Killing two birds with one stone, BioXcel Therapeutics reported this week positive top-line results for the phase 3 trial of BXCL501, its orally dissolving film form of dexmedetomidine, and gave updates via an SEC filing about study mishaps.
This trial, which aimed to examine Alzheimer's-related agitation, had positive data regarding BXCL501’s efficacy. The 60 mg dose reduced symptoms significantly after 2 hours compared to a placebo, meeting its primary endpoint. It also reduced symptoms after one hour during the first agitation episode, and most patients (76%) experienced improvement with this dose.
Simultaneously, BioXcel shared that in December 2022, the U.S. FDA conducted an inspection at a clinical trial site involved in the phase 3 Tranquility II BXCL501 trial. The site, which was responsible for enrolling approximately 40% of the trial's participants, was met with FDA Form 483, outlining three observations.
The observations were related to the failure of the principal investigator to adhere to the approved informed consent form for a small number of subjects, the inadequate maintenance of case histories for specific patients, and noncompliance with the investigational plan in certain cases. In one of the cases, the FDA noted that the principal investigator delayed notification of the sponsor's medical monitor or pharmacovigilance safety vendor about a serious adverse event for one subject, and this report was made outside the 24-hour timeframe specified in the trial protocol. Then in May 2023, BioXcel became aware that the same principal investigator in the Tranquility II clinical trial may have falsified email correspondence to show that the he had submitted a report of an SAE to their pharmacovigilance safety vendor in a timely manner.
Trial hiccups aside, the company 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.