanemdor after a third clinical failure, leading analysts to question the biotech’s future profitability.
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Following FDA rejections, Regeneron and Scholar Rock are turning to other facilities to clear regulatory logjams created by quality problems at an ex-Catalent facility in Indiana. Novo Nordisk, meanwhile, has been tight-lipped about whether its own FDA applications have been affected.
U.K.-based pharmas will not face tariffs as long as Donald Trump is president, according to the agreement.
Following Novo Nordisk’s price cuts for its own GLP-1 medicines, Eli Lilly is offering discounts for the obesity drug purchased through LillyDirect. Both pharmas recently struck a deal with the White House for cheaper prices via the yet-to-be-launched TrumpRx.
Protego Biopharma is advancing a small-molecule drug that helps light chain proteins fold correctly, in turn addressing the underlying biological cause of AL amyloidosis.
The centerpiece of the deal is the in vivo editor TSRA-196, which in preclinical studies has shown robust editing at SERPINA1, the locus linked to alpha-1 antitrypsin deficiency.
The alleged deaths were detected by the FDA’s Vaccine Adverse Event Reporting System, reports from which “generally cannot be used to determine” causation or even contribution, according to the agency.
If approved, Ascendis’ TransCon CNP would become the second therapy for achondroplasia, challenging BioMarin’s Voxzogo.
As bispecifics, ADCs, protein degraders, and AI-designed mini-proteins move into the clinic, discovery teams face a new bottleneck: engineering and producing molecules whose complexity challenges conventional workflows.
As big pharmas including Takeda and Novo Nordisk flee the cell therapy space and smaller biotechs shutter their operations, these players are sticking around to take the modality as far as it can go.
The FDA’s docket in December includes decisions for two big biologic franchises: BMS’s Breyanzi and Amgen’s Uplizna.