Cell Therapy Shows Promise in Progressive Multiple Sclerosis

Key Insights

• A cell therapy is under development for progressive multiple sclerosis (MS) patients with ongoing inflammatory tissue injury and who are HLA-DRB1*15:01 positive.
• GT201, a novel therapy, previously received IND clearance from China’s Center for Drug Evaluation in July 2023, marking a significant regulatory milestone.
• Data from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial demonstrated an 85% median reduction in HS D2S6 levels in the CSF among patients receiving the pivotal dose.

A novel cell therapy is being developed for patients with progressive multiple sclerosis (MS) who exhibit imaging evidence of ongoing inflammatory tissue injury and are HLA-DRB1*15:01 positive. This therapy aims to address the unmet need for effective treatments in progressive MS, where inflammation continues to drive disease progression.

GT201, the investigational therapy, previously received Investigational New Drug (IND) clearance from China’s Center for Drug Evaluation in July 2023. This regulatory milestone facilitates further clinical development and potential future availability of the treatment.

Promising Results from DEVOTE Trial

Data from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial showcased encouraging results. Patients who received the pivotal dose level experienced a median reduction of 85% in HS D2S6 levels within the cerebrospinal fluid (CSF). This reduction suggests a potential impact on disease-related biomarkers.