Drugs

‘Positive Results’ from BioXcel Data Audit

BioXcel Therapeutics says an independent third-party audit of data integrity at one TRANQUILITY II Phase 3 trial site of the company’s BXCL501 for the acute treatment of agitation associated with dementia reported positive findings. “The team of auditors did not identify any findings that they believe impact the data reliability or integrity, nor did they find any evidence of additional misconduct or fraud,” a company statement says. “Based on these findings, BioXcel Therapeutics believes that the positive, statistically significant TRANQUILITY II trial data announced in June potentially support an sNDA for BXCL501 for the acute treatment of agitation associated with dementia in probable Alzheimer’s disease.”

The data integrity audit followed a company 6/29 filing with the Securities and Exchange Commission indicating that a 12/2022 FDA inspection at one of the trial sites led to a form FDA-483 with three observations related to the principal investigator’s failing to adhere to the informed consent form for some subjects, maintain adequate case histories for some subjects, and adhere to the investigational plan in certain instances.

The company also reported it had learned in May that the same principal investigator may have fabricated e-mail correspondence purporting to demonstrate that the investigator timely submitted to the company’s pharmacovigilance safety vendor a report of a serious adverse event from a different subject than the one cited in the form FDA-483.

The company’s investigation led to the decision to retain an independent firm to audit data integrity at the trial site that had enrolled 40% of trial participants.

The company says it recently participated in a Type B/Breakthrough meeting with FDA officials to discuss a way to a potential sNDA for BXCL501 for an agitation associated with dementia indication.

BXCL501 is described as an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. The company says it is under investigation for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting, for the acute treatment of Alzheimer’s-related agitation, and as an adjunctive treatment for major depressive disorder. FDA has granted BXCL501 breakthrough therapy designation for the acute treatment of agitation associated with dementia and fast-track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia.

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