Drug Development
With three FDA approvals in the past 10 months, there is a lot of momentum in the Duchenne muscular dystrophy space. Here are five companies looking to keep it going.
FEATURED STORIES
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
The cholesterol-lowering drug is part of a suite of medicines that also includes MariTide and that Amgen Chief Medical Officer Paul Burton hopes will make the company the “undisputed leader in the management of cardiometabolic risk for patients” by 2030.
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The next frontier in RNA therapies, RNA editing has the potential to treat both genetic and common disorders, and the technology is rapidly expanding beyond the liver.
Following the recommendations of an independent Data Monitoring Committee, Novo Nordisk has halted a Phase III kidney outcomes study of semaglutide ahead of schedule due to strong efficacy signals.
Google and Microsoft are already making inroads into drug development, but smaller biotechs focused on AI may end up leading the charge.
The pharma giant’s anti-PD-1 therapy met its dual primary endpoint of overall survival as a treatment regimen for non-small cell lung cancer patients, as the FDA’s Oct. 16 PDUFA date looms.
Both low and high doses of Akero Therapeutics’ lead candidate efruxifermin failed to significantly outperform placebo at improving liver fibrosis without worsening non-alcoholic steatohepatitis.
Patients treated with Xenon Pharmaceuticals’ investigational potassium channel opener XEN1101 experienced a significant and dose-dependent reduction in seizure frequency.
At the six-month follow-up, about 40% of patients treated with Newron Pharmaceuticals’ evenamide improved so much that they no longer meet the Phase II study’s original eligibility criteria.
Though data became an issue in two separate meetings, the FDA’s Oncologic Drugs Advisory Committee made a potentially precedent-setting decision by voting in favor of US WorldMeds’ neuroblastoma treatment.
Keytruda’s winning streak in bladder continues with positive data from the Phase III AMBASSADOR study, showing significantly better disease-free survival in muscle-invasive urothelial carcinoma.
Patients with treatment-resistant depression treated with Johnson & Johnson’s Spravato were significantly more likely to reach remission and stay relapse-free for up to 32 weeks.