Commentary
Video
Biomarker and Subgroup Insights on Novel CIDP Therapy Riliprubart: Luis Querol, MD, PhD
Author(s):
The neurologist at the Hospital de la Santa Creu, in Barcelona, Spain, provided clinical context on sustained responses and new biomarker findings from exploratory phase 2 data on riliprubart in CIDP. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
"This is the first clinical trial in CIDP to show a reduction in neurofilament light chain—a key biomarker anchoring the observed clinical improvements."
Riliprubart, developed by Sanofi, is a humanized IgG4 monoclonal antibody that selectively inhibits complement component C1s, a key serine protease in the classical complement pathway. By blocking C1s, the agent prevents the activation of downstream complement proteins–such as C4, C2, and C3–that contribute to inflammation and immune-mediated damage to peripheral nerves. Over the years, complement activation has been increasingly recognized as a key driver in antibody- and macrophage-mediated nerve injury in chronic inflammatory demyelinating polyneuropathy (CIDP).
New biomarker and exploratory data from a phase 2 study (NCT04658472) assessing riliprubart in CIDP were presented at the recent Peripheral Nerve Society (PNS) Annual Meeting, held May 17-20, in Edinburgh, Scotland. Overall, the analyses revealed that treatment with the agent resulted in reduced neurofilament light levels, a biomarker of neuroaxonal damage, in patients with CIDP. More notably, greater reductions in NfL levels were associated with higher treatment response rates, as measured by Inflammatory Neuropathy Cause and Treatment (INCAT) Disability score.
To gain greater insights on these data and their implications, NeurologyLive® sat down with study author Luis Querol, MD, PhD, a neurologist at the Hospital de la Santa Creu, in Barcelona, Spain. In the interview, Querol highlighted the consistent efficacy of the agent across patient populations, noting its durable clinical benefit in refractory patients, a historically difficult group to treat. Furthermore, he spoke on the drug’s impact on various CIDP subtypes, as well as the significance of reducing NfL, becoming the first such decrease reported in a CIDP clinical trial.
Click here for more PNS 2025 coverage.
