ADVERTISEMENT
Drug Trial Shows Cognitive Impact of Huntington Disease, but Analysts Express Caution
Scores on a cognitive battery showed a statistically significant difference in patients with Huntington disease and healthy control subjects at baseline in a phase 2 study involving dalzanemdor, an investigational NMDA receptor positive allosteric modulator.
In announcing the topline results from the SURVEYOR study, Sage Therapeutics said the finding—a markedly lower HD-Cognitive Assessment Battery (HD-CAB) composite score before treatment in 40 patients with Huntington disease compared with 29 healthy participants— met the study’s primary endpoint: to determine the magnitude of cognitive impairment in patients with Huntington disease.
The study also aimed to evaluate the safety of dalzanemdor and to investigate the relationship between changes in cognition and changes in function.
>>NEWS: Golodirsen Eases Ambulatory, Pulmonary Function Decline in DMD
After HD-CAB testing, patients with Huntington disease were randomly assigned dalzanemdor or placebo for 28 days. Findings showed dalzanemdor was generally well-tolerated, Sage reported. Although no new safety signals were observed, 11 patients experienced treatment emergent adverse events, most of which were of mild to moderate severity. There were no discontinuations due to treatment emergent adverse events.
The study was not designed or powered to investigate the efficacy of dalzanemdor, the company stated. Nevertheless, it added that results showed a small numerical difference on HD-CAB composite score between treatment with dalzanemdor and placebo.
“Other prespecified analyses suggested the potential for directionally positive signals in a number of individual component tests of the HD-CAB and in some functional assessments,” Sage reported. “Additional work is ongoing to further analyze and understand the data including the relationship of changes in cognition to changes in function.”
Dalzanemdor is in development as a potential oral therapy for cognitive disorders linked with NMDA receptor dysfunction including Huntington disease and Alzheimer disease. According to a report in Fierce Biotech, earlier this year Sage halted research into its potential use in Parkinson disease after a trial failed to show cognitive gains with dalzanemdor in patients with the condition.
Investment analysts from William Blair characterized the SURVEYOR findings as “underwhelming,” Fierce Biotech reported.
“While a small numerical change was observed on the HD-CAB between placebo and Huntington disease participants, we remain cautious on the asset and do not view the small numerical changes as definitive by any means,” the publication quoted from a William Blair note.
References
