Immunic Inc (NASDAQ:IMUX) has reported promising results from its Phase 2 CALLIPER trial of vidofludimus calcium, which showed a 30% reduction in the risk of 24-week confirmed disability worsening in patients with primary progressive multiple sclerosis (PPMS) compared to placebo.
The study enrolled 467 patients with progressive multiple sclerosis (PMS), including subpopulations with PPMS and non-active secondary progressive multiple sclerosis.
Across the full study population, vidofludimus calcium achieved a 20% reduction in 24-week confirmed disability worsening events.
In patients without gadolinium-enhancing lesions at baseline, typically less responsive to anti-inflammatory therapies, the drug reduced 24-week confirmed disability worsening events by 29% compared to placebo.
MRI data from the trial showed a 20% reduction in the annualized rate of thalamic brain volume loss for patients treated with vidofludimus calcium. Thalamic atrophy is a recognized marker of disease progression in PMS.
The overall change in brain volume also showed a 5% improvement over placebo.
Further, vidofludimus calcium demonstrated a beneficial effect on T2 lesion volume, with a mean 3.19% relative improvement over placebo at 24 months.
The therapy's safety profile was consistent with previous trials, with no new safety signals reported.
A novel approach to PMS
"The tremendous reduction of confirmed disability worsening in PMS patients is a wonderful confirmation of the objectives of this exploratory Phase 2 trial,” Immunic CEO Dr Daniel Vitt said in a statement.
“We believe that vidofludimus calcium may represent a novel and exciting approach for people living with PMS, where there continues to be a huge unmet medical need given only one approved therapy.”
Dr Andreas Muehler, Immunic’s chief medical officer, added that vidofludimus calcium is the only medicine being developed for MS that has been shown to be a potent activator of Nurr1, which plays a key role in neuroprotection.
“In particular, the 29% reduction of 24-week confirmed disability worsening events for vidofludimus calcium over placebo in the 391 patients without gadolinium-enhancing lesions at baseline underlines the drug's breakthrough potential in addressing the high unmet need of slowing neurodegeneration in MS patients,” Dr Muehler said.
“We believe today's exciting results of the phase 2 CALLIPER trial further validate vidofludimus calcium's scientific rationale, specifically driven by the impressive numerical benefit in reducing disability worsening, which deserves to be further tested in a Phase 3 registration trial.”
He added that, because nearly all disability events in non-active PMS are due to progression independent of relapse activity (PIRA), the CALLIPER results support the potential of vidofludimus calcium to impact disability progression in relapsing forms of MS, offering a positive read-through to the company’s ongoing Phase 3 ENSURE trials.