GeNeuro announced that the primary analysis of the Phase 2 ProTEct-MS study was presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS 2022) Congress in Amsterdam, Netherlands, by Dr. Fredrik Piehl, Professor of Neurology at the Department of Clinical Neurosciences of the Karolinska Institutet, head of research at the MS clinic of the Academic Specialist Center (ASC), and Principal Investigator of the study. The ProTEct-MS study enrolled a very homogenous cohort of 42 patients being treated with temelimab (18, 36 and 54mg/kg) vs. placebo for 48 weeks. The patients included in the study had confirmed disability progression without relapses, following previous treatment with the anti-CD20 drug rituximab, a highly potent and efficacious drug against relapses and brain lesion formation. The one-year Phase 2 trial, conducted at the Karolinska Institutet's ASC in Stockholm, Sweden, evaluated the administration of temelimab in patients with relapsing remitting MS to address disease progression independent of relapses following treatment with rituximab.
The data presented showed that, after one year of treatment, temelimab appeared to be a safe add-on to anti-CD20 treatment. The drug was well tolerated with no treatment related discontinuations, no serious or severe treatment emergent adverse events, and no differences in overall clinical or laboratory safety findings, which meets the primary endpoint of the study. Also, as already announced in the top-line results in March 2022, MRI biomarkers showed a favourable impact of temelimab in preserving neocortical anatomy and myelin integrity. The effect sizes were of comparable magnitude to those previously observed in the prior CHANGE-MS and ANGEL-MS trials. The combined treatment of temelimab and rituximab protected against loss of cortical thickness by more than 50% relative to rituximab alone. Furthermore, cortical tissue integrity, as measured by magnetization transfer saturation, was improved with temelimab, potentially reflecting remyelination. New exploratory data was presented on soluble biomarkers, as measures of neurodegeneration at one year: the study showed a reduction of GFAP and NfL biomarkers in cerebrospinal fluid (CSF). GFAP is a biomarker for astrocytic activation associated with diffuse neuroaxonal damage leading to MS disease progression. Patients treated with temelimab and rituximab after 48 weeks showed an average 2.5% reduction in GFAP, versus an average increase of 2.5% for those only receiving rituximab. NfL measurement in CSF, another well-established marker of neuroaxonal damage in MS correlating with MS disease evolution, also showed a relative reduction of 33% in patients treated with temelimab and rituximab, compared to those patients treated with rituximab alone. The results on these CSF biomarkers confirm the synergistic potential to treat neurodegeneration with temelimab in addition to a high-efficacy anti-inflammatory therapy in MS.