SAGE-718 named orphan drug in EU for Huntington’s disease

The therapy received fast-track designation in US in 2021

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by Steve Bryson, PhD |

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The European Medicines Agency (EMA) has granted orphan drug status to SAGE-718 as a potential therapy for Huntington’s disease.

The experimental therapy is being developed by Sage Therapeutics for treating cognitive impairment associated with Huntington’s and other neurological disorders.

Orphan drug status is given to treatments with the potential to improve medical care for people with rare, life-threatening, or chronically debilitating diseases. It provides several incentives to the therapy’s developer such as trial protocol assistance, reduced regulatory fees, and, if approved, a 10-year market exclusivity period.

“Huntington’s disease is a serious, debilitating condition that interferes with daily functioning in the prime years of life and is associated with significant morbidity and early mortality, and yet there are currently no approved therapies for the treatment of [Huntington’s disease]-related cognitive impairment,” said Laura Gault, MD, PhD, Sage’s chief medical officer, in a company press release. “With this exciting development of orphan drug designation from the EMA, we have further momentum in our efforts to address this unmet need. Our goal with SAGE-718 is to provide rapid, meaningful, and sustained improvement in cognitive functioning early in the disease so that patients can maintain independence longer.”

SAGE-718 received fast-track designation in the U.S. in 2021 for Huntington’s, which is also expected to accelerate its clinical development and regulatory review.

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Huntington’s is a neurodegenerative disorder marked by uncontrolled movements, cognitive impairment, and psychiatric problems that typically begin in adulthood and worsen over time. While current treatments help patients manage their symptoms, there is no cure or treatment to slow its progression.

The N-methyl-D-aspartate (NMDA) receptor is a protein on the surface of neurons and is involved in memory and learning. In people with Huntington’s and other neurological diseases, abnormal activity of the NMDA receptor is thought to contribute to neurodegeneration.

Currently being evaluated in Sage’s PERSPECTIVE clinical trial program for Huntington’s cognitive problems, SAGE-718 is a first-in-class oral medication designed to modulate the NMDA receptor’s activity and improve cognitive function in the disease’s early stages.

The Phase 2 DIMENSION trial (NCT05107128) is currently recruiting up to 178 patients, ages 25-65, in the U.S., U.K., Canada, and Australia. Eligible participants are those with genetically confirmed Huntington’s in the pre-manifest or early-manifest stages and who are having cognitive problems.

Participants are being randomly assigned to receive either SAGE-718 or a placebo once daily for three months. The trial’s goal is to assess changes in the Huntington’s Disease Cognitive Assessment Battery (HD-CAB) score.

Another Phase 2 study, called SURVEYOR (NCT05358821), is enrolling up to 80 patients in the same age range at sites across the U.S. The placebo-controlled trial will assess the differences in cognitive performance between early Huntington’s patients and healthy volunteers then evaluate the effect of SAGE-718 on real-world functioning in patients.

Those who complete DIMENSION or SURVEYOR can enter the Phase 3 PURVIEW extension trial (NCT05655520), wherein all will receive SAGE-718 and be monitored for safety and efficacy outcomes for up to about a year. The study is also enrolling patients who haven’t participated in a previous SAGE-718 study.