Tag Archive for: endpoints

CSL on Sunday announced that its investigational cholesterol efflux enhancer CSL112 failed the Phase III AEGIS-II trial, unable to significantly improve outcomes in patients who had suffered a heart attack.

The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.

While placebo responses are frequently higher in psychiatric studies, Sumitomo CEO Hiroshi Nomura pointed to COVID-19 as a potential culprit for the high placebo effect as the studies were conducted throughout the pandemic.

Despite positive results Monday from a Phase III trial of KarXT (xanomeline-trospium) and analysts’ recommendations to buy, Karuna Therapeutics’ stock dropped more than 19% in 48 hours this week.

Biomarkers as a surrogate endpoint in ALS will go on trial on March 22 as Biogen and Ionis’s tofersen faces the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee.

Data from the Phase III CONTACT-03 study showed the combination of Roche’s Tecentriq (atezolizumab) and Exelixis’ Cabometyx (cabozantinib) fell short of its primary endpoint of progression-free survival in renal cell carcinoma (RCC).

Mirum Pharmaceuticals announced that Livmarli oral solution met its primary endpoint in the Phase III MARCH study in young patients with progressive familial intrahepatic cholestasis.

Following a favorable vote from an FDA advisory committee, Amylyx is one step closer to seeing its amyotrophic lateral sclerosis (ALS) treatment approved for use in the United States.