The staff neurologist at the Mellen Center for Multiple Sclerosis at Cleveland Clinic discussed open-label extension data from the phase 2 CALLIPER trial of vidofludimus calcium in progressive multiple sclerosis. [WATCH TIME: 6 minutes]
Although the treatment landscape for relapsing
Vidofludimus calcium (Immunic) is an investigational oral agent that directly activates nuclear receptor-related 1 (Nurr1) and selectively inhibits dihydroorotate dehydrogenase (DHODH), a dual mechanism that may allow it to target both focal neuroinflammation and the chronic, compartmentalized inflammation and neurodegeneration that underlie progression. In the phase 2 EMPhASIS trial (NCT03846219), the agent significantly reduced MRI lesions in patients with relapsing-remitting MS.2
To test whether this mechanism translates to progressive disease, the phase 2 CALLIPER trial (NCT05054140) randomly assigned 467 patients with primary or secondary progressive MS to vidofludimus calcium 45 mg once daily or placebo. The double-blind period did not meet its primary end point of percent brain volume change, although exploratory clinical and imaging outcomes showed directionally favorable signals.3
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WATCH TIME: 6 minutes | Captions are auto-generated and may contain errors.
“We want to prevent patients from getting worse, but we also want to try to help them get better.”
To discuss these findings, NeurologyLive® spoke with presenting author
He noted that CALLIPER was modest in size and not powered to detect statistically significant differences in disability outcomes, but described consistent trends toward slower confirmed disability progression in both the secondary progressive and primary progressive subpopulations. Fox emphasized that similar point estimates emerged in patients with and without gadolinium-enhancing lesions at baseline, suggesting the drug may be doing more than suppressing relapsing activity.
From there, Fox walked through the open-label extension, highlighting the low dropout rate as a sign of good tolerability and persistence over several years. He described how patients who switched from placebo to vidofludimus calcium did not appear to catch up to those treated continuously, with similar trends seen for confirmed disability improvement. Fox closed by explaining the rationale for combining confirmed disability progression and improvement into a single scale, noting that both lie along a continuum and that strong trends favored vidofludimus calcium during both the double-blind and open-label periods.
