Riliprubart Treatment Provides Disease-Controlling Benefits in CIDP

Treatment with riliprubart (Sanofi, Bridgewater, NJ) for people with chronic inflammatory demyelinating polyneuropathy (CIDP) was associated with disease-controlling benefits, even for those who experienced failure, inadequate response, or residual disability when treated with standard of care (SOC). The results of the phase 2 study (NCT04658472) evaluating the safety, efficacy, and tolerability of riliprubart treatment for people with CIDP were presented at the 2024 Peripheral Nerve Society (PNS) Annual Meeting.

The global, multicenter, open-label study included participants with CIDP assigned to 1 of 3 cohorts:

• Those treated with SOC with residual disability (SOC-treated; n=48)
• Those who received ≥1 line of treatment with SOC who experienced inadequate or failed response (SOC-refractory; n=18)
• Those who never received SOC treatment (SOC-naïve; n=12)

The study consisted of 2 parts. In part A, participants were treated with riliprubart for 24 weeks. In part B, participants had the option to receive a treatment extension of an additional year of follow-up.

In part A:

• 87% of SOC-treated participants (n=42) improved or remained stable after switching from their previous line of treatment to riliprubart, while 52 (n=25) experienced greater improvement with riliprubart than on their SOC therapy.
• 89% of SOC-refractory participants (n=16) improved or remained stable while receiving riliprubart, with 50% showing improvement.
• 92% of SOC-naïve participants (n=11) improved or remained stable with riliprubart.
• Riliprubart was associated with improvements in patientreported fatigue and quality of life (QoL) outcomes across all 3 cohorts.

In part B:

• 73% (n=29) of SOC-treated participants who continued treatment (n=40) for an additional year experienced a sustained response.
• 89% (n=8) of SOC-refractory participants who continued treatment (n=9) for an additional year experienced a sustained response.
• 71% (n=5) of SOC-naïve participants who continued treatment (n=7) for an additional year experienced a sustained response.

Additionally, treatment with riliprubart was associated with reductions in neurofilament light chain (NfL) levels across all 3 cohorts throughout part A and part B. Treatment-emergent adverse events (TEAEs) occurred in 64.6% of SOC-treated participants and 88.9% of SOC-refractory participants, indicating a manageable safety profile.

According to Luis Querol Gutierrez, MD, PhD, from the Department of Neurology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain, “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. 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.”