Drug for Agitation in Alzheimer Disease Dementia Fails in Phase 3 Trial

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Otsuka announce topline results of the phase 3 clinical trial of AVP-786, a combination of dextromethorphan hydrobromide and quinidine sulfate, in the treatment of agitation associated with Alzheimer disease dementia (NCT03393520). A statistically significant difference was not achieved on the primary efficacy endpoint, which was mean change from baseline to week 12 in the Cohen-Mansfield Agitation Inventory (CMAI) total score compared with placebo.¹

“While the result of this trial is disappointing, we plan to analyze the full data set to determine the future potential of AVP-786 in the treatment of agitation associated with dementia due to Alzheimer’s disease. In 2023, Otsuka became the first company to get a drug approved for this patient population and we are committed to expanding and innovating in this area,” said John Kraus, MD, PhD, executive vice president and chief medical officer at Otsuka.

During the trial, there was 1 treatment-emergent adverse event that appeared with an incidence rate of more than 5% in patients treated with AVP-786 and greater than placebo: falling. In the AVP-786 high dose group, there were 16 participants who had a fall (8.6%), and 18 (9.1%) in the AVP-786 low dose group. In the placebo group, there were only 6 (2.8%). There were also 4 deaths reported in the trial; 1 (0.5%) in the AVP-786 low dose group and 3 (1.4%) in the placebo group.

Agitation associated with dementia has a strong impact on a patient’s quality of life and is reported in about half of all patients with Alzheimer disease dementia, Otsuka shared.²

While full study results are not yet available, further exploratory data analyses will be conducted to determine the full potential of AVP-786 in the treatment of agitation associated with Alzheimer disease dementia. Otsuka will submit the trial results for scientific publication at a later date.

“We are deeply grateful to all of the study participants, their caregivers, and the investigators who took part in this trial and contributed to this research,” said Kraus.

References

1. Otsuka announces phase 3 topline results of AVP-786 in the treatment of agitation associated with dementia due to Alzheimer’s disease. News release. February 12, 2024. Accessed February 14, 2024. https://otsuka-us.com/news/otsuka-announces-phase-3-topline-results-avp-786-treatment-agitation-associated-dementia-due

2. Otsuka's drug for Alzheimer's disease agitation fails in late stage study. Reuters. February 12, 2024. Accessed February 14, 2024. https://www.reuters.com/business/healthcare-pharmaceuticals/otsukas-drug-alzheimers-disease-agitation-fails-late-stage-study-2024-02-13/