A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 ...
San Diego–based Ventyx Biosciences has what pharma wants, according to William Blair: two NLRP3 inhibitors for inflammatory ...
After 27 years in business, Cytokinetics hopes to pit its own cardiac myosin inhibitor against one it initially developed—now owned by Bristol Myers Squibb—in a market worth billions. Aficamten has a ...
BioMarin Pharmaceutical has faced a rocky road, promising and then backing off revenue targets and cutting assets that have underperformed. But Amicus’ rare disease portfolio is already bringing in ...
Insmed’s oral drug Brinsupri did not ease the burden of sinus symptoms in a mid-stage study of chronic rhinosinusitis without nasal polyps, forcing the New Jersey biotech to discontinue its program in ...
These deals radically reshaped the biopharma world, either by one vaccine rival absorbing another, a Big Pharma doubling down ...
Despite falling short of its primary Phase II objective, Nektar plans to push its T cell stimulator rezpeg into Phase III ...
Pfizer CEO Albert Bourla defended his company’s vaccine business as rhetoric from HHS Secretary Robert F. Kennedy Jr. drives ...
Representatives of companies including AbbVie, Eli Lilly, Johnson & Johnson and Merck have voiced concerns about the FDA’s ...
The FDA had previously turned back the heart rhythm nasal spray twice, once in late 2023 with a refusal to file letter and ...
Varegacestat, a gamma secretase inhibitor, significantly improved progression-free survival while also meeting all key ...
For $950 million upfront, Sobi will gain ownership to pozdeutinurad, an oral URAT1 inhibitor that performed well in Phase II ...
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