The novel drug vidofludimus calcium (Immunic Therapeutics) is safe and effective for patients with relapsing-remitting multiple sclerosis (RRMS), new research suggests.
Vidofludimus calcium is an investigational oral second-generation selective dihydroorotate dehydrogenase (DHODH) inhibitor without the off-target effects on kinases seen with drugs of the same class.
In the phase 2 EMPhASIS trial, patients with RRMS who were treated with vidofludimus calcium showed a significant reduction in new MRI lesions compared with those who received placebo. The active drug was also well tolerated.
"This provides an extremely favorable safety and tolerability profile" compared with other DHODH inhibitors, Andreas Muehler, MD, chief medical officer at Immunic, told Medscape Medical News.
"Our goal is to provide patients with an oral MS drug with very good efficacy but that avoids all the safety tolerability problems and therefore maintains their quality of life," Muehler said.
The findings were published online June 14 in the Annals of Clinical and Translational Neurology.
The EMPhASIS trial included 209 adults with RRMS who were randomly allocated to receive once-daily vidofludimus calcium (30 or 40 mg) or placebo for 24 weeks.
At 24 weeks, the mean cumulative number of combined unique active lesions was 6.4 in the placebo group, vs 2.4 in the group that received vidofludimus calcium 45 mg daily.
The study met its primary endpoint, demonstrating a statistically significant 62% reduction in the cumulative number of combined unique active (CUA) MRI lesions up to week 24 in participants who received the 45-mg dose compared with those who received placebo (2.4 vs 6.4 lesions; rate ratio, 0.38; P = .0002), the company notes in a press release.
The study also met its key secondary endpoint, showing a statistically significant 70% reduction in the cumulative number of CUA MRI lesions for the 30-mg once-daily dose in comparison with placebo (4.0 vs 13.2; rate ratio, 0.30; P < .0001).
Other MRI outcomes, including cumulative number of T1, T2, and gadolinium-enhancing lesions up to 24 weeks, favored vidofludimus calcium and were consistent with the study's primary findings, the researchers report.
Trends favoring the active drug were also observed in the clinical outcome of annualized relapse rate and in the clinically relevant biomarker of neurofilament light chain. However, the study was not powered for these outcomes.
The novel drug was also very well tolerated; the safety profile was on par with placebo.
The incidence of the two most common treatment-emergent adverse events (nasopharyngitis and headache) was low and was similar to that of placebo.
"Importantly, vidofludimus calcium was found to be safe and well-tolerated as compared to placebo, with no increase in the rate of infections, effects on liver or blood cell laboratory parameters, and with a very low treatment discontinuation rate," coordinating investigator Robert J. Fox, MD, neurologist, Mellen Center for Multiple Sclerosis, and vice chair for research, Neurologic Institute, Cleveland Clinic, Ohio, said in the release.
The president and CEO of Immunic, Daniel Vitt, PhD, noted in the same release that on the basis of the trial's "strong data," the company has enrolled patients with progressive MS in the phase 2 CALLIPER trial.
CALLIPER was designed "to further explore vidofludimus calcium's neuroprotective potential, as exemplified by a slowing of brain atrophy and delay in disability worsening, which are often caused by axonal and neural damage," he said.
"Equally exciting, we have also been enrolling patients in our phase 3 ENSURE program of vidofludimus calcium as a treatment for relapsing multiple sclerosis (RMS). We remain highly enthusiastic about the potential for this novel therapeutic to become a best-in-class DHODH inhibitor in RMS," Vitt added.
More Research Needed
Commenting on the study for Medscape Medical News, Shaheen E. Lakhan, MD, PhD, a neurologist in Boston, Massachusetts, said the clinical trial results demonstrate that vidofludimus calcium is "safe and tolerable with potential clinical utility against the formation of inflammatory brain lesions in people with relapsing-remitting MS."
However, "longer and larger studies are needed to confirm any effect beyond MRI lesions, including assessing relapse rate and disability," added Lakhan, who was not involved with the current research.
The study was funded by Immunic Therapeutics. Fox and several co-investigators have financial relationships with the company. Lakhan has reported no relevant financial relationships.
Ann Clin Transl Neurol. Published online June 14, 2022. Full article
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Cite this: Megan Brooks. Novel Drug for Relapsing-Remitting MS: New Phase 2 Data - Medscape - Jun 22, 2022.