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Riliprubart Continues to Show Pronounced Impacts on CIDP at 1 Year
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Patients with CIDP treated with riliprubart reported improved quality-of-life and fatigue measures, as well as reduced neurofilament light levels.
Luis Querol Gutierrez, MD, PhD
In a phase 2 study (NCT04658472), newly announced data revealed that Sanofi’s riliprubart resulted in disease-controlling benefits among patients with chronic demyelinating polyneuropathy (CIDP) in both the 24-week treatment period and 52-week open-label extension (OLE). Riliprubart, a complement C1 inhibitor, is currently being evaluated in 2 phase 3 studies, dubbed MOBILIZE (NCT06290128) and VITALIZE (NCT06290141) that will continue to study its effects as a potential treatment for CIDP.1
The study, a global, multicenter trial, assessed riliprubart across 3 cohorts of CIDP: participants who are receiving a standard-of-care (SOC) treatment with residual disability (SOC-treated), participants who had failed or had an inadequate response to SOC treatment (SOC-refractory), and participants who had never received a SOC treatment (SOC-naïve). All participants underwent 24 weeks of treatment (Part A), followed by an optional treatment extension for 52 weeks (Part B).
Results, which were presented at the 2024 Peripheral Nerve Society Annual Meeting, showed improved patient-reported fatigue and quality-of-life measures across all cohorts through the initial 24 weeks and an additional 24 weeks of treatment. These included the RASCH-built Fatigue Severity Scale and the EuroQoL Visual Analogue Scale, a health-related quality of life assessment. In addition, treated patients showed reductions in neurofilament light, a biomarker of neuroaxonal damage, throughout the initial 24 weeks and additional year of treatment.
"Many people living with CIDP do not fully respond to available therapies or do not respond at all, demonstrating a significant unmet need for this community,” study investigator Luis Querol Gutierrez, MD, PhD, a neurologist at the Hospital de la Santa Creu, in Barcelona, Spain, said in a statement.1 "These phase 2 data for riliprubart are encouraging, as they suggest that riliprubart’s unique mechanism of action reduces the overactive, damaging complement pathways that may drive disease progression."
READ MORE: Long-Term Safety and Low Relapse Rate Observed in Phase 3 ADVANCE-CIDP 3 Trial of Hyqvia
At 24 weeks of riliprubart treatment, 87% (42 of 48) of SOC-treated participants reported improved or sustained disease activity, including 52% (25 of 48) who experienced improvement above and beyond their previous therapy. After an additional year of riliprubart, 73% (29 of 40) sustained their response. Similarly, for SOC-naïve patients, 92% (11 of 12) improved or remained stable with riliprubart after 24 weeks, and 71% (5 of 7) sustained their response after an additional 24 weeks of riliprubart treatment.
For SOC-refractory participants, 89% (16 of 18) improved or remained stable with riliprubart after 24 weeks, with 50% (9 of 18) of this cohort showing improvement. In total, 89% (8 of 9) of this group sustained their response after an additional year of riliprubart treatment. Riliprubart was considered well tolerated through the study, with treatment-emergent adverse events (TEAEs) occurring in 64.6% (31 of 48) and 88.9% (16 of 18) of SOC-treated and SOC-refractory participants, respectively. There were 2 reported deaths in the study, both attributable to significant medical comordbidities aside from CIDP.
The original data from part A was presented at the 2023 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting. At baseline, mean INCAT score was 3.3 in the SOC-treated group and 5.4 in the SOC-refractory group. In addition to showing clinically meaningful improvements across primary and secondary efficacy end points, a strong (>90%) and sustained inhibition of complement activity was observed in SOC-treated and SOC-refractory groups.2
CIDP, a rare neurological condition that caused progressive weakness and sensory impairment in the arms and legs, has seen several recent advances in treatment and management of the disease. Earlier this year, the FDA approved Takeda’s immune globulin (IG) infusion 10% with recombinant human hyaluronidase, a liquid medicine, as a maintenance therapy for adults with CIDP. Weeks later, the agency approved another therapy from Takeda, this time approving IG infusion 10% (human)(Gammagard Liquid) as a intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in CIDP.3,4
