BioXcel Therapeutics' shares slumped Thursday after warning that prospects for regulatory approval for its investigational acute treatment for agitation in Alzheimer's patients could be hampered by a fraud investigation at a big study site.
In morning trading, the biopharmaceutical company's shares were 59% lower at $7.31 , having earlier in the day hit a fresh 52-week low of $7.09 . The stock is now down 66% so far this year..
BioXcel reported positive topline results from a late-stage trial that could support the use of BXCL501, an orally dissolving film formulation of dexmedetomidine, as a potential treatment for acute episodes of agitation in patients with mild to moderate Alzheimer's disease.
However, in a regulatory filing with the Securities and Exchange Commission , the company said it had confirmation that the principal investigator at a site that enrolled about 40% of the subjects in a Phase 3 clinical trial allegedly fabricated email correspondence. That came after BioXcel said it launched an investigation after learning in May the principal investigator in the so-called Tranquility II trial may have made up email correspondence purporting to demonstrate that the it timely submitted to the company's pharmacovigilance safety vendor a report of a serious adverse event from a different subject than one cited in a FDA form and purporting to show that the vendor had confirmed receipt.
The Food and Drug Administration inspected the clinical trial site in December, after which it issued a form identifying three observations related to the principal investigator's failure to adhere to an approved informed consent form for a limited number of subjects whose records the FDA reviewed, maintain adequate case histories for certain patients, and adhere to the investigational plan in certain instances.
BioXcel in its filing said it was made aware that fabricated email correspondence was provided to the FDA by the principal investigator's employer during the on-site inspection in December, and after unblinding the data it determined that the serious adverse event that was the subject of the correspondence between the principal investigator and pharmacovigilance vendor occurred in a subject in the placebo arm. It said the investigator hasn't participated in any other clinical trial sponsored or conducted by the company.
Meanwhile, BioXcel said topline results from the trial of BXCL501 were positive and the study met its primary efficacy endpoint. The 60 mcg dose also met the first key secondary endpoint of reducing agitation symptoms at one hour during the first episode of agitation, but didn't meet the other key secondary endpoint of change from baseline in PEC score at 30 minutes, the company said.
