2023-06-29 08:40:41 ET
BioXcel Therapeutics ( NASDAQ: BTAI ) fell ~58% pre-market Thursday even after announcing that its Phase 3 trial for Alzheimer’s candidate BXCL501 reached its main goals as investors reacted to its data integrity issues.
The conduct of a principal investigator at a study site has led to concerns regarding the timeliness of reporting a serious adverse event (SAE) in the placebo arm.
The FDA has also found issues of non-compliance at the site where the same principal investigator enrolled approximately 40% of the subjects in the study named TRANQUILITY II, the company said in a regulatory filing.
BioXcel ( BTAI ) said it plans to launch an investigation into the issues and warned that the developments would impact its plans to seek approval for BXCL501 as an acute treatment of Alzheimer’s-related agitation.
However, TRANQUILITY II, targeted at older adults with mild to moderate dementia, has reached the primary endpoint for BXCL501 at the 60 mcg dose.
The study designed to assess the 2-hour change in Positive and Negative Syndrome Scale-Excitatory Component (PEC) as the primary endpoint indicated a statistically significant 39% reduction in PEC score from baseline against placebo, according to the company.
The PEC change for BXCL501 showed a 7.5-point reduction compared to a 5.4 reduction with placebo at two hours post-dose for the first treated episode of agitation. However, TRANQUILITY II did not meet the other key secondary endpoint of change from baseline in PEC score at 30 minutes.
The experimental therapy was well tolerated, with a side effect profile in line with its prior studies.
BioXcel ( BTAI ) plans to seek a potential marketing application for the treatment in H2 2023, subject to discussions with the FDA.
For further details see:
BioXcel sheds 58% as data integrity issues cloud Alzheimer’s trial win