After misconduct at a key trial site raised questions of data integrity earlier this year, BioXcel Therapeutics’ Alzheimer’s agitation prospect faced a grim outlook. Now, with a third-party audit in hand, the company believes things are looking up.
The company’s June readout for its orally dissolving dexmedetomidine was tainted by major red flags at the site. During a December inspection, the FDA discovered fabricated email correspondences to cover up a missed report of a serious adverse event, plus instances of skirted informed consent protocols and other trial missteps.
Afterward, BioXcel initiated an investigation and contracted an independent third party to audit the data.
The review, which was conducted by a “well-regarded regulatory and quality consulting firm,” looked at records from more than 50% of subjects enrolled at the site to pinpoint additional potential evidence of fraud and determine the data’s integrity and reliability.
According to the company, the auditors didn’t identify any additional instances of misconduct or evidence that would discredit the findings.
With the audit in hand, BioXcel believes that the data “potentially supports” a filing in the acute treatment of agitation associated with dementia in probable Alzheimer’s disease patients.
“We believe these results of an audit by a respected, independent firm validate the integrity of data from the single site in question and add to the body of clinical evidence we intend to include in our sNDA submission,” CEO Vimal Mehta, Ph.D., said in the company’s release.
The company recently met with the FDA to discuss its plan for the drug’s development and expects to provide an update on next steps in its third-quarter financial results.
Even with the confirmation of validity, the trial results were a mixed bag. The drug, which was originally known as Pfizer’s Precedex, missed a key secondary endpoint in the study. The 40 mg dose didn't meet its primary endpoints, either.
For the 60 mg dose, investigators found a significant decrease in Positive and Negative Syndrome Scale-Excitatory Component (PEC) scores compared with placebo. The drug also reduced agitation symptoms after one hour during the first agitation episode.
BioXcel's stock price was up by more than 20% Wednesday after news of the audit.