BioXcel Therapeutics Inc (NASDAQ:BTAI) shares rallied on Wednesday after the artificial intelligence (AI)-powered biopharmaceutical company reported that an independent audit of a site in the late-stage trial of its investigational therapeutic BXCL501 to treat agitation in Alzheimer’s patients found no evidence of misconduct or fraud beyond what has already been reported.
In June 2023, the company flagged issues around the recording of safety data at a site in its TRANQUILITY II Phase 3 trial, specifically that a principal investigator may have fabricated emails showing that they submitted certain safety data in a timely manner. The news saw BioXcel’s shares plummet by more than 63% in a day.
BioXcel said Wednesday that the independent audit consisted of a comprehensive review of records from more than 50% of subjects enrolled at the single trial site to identify any additional instance of misconduct or fraud and to evaluate data integrity and reliability for eligibility, safety, and efficacy data.
The company said this sample size provides 95% confidence that the data reviewed is a representative sample.
The auditors did not identify any findings that they believe impact the data reliability or integrity or any evidence of additional misconduct or fraud, BioXCel said.
As such, the company believes that trial data announced in June could potentially support a supplemented new drug application (sNDA) for BXCL501 for the treatment of agitation associated with dementia in probable Alzheimer’s disease.
“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,” BioXcel CEO Dr. Vimal Mehta said.
The company recently had a Type B/Breakthrough meeting with the United States Food and Drug Administration (FDA) to discuss its development plans for BXCL501.
“We expect to receive the FDA meeting minutes in the first half of November, and intend to provide an update on additional steps for the TRANQUILITY program and a potential sNDA in our upcoming third quarter financial results,” Dr. Mehta said.
BioXcel shares added 32.4% at US$3.12 on Wednesday morning but the company still has a long way to go in rebounding to its share price of about US$17 before its trial issues were revealed in June. The stock is down 85.4% in the year to date.
Contact the author at emily.jarvie@proactiveinvestors.com
Follow her on X, formerly known as Twitter, @emilyjjarvie