Investigational Small Molecule Therapy for Progressive MS Associated with Reduced Serum NfL Levels Compared with Placebo
According to interim analysis of the CALLIPER clinical trial (NCT05054140), people with progressive multiple sclerosis (PMS) subtypes who were treated with VidoCa (vidofludimus calcium; Immunic, New York, NY) as an oral tablet for 24 weeks showed a significant reduction in serum neurofilament light chain (NfL) levels, compared with those receiving placebo. VidoCa is an investigational small molecule therapy with neuroprotective, anti-inflammatory, and anti-viral effects. These results were presented at the Americas Committee for Research and Treatment in Multiple Sclerosis (ACTRIMS) Forum 2024.
CALLIPER is an active phase 2, multicenter, randomized, double-blind, placebo-controlled study that is assessing the safety, efficacy, and tolerability of VidoCa as a treatment for people with PMS. The study enrolled 467 partcipants with primary PMS (PPMS), non-active secondary PMS (n-aSPMS), and active secondary PMS (SPMS). Participants were randomized to receive either VidoCa oral tablets once daily or placebo and will be monitored for a total of 120 weeks. The interim analysis, which was conducted after 203 participants completed 24 weeks of treatment, assessed serum NfL levels using the Quanterix Simoa assay (Quanterix, Billerica, MA).
In terms of interim analysis results:
- 61% of the interim population had naSPMS , 29% had PPMS, and 10% had SPMS.
- The mean age was 49.7 years with a disease duration of 4.6 years.
- Compared to the placebo group, participants who received VidoCa showed a 22.4% decrease in serum NfL levels across all PMS subtypes:
- There was a 18.8% decrease for PPMS compared with placebo.
- There was a 20.1% decrease for n-aSPMS compared with placebo.
- There was a 43.3% decrease for SPMS compared with placebo.
The study authors are affiliated with the Cleveland Clinic, Lycalis, and Immunic.