Biohaven’s troriluzole did not differentiate from placebo in Alzheimer’s trial

Biohaven Pharmaceutical announced that it has completed a focused analysis of the topline co-primary and key secondary data from its Phase 2/3 clinical trial of troriluzole as a symptomatic treatment in mild-to-moderate Alzheimer’s disease. Additional secondary and exploratory efficacy analyses and biomarker data including neurofilament light chain, neurogranin, tau and amyloid are still pending and expected in the coming months. Troriluzole did not statistically differentiate from placebo at 48 weeks on the study’s prespecified co-primary endpoints on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 and the Clinical Dementia Rating Scale Sum of Boxes in study participants with mild-to-moderate Alzheimer’s disease. Troriluzole also did not differentiate from placebo on the key secondary measure of hippocampal volume assessed by magnetic resonance imaging in the overall population, Biohaven added. A subgroup analysis consisting only of mild Alzheimer’s patients did, however, reveal that troriluzole exhibited a “nonsignificant numerical difference” of a potential benefit at week 48 on both the ADAS-cog and hippocampal volumetric MRI. Troriluzole treated participants with mild AD had a mean deformation change from baseline hippocampal volume of -1.1% versus -1.6% for placebo treated participants at week 48, Biohaven said. The company added, “Our goal was to efficiently assess whether troriluzole could benefit patients relatively late in the disease process with mild-to-moderate AD. This study was well-conducted but unfortunately it is clear from this preliminary analysis that troriluzole is not efficacious as a symptomatic treatment in a mixed population of patients with mild and moderate AD. We are awaiting additional biomarker data and other secondary analyses that will help inform whether troriluzole may provide benefit in early AD as a disease modifying agent.”