To investigate the efficacy and safety of the glycolipid OCH-NCNP1 (OCH), in patients with relapsing multiple sclerosis (RMS).

First-in-human clinical trial of OCH, orally administered to healthy subjects and multiple sclerosis (MS) patients has demonstrated immunoregulatory effects through activation of NKT cells. 

A randomised, placebo-controlled study was conducted with a placebo group (n=15) and an OCH group (n=15) in 30 RMS patients. OCH (3.0 mg) or the placebo were orally administered weekly for 24 weeks. The primary outcomes were changes in magnetic resonance imaging results. Secondary outcomes were clinical observations, safety profiles, and exploratory biomarkers.

Of the 30 enrolled patients [relapsing-remitting MS (RRMS): 18 patients and secondary progressive MS (SPMS): 12 patients], 25 completed the trial, while 5 discontinued. In comparison with the placebo group, patients in the OCH group tended to have fewer new lesions or existing enlarged lesions, a lower annual recurrence rate, and more cases achieving NEDA, although there were no significant differences. Regarding the cumulative recurrence-free rate, MS activity was more suppressed in the OCH group than in the placebo group. The MS subtype analysis revealed that patients with SPMS in the OCH group significantly achieved NEDA (5/6 patients; 83.3%) compared with those in the placebo group (0/6 patients; 0%; p=0.015). Biomarker analysis revealed a significant increase in IL-4-producing helper T cells on day 1 after OCH administration compared with the placebo (p=0.010). The frequency of granulocyte-macrophage colony-stimulating factor (GM-CSF)-producing helper T-cells at 6 months after OCH administration significantly decreased compared with pre-treatment values (p=0.0056). A more significant decrease was observed in the SPMS group than in the RRMS group. There was no significant difference between the OCH and placebo groups in the safety profile.

OCH is a promising treatment for multiple sclerosis, especially for SPMS.