SAGE-718 on FDA Fast Track as Potential Huntington’s Therapy
The U.S. Food and Drug Administration (FDA) has granted fast track designation to SAGE-718 as a potential therapy for Huntington’s disease.
The FDA gives this designation to experimental medications with the potential to improve medical care for serious health conditions and fill unmet needs. The designation gives the therapy’s developer — Sage Therapeutics — access to more frequent meetings with the FDA to review the development plan for the medicine, with the goal of getting effective medicines to market faster.
“The FDA Fast Track Designation is an important milestone in the development of SAGE-718, as it provides opportunities to engage collaboratively with the FDA to further clinical development and future regulatory review of SAGE-718 for the treatment of [Huntington’s disease],” Jim Doherty, PhD, chief research officer at Sage, said in a press release.
SAGE-718 is a first-in-class oral medication that works by modulating the activity of the N-methyl-D-aspartate (NMDA) receptor, a protein that’s present on the surface of neurons (nerve cells) and plays key roles in learning and memory. Abnormal activity of the NMDA receptor is thought to contribute to the death and dysfunction of neurons in people with Huntington’s and other neurological diseases.
The safety, tolerability, and pharmacologic properties of SAGE-718 in healthy subjects has been assessed in several clinical trials. Sage sponsored a Phase 1 trial (NCT03787758) that tested the experimental medication’s safety in six people with Huntington’s, which concluded in 2019.
Sage is planning to launch a placebo-controlled Phase 2 trial to test whether SAGE-718 may improve cognition in people with early to moderate Huntington’s later this year.
“In studies to date, treatment with SAGE-718 has been associated with improved cognitive performance, particularly in the domain of executive functioning,” Doherty said.
“Cognitive decline is often one of the earliest signs of [Huntington’s] disease and this decline, in addition to other symptoms, results in a devastating impact on independence, general functioning, and quality of life,” Doherty added. “We believe that improving cognitive function is one of the core paths to maintaining quality of life in [Huntington’s] and remains an area of significant unmet medical need.”