Hesperos' CIDP-on-chip evidence instrumental to achieving orphan drug designation

30 June 2025

The Ministry of Health, Labour and Welfare in Japan has granted orphan drug designation to riliprubart for treating chronic inflammatory demyelinating polyneuropathy (CIDP), Sanofi reports. Riliprubart also holds this designation for CIDP in the EU and US.

By engineering its human-relevant CIDP-on-chip as preclinical tool for disease modelling and drug efficacy testing, Hesperos has played an instrumental role in achieving this outcome.

CIDP is an inflammatory neuropathy that affects the peripheral nervous system, causing progressive muscle weakness and sensory impairment. Over the last decades, several animal species were employed to model CIDP, including mice, guinea pigs, rats, rabbits and non-human primates. However, owing to myriad inter-species differences in nervous, immune and muscular systems, animal models do not faithfully recapitulate the pathophysiology of this rare autoimmune disorder.

To date, inflammatory neuropathies remain an area of substantial unmet medical need, with very few approved therapies, variable treatment responses, and a lack of targeted, disease‑modifying options. Thus CIDP is currently managed through treatment with corticosteroids and intravenous immune globulins. Approximately 70% of CIDP patients respond to this first‑line therapy, typically showing moderate functional improvement (grip strength, walking distance). Less than a third of patients achieve long-term treatment-free remission and may relapse years later.

In their 2022 study, Hesperos established that C1s inhibition can rescue complement‑mediated neuromuscular dysfunction in human-based CIDP-on-chip. Building on this platform, Hesperos later generated proprietary riliprubart‑specific data for Sanofi that supported the authorization of Sanofi’s clinical study NCT04658472.

Riliprubart is an IgG4 humanized monoclonal antibody that targets activated C1s serine protease which initiates the classical complement pathway of the innate immune system. By selectively inhibiting C1s,  this antibody may attenuate the inflammatory mechanisms that drive demyelination and axonal injury in CIDP, thereby preserving neuromuscular function. Safety and efficacy of riliprubart is currently under phase 3 clinical investigation. Its mechanism of action, supportive human-on-chip data, significant unmet need and disease rarity have provided a solid scientific rationale for orphan drug designation.

Orlando-based CRO Hesperos, Inc is a pioneer and an industry leader in Human-on-a-Chip microfluidic systems for disease modelling, drug discovery, and efficacy-toxicity testing. Servicing global pharmaceutical, chemical and regulatory industries, Hesperos empowers researchers with human physiology-relevant interconnected multi-organ devices to accelerate and de-risk drug development.