BioXcel Therapeutics Inc (NASDAQ:) shares plunged 65% after releasing data from a Phase 3 trial for the acute treatment of Alzheimer’s disease-related agitation. The trial for BXCL501 met the primary endpoint with the 60 mcg dose, but the primary endpoint was not met for the 40 mcg dose, and BioXcel disclosed a data integrity issue.
The 60 mcg dose of BXCL501 showed a statistical significance reduction in acute agitation after two hours compared with a placebo, and the drug was well tolerated. The 60 mcg dose was also statistical significance at 1 hour, a key secondary endpoint, but not at 30 minutes.
BioXcel disclosed the data integrity issue in an SEC filing that listed three problems found by the FDA during an inspection. It was later discovered that the person in charge of the trial may have made up email conversations related to timely reports on serious adverse events.
Mizuho Securities commented on the development, stating, “Overall, in our view, we came away very disappointed on the totality of BTAI’s T2 data update. On the surface, while the higher 60ug dose did demonstrate stat sig, (and hence a win on paper), in our view, we believe that in the worst-case scenario, the ongoing FDA investigation calls into question the data where the clinical site enrolled 40% of patients.”
Guggenheim analysts said, “We are lowering our estimates and chances of approval in ADA until the ongoing FDA issues are resolved, and we have more clarity on filing and approval timelines.”
Despite the problems, BioXcel Therapeutics plans to develop a path to potential sNDA submission for the acute treatment of agitation associated with Alzheimer’s disease in the second half of 2023, subject to further discussions with the FDA.
