In concluding benefits do not outweigh risks in the drug’s current indication, US FDA advisory committee members cite concerns about survival data from the Phase III DUO trial, as well as the drug’s toxicities, safety issues with the PI3K inhibitor class, and the changing treatment landscape for chronic lymphocytic leukemia and small lymphocytic lymphoma.

Office of Neuroscience Director Billy Dunn touted FDA’s ability to withdraw approval for AMX0035 if subsequent data indicate a lack of substantial evidence of effectiveness. However, this provision appears to have been infrequently used and involves a potentially time-consuming hearing.

Public Company Edition: William Blair analysts note that stock prices are rising due to a Q2 boost in dealmaking after a lull in Q1. Nevertheless, layoffs and portfolio prioritizations are ongoing, including at X4 Pharmaceuticals, but others have raised cash following good news, like Revolution Medicines.

RA drug development has been slow to revise the trial design paradigm despite agreement on the drawbacks of placebo controls and crossover elements, but FDA biostatistician Levin sees promise in non-inferiority designs.