New Approved Therapy for Huntington’s Disease

The N-methyl-D-aspartate (NMDA) receptor is a protein that is present on the surface of neurons and plays a vital role in learning and memory. Abnormal activity of the NMDA receptor is thought to contribute to the death and neurologic dysfunction observed in people with Huntington’s disease (HD) and other neurological diseases. Cognitive decline is often one of the earliest signs of HD and results in a devastating impact on independence, general functioning, and quality of life. Improving cognitive function is one of the core paths to maintaining quality of life in HD and remains an area of significant unmet medical need.

On April 1, 2022, Sage Therapeutics announced positive results for SAGE-718 in patients with mild cognitive impairment (MCI) and mild dementia. SAGE-718 is an oral, positive allosteric modulator of the NMDA receptor to treat cognitive disorders associated with NMDA receptor dysfunction, including HD, Parkinson’s disease, and Alzheimer’s disease. Results from the Phase 2 LUMINARY Study found SAGE-718 to be generally well-tolerated and associated with improvement on multiple tests of executive performance and learning and memory.

These results are consistent with improvement seen in cognitive performance across the SAGE-718 program, including in people with Parkinson’s and Huntington’s disease. Last year the FDA granted Fast Track Designation to SAGE-718 for development as a potential treatment for HD, a process designed to facilitate the development and review of new treatments for conditions with unmet medical needs. SAGE-718 is currently being studied in the ongoing Phase 2 DIMENSION Study, a double blind placebo-controlled study in people with early to moderate cognitive impairment due to HD that is designed to evaluate the efficacy of once daily dosed SAGE-718 over 3 months.

Sources: Sage Therapeutics Announces Presentation of Promising Results from the Phase 2 LUMINARY Study of SAGE-718 in Patients with Mild Cognitive Impairment and Mild Dementia

LUMINARY Trial: https://clinicaltrials.gov/ct2/show/NCT04602624

DIMENSION Study: https://clinicaltrials.gov/ct2/show/NCT05107128