Clinical Trials of Vidofludimus for Relapsing and Progressive Multiple Sclerosis Get Green Light
The Food and Drug Administration (FDA) cleared phase 3 ENSURE (NCT identifier not yet available) and phase 2 CALLIPER (NCT identifier not yet available) clinical trials of vidofludimus calcium (VFC)(IMU-838; Immunic, New York, NY) for relapsing and progressive multiple sclerosis (MS).
“IND clearance for our phase 3 program in relapsing MS is yet another seminal moment for Immunic as it progresses our lead asset, IMU-838, into a pivotal program and heralds the final phase of clinical development in MS. We believe the phase 3 ENSURE program meaningfully simplifies IMU-838’s regulatory approval path in MS as it applies a very clean and straightforward study design,” stated Daniel Vitt, PhD, chief executive officer and president of Immunic. “In addition, together with our MS expert panel, we designed the CALLIPER trial to study patients who currently are not typically treated with relapse-preventing therapies. Our goal is to highlight IMU-838 as a therapy that combines truly differentiated safety and tolerability with neuroprotective activity such as slowing of brain atrophy and disability worsening.
In the phase 2 EMPhASIS trial (NCT03846219), individuals with relapsing MS (n=138) were randomly assigned 1:1 to received 45 mg/day of vidofludimus vs placebo. Vidofludimus 30 or 45 mg/day resulted in statistically significant relative reductions of 70% (P<.0001) and 62% (P=.0002), respectively, in cumulative combined unique active (CUA) lesions on MRI over 24 weeks compared with placebo.
ENSURE comprises 2 multicenter randomized double-blind phase 3 trials designed of VFC vs placebo in participants with relapsing MS with a combined expected enrollment of 1,050 people. Participants will be randomly assigned to receive 30 mg/day VFC or placebo for 72 weeks with a planned outcome measure of time to first relapse. Volume of new T2-lesions, time to confirmed disability progression, time to sustained clinically relevant changes in cognition, and percentage of whole-brain volume change, grey matter volume, and white matter volume will also be measured. In addition, a pooled analysis of confirmed disability worsening will be completed. Interim analysis will be used to assess event rates and adjust sample size and outcome dates as needed.
CALLIPER is focused on the potential of vidofludimus to for progressive MS and will evaluate rates of brain atrophy and disability worsening with 45 mg/day vidofludimus vs placebo. Planned enrollment is 450 individuals and the trial will last 120 weeks.