Oral glycolipid shows promise in the treatment of MS, especially SPMS

The glycolipid OCH-NCNP1 is an oral drug with a unique mechanism that acts on immunoregulatory cells and is a promising novel treatment for MS, especially for secondary-progressive MS (SPMS). This was concluded from a randomised, placebo-controlled, phase 2 trial for patients with relapsing MS.

The study results were presented by Dr Tomoko Okamoto (National Center Hospital, Tokyo, Japan) [1]. Dr Okamoto explained that the first in-human trial of the glycolipid OCH was conducted in her own hospital between 2012 and 2017. This was a single-dose study in healthy adults, followed by once weekly administration in MS patients for a period of 4 or 13 weeks [2]. The results showed that OCH exerts immunoregulatory effects through activation of natural killer T cells, to selectively produce IL-4.

Dr Okamoto went on to present the results of an investigator-initiated, randomised, placebo-controlled, phase 2 trial of OCH (UMIN000009382) in 30 MS patients (10 men and 20 women). Of these, 18 had relapsing-remitting MS, and 12 had SPMS; median disease duration was 11 years. Half of the participants (n=15) received OCH granules (3 mg), and the other half received matching placebo, once weekly for 24 weeks. The primary outcomes were new or enlarged T2 lesions on MRI. Secondary outcomes were annual relapse rate (ARR), relapse-free period, sustained reduction in disability (SRD), the period until SRD, and no evidence of disease activity (NEDA).

In the treatment and the placebo group, 2 and 3 participants, respectively, discontinued treatment before the end of the study. After 182 weeks, 1 of 15 (6.7%) participants in the experimental group and 3 of 15 (20%) in the control group had new lesions or existing enlarged T2 lesions. This difference did not reach statistical significance (difference -13.4%; 95 CI -43.3 to 14.9; P=0.598). The same was true for all secondary endpoints. The number of relapses was also lower in the OCH group (4 vs 8; ARR 0.565 vs 1.172). Dr Okamoto added that 10 participants in the OCH group (66.7%) and 5 in the placebo group (33.3%) achieved NEDA (difference -33.3%; 95 CI -65.4 to 5.6; P=0.143). Of note, 5 NEDA cases in the OCH group were among SPMS patients, versus 0 in the placebo group.

Biomarker analysis revealed a significant increase in IL-4-producing cells after 1 day in the OCH group compared with the placebo group (P=0.098). The number of granulocyte-macrophage colony-stimulating factor (GM-CSF)-producing helper T cells decreased significantly 6 months after OCH administration compared with baseline (P=0.0056), especially among SPMS patients.

1. Okamoto T, et al. Phase II clinical trial of NKT cell-targeting glycolipid OCH-NCNP1 for patients with Relapsing Multiple Sclerosis. O059, MSMilan 2023, 11–13 October, Milan, Italy.
2. Sato W, et al. Ther Adv Neurol Disord. 2023;16:17562864231162153.

 

Copyright ©2023 Medicom Medical Publishers



Posted on 4 December, 20234 December, 2023