On 29 June, BioXcel announced positive top-line results from TRANQUILITY II (NCT05271552), a Phase III study of Igalmi (dexmedetomidine sublingual film) for the acute treatment of agitation episodes associated with Alzheimer’s disease (AD). The trial met its primary endpoint, with treatment using the higher dose of Igalmi resulting in a statistically significant reduction in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) score after two hours from baseline compared with placebo. Several secondary endpoints were also met such as a reduction in agitation symptoms at one hour, and the drug was well tolerated with the safety profile consistent with previous trials of the drug.

However, these positive results have been overshadowed by several counts of bad clinical practice disclosed by the company. It has emerged that a principal investigator failed to follow the informed consent plan for four of the trial participants, in addition to insufficient case histories being kept for some patients. The clinical trial site failed to report a serious adverse event within the 24-hour time frame stated in the trial protocol, and it is alleged that the principal investigator also provided fabricated emails to the FDA regarding the reporting of a serious adverse event. Although BioXcel does not believe that all of the data from the trial will be unusable, the trial site run by that principal investigator enrolled around 40% of the trial participants and the company is carrying out a full investigation and audit of the trial site; concerns surrounding the validity of some of the data from the trial remain.

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Igalmi was approved by the FDA for agitation associated with bipolar disorder and schizophrenia in April 2022. A label expansion into AD is a key strategy for BioXcel, as there is a larger potential patient population in this indication, particularly with an ageing global population, combined with a lack of treatments targeting agitation associated with AD. If an additional clinical trial was to be required by the FDA, it would put BioXcel on the back foot in the market for treating agitation associated with AD, delaying Igalmi’s launch behind many potential competitors. Further, even if the clinical trial data is deemed sufficient to allow a supplemental drug application and subsequent approval, there could still be negative opinions surrounding BioXcel and Igalmi, resulting in preferential use of other products by physicians and patients, damaging potential sales for the drug.

There are several other products currently in Phase III development for AD-related agitation, including Avanir Pharmaceuticals’s deuterated form of dextromethorphan/quinidine AVP-786, Axsome Therapeutics’s NMDA-R antagonist Auvelity (bupropion + dextromethorphan), and Suven Life Sciences’ 5-hydroxytryptamine receptor 6 antagonist masupirdine. Additionally, Otsuka’s Rexulti (brexpiprazole) became the first drug to be approved by the FDA for agitation associated with AD on 10 May 2023.

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